Spe­cial re­port: Twen­ty ex­tra­or­di­nary women blaz­ing trails in bio­phar­ma R&D — Covid-19 and be­yond

In the sec­ond year of End­points News’  bud­ding tra­di­tion of high­light­ing women blaz­ing trails in bio­phar­ma R&D, we’ve seen a num­ber of firsts.

For the first time, the biggest sto­ry in R&D is al­so top of mind for a world anx­ious to end the most dev­as­tat­ing health cri­sis in decades. With its sweep­ing ef­fects, the Covid-19 pan­dem­ic is turn­ing the dai­ly rou­tines for many work­ing women up­side down, tak­ing a toll on not just their phys­i­cal and men­tal health, but al­so their ca­reer prospects. At the same time, the biotech in­dus­try is do­ing some se­ri­ous soul search­ing with a new score­card and plan for di­ver­si­ty and in­clu­sion.

It is more im­por­tant than ever to shine light on the grow­ing num­ber of women who have scaled the heights of drug dis­cov­ery and de­vel­op­ment even though the odds are stacked against them, break­ing open paths in labs and C-suites that can be fol­lowed by fu­ture gen­er­a­tions, some of whom they are al­so ac­tive­ly nur­tur­ing and men­tor­ing.

We set out this year with a dual goal: to cel­e­brate women at the fore­front of sub­du­ing Covid-19 — ei­ther with di­ag­nos­tics, vac­cines or treat­ments — and to hon­or those work­ing day in and day out to ad­dress oth­er equal­ly press­ing med­ical needs.

Pro­files are, by de­f­i­n­i­tion, snap­shots. Some of the women rec­og­nized in our 2019 spe­cial re­port have since be­come house­hold names: Jen­nifer Doud­na be­came a No­bel lau­re­ate for her pi­o­neer­ing work in CRISPR gene edit­ing and Özlem Türe­ci helped cre­ate the world’s first vac­cine proven ef­fec­tive against Covid-19. Even in the short pe­ri­od since we in­ter­viewed this year’s hon­orees, we’ve seen them mark new mile­stones, from tak­ing a biotech pub­lic to scor­ing his­toric clin­i­cal da­ta and reg­u­la­to­ry au­tho­riza­tions.

We hope our pro­files cap­ture a unique mo­ment in his­to­ry as these high­ly ac­com­plished fig­ures take us with them down mem­o­ry lane to il­lus­trate what brought them to this mo­ment and how they are help­ing oth­er women do the same — if not even more.

Hear from the hon­orees at our event

We host­ed an on­line live event to in­tro­duce this year’s top women in bio­phar­ma R&D, fol­lowed by a pan­el dis­cus­sion on what it will take to tru­ly achieve gen­der di­ver­si­ty in the in­dus­try. You can watch the full event here.

  • Car­olyn Bertozzi: Twen­ty one years af­ter her ‘ge­nius’ break­through, Car­olyn Bertozzi keeps push­ing the lim­its of gly­co­bi­ol­o­gy
  • Kathy Bowdish: Kathy Bowdish flouts a misog­y­nist in­vestor ac­quain­tance with big prof­its and a big­ger ca­reer
  • Di­ana Brainard: Once an as­pir­ing pro­fes­sor, Di­ana Brainard is in­stead lead­ing Gilead’s Covid-19 charge
  • Jan­ice Chen: Mam­moth’s Jan­ice Chen, a Jen­nifer Doud­na acolyte, wants to bring di­ag­nos­tics to your doorstep
  • Ann Che­ung: Ann Che­ung builds next-gen I/O be­neath a Cam­bridge bar
  • Lynn Con­nol­ly: Chase for Covid-19 an­ti­bod­ies brings Lynn Con­nol­ly back full cir­cle
  • Kizzmekia Cor­bett: Six years in­to her job at the NIH, Kizzmekia Cor­bett lends an im­por­tant hand in Covid-19 vac­cine de­vel­op­ment
  • Leena Gand­hi: An I/O pi­o­neer re­turns from phar­ma to find the next big break­through
  • Pearl Huang: From phar­ma en­claves to Chi­na to biotech start­up, Pearl Huang fol­lows her cu­rios­i­ty in­to new ad­ven­tures
  • Kathrin Jansen: Al­ways look­ing for the prob­lems, Kathrin Jansen found a his­toric Covid-19 so­lu­tion
  • Katal­in Kariko: The co-in­ven­tor of mod­i­fied mR­NA, Katal­in Karikó is fi­nal­ly get­ting her mo­ment
  • Sara Kenkare-Mi­tra: In­spired by her grand­fa­ther, Sara Kenkare-Mi­tra finds her high­er call­ing in med­i­cine
  • Lynne Krum­men: Af­ter climb­ing the ranks at Genen­tech, Lynne Krum­men digs in­to im­munol­o­gy at Vir
  • Leah Lip­sich: Re­gen­eron’s mas­ter plan­ner finds pow­er in lis­ten­ing and be­ing the most in­flu­en­tial voice in the room
  • Stacey Ma: Stacey Ma fol­lows great-grand­fa­ther’s foot­steps to a top seat at start­up Sana
  • Jen­nifer Pet­ter: ‘Nev­er had a grand plan’: A wind­ing ca­reer path led Jen­nifer Pet­ter to launch her own com­pa­ny
  • Aviv Regev: Genen­tech taps an am­bi­tious pi­o­neer to guide them through bi­ol­o­gy’s fu­ture
  • Mar­go Roberts: An ear­ly CAR re­searcher and Yescar­ta leader, Mar­go Roberts is back in the game at Lyell
  • Lau­ra Shawver: ‘Just say yes’: Lau­ra Shawver leads her fifth biotech with an all-fe­male C-suite
  • Kim­ber­ly Smith: Kim­ber­ly Smith goes in­side the in­dus­try to build a new kind of HIV biotech ex­ec­u­tive

Twen­ty one years af­ter her ‘ge­nius’ break­through, Car­olyn Bertozzi keeps push­ing the lim­its of gly­co­bi­ol­o­gy

Car­olyn Bertozzi wasn’t ex­act­ly look­ing to start a com­pa­ny when she tweet­ed about a preprint her group post­ed on Chem­Rx­iv last No­vem­ber. The pa­per de­scribed LY­TACs — mol­e­cules that can tag ex­tra­cel­lu­lar pro­teins for degra­da­tion.

She was head­ing out to the Amer­i­can Chem­i­cal So­ci­ety Con­fer­ence in Or­lan­do to talk about the project, led by a post­doc in her Stan­ford lab named Steve Banik.

“(Steve) was about to go on the aca­d­e­m­ic job mar­ket, so it’s a good time for me to go and sort of talk about his work pub­licly and drop his name and get him some name recog­ni­tion,” she re­called.

With­in 24 hours, though, she got a dozen phone calls from ven­ture cap­i­tal groups who picked up on the preprint. There was a pro­lif­er­a­tion of star­tups uti­liz­ing and im­prov­ing on PRO­TACs — a tar­get­ed degra­da­tion tech­nol­o­gy born out of Craig Crews’ aca­d­e­m­ic work at Yale — but that mag­ic was lim­it­ed to in­tra­cel­lu­lar pro­teins. Bertozzi was a self-de­scribed jeal­ous fan of Crews’ work be­cause gly­co­pro­teins, the sub­ject of her own re­search, re­side ex­clu­sive­ly on cell sur­faces; it turned out VCs had al­so been wish­ing there were ways to tar­get se­cret­ed and mem­brane pro­teins.

In the end, she found Ver­sant Ven­tures to be the best fit for the sci­ence. Now, Ly­cia Ther­a­peu­tics, which un­veiled a $50 mil­lion Se­ries A just a few months ago, has been “tak­ing off like a rock­et ship,” she said.

The en­thu­si­asm from in­vestors might be ex­pect­ed for a sci­en­tist and en­tre­pre­neur whose last biotech cre­ation, Palleon Phar­ma­ceu­ti­cals, re­cent­ly reeled in $100 mil­lion in fresh fi­nanc­ing. Red­wood, the one that came be­fore, re­cent­ly re­port­ed Phase I da­ta as a sub­sidiary of the CRO gi­ant Catal­ent. Then there are the hand­ful of di­ag­nos­tics play­ers that she cre­at­ed with re­searchers un­der her wing.

But for Bertozzi, it’s all about tim­ing.

Her first ex­pe­ri­ence with en­tre­pre­neur­ship — a biotech start­up she co-found­ed in the late 1990s af­ter com­plet­ing a post­doc with Steve Rosen at UCSF — ac­tu­al­ly end­ed in fail­ure. Thios Phar­ma­ceu­ti­cals, as it was known, had ze­roed in on a tar­get for sick­le cell acute va­so-oc­clu­sive cri­sis and in-li­censed a mol­e­cule that worked in a sim­i­lar way as two ther­a­pies ap­proved by the FDA late last year.

They raised $10 mil­lion for it, a re­spectable amount for a Se­ries A at that time, but it wasn’t enough to sus­tain the 30-per­son com­pa­ny when in­vestors de­cid­ed not to con­tin­ue fund­ing it. The drug was shelved as Thios closed up shop.

“It was al­ways very heart­break­ing to us that we couldn’t have brought that to those pa­tients 15 years ear­li­er,” Bertozzi said. “But that’s how it goes in this busi­ness, and every com­pa­ny has a sto­ry like that.”

In her day job as an aca­d­e­m­ic, Bertozzi is known for spear­head­ing a “gly­corev­o­lu­tion:” de­vel­op­ing chem­i­cal tools and tech­nolo­gies to elu­ci­date the roles that com­plex sug­ar struc­tures play in bi­o­log­i­cal sys­tems. Gly­cans, as she learned dur­ing her doc­tor­ate, are an or­der of mag­ni­tude more tech­ni­cal­ly com­plex than polypep­tides and oligonu­cleotides be­cause of their struc­tur­al di­ver­si­ty. But pa­pers dat­ing as far back as the 1950s have sug­gest­ed they do play a role in dis­eases such as can­cer, and spec­tac­u­lar break­throughs in chem­i­cal syn­the­sis — the chal­lenge that drew her to the space ini­tial­ly — have helped sci­en­tists make in­roads in their study.

Her biggest break­through — which won her a MacArthur “ge­nius grant” at age 33 — came in what she coined “bioorthog­o­nal chem­istry,” a way for chem­i­cal re­ac­tions to take place with­in bi­o­log­i­cal sys­tems.

Both the tools and her deep knowl­edge of gly­co­bi­ol­o­gy pro­vid­ed the foun­da­tion for the sub­se­quent tech­nolo­gies her group would lat­er de­vel­op. Site-spe­cif­ic chem­i­cal mod­i­fi­ca­tion of re­com­bi­nant pro­teins promised to make bet­ter an­ti­body-drug con­ju­gates (ADC) than the “very slop­py mol­e­cules” that char­ac­ter­ized the first gen­er­a­tion of AD­Cs; dig­ging deep­er in­to new find­ings on how can­cer cells sprouts sug­ars on their sur­faces to evade the im­mune sys­tem led them to bis­pe­cif­ic an­ti­body-en­zyme con­structs that tar­get the siglec-sial­ic acid path­way, billed as the next big I/O check­point; and uti­liza­tion of lyso­so­mal traf­fick­ing shut­tles — “one of the first things you learn when you study gly­co­bi­ol­o­gy”— gave birth to chimeras that send un­want­ed ex­tra­cel­lu­lar pro­teins to the lyso­some for tar­get­ed degra­da­tion.

More so than any par­tic­u­lar tech­nol­o­gy, though, she con­sid­ers the young sci­en­tists who trained with her and are now stand­ing on their own feet her most im­por­tant achieve­ment.

“Car­olyn’s ge­nius is not on­ly in the re­search she leads, but al­so in those she re­cruits,” Mireille Ka­ma­riza, a for­mer PhD stu­dent, said. Now a ju­nior fel­low at Har­vard Uni­ver­si­ty, she co-found­ed with Bertozzi a pub­lic ben­e­fit cor­po­ra­tion to ad­vance a point-of-care di­ag­nos­tic de­vice for tu­ber­cu­lo­sis.

“She has an un­can­ny abil­i­ty to bring out the best in peo­ple,” Ka­ma­riza added. “She leads by ex­am­ple and gives un­yield­ing sup­port to her stu­dents’ in­ter­ests and pur­suits.”

Now 54, Bertozzi re­cent­ly found her­self re­flect­ing on her ca­reer af­ter the death of late Supreme Court Jus­tice Ruth Bad­er Gins­burg. Har­vard, af­ter all, had on­ly in­te­grat­ed with Rad­cliffe a few years be­fore she’d start un­der­grad­u­ate stud­ies there.

“I con­sid­er my­self to be in sort of the first gen­er­a­tion where you see a crit­i­cal mass of women who made it through the aca­d­e­m­ic pipeline and in­to in­dus­try,” she said. “There aren’t many of us, but you can count us. We’re vis­i­ble.”

Hav­ing seen every shade of gen­der dis­crim­i­na­tion and bias (“show me a woman who hasn’t”), Bertozzi is lever­ag­ing her in­flu­ence as the co-di­rec­tor of Stan­ford ChEM-H to bring in more voic­es from out­side the usu­al sus­pects — reach­ing out and forg­ing re­la­tion­ships with pub­lic uni­ver­si­ties and in­sti­tu­tions serv­ing un­der­rep­re­sent­ed com­mu­ni­ties to find “tal­ent that is hid­ing in plain sight.”

In pre­vi­ous in­ter­views, she’s not­ed that part of what got her hooked in those first or­gan­ic chem­istry class­es was how mol­e­cules have per­son­al­i­ties like peo­ple. So I couldn’t help but ask: What mol­e­cule would she be?

The an­swer was sur­pris­ing­ly sim­ple. She as­pires to be a mon­o­clon­al an­ti­body.

“They mul­ti­task all over the place,” she said. In fact, all of the next-gen­er­a­tion bi­o­log­ics out of her lab have had an­ti­bod­ies as their back­bones.

“I have got­ten so much mileage out of an­ti­bod­ies,” she said. “And so I would like to think maybe my stu­dents could get the mileage out of me that same way. That would be my as­pi­ra­tion.”

That’s not all: “Oh, and they are gly­co­sy­lat­ed.” — Am­ber Tong

Kathy Bowdish flouts a misog­y­nist in­vestor ac­quain­tance with big prof­its and a big­ger ca­reer

Kathy Bowdish was sit­ting down at a San Diego restau­rant af­ter her first pitch meet­ing in 1997 when one of the po­ten­tial in­vestors turned and asked her a ques­tion: “Does your hus­band know you’re do­ing this?”

Tak­en aback, Bowdish, 39 at the time, said yes, and that part of this was his idea. Of the 12 an­gel in­vestors at the ta­ble that day, the con­cerned cit­i­zen was the on­ly one who didn’t de­cide to back her first start­up. And he was the on­ly one who didn’t get a cut of the prof­its three years lat­er, when Bowdish sold the start­up to Alex­ion for 10 times their in­vest­ment.

When the press re­lease for the buy­out hit the wire, her phone rang. It was the lone in­vestor.

“If you do this again, I want to be on speed di­al,” the in­vestor told her. Bowdish looks back on that mo­ment with vic­to­ry-tinged hu­mor: “Clear­ly, his ques­tion had been trou­bling him.”

The seed in­vestor had made two mis­takes. One should’ve been ob­vi­ous at the time: plain misog­y­ny. But the oth­er would on­ly be­come ful­ly ap­par­ent over the en­su­ing 23 years as the young sci­en­tist-ex­ec­u­tive en­meshed her­self in a se­ries of top biotechs and even­tu­al­ly was hand­picked by Elias Zer­houni to lead a unique and — un­der her di­rec­tion — fruit­ful ven­ture in Sanofi’s up­per ranks.

He had un­der­es­ti­mat­ed Kathy Bowdish.

Bowdish would prove over the years a rare triple-threat in biotech: a bril­liant sci­en­tist who could han­dle the busi­ness side but al­so had the poise and emo­tion­al IQ to mas­sage need­ed per­son­al re­la­tion­ships and whee­dle folks who could get things done. Af­ter near­ly two decades in biotech, she used those skills to build pow­er­ful new com­pa­nies at Sanofi. And when the winds changed there, she piv­ot­ed mid-ca­reer to what she first learned to do in San Diego: build a com­pa­ny with cut­ting-edge sci­ence from the ground up.

“Kathy im­pressed me be­cause she had been CSO, she was a very good sci­en­tist,” Zer­houni told me. “But she had this very spe­cial nat­ur­al calm … A very thought­ful calm, a re­silience. She had this an­a­lyt­i­cal mind.”

Bowdish is now work­ing from her home in Cam­bridge, try­ing to get a three-per­son, RNA-based start­up off the ground with $5 mil­lion in seed cash. It’s a sig­nif­i­cant de­par­ture from her Sanofi perch, but it’s a po­si­tion she knows and a place she’s thrived be­fore.

The daugh­ter of an en­gi­neer and an artist, Bowdish was se­duced by the cere­bral yet cre­ative chal­lenge that sci­en­tif­ic prob­lems posed. Af­ter study­ing bi­ol­o­gy at William & Mary, she moved to the West Coast, where she was quick­ly re­cruit­ed for a po­si­tion at Scripps Re­search and worked with Richard Lern­er on the then-pi­o­neer­ing field of cat­alyt­ic an­ti­bod­ies.

She was clever and am­bi­tious, de­vis­ing new ways of se­quenc­ing an­ti­bod­ies in an era be­fore you could sim­ply plug them in­to RNA-Seq. She left to get a PhD at Co­lum­bia Uni­ver­si­ty, and picked a yeast lab be­cause yeast mul­ti­plies like mi­cro­bial rab­bits, which meant you could do a lot of ge­net­ics work in a sin­gle pro­gram.

A post­doc fol­lowed, but she soon grew im­pa­tient. These were still ear­ly days for an­ti­bod­ies. She pitched Lern­er on the idea of build­ing a com­pa­ny around the work they did on com­bi­na­to­r­i­al an­ti­body li­braries — ba­si­cal­ly dis­cov­ery en­gines in a test tube.

“His im­me­di­ate ques­tion to me was ‘can you live with­out a salary for 6 months? ‘Cause that’s what it’s go­ing to take in or­der to raise the mon­ey.’ And I said yes,” she said. “I was pret­ty naive — maybe the naiveté was a good thing.”

The in­vestors (but one) came on board, Bowdish fi­na­gled her way in­to some cheap equip­ment, hired an­oth­er sci­en­tist — a woman — and for a year the pair worked at the bench to dis­cov­er an­ti­bod­ies that would ac­ti­vate, rather than in­ac­ti­vate, re­cep­tors. She shift­ed over to a full CEO role af­ter year one, and af­ter year three Alex­ion bought the com­pa­ny for $41 mil­lion, bet­ting that it could serve as their dis­cov­ery en­gine.

She stayed at Alex­ion un­til 2007, by which time she was a sought-af­ter name for biotechs with big ideas. In­vestors re­cruit­ed her to re­launch Anaphore, a Dan­ish biotech try­ing to de­vel­op an­ti­body-like al­ter­na­tives that were more se­lec­tive or more po­tent. They strug­gled for years, which wor­ried Bowdish un­til she re­al­ized every com­pa­ny in the space was strug­gling. It taught her a valu­able les­son about what makes for good trans­la­tion­al sci­ence.

“You just can’t beat evo­lu­tion,” she said.

Then ARCH and Flag­ship came call­ing. They had fund­ed a bold Bob Langer idea for de­liv­er­ing pro­teins in­side cells. Quick­ly, she re­al­ized they had to start test­ing this in an­i­mals to have any idea if it worked. And quick­ly they learned that it didn’t every­thing went to the liv­er. So she lever­aged their tech in­to an­ti­body-drug con­ju­gates.

“It looked re­al­ly good in vit­ro,” she said. “In vi­vo, not so much.”

By then it was 2013. and Bowdish had been in the league for 16 years, help­ing run five biotechs on both coasts. She knew the game, but she was cu­ri­ous: about phar­ma, that be­he­moth in the back­ground, and about in­vest­ment, a gen­er­al-es­que perch from which she could pon­der and eval­u­ate. At the same time, Zer­houni was con­ceiv­ing a new ini­tia­tive at Sanofi: Sun­rise, a way of giv­ing biotechs the re­sources and ex­per­tise of phar­ma while al­low­ing them to flour­ish on their own. It mar­ried the two.

Bowdish got the job, beat­ing out such biotech ti­tans as Michael Gilman. In­side, she learned how phar­ma worked and she built new com­pa­nies. She was, Zer­houni said, sin­gle­hand­ed­ly re­spon­si­ble for MyoKar­dia, the car­dio­vas­cu­lar up­start that Bris­tol My­ers Squibb re­cent­ly pur­chased for $13 bil­lion. She found them, he said, de­signed a deal to get them the best re­sources and con­vinced Sanofi lead­er­ship to get on board.

“She’s able to move the nee­dle from a pret­ty es­tab­lished sort of ap­proach to an in­no­v­a­tive one,” Zer­houni said. “She’s a change agent.”

Then Paul Hud­son came in, reshuf­fled Sanofi, and Bowdish found her­self on the outs (Sanofi al­so pulled out of her MyoKar­dia deal, a blun­der that cost them a cool $1 bil­lion). She went home, re­laxed and did what she al­ways does — dove back in­to the lit­er­a­ture: pa­pers on im­munol­o­gy, di­a­betes, bio­mol­e­c­u­lar con­den­sates, what­ev­er fas­ci­nat­ed her.

Even­tu­al­ly, a re­cruiter called. She re­al­ized it was time to get mov­ing. She men­tioned to Gilman they had stayed in touch af­ter a con­fer­ence — that she was on a hunt, and Gilman put her in touch with Gen­zyme Ven­tures vet Alan Waltz, who was look­ing for a CEO for a com­pa­ny that would de­vel­op small mol­e­cules that tar­get struc­tures on mR­NA in an ef­fort to block mul­ti­ple onco­genes. She liked the sci­ence and the po­ten­tial im­pact.

“It was back to some of my ear­li­er learn­ings, in terms of the right con­stel­la­tion of peo­ple around the ta­ble and the sci­ence that was ready for this very di­rect­ed ef­fort,” she said.

So now she works from home, por­ing over the lit­er­a­ture, di­rect­ing CROs and her team of three, prepar­ing for a fundrais­ing round and build­ing a whole team. It’s a small­er com­pa­ny than she had led since she was just out of grad school, ig­nor­ing the pre­ten­sions of a sex­ist in­vestor, and she couldn’t be hap­pi­er.

“It’s ac­tu­al­ly a lot of fun,” she said. — Ja­son Mast

Once an as­pir­ing pro­fes­sor, Di­ana Brainard is in­stead lead­ing Gilead’s Covid-19 charge

Di­ana Brainard was study­ing abroad in France when she re­al­ized some­thing just didn’t quite feel right.

In­tend­ing to be­come a com­par­a­tive lit­er­a­ture pro­fes­sor while at­tend­ing Brown, Brainard en­rolled at a French uni­ver­si­ty as part of her ma­jor and ful­ly im­mersed her­self for the first time in lit­er­a­ture and lit the­o­ry class­es. Brainard had main­ly tak­en a bal­anced di­et of cours­es state­side — every­thing from math and sci­ence to Eng­lish — and the Study Abroad pro­gram was a nec­es­sary part of her pro­gram. She went in­to it head-on as her lit­er­a­ture sem­i­nars were her fa­vorite un­der­grad class­es, with pas­sion­ate pro­fes­sors seem­ing­ly putting on a “pro­duc­tion” every class.

But af­ter div­ing in, Brainard felt her stud­ies had lost their at­tach­ments to the re­al world.

“All of a sud­den there was this re­al­iza­tion of ‘Wow, what I’m re­al­ly be­ing as­sessed on in these class­es, and how my ca­reer will be as­sessed in this field, is com­ing up with a the­o­ry and mak­ing that the­o­ry sexy,’” Brainard said. “But that the­o­ry doesn’t have to be true. It just has to be sexy and in­ter­est­ing … and I can do all of that, but there is no truth here.”

Since then, Brainard has moved past the days of Thomas Mann’s The Mag­ic Moun­tain, though she hasn’t lost her love of lit­er­a­ture. Now, she’s a se­nior ex­ec­u­tive at Gilead and has been a ma­jor part of the team that de­vel­oped remde­sivir, re­cent­ly ap­proved by the FDA as the first Covid-19 treat­ment for hos­pi­tal­ized pa­tients.

The path from France to Gilead wasn’t a straight line, how­ev­er. Brainard notes that af­ter Brown, she even­tu­al­ly went to med school ex­pect­ing to be­come a physi­cian. But that didn’t end up be­ing the right fit ei­ther, es­pe­cial­ly af­ter Brainard “fell in love” with in­fec­tious dis­eases. She end­ed up as a re­searcher at Har­vard fo­cused on HIV, con­tent to work off NIH-backed grants for the rest of her ca­reer.

Then bio­phar­ma came call­ing.

“Leav­ing aca­d­e­mics was re­al­ly hard be­cause I was hap­py do­ing what I was do­ing, and I hadn’t en­vi­sioned in the fu­ture that I would be go­ing to in­dus­try,” Brainard said. “There weren’t a lot of peo­ple at Har­vard who had done that be­fore, and so I felt like I was tak­ing a big risk, I was leav­ing be­hind NIH fund­ing, we were leav­ing Boston where we’d been for a long time, and I didn’t know if I would wind up re­gret­ting all of the things that I was let­ting go of.”

Brainard jumped aboard first at Mer­ck, then joined Gilead in 2010. She’s spent her en­tire ca­reer, in acad­e­mia and oth­er­wise, work­ing in in­fec­tious dis­eases and im­munol­o­gy, and much of her time at Gilead has cen­tered on he­pati­tis C treat­ments. When she was first alert­ed to remde­sivir’s po­ten­tial ben­e­fits for a new coro­n­avirus strain out of Chi­na al­most a year ago, Brainard moved to get it in­to the lab as quick­ly and ef­fi­cient­ly as pos­si­ble.

Remde­sivir was a team ef­fort, Brainard said, and she’s re­luc­tant to take cred­it for spear­head­ing the drug’s piv­ot to Covid-19. Orig­i­nal­ly test­ed in he­pati­tis C and then Ebo­la, remde­sivir showed ear­ly signs of in vit­ro ac­tiv­i­ty in SARS and MERS. Brainard pushed for Gilead to be­gin a com­pas­sion­ate use pro­gram for the drug, and it re­ceived par­tial emer­gency au­tho­riza­tion in May for se­vere Covid-19 cas­es.

That EUA was ex­pand­ed in Au­gust to in­clude mod­er­ate cas­es, and the FDA hand­ed down a full ap­proval in Oc­to­ber for pa­tients who have been hos­pi­tal­ized with the dis­ease.

“It’s been ex­hil­a­rat­ing to do [this] for Covid be­cause of the acute, un­prece­dent­ed med­ical need, and be­cause sci­en­tif­i­cal­ly it’s fas­ci­nat­ing to have a dis­ease, as an in­fec­tious dis­ease ex­pert, to learn about a dis­ease at the same time you’re try­ing to fig­ure out if a ther­a­py for a dis­ease works,” Brainard said a few weeks be­fore the ap­proval. “It’s a re­al­ly dif­fi­cult prob­lem, and one that doesn’t come around very of­ten.”

The drug has re­ceived some push­back, as a study backed by the WHO found in mid-Oc­to­ber that remde­sivir had lit­tle to no ef­fect in re­duc­ing mor­tal­i­ty rates or the need for ven­ti­la­tors.

Though Brainard was orig­i­nal­ly hes­i­tant to move from acad­e­mia, she’s hap­py to have found a home at Gilead and not have to deal with what she says are li­a­bil­i­ties in that are­na. She nev­er re­al­ly en­joyed the struc­ture of how aca­d­e­m­ic ca­reers are tra­di­tion­al­ly judged, where the main goal is to work your way up the au­thor­ship ranks and get a cou­ple of pa­pers un­der your belt.

Brainard much prefers the col­lab­o­ra­tive en­vi­ron­ment in in­dus­try. And at Gilead, she thinks that col­lab­o­ra­tion helps her be a bet­ter role mod­el for younger women ris­ing through the field.

“The im­pact that I have, a lot of it is the drugs,” Brainard said, “but a lot of it is the peo­ple. The peo­ple who I work with, a lot of the peo­ple who come to Gilead, for ex­am­ple, come right out of train­ing … and they make that tran­si­tion in the same way that I did. Help­ing them learn how to think in this new en­vi­ron­ment and thrive, and flour­ish in a new en­vi­ron­ment, that’s re­al­ly mean­ing­ful to me.” — Max Gel­man

Mam­moth’s Jan­ice Chen, a Jen­nifer Doud­na acolyte, wants to bring di­ag­nos­tics to your doorstep

Jan­ice Chen is all about de­moc­ra­tiz­ing di­ag­nos­tics.

The Mam­moth Bio­sciences co-founder has made that her mis­sion over the last three and a half years, work­ing along­side new­ly-mint­ed No­bel lau­re­ate Jen­nifer Doud­na to de­vel­op di­ag­nos­tic tools cen­tered around CRISPR’s gene edit­ing tech­niques. But what might that look like in prac­tice? Chen the­o­rized some po­ten­tial us­es in a TEDx CERN Talk two years ago.

“What if you could di­rect­ly and ac­cu­rate­ly test for the flu at home?” Chen said. “What if you re­ceive the pre­scrip­tion and treat­ment plan with­out hav­ing to step foot in­to a clin­ic? And what if the same prin­ci­ple could be ap­plied to oth­er dan­ger­ous dis­eases such as Ebo­la?”

Chen kicked off her sci­en­tif­ic ca­reer as an un­der­grad at Johns Hop­kins, where she took a course in which stu­dents built the yeast genome from scratch. She cred­its that class with get­ting her the tech­ni­cal skills need­ed to ap­ply to re­search and lab as­sis­tant po­si­tions, as well as ex­plore how syn­thet­ic bi­ol­o­gy could lead to a swath of ap­pli­ca­tions.

It al­so served as a jump­ing-off point for Chen’s am­bi­tions, where she said she was able to get her hands wet in some­thing with re­al-world ap­pli­ca­tions rather than read­ing lines from a text­book. Af­ter a year­long stint as a re­search tech­ni­cian at Har­vard, where her work fo­cused on Ral­sto­nia eu­tropha bac­te­ria, Chen moved on to her doc­tor­ate at UC Berke­ley and joined Doud­na’s lab.

It’s there where she first hooked on to CRISPR re­search.

“My­self and col­leagues in the lab, in­clud­ing co-founder Lu­cas Har­ring­ton, came across this un­ex­pect­ed find­ing that some of these Cas pro­teins were able to de­tect DNA,” Chen told End­points News. “A lot of it was serendip­i­tous in terms dis­cov­er­ing this ac­tiv­i­ty, and be­ing able to demon­strate it on re­al pa­tient sam­ples I think was re­al­ly an ex­cit­ing mo­ment for me in think­ing about ‘OK, can we ac­tu­al­ly take this out­side the aca­d­e­m­ic lab and try to do some­thing with it?’”

Though CRISPR has large­ly set­tled in the main­stream con­scious­ness as a way to po­ten­tial­ly cure a range of se­vere dis­eases — along with the oc­ca­sion­al head­line that sci­en­tists are out to cre­ate gene-edit­ed em­bryos in a lab — Chen and the Mam­moth team are look­ing at ways these tools can be used for dis­ease de­tec­tion. In­stead of us­ing the “scis­sors” nor­mal­ly as­so­ci­at­ed with CRISPR to ed­it genes, Chen pro­grams the tech to find a de­fined gene se­quence and sends out a sig­nal once it’s been lo­cat­ed.

With the Covid-19 pan­dem­ic in full swing, Mam­moth has al­so steered its re­search to­ward cre­at­ing ac­ces­si­ble and easy-to-use tests for the de­tec­tion of SARS-CoV-2. In that in­stance, the plat­form is pro­grammed with a guide RNA, and Chen hopes by work­ing on tests that can func­tion out­side of the typ­i­cal lab set­ting, this can be one of the ways to “de­moc­ra­tize” the tech­nol­o­gy.

“It’s been suc­cess­ful be­cause it’s pro­gram­ma­ble,” Chen said. “There’s a whole world of test­ing that’s just start­ing to be clos­er to re­al­i­ty … that has nev­er quite been able to be done be­cause of the lim­i­ta­tions of in­fra­struc­ture needs, the ac­cu­ra­cy to ac­tu­al­ly be a vi­able so­lu­tion. With CRISPR now on the scene, we’re re­al­ly ex­cit­ed about the po­ten­tial to ad­dress these point-of-need en­vi­ron­ments that don’t re­ly on your tra­di­tion­al clin­i­cal lab­o­ra­to­ry.”

Chen cred­its her doc­tor­al ad­vi­sors from Berke­ley, Doud­na in­clud­ed, with keep­ing her fo­cused and tran­si­tion­ing from acad­e­mia to be­com­ing an en­tre­pre­neur at Mam­moth. Ul­ti­mate­ly, that led Chen to her “north star” of not just en­gi­neer­ing new us­es for CRISPR, but be­ing able to cre­ate im­pact­ful tech­nol­o­gy in gen­er­al.

“(Doud­na) her­self is a se­r­i­al en­tre­pre­neur, and so it’s been re­al­ly im­por­tant to have her men­tor­ship, and see­ing her as a role mod­el be­ing very suc­cess­ful in both the aca­d­e­m­ic world as well as the start­up world,” Chen said, a few weeks be­fore Doud­na won the No­bel Prize.

As some­one who’s still in the ear­ly stages of her ca­reer, Chen rec­og­nizes the op­por­tu­ni­ty she has to make an im­pact on the women who fol­low in her foot­steps. She’s al­ready been named to Forbes’ 30 un­der 30 health­care list from 2018 for be­ing among the “most in­flu­en­tial mil­len­ni­als” in the sec­tor. By co­in­ci­dence, all of her ma­jor re­search ex­pe­ri­ences from Johns Hop­kins through Berke­ley were led by women prin­ci­pal in­ves­ti­ga­tors.

And while Chen says there’s not go­ing to be one “mag­ic bul­let” that fix­es every­thing in the in­dus­try, the best place to get start­ed is by re­cruit­ing tal­ent from all sorts of dif­fer­ent back­grounds.

“Once you have that tal­ent and have them in the com­pa­ny it’s re­al­ly im­por­tant to en­sure that they have the sup­port sys­tems and men­tor­ship in­ter­nal­ly to help them grow,” Chen said. “It’s im­por­tant to rec­og­nize that there are re­al­ly great lead­ers in com­pa­nies that might not fit the tra­di­tion­al mold, and I think that there are a lot of com­pa­nies that are start­ing to fig­ure that out.” — Max Gel­man

Ann Che­ung builds next-gen I/O be­neath a Cam­bridge bar

In a cramped, win­dow­less lab base­ment be­side Cam­bridge’s Blue Room bar, Ann Che­ung stuck her head in the freez­er and, with a vial of cells in one hand, used the oth­er to phone a world-fa­mous pro­fes­sor across town and a tech en­tre­pre­neur in San Fran­cis­co.

Che­ung was 34. She had the aca­d­e­m­ic pedi­gree: Brown, MIT, a post­doc at Cal­Tech where she built nanopar­ti­cles and stud­ied im­munother­a­py with a No­bel lau­re­ate. But it had been over four years since she worked with flasks and cen­trifuges. Her last job was at MIT— but as an ad­min­is­tra­tor and com­mu­ni­ca­tor. It in­volved a lot of tweet­ing.

Then a call came from Tyler Jacks, the renowned head of the MIT Cen­ter for Can­cer Re­search (now called the Koch In­sti­tute) and her old doc­tor­al ad­vi­sor. Along with an old col­lege friend, the Sil­i­con Val­ley in­ven­tor Bill Haney, they want­ed to boot­strap this new idea out of Jacks’ lab, a jack­knife way of get­ting the im­mune sys­tem to turn on can­cer. And Jacks knew Che­ung was itch­ing to get back to the bench.

Soon she found her­self in the base­ment of a Kendall Square bar, in a lab no big­ger than a uni­ver­si­ty of­fice, grow­ing an­ti­bod­ies and nat­ur­al killer cells with a sin­gle oth­er em­ploy­ee and hear­ing the sounds of eat­ing and loud talk­ing when­ev­er they went in the hall. She was the grit­ty and bril­liant, if un­like­ly, CSO of Drag­on­fly Ther­a­peu­tics, and her work there and in the shiny of­fices they’ve since moved to would prove and de­vel­op ideas that even­tu­al­ly land­ed col­lab­o­ra­tions with Cel­gene, Ab­b­Vie and Bris­tol My­ers Squibb. Last year, it en­tered pa­tients for the first time.

“Her growth has nev­er stopped and this has been true for her en­tire ca­reer,” Jacks told me. “There’s no chal­lenge that dis­cour­ages her. She’s kind of fear­less.”

Che­ung didn’t ar­rive as a grad­u­ate stu­dent at Jacks’ MIT lab want­i­ng to study im­muno-on­col­o­gy. This was 2002. Check­point in­hibitors were still a fringe idea in the head of a wild-haired Berke­ley pro­fes­sor. No one stud­ied im­muno-on­col­o­gy in Jacks’ lab.

One evening, though, Bob Schreiber came in for a lec­ture. Schreiber’s mice work was just be­gin­ning to res­ur­rect the idea of us­ing T cells to at­tack tu­mors, and Che­ung was cap­ti­vat­ed. On the walk back that night, she turned to Jacks with an epiphany. “Of course the im­mune sys­tem can fight can­cer,” Che­ung said. “Can­cer is some­thing that is for­eign to the body even though it comes from the body, so it ab­solute­ly makes sense.”

Jacks was fa­mous for mak­ing mouse mod­els to study can­cer ge­net­ics, paving the way for pre­ci­sion drugs that tar­get tu­mor pro­teins, but he had had his eye on im­munol­o­gy for a while. He was just wait­ing for a grad stu­dent will­ing to take on a field that was still on­ly main­stream-ad­ja­cent. Che­ung be­came that stu­dent, work­ing alone in a pres­ti­gious lab on an idea that the rest of the grad stu­dents and post­docs thought would like­ly fail.

“I re­mem­ber a lot of con­ver­sa­tions and talks I gave where es­sen­tial­ly there was a lot of skep­ti­cism,” Che­ung told me. “Many peo­ple thought that im­mune re­spons­es that fought can­cer and oc­ca­sion­al­ly even caused spon­ta­neous re­mis­sions were just anec­do­tal things and not some­thing that could be har­nessed.”

In time, of course, im­muno-on­col­o­gy would take over the can­cer field, and Che­ung would move from an out­sider to a mi­nor pi­o­neer; half of Jacks’ lab is now fo­cused on im­munother­a­py. Many mem­bers are con­duct­ing sim­i­lar work to Che­ung, de­vel­op­ing mouse mod­els for im­muno-on­col­o­gy, a no­to­ri­ous­ly dif­fi­cult task be­cause the im­mune sys­tem is more dif­fi­cult to sim­u­late than any in­di­vid­ual can­cer pro­tein or tu­mor type.

Che­ung had a back­ground in en­gi­neer­ing, too, and af­ter MIT, she worked at a nan­otech lab at Cal­Tech. She helped de­vel­op mi­croflu­idic de­vices that could an­a­lyze in­di­vid­ual cells and used them to an­a­lyze the T cells of can­cer pa­tients who re­ceived a new cell re­cep­tor ther­a­py from No­bel lau­re­ate David Bal­ti­more.

From there, she took a job at Cell press — an amaz­ing one, she said, that al­lowed her to read and think wide­ly across can­cer and bi­ol­o­gy, but one that even­tu­al­ly grew weari­some. She was a crit­ic there, but she want­ed to be a builder. She went back to MIT, this time as an ad­min­is­tra­tor for col­lab­o­ra­tions be­tween the Koch In­sti­tute and oth­er cen­ters. She knew ear­ly on it’d be a way sta­tion to biotech. She was about to ac­cept a dif­fer­ent of­fer when Jacks called, talk­ing about this idea of de­vel­op­ing “TriN­Kets” that can grab tu­mors and link them with the body’s own nat­ur­al killer cells.

Che­ung be­came Em­ploy­ee No. 1. The three met in the Cam­bridge base­ment. Jacks drew a big blue dot in the cen­ter of the white­board — the NK cell — and the three chart­ed out all they could imag­ine, sketch­ing di­a­grams that un­der­lay a drug now in the clin­ic, di­a­grams they would nev­er build, di­a­grams that still rep­re­sent some of their biggest dreams.

Haney es­chewed VC mon­ey, re­ly­ing in­stead on fam­i­ly in­vestors and ear­ly col­lab­o­ra­tions. So in year 1, it was just Che­ung and her per­son­al Em­ploy­ee No. 1, Gre­go­ry Chang, in the base­ment, try­ing to kill can­cer cells in test tubes and call­ing back with the re­sults. It was a lot of re­spon­si­bil­i­ty and a lot of work for some­one in their first biotech job, but Che­ung said she nev­er thought of it that way. In fact, she nev­er re­al­ly thought of it.

“I come from an im­mi­grant fam­i­ly. My par­ents, they had a Chi­nese take­out, so in a way they were en­tre­pre­neurs,” she said. “And for a take­out or even a Chi­nese restau­rant, you don’t have a lot of peo­ple but there are a lot of po­si­tions that need to be filled. So this kind of men­tal­i­ty of just step­ping in and do­ing what’s need­ed, I’ve pret­ty much car­ried with me through life. And that’s what start­up’s all about.”

The re­sults came, and so did a lab with win­dows and breath­ing space. Jacks said Che­ung not on­ly proved the ear­ly sci­ence but al­so proved shrewd, con­fi­dent and in­valu­able at the ta­ble with Big Phar­mas as they pre­sent­ed and ne­go­ti­at­ed col­lab­o­ra­tions.

She now di­rects a size­able team as head of bi­ol­o­gy and she no longer has her head in the freez­er on a dai­ly ba­sis. The thrills are big­ger, too: not crude proof-of-con­cepts, but emails from the FDA con­firm­ing they can start a tri­al. She re­mem­bers when the first two OKs came, and she drew up a plan for when, hope­ful­ly, the big call comes — con­fir­ma­tion that the thing they first drew up be­neath a Cam­bridge bar ac­tu­al­ly works.

“When that day comes, I’ve de­cid­ed I’m go­ing to tat­too my­self with the drag­on­fly,” she said. “I’ve said it many times, this is go­ing to hap­pen.” — Ja­son Mast

Chase for Covid-19 an­ti­bod­ies brings Lynn Con­nol­ly back full cir­cle

When Lynn Con­nol­ly, in­spired by her un­der­grad­u­ate ex­pe­ri­ence work­ing with both ba­sic sci­en­tists and clin­i­cians, was in­ter­view­ing for MD/PhD pro­grams at the turn of 1990, she had two dif­fer­ent pro­gram di­rec­tors tell her point-blank: Her as­pi­ra­tional dual-ca­reer would not be pos­si­ble for a woman.

It was long be­fore Con­nol­ly would come to Ada­gio Ther­a­peu­tics, where she is now work­ing with a team of se­nior women lead­ers to de­vel­op an­ti­bod­ies against SARS-CoV-2. Al­though she had nev­er met a woman MD/PhD in per­son, it had al­ways struck her as a pos­si­bil­i­ty. She was im­me­di­ate­ly tak­en aback.

“My re­sponse was like, ‘I re­spect­ful­ly dis­agree.’ And amaz­ing­ly I got in­to one of those pro­grams,” she said. “I didn’t go there, clear­ly.”

She end­ed up at the Uni­ver­si­ty of Cal­i­for­nia, San Fran­cis­co, where she be­gan con­sid­er­ing a piv­ot in her re­search fo­cus from hu­man ge­net­ics to in­fec­tious dis­eases. The HIV epi­dem­ic was in full force while drug­mak­ers still had lit­tle to of­fer, and she found her­self en­grossed in med­ical mi­cro­bi­ol­o­gy and epi­demi­ol­o­gy class­es as well as Lau­rie Gar­rett’s book on the com­ing plague.

Vi­rol­o­gy was a nat­ur­al path to go down af­ter work­ing on vac­cines for her­pes and HIV dur­ing short stints at Ch­i­ron. But her su­per­vi­sor’s de­par­ture led Con­nol­ly to Car­ol Gross’ lab in the mid­dle of her PhD pro­gram, where she would switch her fo­cus to bac­te­ri­ol­o­gy — specif­i­cal­ly to es­tab­lish a chain of pro­teins in­volved in a trans­duc­tion path­way for E coli.

“She just con­cep­tu­al­ized the prob­lem so clear­ly that as soon as she said it I said, “You know, of course, it should’ve been this way,’” re­called Gross, whom Con­nol­ly cred­its as a key men­tor. They grew so close, in fact, that Con­nol­ly said she’s al­so the one who in­spired a then-un­usu­al de­ci­sion to have a child while in grad­u­ate school.

That back­ground served her well when she even­tu­al­ly moved to her first in­dus­try job as med­ical di­rec­tor at the an­tibi­otics shop Achao­gen. Dur­ing the decade-plus in be­tween as Con­nol­ly com­plet­ed her res­i­den­cy, fel­low­ship and lat­er be­came ju­nior fac­ul­ty at UCSF, the whole in­fec­tious dis­ease field had a “huge tech­no­log­i­cal rev­o­lu­tion that has al­lowed us to study some of these pathogens that have been his­tor­i­cal­ly very chal­leng­ing to study,” she not­ed. In­tran­si­gent bac­te­ria like My­cobac­teri­um tu­ber­cu­lo­sis can now be eas­i­ly ma­nip­u­lat­ed; there were now pro­tease in­hibitors for HIV pa­tients.

At the same time, she’d learn at Achao­gen, the tra­di­tions hadn’t changed much for the de­vel­op­ment of a new an­tibi­ot­ic. Large non-in­fe­ri­or­i­ty tri­als were re­quired even for agents tar­get­ing mul­tidrug-re­sis­tant gram-neg­a­tive pathogens, re­cruit­ing pa­tient pop­u­la­tions that don’t ex­act­ly re­flect who they’re try­ing to ad­dress.

Con­nol­ly says play­ing a part in chang­ing that stan­dard process is one of her proud­est ac­com­plish­ments, as she came up with pro­pos­als for stream­lined clin­i­cal tri­als that not just worked with reg­u­la­tors to nail down that new path, but worked in the trench­es with physi­cians to en­roll these spe­cif­ic, of­ten very weak, pa­tients. Then there was all the ad­vo­ca­cy work with pay­ers to de­vise new re­im­burse­ment mod­els.

“I think as a so­ci­ety, as not just in the US, but on the whole, I think folks have come to think of an­tibi­otics as things that should be read­i­ly avail­able, low cost — where in fact these are very pre­cious com­modi­ties,” she said. “These drugs are not de­signed to be block­busters. They’re re­al­ly de­signed to be re­served for the pa­tients who need them most.”

Ul­ti­mate­ly it was all too lit­tle, too late for Achao­gen, which strug­gled fi­nan­cial­ly de­spite get­ting a drug ap­proved. By the time it de­clared bank­rupt­cy, Con­nol­ly had land­ed a new clin­i­cal re­search role at Vir, the big-mon­ey in­fec­tious dis­ease start­up helmed by George Scan­gos.

The many facets of in­fec­tious dis­ease drug de­vel­op­ment on dis­play at Vir, from mon­o­clon­al an­ti­bod­ies to siR­NA to live at­ten­u­at­ed vac­cines tar­get­ing a wide range of pathogens, was a “can­dy shop” for some­one in­ter­est­ed in tack­ling some of those glob­al health chal­lenges.

Just a few months af­ter she was pro­mot­ed to se­nior vice pres­i­dent, though, Till­man Gern­gross of Adimab called.

To­geth­er with Lau­ra Walk­er, the di­rec­tor of an­ti­body sci­ences as Adimab, he’d spun out a start­up named Ada­gio ded­i­cat­ed to tack­ling not just the coro­n­avirus caus­ing this pan­dem­ic but the oth­ers to come. For Con­nol­ly, the move would mean re­turn­ing to a group of il­lus­tri­ous women whom she had got­ten to know from work­ing in the an­tibac­te­r­i­al space.

There’s René Rus­so, the for­mer CEO of Ar­sa­nis who’s com­ing in as co-founder and chair; Cu­bist vet El­lie Her­sh­berg­er is the chief de­vel­op­ment of­fi­cer; and the rest of the C-suite fea­tures CSO Walk­er, chief tech­nol­o­gy & man­u­fac­tur­ing of­fi­cer Becky Dab­o­ra and chief op­er­at­ing & busi­ness of­fi­cer Hal­ley Gilbert.

“Here’s a com­pa­ny where 80% of the C-lev­el lead­er­ship is fe­male. You don’t find that of­ten, and you cer­tain­ly don’t find it in this pan­dem­ic,” Gern­gross said. “We were not try­ing at all to make this a pre­dom­i­nant­ly fe­male lead­er­ship team, but that’s where we end­ed up, in a very or­gan­ic process.”

These are al­so peo­ple who like­ly share Con­nol­ly’s ex­pe­ri­ence of get­ting called out for ques­tion­ing deeply around sci­en­tif­ic da­ta — even as men around her were ask­ing the same ques­tions.

“That was re­al­ly the first time where I re­al­ly un­der­stood, aha! This is what every­one is talk­ing about, where cer­tain be­hav­ior in the con­text of a woman is seen as ag­gres­sive and neg­a­tive, where­as male col­leagues par­tic­i­pat­ing in this be­hav­ior is re­ward­ed,” she said. “That dou­ble stan­dard be­came just in­cred­i­bly clear to me at that point.”

Ad­dress­ing these dou­ble stan­dards head-on is some­thing bio­phar­ma com­pa­nies can do to sup­port women, ac­cord­ing to her, in ad­di­tion to hav­ing pro­grams to sup­port child­care.

“It re­quires open di­a­logue,” she said. “You have to ac­knowl­edge that it’s hap­pen­ing.” — Am­ber Tong

Six years in­to her job at the NIH, Kizzmekia Cor­bett lends an im­por­tant hand in Covid-19 vac­cine de­vel­op­ment

NIH re­search fel­low Kizzmekia Cor­bett has been prepar­ing for this mo­ment for six years.

On a Fri­day in Oc­to­ber 2014, Cor­bett de­fend­ed her PhD the­sis at the Uni­ver­si­ty of North Car­oli­na at Chapel Hill, then packed her bags for a road trip. The fol­low­ing Mon­day, she was off to Mary­land, where she was start­ing a job at the NIH’s Vac­cine Re­search Cen­ter study­ing coro­n­avirus­es.

Be­fore the pan­dem­ic, Cor­bett was fo­cused on MERS vac­cine de­vel­op­ment. Her team takes a “pro­to­type pathogen ap­proach,” she ex­plained, in which they pick a pro­to­typ­ic virus from a huge vi­ral fam­i­ly, then de­sign and eval­u­ate vac­cines that could be used as a start­ing point in case of a pan­dem­ic.

“There has al­ways been this idea that coro­n­avirus­es would have pan­dem­ic po­ten­tial,” she said. “I think at this point, we can say ‘I told you so.’”

When the Covid-19 cri­sis struck, Cor­bett sprang in­to ac­tion. A lot of what she does is “or­ga­niz­ing small pieces of the big­ger thing”: an­a­lyz­ing and in­ter­pret­ing da­ta, plan­ning, de­sign­ing ex­per­i­ments, speak­ing and writ­ing.

When sci­en­tists first re­al­ized that Covid-19 is re­lat­ed to SARS, it was “ex­cit­ing and scary,” Cor­bett said.

“Ex­cit­ing be­cause at that time in Jan­u­ary … we weren’t in a pan­dem­ic, but it was re­al­ly ex­cit­ing to be able to uti­lize the knowl­edge that we have for a proof of prin­ci­ple around this en­tire pan­dem­ic pre­pared­ness plan. And then it be­came scary when it got clos­er to home in the US and then peo­ple start­ed to die.”

The last few months have been sur­re­al, she said. And ex­haust­ing. Since her team large­ly fo­cus­es on non-clin­i­cal de­vel­op­ment, they’ve had a chance to breathe and get back to oth­er work. Cor­bett will still be work­ing on coro­n­avirus­es, “just what­ev­er the next one is,” she said.

When asked about her great­est ac­com­plish­ment in the field, the 34-year-old paused. “I don’t think I’ve had it yet,” she said af­ter some thought. “I still haven’t ful­filled my dreams yet.”

One of those dreams, she said, is a vac­cine. But she’d al­so like to be a prin­ci­pal in­ves­ti­ga­tor and have her own lab, or maybe tap in­to en­tre­pre­neur­ship.

If she had to choose, though, she’d choose her PhD, which she earned in mi­cro­bi­ol­o­gy and im­munol­o­gy. “PhDs are a dime a dozen in the board­rooms that I sit in now, but where I’m from they’re not. So I think maybe that is my great­est ac­com­plish­ment,” she said.

Grow­ing up in Hills­bor­ough, NC, Cor­bett was al­ways gift­ed in school. But it was a high school in­tern­ship through the Amer­i­can Chem­i­cal So­ci­ety’s Pro­ject SEED that ce­ment­ed her love of sci­ence. The pro­gram pro­vides dis­ad­van­taged stu­dents with paid lab­o­ra­to­ry in­tern­ships for 8 to 10 weeks over the sum­mer. Cor­bett worked in an or­gan­ic chem­istry lab at UNC when she was just 16, un­der the men­tor­ship of pro­fes­sor James Morken.

“Kizzy was pas­sion­ate about sci­ence, en­thu­si­as­tic about learn­ing and re­al­ly strove for ex­cel­lence,” Morken said in a state­ment. “What she did not have were many op­por­tu­ni­ties to do re­search, and Pro­ject SEED of­fered that.”

For Cor­bett, that was the mo­ment “it sort of clicked that it was pos­si­ble.”

Af­ter grad­u­at­ing from Or­ange High School, she got her bach­e­lor’s in bi­ol­o­gy from the Uni­ver­si­ty of Mary­land, Bal­ti­more Coun­ty, then re­turned to her home state for her PhD.

To at­tract women to the field and not lose them in the pipeline, Cor­bett urges com­pa­nies to pro­mote di­ver­si­ty and in­clu­sion, and “put your mon­ey where your mouth is.”

“Every time some­one hears about a di­ver­si­ty and in­clu­sion prob­lem, they form a com­mit­tee, but then noth­ing comes from it … A lot of times places fo­cus on di­ver­si­ty so much that they for­get the in­clu­sion part. So … if you’re go­ing to hire women, then shouldn’t you have a lac­ta­tion room?”

There are iden­ti­ty in­ter­sec­tions, Cor­bett said, adding that she iden­ti­fies more so with the strug­gle of be­ing Black than the strug­gle of be­ing a woman. The pan­dem­ic has been the longest pe­ri­od of time in the last 6 years that she’s worn her hair straight. Nor­mal­ly, she feels com­fort­able wear­ing it in braids, or how­ev­er she wants at work.

“But now that I am vis­i­ble out­side of that work­place, know­ing how that will be ac­cept­ed every­where is not nec­es­sar­i­ly sure,” she said.

Be­ing held to stan­dards — such as look­ing or speak­ing a cer­tain way — can be drain­ing. “The way that we tend to think about pro­fes­sion­al­ism and work, that just doesn’t fit in­to every­one’s cul­tur­al per­cep­tion,” she said.

Then there’s the so­ci­etal pres­sure to do “all of the things that women should be do­ing,” like get­ting mar­ried and hav­ing chil­dren, she added. “It’s okay to shake up that nar­ra­tive,” she said.

Her ad­vice to young fe­male sci­en­tists? “Find your voice re­al­ly ear­ly and speak very loud.” — Nicole De­Feud­is

An I/O pi­o­neer re­turns from phar­ma to find the next big break­through

If you or some­one you know has ben­e­fit­ed from the check­point in­hibitors that have re­made can­cer treat­ment over the last decade, there are a few peo­ple you might con­sid­er thank­ing: Jim Al­li­son, of course, the No­bel Prize win­ner. Or maybe Mer­ck, the phar­ma that de­vel­oped the most suc­cess­ful in­hibitor, which now an­nu­al­ly earns them the GDP of a small East­ern Eu­ro­pean na­tion. But some­where on the list, you might al­so con­sid­er a less­er known name, a tho­racic on­col­o­gist named Leena Gand­hi.

Gand­hi has done much over the past few years — she’s got a gaudy project up go­ing right now — but to un­der­stand the mag­ni­tude of her con­tri­bu­tion, it’s prob­a­bly worth go­ing back near­ly a decade, to a time when the first check­point had been ap­proved but the ques­tion of just how big an im­pact they would have, and for whom, was still un­clear. Specif­i­cal­ly, to AS­CO 2014, and a small con­fer­ence room in­side Chica­go’s glassy Mc­Cormick Place ho­tel.

Gand­hi, then the young di­rec­tor of Dana-Far­ber’s tho­racic on­col­o­gy tri­als, stood up and pre­sent­ed da­ta sug­gest­ing that an ex­per­i­men­tal check­point in­hibitor known as MK-3475 worked far bet­ter in lung can­cer pa­tients whose tu­mors ex­pressed the pro­tein PD-L1 than in those whose didn’t.

It was a de­cep­tive­ly ob­scure find­ing. Check­point in­hibitors were sup­posed to take the “brakes” off the im­mune sys­tem, un­leash­ing T cells to at­tack tu­mors. But ear­ly tri­als pro­duced mixed and wild­ly vary­ing re­sults: Some pa­tients’ tu­mors van­ished and oth­ers didn’t and there was lit­tle way of pre­dict­ing which would do which. That was trou­ble; with­out a good pre­dic­tor for who would ben­e­fit, the drug would look mediocre in the ag­gre­gate, and doc­tors wouldn’t pre­scribe it or would pre­scribe it less.

On­col­o­gists weren’t con­vinced PD-L1 was the pre­dic­tor. For one, it was dif­fi­cult to mea­sure; the tech­nol­o­gy used at the time to ex­am­ine tu­mors was rel­a­tive­ly rudi­men­ta­ry and of­ten missed the pro­tein when it was there. “We thought we’d nev­er be able to use PD-L1,” Gand­hi told me.

There were al­so vary­ing re­sults de­pend­ing on the can­cer; on the same day Gand­hi pre­sent­ed her re­sults, UCSF’s Adil Daud pre­sent­ed da­ta show­ing that in melanoma, many pa­tients re­spond­ed re­gard­less of whether they ex­pressed PD-L1.

Yale’s Mario Sznol, a melanoma spe­cial­ist who was mod­er­at­ing the ses­sion, dis­cussed both and threw up his hands. “Well I’m not sure,” Gand­hi re­called him say­ing. “It’s not clear cut.”

MK-3475, of course, would be­come Keytru­da. And soon in­ves­ti­ga­tors would get re­sults from Keynote-024, the land­mark study that proved that, for PD-L1 pos­i­tive lung can­cer pa­tients, the check­point in­hibitor was bet­ter than chemother­a­py.

That’s when the ques­tions stopped. Now vir­tu­al­ly every tri­al for PD-1 in­hibitor con­tains a bio­mark­er break­down and most non-small cell lung can­cer pa­tients are screened.

“Why throw the ba­by out with the bath­wa­ter?” she said. “It’s an im­per­fect bio­mark­er. But it works.”

Gand­hi was well-po­si­tioned to spot PD-L1’s po­ten­tial. She had stud­ied to be a tho­racic on­col­o­gist in the ear­ly 2000s, a time when the field was be­gin­ning to un­der­go a rad­i­cal trans­for­ma­tion. When Gand­hi fin­ished med school, lung can­cer spe­cial­ists were still ex­perts in pal­lia­tive care; a di­ag­no­sis was a death sen­tence, one that chemo could on­ly de­lay.

Be­gin­ning in 2004, though, with the first EGFR in­hibitor, the sto­ry changed. Re­searchers learned to screen for tu­mor pro­teins and de­vel­op drugs to tar­get them, of­ten ex­tend­ing lives by years.

Gand­hi, who as a res­i­dent picked the field be­cause she want­ed to help pa­tients nav­i­gate the tur­moil of a ter­mi­nal di­ag­no­sis, spent her first year af­ter fel­low­ship help­ing run a Phase I tri­al on crizo­tinib. The re­sults on pa­tients with ALK+ tu­mors were so pro­found the FDA gave the drug ac­cel­er­at­ed ap­proval on two Phase Is alone.

“I re­mem­ber hav­ing a pa­tient who had such a great re­sponse, he was a young man, and he end­ed up hav­ing two chil­dren af­ter be­ing di­ag­nosed,” she said. “It was very grat­i­fy­ing to be a part of that.”

That led her to the ear­ly im­munother­a­py tri­als, a place that gave her not on­ly time with pa­tients but a unique im­pact on the sci­ence. You can’t mod­el the im­mune sys­tem well in an­i­mals; the Phase I tri­als tell you every­thing.

Over the next decade, Gand­hi would help lead dozens of tri­als, help­ing de­fine and ex­ca­vate the lim­its of PD-1, show­ing more da­ta on bio­mark­ers, ex­plor­ing the tu­mor en­vi­ron­ment, and pos­ing key ques­tions about com­bi­na­tions with chemother­a­py: Would the old and new in­ter­ven­tions be syn­er­gis­tic, or could the chemo kill the im­mune re­sponse PD-1s were sup­posed to boost? (Syn­er­gis­tic, it seems.)

Then in 2018, a call came from Levi Gar­raway, the Broad In­sti­tute re­searcher re­cent­ly tapped to run Eli Lil­ly’s on­col­o­gy de­part­ment. It would net the slow-foot­ed phar­ma one of their biggest hires, and soon af­ter, one of their loud­er ex­its.

Gand­hi took the job run­ning their im­muno-on­col­o­gy pro­gram to work with Gar­raway, some­one she saw as hav­ing a proven track record and with whom she could make an im­pact. But then Gar­raway left for Roche and Lil­ly hand­ed their can­cer work to the Loxo team, who was known for push­ing low-risk tar­get­ed drugs that could move quick­ly through the clin­ic.

Gand­hi left in June, hop­ing to re­turn to her long­time fo­cus on im­muno-on­col­o­gy. She had land­ed a for­mi­da­ble new op­por­tu­ni­ty. She’s work­ing to build a Phase I trans­la­tion­al cen­ter at Dana-Far­ber. The goal is to cre­ate a place that can test new I/O ideas, putting them in hu­mans be­cause, as she learned ear­ly in her ca­reer, you can’t test them in mice. She’ll work with oth­er in­sti­tu­tions and phar­ma, us­ing knowl­edge she learned from two years in the in­dus­try.

Im­muno-on­col­o­gy has be­come foun­da­tion­al in the last decade, in a way chemo once was, she said. That suc­cess de­pend­ed on fig­ur­ing out on which pa­tients the drugs work best; the next suc­cess­es just might de­pend on the same thing.

“We haven’t seen a lot of change the field, in terms of im­munother­a­peu­tics, since PD-1 in­hibitors,” she said. “And I think part of that is the mod­el of drug de­vel­op­ment that’s be­ing used for a lot of that is the old mod­el of drug de­vel­op­ment.” — Ja­son Mast

From phar­ma en­claves to Chi­na to biotech start­up, Pearl Huang fol­lows her cu­rios­i­ty in­to new ad­ven­tures

Cu­rios­i­ty keeps Pearl Huang go­ing.

It was what drove her to study mol­e­c­u­lar ge­net­ics al­most 35 years ago; make the un­fash­ion­able jump in­to phar­ma right af­ter fin­ish­ing her PhD at Prince­ton (learn­ing first­hand as a moth­er of two what it took to get to tenure and see­ing few suc­cess­ful women on that tract there, to be sure, was al­so a big push fac­tor); move from the front­lines of can­cer drug dis­cov­ery deep­er in­to po­si­tions where she was more in­volved in strat­e­gy and de­ci­sion mak­ing, through a se­ries of fruit­ful jobs at Mer­ck, DuPont, Glax­o­SmithK­line and then Roche; and take up an in­vi­ta­tion from Flag­ship to now lead Cyg­nal, a start­up fo­cused on ex­oneur­al bi­ol­o­gy.

Grow­ing up in the Mid­west, there was al­so a spe­cif­ic place Huang yearned to know more about: Chi­na, her par­ents’ home­land.

They met in the US, and un­til Richard Nixon and Deng Xi­aop­ing opened up re­la­tions around 1977, they had nev­er vis­it­ed each oth­er’s fam­i­lies in Chi­na. “There were let­ters,” she said. “I re­mem­ber the let­ters, but they were few and far be­tween.”

Huang vis­it­ed the coun­try for the first time in 1999, to­geth­er with her five sib­lings and all their fam­i­lies — a three-week tour that she con­sid­ered trans­for­ma­tive.

She knew she would go back. The op­por­tu­ni­ty came in the ear­ly 2000s, when GSK want­ed to ex­plore ex­pand­ing clin­i­cal tri­als to Chi­na for the pipeline she helped build. To­geth­er with Pe­ter Ho, a Chi­nese Amer­i­can col­league on the clin­i­cal phar­ma­col­o­gy team, they made trips to var­i­ous na­tion­al hos­pi­tals in dif­fer­ent provinces.

“And we were both over­whelmed by the num­ber of peo­ple who need­ed ac­cess to can­cer med­i­cines,” she said. “You go to a place like Guangzhou, and you go to the can­cer hos­pi­tal, you can see the pa­tients lin­ing up every morn­ing to get in line to see the doc­tors. And so the need was so pow­er­ful, but al­so we were try­ing to de­vel­op our mol­e­cules that had al­ready been proven to work in the US and in Eu­rope, and it would be years be­fore — at that time — pa­tients in Chi­na would have ac­cess.”

As they talked about ways to bring med­i­cines to Chi­nese pa­tients faster, John Oyler — whom they knew as the chief of a CRO that did a lot of busi­ness with Big Phar­ma — called with the idea of what would be­come BeiGene. At that time, it was a pi­o­neer­ing idea: a biotech com­pa­ny in Chi­na, for Chi­na, by Chi­na.

The year was 2010, and the stars were aligned. Over­seas sci­en­tists were in­creas­ing­ly re­turn­ing, the con­tract busi­ness was boom­ing and Chi­na of­fered the most cap­i­tal-ef­fi­cient way to do drug dis­cov­ery — pick tar­gets, make mol­e­cules — for some­one who knows what they’re do­ing.

With Oyler and an­oth­er co-founder, Xi­aodong Wang, trav­el­ing back and forth, Huang and Ho opened the first of­fice at the Ben Franklin Cen­ter in Philadel­phia, re­cruit­ing and draft­ing busi­ness plans in a tiny room for two months be­fore get­ting on a plane to Bei­jing. There, she filed the first two patents for BeiGene, one for a BRAF in­hibitor and the oth­er for a PARP in­hibitor, and ne­go­ti­at­ed with phar­ma on li­cens­ing deals over 15 months. Af­ter go­ing through sev­er­al places she end­ed up sub­let­ting from the Wash­ing­ton Post an apart­ment in an area by the Sec­ond Ring, where diplo­mats and for­eign ser­vice of­fi­cers lived, rid­ing the sub­way every­where to ex­plore the Chi­nese cap­i­tal.

“I al­ways thought that that re­flect­ed a very ad­ven­tur­ous spir­it in Pearl to do that” in­stead of stay­ing at the ho­tel arranged for ex­pats like them, Ho re­called. “I al­ways thought that that was some­thing that — I don’t think many peo­ple would have cho­sen to do. I cer­tain­ly didn’t.”

But it was clear from the be­gin­ning that a com­pa­ny whose cen­tral plat­form is ge­og­ra­phy wasn’t go­ing to be a long-term fit for Huang, who made the hard de­ci­sion to go back to GSK once BeiGene’s crew grew to a de­cent 110.

Build­ing teams that could stand on their own feet and break through bar­ri­ers, af­ter all, amount to Huang’s great­est ac­com­plish­ments in her own mind.

“I would say for all the teams that I’ve worked on for drug dis­cov­ery, the ones that suc­ceed­ed you hear about, the ones that got closed and didn’t make it you don’t hear about, but those teams worked just as hard and made great de­ci­sions be­cause they had the ex­per­tise and the abil­i­ty to do that,” she said.

Her first time at GSK, for in­stance, she led a col­lab­o­ra­tion with Cy­to­ki­net­ics around ki­nesin spin­dle pro­tein in­hibitors. It was some of the best sci­ence she’d ever par­tic­i­pat­ed in. Yet, the “beau­ti­ful ther­a­peu­tic in­dex” in mice didn’t trans­late in­to the pa­tient pop­u­la­tion they test­ed the drug in. In the end, they killed it.

She cred­its the sci­en­tists on her team who “de­fined the prob­lem in great pre­ci­sion” and gen­er­at­ed the da­ta to an­swer it. To this day, she’d take the Cyg­nal team out to cel­e­brate a de­ci­sion to drop a pro­gram.

So how do you en­cour­age a cul­ture like that on your team? Huang draws lessons from her first stint in the in­dus­try, as a se­nior re­search bio­chemist at Mer­ck Re­search Labs. Be­tween 1990 and 1992, can­cer re­search was a grow­ing area of sci­ence and she was brought in along­side over half a dozen new young PhDs, all an­gling each oth­er to fig­ure out the right tar­get, ex­per­i­ment and mol­e­cule.

“It was a very live­ly, live­ly place, but I was pret­ty qui­et,” she said. “And I re­mem­ber this per­son who be­came my men­tor, sit­ting in a room where every­body was ar­gu­ing about this, that and the oth­er, and this per­son said, ‘I want to know what Pearl thinks.’”

That per­son was Allen Oliff, who now heads GSK’s Cen­ter of Ex­cel­lence for Drug Dis­cov­ery. The sea­soned drug hunter calls Huang one of the best sci­en­tists he’s worked with in acad­e­mia, gov­ern­ment or phar­ma in­dus­try po­si­tions, im­pressed by the cre­ative so­lu­tions she brings to pre­clin­i­cal eval­u­a­tion of drugs.

“It was clear that she was a very bright and care­ful thinker, un­der­stood the bi­ol­o­gy and the mol­e­c­u­lar ge­net­ics of the pro­grams she was at,” he said, not­ing that he re­mem­bered the ex­act meet­ing be­cause he re­called try­ing to pull ideas out of Huang mul­ti­ple times. “Many peo­ple, not just me, many of the oth­er peo­ple in the lab go to Pearl and ask her for ad­vice.”

It is a prac­tice that she now em­ploys as a leader. En­gag­ing peo­ple like her­self — a dou­ble mi­nor­i­ty as an Asian woman — and get­ting rid of rules on how some­one should be­have, she be­lieves, will be the key to com­bat­ing all shades of gen­der bias.

“The clos­er you get to par­i­ty, the clos­er you get to these prob­lems go­ing away,” she said. — Am­ber Tong

Al­ways look­ing for the prob­lems, Kathrin Jansen found a his­toric Covid-19 so­lu­tion

Be­fore Kathrin Jansen cel­e­brat­ed the his­tor­i­cal mo­ment when her team at Pfiz­er, to­geth­er with part­ners at BioN­Tech, de­liv­ered the good news that their Covid-19 vac­cine may in­deed help put an end to the pan­dem­ic, she re­mem­bered the sirens.

“I’m lo­cat­ed in New York, be­ing in the hot zone so to speak, when the virus cut loose in the Unit­ed States,” she re­called around a month be­fore the ground­break­ing read­out. “We’re close to sev­er­al hos­pi­tals where we live. Hear­ing the sirens all day, all night, and you know ex­act­ly what that meant, right? It was peo­ple, you know, fight­ing for their lives, get­ting in­to the hos­pi­tal to be treat­ed for Covid-19. See­ing the re­frig­er­a­tion trucks, where in­di­vid­u­als that suc­cumbed to Covid-19 pile up, in the streets in front of the hos­pi­tals.”

It all put the sea­soned sci­en­tist right in the midst of a com­plete­ly dif­fer­ent di­men­sion than what she’s weath­ered through close to three decades in the in­dus­try. All bets are off; how do you ap­proach it dif­fer­ent­ly than nor­mal cir­cum­stances, yet still main­tain the stan­dards re­quired to de­vel­op a safe and ef­fec­tive in­oc­u­la­tion?

How to do things right un­der nor­mal cir­cum­stances, to be sure, is some­thing Jansen knows well. With three mar­ket­ed vac­cines un­der her belt — Gar­dasil for pre­ven­tion of can­cers and oth­er dis­eases caused by HPV; Tru­men­ba for menin­gi­tis B; and the pneu­mo­coc­cal con­ju­gate vac­cine Pre­vnar 13 — and 11 years of ex­pe­ri­ence as Pfiz­er’s head of vac­cine R&D, she was over­see­ing a pipeline of eight ex­per­i­men­tal im­mu­niza­tions against both bac­te­r­i­al and vi­ral pathogens be­fore switch­ing all her at­ten­tion to the mys­te­ri­ous coro­n­avirus that dev­as­tat­ed first Chi­na, then Italy, then even­tu­al­ly the US.

But her ca­reer al­most didn’t turn out that way.

Jansen ar­rived at her first vac­cine job, in Mer­ck’s vac­cine group, at a time the phar­ma gi­ant was think­ing about get­ting out of vac­cines. The unit was look­ing to rein­vent it­self, and Jansen was hired to work on a non-vac­cine re­lat­ed top­ic.

Af­ter com­plet­ing a post­doc at Cor­nell on a Hum­boldt Fel­low­ship fo­cused on yeast ge­net­ics, she had spent sev­er­al years ex­press­ing in a re­com­bi­nant sys­tem a re­cep­tor be­lieved to be a drug tar­get to pre­vent al­ler­gies and asth­ma with col­leagues at Glaxo In­sti­tute for Mol­e­c­u­lar Bi­ol­o­gy in Gene­va. The pro­gram didn’t end up lead­ing to a drug — a les­son she had by then learned about the in­dus­try she’d al­ways dreamed of join­ing. But it equipped her for the new task.

Soon enough, though, Jansen and her Mer­ck col­leagues re­al­ized that the da­ta they’d been hand­ed were what she called “ar­ti­facts.” They couldn’t re­pro­duce a sin­gle one of the re­sults.

At around the same time, Mer­ck was bring­ing in a new tech­nol­o­gy dis­cov­ered at the Uni­ver­si­ty of Queens­land out in Aus­tralia, sug­gest­ing that the cap­sid pro­teins de­rived from the out­er coat of the hu­man pa­pil­lo­mavirus would in­duce an­ti­bod­ies to the virus.

“I told my boss, ‘This is not work­ing, this makes no sense,’” Jansen said and, be­cause she had an idea of how to make a vac­cine us­ing the in­fra­struc­ture Mer­ck al­ready had, added: “I want to work on the hu­man pa­pil­lo­mavirus.”

It was an in­stru­men­tal vac­cine for hu­man­i­ty as well as for her who, as a first-time pro­gram lead, ex­pe­ri­enced every­thing and learned what it means to cre­ate a con­struct, de­vel­op process­es, in­ter­act with reg­u­la­tors, and think from a com­mer­cial stand­point.

Two habits formed that would stick even af­ter she left Mer­ck, through short stints at the ill-fat­ed biotech Vax­Gen and then pre-merg­er Wyeth: Nev­er make as­sump­tions, and fo­cus on where prob­lems could lurk.

“I al­ways say, I’m al­ways look­ing for the prob­lems,” she said. “The so­lu­tions will come — I’m sure about this. I’m wor­ried about the prob­lems, and where do we run in­to prob­lems.”

Some of those prob­lems they were able to avoid in de­vel­op­ing the BNT162 pro­gram, be­cause Jansen’s team was told by Pfiz­er CEO Al­bert Bourla, whom she’d meet twice a week, that they could get what­ev­er they need­ed. Oth­ers were ad­dressed quick­ly once they were raised. There were no com­pet­ing pro­grams, no need to scram­ble for re­sources, noth­ing stand­ing in the way.

“The on­ly con­straint is time,” she said, “and time you don’t have. So you be­come very cre­ative.”

They made no as­sump­tions pre­clin­i­cal­ly, tak­ing not one but four slight­ly dif­fer­ent mR­NA con­structs in­to the clin­ic — which turned out to be a good de­ci­sion be­cause “we would’ve been so wrong” — col­laps­ing a se­quen­tial work­flow in­to a par­al­lel one, and mak­ing de­ci­sions on the fly. The last da­ta from the Phase II study came in on Ju­ly 24; the team had a meet­ing the next day to de­bate on the two fron­trun­ners that had the best da­ta; and they locked in the b2 can­di­date. The Phase III por­tion of the study start­ed on Ju­ly 27.

Jansen, who was born in East Ger­many and ed­u­cat­ed in West Ger­many be­fore set­tling in the US with her Amer­i­can hus­band, cred­its com­pa­ny cul­ture as a big dri­ver in all this — both at Pfiz­er and BioN­Tech.

As a woman in bio­phar­ma, sup­port­ive en­vi­ron­ments mat­ter. She likes to tell the sto­ry from her PhD days when her mi­cro­bi­ol­o­gy class vis­it­ed a com­pa­ny in Ger­many. The man show­ing them around saw that half of the group were women and asked: “So you want your PhD, why do you both­er? You know you’re not go­ing to work any­way.”

De­spite that jaw-drop­ping ex­pe­ri­ence and the dearth of se­nior women lead­ers in phar­ma at the time, she not­ed that the US felt dif­fer­ent from the get-go. And even if you can’t change a dis­crim­i­na­to­ry en­vi­ron­ment, Jansen is of the be­lief that you can change your own.

“I think one al­ways has to make de­ci­sions of where — where do you want to be,” she said.

Right now, right here at the cross­ways of his­to­ry is ex­act­ly where she wants to be. — Am­ber Tong

The co-in­ven­tor of mod­i­fied mR­NA, Katal­in Karikó is fi­nal­ly get­ting her mo­ment

Katal­in Karikó re­mem­bers sit­ting in her fa­ther’s butch­er shop as a lit­tle girl, un­afraid of the blood and en­trails.

She’d sit in the small store in Hun­gary, watch­ing an­i­mal af­ter an­i­mal get chopped up and sold to cus­tomers. Her fa­ther would have to kill the an­i­mals as well, and she’d sit and watch while her moth­er and sis­ter couldn’t bear to even lis­ten to the nois­es. Just sit­ting, just cu­ri­ous about the in­ner work­ings of liv­ing be­ings.

It’s that in­nate cu­rios­i­ty that’s dri­ven Karikó her sci­en­tif­ic en­tire ca­reer, a ca­reer that’s led to help­ing solve the rid­dle of one of the tech­nolo­gies at the fore­front of the Covid-19 pan­dem­ic fight: mod­i­fied mes­sen­ger RNA.

“I wrote a lit­tle pre­view called ‘Out of the Shad­ow, In­to the Spot­light,’ this was about the mes­sen­ger RNA be­cause it was al­ways in the shad­ow and it’s fi­nal­ly in the spot­light,” Karikó said. “Not many sci­en­tists can say that I was think­ing of some­thing, proved it, pub­lished it, had a patent on it, every­thing and then it end­ed up fi­nal­ly in the high­ly-seen prod­uct.”

The road to the dis­cov­ery that’s come to de­fine her re­search was not al­ways smooth. Karikó first came to the Unit­ed States in 1985, tak­ing a job at the Uni­ver­si­ty of Penn­syl­va­nia. She had been fo­cus­ing on mR­NA tech­nol­o­gy even then, but ran in­to head­winds as ear­ly syn­thet­ic RNA proved high­ly sus­cep­ti­ble to vi­o­lent im­mune re­spons­es.

De­spite sub­mit­ting her first mR­NA ther­a­py ap­pli­ca­tion in 1989, she couldn’t get any grant fund­ing to de­vel­op it. Af­ter a few years, Karikó’s boss­es at UPenn de­mot­ed her.

But Karikó kept plug­ging away at the re­search, con­vinced of the tech­nol­o­gy’s po­ten­tial. Even­tu­al­ly, she teamed up with one of her col­leagues at Penn, Drew Weiss­man, and came up with a so­lu­tion to the im­mune re­sponse prob­lem — by mod­i­fy­ing one of the nu­cle­o­sides that make up the RNA and us­ing that ver­sion of the ther­a­py in­stead, Karikó and Weiss­man cre­at­ed a way to evade the body’s nat­ur­al de­fens­es.

In 2006, she and Weiss­man used the ba­sis of that dis­cov­ery to found a com­pa­ny called RNARx, where Karikó served as CEO. By the time their patent for the tech­nol­o­gy was ac­cept­ed in 2012, how­ev­er, Penn sub­li­censed it out to an­oth­er com­pa­ny. A few months lat­er, Mod­er­na — which at the time was still a nascent Flag­ship biotech — signed a $240 mil­lion deal with As­traZeneca to de­vel­op a VEGF mR­NA.

Those blows es­sen­tial­ly forced Karikó’s out­fit to close up shop, and Kariko her­self de­cid­ed she’d had enough of acad­e­mia. She took a role at BioN­Tech soon af­ter as se­nior vice pres­i­dent.

Now that mR­NA vac­cines are two of the lead­ing can­di­dates in the race for a Covid-19 cure at BioN­Tech/Pfiz­er and Mod­er­na, whose tech­nol­o­gy is based on her old patent, Karikó is hap­py that her re­search is a part of the an­swer. But she al­so feels val­i­dat­ed af­ter so many pro­fes­sion­al set­backs.

“I wish to tell some of those peo­ple who put me down and ridiculed me and what­not to see that, ‘You see?’” Karikó said. “But that’s OK. I am hap­py that the two lead­ing mR­NA vac­cines, Mod­er­na and BioN­Tech with Pfiz­er prod­ucts, both of them are in­clud­ing some­thing that I con­tributed. Even oth­er peo­ple who will not know be­cause Mod­er­na usu­al­ly says that they dis­cov­ered every­thing, but they did pay for that patent and sub­li­censed it from Penn.”

The tech­nol­o­gy that Karikó co-in­vent­ed could end up sav­ing thou­sands of lives and has sig­nif­i­cant ram­i­fi­ca­tions in ar­eas out­side Covid-19. Karikó said she orig­i­nal­ly be­gan look­ing in­to the re­search aim­ing to de­vel­op an HIV vac­cine, but mR­NA-based ther­a­pies have been pop­ping up in oth­er rare dis­eases and cer­tain can­cers.

Karikó pre­dicts that in the near fu­ture, it’s very like­ly mR­NA will suc­cess­ful­ly be ap­plied to oth­er in­fec­tious dis­eases. It’s a bit far­ther off in can­cer, as the sci­ence needs to be im­proved and un­der­stood bet­ter, she says.

One pos­si­ble mR­NA-based treat­ment that could come far in the fu­ture is a ther­a­peu­tic spray or cream, sim­i­lar to Neosporin, that peo­ple can keep in their freez­ers at home and ap­ply to open wounds. In­di­vid­u­als would use the spray as need­ed, and it would ac­cel­er­ate col­la­gen re­pair and lessen their pain.

“Many times be­fore, the mR­NA has been re­ject­ed be­cause it is tem­po­rary, be­cause it de­grades. But it is a very good thing that it de­grades,” Karikó said. “It can be ap­plied like med­i­cine. If you need more, then you take more. But it de­grades, and it will not have a con­tin­u­ous ef­fect.”

Re­gard­less of what comes next for mR­NA, it’s al­ready had a huge im­pact on the biotech and phar­ma­ceu­ti­cal world, par­tic­u­lar­ly in 2020. No­tably, Mod­er­na’s own co-founder Der­rick Rossi has called for Karikó and Weiss­man to win the No­bel Prize in Chem­istry for their mR­NA re­search.

And for the next gen­er­a­tion of women in biotech, Karikó wants them to know that you don’t have to choose be­tween a ca­reer and your fam­i­ly. Karikó re­mem­bers sev­er­al in­stances where she’d be asked who her boss was, as many sim­ply as­sumed that the “woman with the ac­cent” had to re­port to some­body else. But the times are chang­ing.

“In Ger­many, I gave sev­er­al lec­tures for stu­dents, and some of them ap­proached me that they were told that if they want to have a ca­reer then for­get about fam­i­ly. I al­so say, ‘You don’t have to do that,’” Karikó said. “My daugh­ter … she al­ways said that our work eth­ic was help­ing her, so my mes­sage was for the younger gen­er­a­tion that they don’t have to give up fam­i­ly life, es­pe­cial­ly when things are hard you need sup­port, moral sup­port from your fam­i­ly, af­ter an­oth­er grant re­jec­tion.” — Max Gel­man

In­spired by her grand­fa­ther, Sara Kenkare-Mi­tra finds her high­er call­ing in med­i­cine

Sara Kenkare-Mi­tra grew up lov­ing the sto­ries her grand­fa­ther would tell her of his time as a physi­cian in In­dia.

Work­ing in a very rur­al part of the coun­try, he would of­ten end up as the on­ly physi­cian pa­tients could turn to in a cri­sis. Kenkare-Mi­tra re­called a time where he spoke of be­ing awak­ened in the mid­dle of the night to help peo­ple on a ship who had nowhere else to turn, giv­en that he was the on­ly doc­tor around for miles.

That pas­sion for med­i­cine was ul­ti­mate­ly passed down to Kenkare-Mi­tra, now a 22-year vet­er­an at Genen­tech where she serves as se­nior vice pres­i­dent of de­vel­op­ment sci­ences.

“He be­came the physi­cian of the com­mon peo­ple,” Kenkare-Mi­tra told End­points News. “The sto­ries of him im­pact­ing peo­ple’s lives through the skillsets he had got­ten was just amaz­ing and thrilling to me. We used to talk about med­i­cines, I grew a deep in­ter­est in med­i­cine, sort of ask­ing just what med­i­cines you take and when, even as a young girl, I re­mem­ber that that was some­thing I had a lot of ques­tions around.”

Be­ing ex­posed to her grand­fa­ther’s tales set Kenkare-Mi­tra on the path where she finds her­self to­day. She al­ways knew she want­ed a ca­reer in a sci­ence-re­lat­ed field, but veered to­ward drug R&D rather than work di­rect­ly with pa­tients in or­der to get in­volved in the re­search. When she was first look­ing to go to grad school, phar­ma­ceu­ti­cals was still a nascent field in In­dia with most pro­grams fo­cus­ing on phar­ma tech and man­u­fac­tur­ing.

Kenkare-Mi­tra soon found her­self at the Uni­ver­si­ty of Texas at Austin, but didn’t feel like that was a great match. Com­ing to the US was no small task ei­ther in the era be­fore cell phones and the in­ter­net, Kenkare-Mi­tra says, so ad­just­ing on the fly proved to be an­oth­er chal­lenge. Even­tu­al­ly, she set­tled in­to a PhD pro­gram at UCSF where Leslie Benet, the “Fa­ther of Phar­ma­co­ki­net­ics,” was one of her pro­fes­sors.

Re­al­iz­ing that most of that work was equiv­a­lent to pre­clin­i­cal de­vel­op­ment and that she like­ly wouldn’t see the ef­fect on pa­tients for years, Kenkare-Mi­tra still felt some­thing was miss­ing. Un­til the HER2-pos­i­tive breast can­cer ther­a­py Her­ceptin made head­lines in the late 1990s.

“[Her­ceptin] told a sto­ry that, for me, as a phar­ma­ceu­ti­cal sci­en­tist, some­one that’s al­ways been in­ter­est­ed in med­i­cine, I thought, ‘Wow, that’s in­no­v­a­tive,’” Kenkare-Mi­tra said. “We were all used to drugs that aren’t re­al­ly tar­get­ed, they aren’t re­al­ly per­son­al­ized, but now here’s a drug that’s to be giv­en to women that have a spe­cif­ic type of tu­mor that shows the HER2 pos­i­tiv­i­ty, and it ac­tu­al­ly works. To me, it was a big leap in med­i­cine.”

It was around that time when some­one from Genen­tech reached out to her with a job of­fer. Kenkare-Mi­tra ac­cept­ed and start­ed as a sci­en­tist, but worked in sev­er­al dif­fer­ent roles, in­clud­ing lead­er­ship po­si­tions. She’s worked in the same or­ga­ni­za­tion with­in Genen­tech for most of her time there, and said her teams have brought 15 drugs to mar­ket over her two-plus decades.

The theme that’s strung her ca­reer to­geth­er has been trans­la­tion, or en­sur­ing that dur­ing the R&D process, the ex­per­i­men­tal drugs will con­tin­ue to pro­duce mean­ing­ful re­sults as they ad­vance through the clin­ic. Kenkare-Mi­tra says she’s al­ways been able to re­cruit the right tal­ent from many dis­ci­plines for her projects, cred­it­ing her post­doc ex­pe­ri­ence where she was one of on­ly a few PhDs in a pro­gram nor­mal­ly re­served for clin­i­cians.

Kenkare-Mi­tra said one of the biggest chal­lenges in her job is when, af­ter re­al­iz­ing that a cer­tain pro­gram may not trans­late as well as orig­i­nal­ly hoped, she has to make a de­ci­sion on whether or not to punt.

“That trans­la­tion is not triv­ial. It’s re­al­ly un­der­stand­ing the sci­ence and da­ta enough to say, ‘Ah, I think this has got legs and should move for­ward,’” she said. “And then al­so learn­ing from the for­ward and bring­ing it back, re­verse-trans­lat­ing that. Say­ing, ‘These are the lessons we’ve learned, and this is how it ap­plies to the world.’

“The best thing that you can do is if a par­tic­u­lar med­i­cine is not go­ing to move for­ward, you need to make that de­ci­sion and stop it, with­out wast­ing more mon­ey and re­search, and then find­ing out it doesn’t work,” she added.

Now, though, Kenkare-Mi­tra says she’s found her high­er pur­pose in life help­ing pa­tients, though in a dif­fer­ent way than her grand­fa­ther. It took her a while to get to this stage, as ear­li­er in her ca­reer she felt some­what over­whelmed and wasn’t think­ing too much about the fu­ture.

And if there’s one piece of ad­vice she could give to up-and-com­ing women in the in­dus­try, it’s to make sure one keeps the big pic­ture in mind.

“Sci­ence, as well as work in this in­dus­try, re­al­ly re­quires long-term think­ing and re­quires a lev­el of re­silience,” Kenkare-Mi­tra said. “You have to be in it for the long run, but the re­wards in terms of sat­is­fac­tion, you’ll get in life, are so very high. But you have to re­al­ize that you won’t have re­wards to­mor­row.” — Max Gel­man

Af­ter climb­ing the ranks at Genen­tech, Lynne Krum­men digs in­to im­munol­o­gy at Vir 

Lynne Krum­men didn’t know a whole lot about drug de­vel­op­ment when she start­ed at Genen­tech in 1990 — but she re­mem­bers the mo­ment she was hooked.

It was in a side­bar meet­ing, af­ter a guest sci­en­tist spoke about work re­lat­ed to the cloning of the LH re­cep­tor. Ideas were fly­ing around the room: “We could start PK stud­ies,” some cowork­ers said, while oth­ers brain­stormed pos­si­ble in­di­ca­tions.

“Wow, this is where this hap­pens,” Krum­men thought to her­self. “I just got ex­posed to this whole world of: How do you take an idea in re­search and turn it in­to a med­i­cine that helps peo­ple?”

When Krum­men grad­u­at­ed with her PhD in en­docrinol­o­gy from the Uni­ver­si­ty of Cincin­nati Col­lege of Med­i­cine in 1987, the biotech field was still warm­ing up. But she knew of one com­pa­ny that was a “mav­er­ick,” and that was Genen­tech. They were work­ing on a growth hor­mone at the time, which is an en­docrine mol­e­cule, and Krum­men was set on join­ing them.

“I sort of tried every av­enue I could to get a job at that com­pa­ny,” she said.

She land­ed a post­doc spot in 1990, then tran­si­tioned in­to sci­en­tist and man­age­ment roles, work­ing her way up to VP of tech­ni­cal de­vel­op­ment be­fore leav­ing for her cur­rent po­si­tion: se­nior VP of reg­u­la­to­ry and de­vel­op­ment pro­gram lead­er­ship and man­age­ment at Vir Biotech­nol­o­gy.

Krum­men’s ca­reer with Genen­tech spanned 27 years. She was there for the Roche merg­er, and to work on block­buster Avastin, which was first ap­proved in 2004 for colon can­cer, and has since snagged OKs in ad­vanced lung, kid­ney and brain can­cers. While de­vel­op­ing the drug, Krum­men and her cowork­ers lost sev­er­al fam­i­ly mem­bers to can­cer.

“It was just very im­por­tant that we could be do­ing work that was use­ful in some­thing that was … af­fect­ing us so per­son­al­ly,” she said.

She al­so got the chance to learn from men­tors, some of whom “prob­a­bly nev­er knew they were,” like on­col­o­gy ex­pert Sue Desmond-Hell­mann. The for­mer Bill & Melin­da Gates Foun­da­tion CEO worked at Genen­tech for 14 years, end­ing as pres­i­dent of prod­uct de­vel­op­ment.

“I al­ways felt smarter af­ter hav­ing been in a meet­ing that she was chair­ing,” Krum­men said of Desmond-Hell­mann. “She made you feel like you were telling her some­thing that was im­por­tant when in fact she knew the an­swer all along.”

Krum­men was for­tu­nate. As she climbed the lad­der at Genen­tech, she says there were al­ways women in lead­er­ship roles at se­nior lev­els, though they were in the mi­nor­i­ty. And she rec­og­nizes that is a unique ex­pe­ri­ence.

“Even though I think there have been pur­pose­ful ef­forts to try to bal­ance re­cruit­ing pools and oth­er kinds of things … It still comes down to peo­ple mak­ing choic­es at the end of the day. And you can in­ter­view a lot of women, but the choice to hire that woman is one that I think we have to hold lead­er­ship ac­count­able for,” she said.

Krum­men picked up var­i­ous ti­tles at Genen­tech: se­nior di­rec­tor of process de­vel­op­ment, VP and glob­al head of bi­o­log­ics reg­u­la­to­ry CMC, and VP of tech­ni­cal de­vel­op­ment. But one of the great­est chal­lenges was fig­ur­ing out how to add mom to the list, she says.

Genen­tech was one of the few com­pa­nies at the time with its own day­care just a cou­ple blocks from the of­fice, which Krum­men said was help­ful be­fore her two kids, now 23 and 26 years old, were school age. Be­cause the day­care was so close, she could swing by for lunch, or the Hal­loween pa­rade. Once her kids start­ed school, the rou­tine got more com­pli­cat­ed. Krum­men and her hus­band split up the af­ter-school games and bal­let, and they leaned on oth­er par­ents and a nan­ny when pos­si­ble.

“There’s on­ly so much you can do,” she said, adding that there’s no shame in out­sourc­ing. “Fig­ure out where you need the help and don’t be afraid to ask for the help so that you can fo­cus on the things that are most im­por­tant.”

About three years ago, Krum­men jumped to Vir. There’s a much deep­er un­der­stand­ing of the im­mune sys­tem now than there was when she stud­ied im­munol­o­gy in school, and she want­ed to dig in.

“The tools now are just as­tound­ing and I think they’re go­ing to con­tin­ue to be­come more and more so­phis­ti­cat­ed over the next sev­er­al years, and there will be new types of modal­i­ties that we don’t even have avail­able to us to­day,” she said.

Vir cur­rent­ly has sev­er­al can­di­dates in the clin­ic, in­clud­ing two Phase II Al­ny­lam-part­nered drugs for he­pati­tis B, and a GSK-part­nered ear­ly Covid-19 treat­ment in Phase III.

“I would like Vir to re­al­ize its mis­sion state­ment of … a world with­out in­fec­tious dis­ease,” Krum­men said. “It’s so im­por­tant … we couldn’t have lived in a time when that is not clear­er how im­por­tant the mis­sion of the com­pa­ny is.” — Nicole De­Feud­is

Re­gen­eron’s mas­ter plan­ner finds pow­er in lis­ten­ing and be­ing the most in­flu­en­tial voice in the room

One of the biggest chal­lenges Leah Lip­sich has faced in her ca­reer dates back to her lengthy tenure at Boehringer In­gel­heim. She had just be­come the di­rec­tor of phar­ma­ceu­tics, a de­part­ment tasked with for­mu­lat­ing drugs. But the heads of R&D and com­mer­cial came to her with a com­plete­ly dif­fer­ent prob­lem: The Ger­man phar­ma had just got­ten an asth­ma in­haler ap­proved by the FDA, yet they were hav­ing trou­ble man­u­fac­tur­ing it to spec­i­fi­ca­tions re­quired for com­mer­cial launch.

The men, Stephen Carter and Hans Leuchs, want­ed Lip­sich to han­dle it. Fast.

“This was so far out of my area of ex­per­tise, I knew noth­ing about it,” she said. “I pret­ty much said to them, ‘Are you kid­ding me? I know noth­ing about this. How can I pos­si­bly do this?’ And they said, ‘You’ll sur­round your­self with the peo­ple who do know things tech­ni­cal­ly and you’ll use your lead­er­ship skills to solve the prob­lem.’”

The daunt­ing task, in a way, would serve as a test run for many of the projects Lip­sich has since spear­head­ed. It was a turn­ing point where she re­al­ized that bring­ing the best out of sub­ject mat­ter ex­perts, rather than know­ing every­thing, is the key to get­ting the job done.

That same les­son was true through stints at Pur­due, UCB and Elan — and es­pe­cial­ly so now. As the VP of strate­gic pro­gram di­rec­tion re­spon­si­ble for in­fec­tious dis­eases, she is man­ag­ing the same team of sci­en­tists, ops and man­u­fac­tur­ing ex­perts that helped de­vel­op an ef­fec­tive Ebo­la drug in pur­suit of an an­ti­body cock­tail that works against Covid-19.

Be­fore she em­barked on a ca­reer in drug de­vel­op­ment, Lip­sich said, a bi­ol­o­gy teacher in high school had sparked her in­ter­est in sci­ence, so much so that she had ac­tu­al­ly want­ed to teach. But her fa­ther — an aca­d­e­m­ic him­self — en­cour­aged her to first get a de­gree.

A love of lab­o­ra­to­ry sci­ence soon turned in­to a pas­sion for clin­i­cal sci­ence. Her first job as a fresh­ly mint­ed PhD, at a Seat­tle-based biotech named Ge­net­ic Sys­tems, tech­ni­cal­ly last­ed one day be­fore Bris­tol My­ers Squibb ac­quired it. So Lip­sich was im­me­di­ate­ly plugged in­to Big Phar­ma, work­ing on none oth­er than mon­o­clon­al an­ti­bod­ies for in­fec­tious dis­eases. For bac­te­ria, no less.

Hav­ing stud­ied an­ti­bod­ies on and off through the years, it was easy to ap­pre­ci­ate Re­gen­eron’s in­vest­ment in­to tech­nolo­gies that could re­li­ably iden­ti­fy and man­u­fac­ture ful­ly hu­man an­ti­bod­ies, elim­i­nat­ing all the road­blocks that marked the be­gin­ning of her ca­reer.

Lip­sich took pride in the com­pa­ny’s de­ci­sion to ap­ply that plat­form on Ebo­la, which paid off last year when the three-drug cock­tail was shown to work in a tri­al across West Africa. Yet she and the team had just a few months to sa­vor and cel­e­brate the mo­ment be­fore she found her­self talk­ing to Chris­tos Kyrat­sous, Re­gen­eron’s chief sci­en­tist in in­fec­tious dis­eases, fright­ened by re­ports about nurs­ing homes in Seat­tle be­ing over­run with the mys­te­ri­ous new virus.

They were able to best their own record and go a lit­tle bit faster this time around — an im­prove­ment Lip­sich chalks up to bet­ter knowl­edge of the tech­nol­o­gy and an abil­i­ty to in­ter­pret the da­ta faster. Col­lab­o­ra­tions with re­searchers as phys­i­cal­ly far as Sin­ga­pore al­so helped, as did the gov­ern­ment step­ping in to re­move ob­sta­cles.

Kyrat­sous might add a third fac­tor: Lip­sich’s abil­i­ty to pull all the strings to­geth­er, pri­or­i­tize them prop­er­ly for pre­sen­ta­tion to se­nior man­age­ment, gov­ern­ment of­fi­cials or reg­u­la­tors, while high­light­ing the in­di­vid­ual com­po­nents as need­ed.

“It’s not just that she’s look­ing at a task on hand, but she’s — it’s the task on hand that is be­ing done by a per­son,” he told End­points News just af­ter re­spond­ing to an email from Lip­sich. “So she un­der­stands how this per­son op­er­ates, she un­der­stands the spe­cif­ic. Every­body’s dif­fer­ent, every­body’s work­ing dif­fer­ent times of day, they have com­plete­ly dif­fer­ent sched­ules, they have dif­fer­ent styles of work­ing, and she is able ba­si­cal­ly to per­son­al­ize the pro­gram, ba­si­cal­ly, for each in­di­vid­ual team.”

Hav­ing en­joyed life­long re­la­tion­ships with men­tors, she finds it equal­ly sat­is­fy­ing to ad­vise and coach young peo­ple work­ing un­der her, many of whom are women.

“I still think women strug­gle be­ing heard,” she said. “We’re not com­fort­able be­ing the loud­est voice in the room. And I don’t know that that will ever change. But I do think that there are ways to be the most pow­er­ful voice, or the most in­flu­en­tial voice, in the room.”

Women, in her ob­ser­va­tion, gen­er­al­ly feel un­com­fort­able talk­ing about their salary. She would know: Ear­ly in her ca­reer at Bris­tol My­ers, she hadn’t re­al­ized that she wasn’t com­pen­sat­ed as well as her male coun­ter­parts un­til her boss, a man named Philip Sauer, point­ed it out to her and took steps to change it. The sense of recog­ni­tion even­tu­al­ly em­bold­ened her to ask for a pro­mo­tion in a lat­er role, even though she has still nev­er ne­go­ti­at­ed her salary in any of her roles — some­thing she now press­es her mentees to al­ways do.

As the Covid-19 sched­ule push­es her in­to 10, 12 or even 16-hour days, the self-de­scribed avid veg­etable gar­den­er makes sure to carve out 10 to 15 min­utes a day just for her­self, go­ing out to see what she can har­vest in her gar­den out on Shel­ter Is­land, NY — and just breathe.

“I’m sure that women take care of oth­ers be­fore they take care of them­selves,” she said. “But it’s re­al­ly im­por­tant, even if it’s just 5 min­utes, take 5 min­utes, walk around the block, breathe. And take care of your­self.” — Am­ber Tong

Stacey Ma fol­lows great-grand­fa­ther’s foot­steps to a top seat at start­up Sana

Stacey Ma’s en­tire ca­reer traces back to Shang­hai in the 1980s, her great-grand­fa­ther and a wood­en Chi­na cab­i­net.

While liv­ing with her great-grand­par­ents, 10-year-old Ma was cap­ti­vat­ed by Yi-Fan Jiang’s cab­i­net of med­i­cines. The for­mer med­ical school dean was around 90 years old at the time and in a wheel­chair — but that didn’t stop him from tin­ker­ing with his oint­ments and oth­er con­coc­tions.

Decades ear­li­er, around 1915, Jiang was on his way to study med­i­cine in Ger­many when he got side­tracked in the US. He stayed for 17 years, study­ing at the Uni­ver­si­ty of Min­neso­ta and the Uni­ver­si­ty of Chica­go be­fore re­turn­ing to Chi­na to teach. He was the dean of two med­ical schools. He spoke nine lan­guages. And even in his 90s, he was cu­ri­ous, his head of­ten buried in a news­pa­per or mag­a­zine.

He taught Ma to dream big. And she dreamed of be­ing like him.

So in 1987, she, too, packed her bags for the Uni­ver­si­ty of Min­neso­ta. “The orig­i­nal in­tent was to ac­tu­al­ly prac­tice med­i­cine, but some­how I end­ed up be­ing an en­gi­neer … mak­ing med­i­cine,” Ma said.

The tra­jec­to­ry change was prac­ti­cal: Go­ing to med­ical school as an in­ter­na­tion­al stu­dent was ex­pen­sive, and Min­neso­ta had a good chem­i­cal en­gi­neer­ing pro­gram.

Ma grad­u­at­ed in 1991, then con­tin­ued on to Yale and even­tu­al­ly Genen­tech, where she stayed for 23 years. She ar­rived just as the com­pa­ny was prepar­ing its fil­ing for Rit­ux­an, the mon­o­clon­al an­ti­body first ap­proved in 1997 for Hodgkin’s lym­phoma. Last year, the drug raked in $6.75 bil­lion in net sales.

“I kind of rode the wave of mon­o­clon­al an­ti­bod­ies,” Ma said.

She served var­i­ous roles in tech de­vel­op­ment, qual­i­ty and sup­ply chain, and was named glob­al head of phar­ma­ceu­ti­cal tech­ni­cal in­no­va­tion and man­u­fac­tur­ing sci­ences and tech­nol­o­gy in 2018.

“It’s been a ca­reer that I def­i­nite­ly did not imag­ine, and I have been re­al­ly en­joy­ing it,” she said.

To­ward the end of her time at Genen­tech, Ma was in­trigued by the prospects of cell and gene ther­a­py. “It kind of … re­mind­ed me of the ear­ly days of mon­o­clon­al an­ti­bod­ies,” she said.

That cu­rios­i­ty brought her to Sana Biotech­nol­o­gy, a biotech found­ed in 2018 on a mis­sion to ma­nip­u­late or con­trol genes to re­place or re­pair any cell in the body. She was tapped last year as ex­ec­u­tive VP and head of tech­ni­cal op­er­a­tions, and has since watched the start­up ex­pand to more than 250 em­ploy­ees. By June, a syn­di­cate had raised more than $700 mil­lion to sup­port the fledg­ling biotech that CEO Steve Harr said is still sev­er­al years away from the clin­ic.

“One of the key rea­sons for me join­ing Sana is be­cause I tru­ly be­lieve cell and gene ther­a­py is the next kind of ex­cite­ment in terms of re­al­ly be­ing able to bring some more in­no­v­a­tive so­lu­tions to these pa­tients,” Ma said.

One thing Ma has no­ticed on her climb to the top? The high­er she got, the few­er women there were.

It’s like a pyra­mid, she said. At the en­try lev­el, right out of school, Ma saw a strong base of women en­ter­ing the field. But as she moved up, she wit­nessed a “very clear, oh-so-ob­vi­ous shift”: Most of her man­agers were men. Few women in the in­dus­try were mak­ing it to the ex­ec­u­tive lev­el.

“I see that sort of glass ceil­ing for fe­males … we still have a long ways to go,” Ma said.

The VP en­cour­ages women to pull each oth­er up. “How do we al­so net­work more and … cre­ate that strong sup­port sys­tem for each oth­er?” she asked. “We don’t have to be like men and lead like men … lead­ing the way we are can be just as ef­fec­tive.”

When faced with a chal­lenge, Ma said she thinks back to that old, wood­en Chi­na cab­i­net in her great-grand­fa­ther’s house. “It’s the yin and yang right?” She re­minds her­self of an­oth­er one of Jiang’s lessons: With every chal­lenge, there is op­por­tu­ni­ty.

“I do see the in­dus­try has evolved,” Ma said. “The fact that I’m here talk­ing to you I think is one (ex­am­ple). So I think there’s def­i­nite­ly hope that we will get there.” — Nicole De­Feud­is 

‘Nev­er had a grand plan’: A wind­ing ca­reer path led Jen­nifer Pet­ter to launch her own com­pa­ny

Jen­nifer Pet­ter didn’t re­al­ize it at the time, but not get­ting tenure was ac­tu­al­ly a good thing — a great thing, even.

It spurred her jump from aca­d­e­mics to drug mak­ing, send­ing her on a decades-long path that led to the launch of her own com­pa­ny, Ar­rakis Ther­a­peu­tics.

As with any set­back, “you have to spend at least one week­end just watch­ing TV and eat­ing ice cream,” she said. “When­ev­er you’ve dried your tears, you can kind of stand up and look around (and say), ‘Ok, well, what are we go­ing to do now?’”

With her days teach­ing chem­istry at the Uni­ver­si­ty of Pitts­burgh be­hind her, Pet­ter land­ed a lab job at San­doz in 1991. Tran­si­tion­ing from aca­d­e­mics to lab work was easy, she said. “You put some mu­sic on and you get to work.” To­day, her playlist ranges from Mon­tever­di’s Ves­pers to Paul Si­mon.

Pet­ter stayed at San­doz for 5 years, leav­ing just as it was be­com­ing No­var­tis. Then she spent 9 years at Bio­gen, where she was di­rec­tor of small mol­e­cule drug dis­cov­ery un­til los­ing her spot in a 650-per­son lay­off in 2006. Af­ter that, she served as VP of re­search, drug dis­cov­ery and chem­istry at Mer­sana, Avi­la and Cel­gene, re­spec­tive­ly.

When asked if launch­ing a com­pa­ny was al­ways part of her plan, Pet­ter chuck­led.

“I nev­er had a grand plan for my life, or even my ca­reer,” she said. “I’ve just com­plete­ly thrown my­self in­to the things that I’ve done and pur­sued them un­til I had to stop. And then I threw my­self in­to some­thing new.”

Ar­rakis — named af­ter the desert plan­et from the nov­el Dune — is on a mis­sion to de­vel­op small mol­e­cules that tar­get dis­ease-caus­ing RNA. It emerged back in 2017 with a $38 mil­lion Se­ries A round, and re­cent­ly pulled in $190 mil­lion up­front in a Roche deal.

Pet­ter, now CSO, said:

When I first heard about RNA be­ing ad­dressed by small mol­e­cules, I was im­me­di­ate­ly gal­va­nized — I want to do that. And it wasn’t be­cause I knew what the an­swer was, but be­cause I loved the prob­lem.

In 2018, over sushi and munchies at a com­pa­ny hap­py hour, Pet­ter came out to her cowork­ers as trans­gen­der. Ar­rakis CEO Michael Gilman ap­plaud­ed her brav­ery in a blog post, and the biotech got right to work on a doc­u­ment out­lin­ing the oblig­a­tions of em­ploy­ees, lead­er­ship and the com­pa­ny as a whole.

While near­ly every­thing in the doc­u­ment was al­ready guar­an­teed by law, Pet­ter saw val­ue in the sim­ple act of draw­ing it up.

“Some­times the fact that some­thing is al­ready man­dat­ed by law, at least here in Mass­a­chu­setts, doesn’t mean there isn’t some val­ue in af­firm­ing it,” she said. “I think in some cas­es it’s just a mat­ter of go­ing, you know, not even that ex­tra mile — the ex­tra cou­ple hun­dred feet just to make sure that peo­ple un­der­stand … what your views are in this mat­ter.”

Pet­ter doesn’t doubt that com­ing out ear­li­er in her ca­reer would have made the jour­ney more dif­fi­cult. Many le­gal pro­tec­tions weren’t in place years ago, even in Mass­a­chu­setts. And “there’s no ques­tion” that mak­ing progress in the bio­phar­ma field is more chal­leng­ing as a woman, she said.

“Just from a pure­ly sta­tis­ti­cal per­spec­tive, it is un­de­ni­able that women are un­der­rep­re­sent­ed in se­nior lead­er­ship po­si­tions across our in­dus­try and oth­ers,” Pet­ter said.

The tim­ing of her de­ci­sion to come out had to do with “in­ter­nal dri­vers,” and not the ca­reer chal­lenges she would have faced. Nev­er­the­less, it’s on com­pa­nies to pro­mote di­ver­si­ty and in­clu­sion, she said.

“Di­ver­si­ty is about get­ting peo­ple in the door,” she said. “In­clu­sion is about ac­tu­al­ly get­ting them in­volved and … mov­ing along in their ca­reers while they’re in your com­pa­ny or in your in­sti­tu­tion.”

Get­ting women through the door can be chal­leng­ing when there are sim­ply more men wait­ing on the stoop. For top po­si­tions, there are of­ten far few­er women in the run­ning, Pet­ter said.

It’s our re­spon­si­bil­i­ty to use net­work­ing as a way to bring peo­ple along, and make them more ready for these op­por­tu­ni­ties. And to go out and cul­ti­vate sci­en­tists that you know are not ready for the job you have now, but might be good for a job like that in five years.

As for her own fu­ture, Pet­ter said she wants to see Ar­rakis bloom. “If there’s an op­por­tu­ni­ty to start an­oth­er com­pa­ny some­where in there, that might be fun too,” she added. — Nicole De­Feud­is 

Genen­tech taps an am­bi­tious pi­o­neer to guide them through bi­ol­o­gy’s fu­ture

To un­der­stand Genen­tech’s new head of re­search, be­gin on a cold, Tel Aviv win­ter, when Aviv Regev is not yet 30, not yet a world-fa­mous re­searcher, just a promis­ing grad­u­ate stu­dent whose friend is on the verge of quit­ting.

Dana Pe’er stud­ied com­put­er sci­ence at He­brew Uni­ver­si­ty in Jerusalem. The two had struck up a friend­ship at a con­fer­ence, each of them in­ter­est­ed in sim­i­lar ques­tions about ge­net­ics and com­pu­ta­tion­al bi­ol­o­gy. But in the months since, Pe’er’s da­ta had come in and pro­vid­ed, rather than an an­swer, a be­wil­der­ing mess that the com­put­er sci­en­tist, new to bi­ol­o­gy, couldn’t make heads or tails of. She called Regev.

“I said my da­ta failed, my project failed, I’m a fail­ure and I’m go­ing to quit,” Pe’er re­called. “And she said, let me look at the da­ta.”

Regev would make her name as a com­pu­ta­tion­al­ist, but her grasp of bi­ol­o­gy was al­most philo­soph­i­cal — the kind of in­tu­itive think­ing that al­lows quan­tum physi­cists to dis­cern deep, ab­stract prin­ci­ples of re­al­i­ty by look­ing at par­ti­cles break­ing in a col­lid­er. She looked over the re­sults and then sat down with Pe’er at a con­fer­ence: “What do you mean it failed?”

Pe’er didn’t get the an­swer she was look­ing for, but, Regev told her, she had man­aged to re­ca­pit­u­late much of the ge­net­ic cir­cuit­ry for cell me­tab­o­lism. The two would co-write a study for Ox­ford’s Bioin­for­mat­ics, one of the first pa­pers ei­ther of them ever pub­lished.

“She gets ex­cit­ed, it’s very in­fec­tious — her tone,” Pe’er, who now runs a com­pu­ta­tion­al bi­ol­o­gy lab at Memo­r­i­al Sloan Ket­ter­ing, said. “The pace of her words just quick­ened and it’s amaz­ing.”

Pe’er said Regev hasn’t changed much over the two decades since, a re­mark­able thing giv­en how far those years have tak­en her: through a PhD at Tel Aviv Uni­ver­si­ty, a pro­fes­sor­ship at MIT and the Broad In­sti­tute, one of the most au­da­cious bi­ol­o­gy projects of the last decade and, now, one of the most-cov­et­ed and watched perch­es in bio­phar­ma.

It was a no­table se­lec­tion, re­flec­tive of how much the in­dus­try has moved over Regev’s ca­reer (dri­ven, in no small part, by Regev). Michael Var­ney, her pre­de­ces­sor, was a tra­di­tion­al or­gan­ic chemist who had come up through Agouron and Pfiz­er and start­ed at Genen­tech as a small mol­e­cule dis­cov­er­er. Regev’s hir­ing re­flect­ed a bet on where the best com­pu­ta­tion­al tools and minds are al­ready pulling biotech and where Genen­tech will have to move to stay atop the in­dus­try they birthed.

Ar­guably more than that, though, it was a bet on the per­son. Over Zoom, Regev is ef­fer­ves­cent yet sharp, lay­ing out the fu­ture of AI in bi­ol­o­gy while di­rect­ing her very pa­tient daugh­ter on where to find col­ored pen­cils and laugh­ing about the strug­gles of com­ing on­to a job that’s still ful­ly re­mote. Col­leagues and for­mer men­tors de­scribe a fig­ure they’ve rarely en­coun­tered: a com­put­er sci­en­tist who thinks about and is fas­ci­nat­ed first and fore­most by bi­ol­o­gy, an aca­d­e­m­ic supreme­ly con­fi­dent of her own con­vic­tions and yet clear-head­ed about what she can do — and al­so what you can do, which of­ten turns out to be a lot, with a lit­tle help. She is, they say, cu­ri­ous and fierce­ly kind.

“I had many stu­dents — be­lieve me I had some very bril­liant peo­ple, no less good in terms of in­tel­lect than she is,” Eva Jablon­ka, her the­sis ad­vi­sor and a well-known ge­neti­cist at Tel Aviv Uni­ver­si­ty, told me. “But I nev­er had some­body like her.”

Com­pu­ta­tion­al bi­ol­o­gy is big these days — big enough that some forms, such as ar­ti­fi­cial in­tel­li­gence, get knocked as over­hyped. But that wasn’t yet the case when Regev start­ed out. She hadn’t set out to be a bi­ol­o­gist. Af­ter her mil­i­tary ser­vice, she was ac­cept­ed in­to Tel Aviv Uni­ver­si­ty’s In­ter­dis­ci­pli­nary Pro­gram for Out­stand­ing Stu­dents, a se­lect pro­gram that took 15 stu­dents and ac­cel­er­at­ed them to a four-year mas­ter’s, al­low­ing them to study what­ev­er they want­ed in the process. She loved com­put­er sci­ence, math, phi­los­o­phy, law. “I didn’t want to choose,” she told me.

Jablon­ka’s ge­net­ics course changed that. It was ab­stract, its puz­zles cracked by in­fer­ence. “I said, ‘Oh that’s fan­tas­tic.’ Bi­ol­o­gy is ex­act­ly the way I like to think about the world,” she re­called.

Jablon­ka, too, was a com­pelling fig­ure. She had al­most sin­gle-hand­ed­ly re­vived the Lamar­ck­ian idea that ac­quired traits could be passed on, show­ing amid ex­treme skep­ti­cism that epi­ge­net­ics made it not on­ly fea­si­ble but piv­otal in evo­lu­tion. It taught Regev that sci­ence was not a pop­u­lar­i­ty con­test; you could be de­rid­ed and still be right.

In time, of course, would come mi­cro­bi­ol­o­gy with its pipettes and PCR ma­chines, its messy re­sults muck­ing those ab­stract prin­ci­ples. And yet Regev saw a sym­me­try be­tween the his­to­ry of mi­cro­bi­ol­o­gy and the fu­ture of com­pu­ta­tion­al bi­ol­o­gy. Mi­cro­bi­ol­o­gy gave bi­ol­o­gists new tools, but it al­so de­mand­ed a new way of think­ing and forced re­sults to be ex­plained in new terms. Com­pu­ta­tion could do the same.

She tried that with her PhD pro­pos­al: a project to use nat­ur­al lan­guage pro­cess­ing — the same AI tech­nique that al­lows com­put­ers to “un­der­stand” the con­tents of a doc­u­ment or chat­box — to mod­el the cir­cuits that gov­ern how cells dif­fer­en­ti­ate and func­tion. Jablon­ka thought it was bril­liant, al­low­ing bi­ol­o­gists to look be­yond the messy de­tails and see the over­all log­ic of the sys­tem, but Regev soon ran in­to trou­ble. Bi­ol­o­gists didn’t yet know enough to de­sign such a mod­el. “In re­al­i­ty, we on­ly knew a smidge,” she said. “There was just so much we didn’t know. ”

With in­spi­ra­tion from Pe’er, she took a dif­fer­ent ap­proach: not try­ing to mod­el the sys­tem di­rect­ly, but de­vis­ing ma­chine learn­ing tech­niques that would let the da­ta tell you what the sys­tem is. It’s al­so how she even­tu­al­ly end­ed up with her own wet lab at the Broad In­sti­tute. Need­ing to per­turb the sys­tem to prove the mod­el was cor­rect, she be­came the rare com­pu­ta­tion­al­ist stand­ing over a petri dish.

“You do com­pu­ta­tion­al bi­ol­o­gy, you come up with meth­ods and then you search for ways to ap­ply those meth­ods,” Ram­nik Xavier, an im­mu­nol­o­gist and col­league at the Broad, told me. “Where­as Aviv went the oth­er way, she start­ed with the hu­man bi­ol­o­gy ques­tion and she want­ed to find the so­lu­tion to that ques­tion and de­vel­op meth­ods that would al­low one to an­swer (it).”

In many ways, Regev’s goals haven’t changed since. She’s still try­ing to un­cov­er those sys­tems in search of bet­ter drugs.

“If we know how the sys­tem works, then we can pre­dict it, and if we can pre­dict it then we should be able to de­vel­op bet­ter drugs, more safe­ly, fast,” Regev said. “I don’t think it was a new in­sight but I al­so don’t think it was that ex­e­cutable a decade ago.”

A decade ago, Regev said, three things changed: On the floor above her, Feng Zhang helped pi­o­neer CRISPR, mak­ing ge­net­ic knock­out ex­per­i­ments vast­ly eas­i­er. Ma­chine learn­ing ad­vanced to an in­flec­tion point. And Regev her­self pi­o­neered RNA se­quenc­ing, al­low­ing re­searchers for the first time to close­ly probe the ge­net­ic net­works in­side cells.

In a ground­break­ing 2013 Na­ture study, Regev and a team se­quenced 18 in­di­vid­ual im­mune cells. Then, in 2014, she was in­vit­ed as one of sev­er­al speak­ers to pro­pose a hy­po­thet­i­cal yet re­al­is­tic project for the NIH to spend $50 mil­lion on. She pro­posed pro­fil­ing every cell in the hu­man body — a Google Maps for the cel­lu­lar body.

“It was a com­plete­ly crazy, crazy idea,” Sarah Te­ich­mann, who was then di­rec­tor of Trin­i­ty Col­lege, Cam­bridge, told me. Five years pri­or, sci­en­tists broke ground se­quenc­ing 5 cells. The hu­man body has 37.2 tril­lion cells. “It was com­plete­ly crazy at that time to think about some­thing like this or any­thing at this scale.”

Te­ich­mann, though, had toyed with a sim­i­lar idea. So had a cou­ple of oth­ers, and when Te­ich­mann two years lat­er was ap­point­ed head of cel­lu­lar ge­net­ics at the Well­come Sanger In­sti­tute, she emailed Regev. With a cou­ple of dozen oth­er sci­en­tists they found­ed the Hu­man Cell At­las, which has al­ready had ther­a­peu­tic im­pli­ca­tions.

Regev helped fig­ure out that, con­trary to years of pub­lished work, the gene be­hind cys­tic fi­bro­sis, CFTR, is ac­tu­al­ly on­ly ex­pressed in a small sub­set of spe­cial­ized cells, po­ten­tial­ly af­fect­ing how you would build a gene ther­a­py. With Xavier, she mapped GI cells, study­ing rare neu­rons in­stru­men­tal in dis­eases like IBS. We’ve long viewed mul­ti­ple scle­ro­sis or Crohn’s as a sin­gle dis­ease, but sin­gle-cell se­quenc­ing is show­ing how di­verse they are and how di­verse the treat­ments have to be. “Text­books will be re-writ­ten,” Xavier said.

The at­las al­so put her at the head of an in­ter­na­tion­al coali­tion that spanned dis­ci­plines, re­quir­ing fi­nesse, or­ga­ni­za­tion and in­ter­dis­ci­pli­nary knowl­edge that few aca­d­e­mics have to deal with  — but which big biotech ex­ecs need. “I can’t imag­ine a bet­ter per­son to tran­si­tion,” Ja­yaraj Ra­jagopal, a Har­vard stem cell bi­ol­o­gist who col­lab­o­rat­ed with Regev on the CFTR work, told me.

But why leave? Regev puts it blunt­ly: “Be­cause I ac­tu­al­ly be­lieve the things I say.”

For two decades, she had ar­gued that if bi­ol­o­gists could un­der­stand mol­e­c­u­lar sys­tems, you could turn them in­to bet­ter drugs. Now, re­searchers had amassed ways of look­ing at cells in un­par­al­leled res­o­lu­tion, col­lect­ed un­heard-of hu­man ge­net­ic da­ta and hu­man biop­sies, and de­vised in­cred­i­bly pow­er­ful ma­chine learn­ing al­go­rithms. There were few oth­er places where she would be able to stitch it all to­geth­er.

Now, she plots build­ing an en­gine grander than the small mol­e­cule AI ma­chines that dom­i­nate head­lines. The idea is that you can build drugs dif­fer­ent­ly at every step, de­sign­ing pre­clin­i­cal and clin­i­cal tri­als to get the da­ta ma­chine learn­ing can then turn in­to an­swers. And with enough ex­per­i­ments, you could be pre­dic­tive in a way few bi­ol­o­gists are, col­lect­ing enough da­ta and feed­ing it through enough al­go­rithms to turn the con­found­ing messi­ness of bi­ol­o­gy in­to your friend and al­low you to see, for ex­am­ple, what hap­pens if you dis­turb 3 genes.

Some­times, she says, she pon­ders mov­ing away from the sin­gle mol­e­c­u­lar “tar­get” al­to­geth­er, to use these tools to try to un­der­stand the bird’s-eye sys­tem of cells, as she tried to do as a grad­u­ate stu­dent, and drug ac­cord­ing­ly. But that’s still spec­u­la­tive, a far-off pos­si­bil­i­ty. She’s cer­tain the rest will hap­pen.

“I whole­heart­ed­ly think the in­flec­tion point is up­on us,” Regev said. “We are not ex­act­ly in­flect­ed yet, though, and I want to make that in­flec­tion hap­pen.” — Ja­son Mast

An ear­ly CAR re­searcher and Yescar­ta leader, Mar­go Roberts is back in the game at Lyell

Mar­go Roberts nev­er thought she’d end up at Lyell af­ter leav­ing her pre­vi­ous job.

The long­time biotech vet spent decades work­ing on CAR ther­a­pies and had just wrapped up a suc­cess­ful run at Kite Phar­ma in 2018, where she was CSO and put to­geth­er the team that de­vel­oped the ground­break­ing can­cer drug Yescar­ta. One of two CAR-T treat­ments ap­proved in late 2017, along with No­var­tis’ Kym­ri­ah, the drug ap­peared to be the cap­stone for Roberts’ decades-long ca­reer as a leader in CAR re­search.

But when she heard that the Rick Klaus­ner-backed Lyell want­ed to take the cell ther­a­py tech­nol­o­gy and use it to treat sol­id tu­mors, Roberts came down with a case of cu­rios­i­ty, and per­haps a lit­tle bit of FO­MO. And there are no FDA-ap­proved drugs for that — not yet, any­way.

“I just felt like I re­al­ly need­ed a break,” Roberts said. “And I wasn’t ex­pect­ing to come back to a po­si­tion like this, but I did, be­cause … when I heard the vi­sion of the com­pa­ny, I thought to my­self, ‘I can’t let this hap­pen with­out me.’ I’d be so up­set if I had to read about this com­pa­ny in the news and I’m not part of it, I’d be pissed at my­self.”

So Roberts joined the squad at Lyell a lit­tle over a year ago, where she’s again serv­ing as CSO and lead­ing an­oth­er CAR charge. Klaus­ner brought her on with the goal of as­sem­bling one of the most im­pres­sive teams of sci­en­tists to tack­le cell ther­a­py 2.0, while rais­ing an im­pres­sive $600 mil­lion to get the re­search off the ground. In­clud­ed in that heap of cash is a con­tri­bu­tion from GSK well with­in the nine-fig­ure range.

Roberts has worked with sev­er­al smart in­di­vid­u­als through­out her ca­reer, but there’s more to R&D than sim­ple ex­per­tise, she says. There are a lot of strong opin­ions out there but what’s most im­por­tant is find­ing an en­vi­ron­ment where peo­ple are will­ing to ques­tion the sci­en­tif­ic dog­ma.

That’s ul­ti­mate­ly what led Roberts to what she con­sid­ers one of the great­est ac­com­plish­ments in her ca­reer pre-Yescar­ta: ap­ply­ing the con­cept of CAR-Ts and T cell man­u­fac­tur­ing to HIV treat­ment.

“I re­mem­ber the re­views com­ing back from the NI­AID, and they were say­ing, ‘Wow this is re­al­ly in­ter­est­ing, this is a re­al­ly cool thing that you’re propos­ing.’ But one re­view­er said, ‘But this is Star Wars, and it will nev­er work,’” Roberts said. “I wish I’d kept that be­cause I’d love to have framed it and have lights flash­ing all around it. We were in the clin­ic 16 to 18 months lat­er.”

Since then, the tech­nol­o­gy has proven it­self to be one of the most promis­ing ar­eas in the phar­ma in­dus­try. As ev­i­denced by Lyell’s own fund­ing, there’s a lot rid­ing on the type of re­search where Roberts has spent most of her ca­reer. It’s been a long and rocky road fight­ing against that dog­ma of what’s be­lieved to be pos­si­ble.

She re­mem­bers work­ing on an ear­ly project in­volv­ing co-stim­u­la­tion sig­nals in the con­text of a CAR, an­oth­er of her ma­jor con­tri­bu­tions to the field. At the time, her patent for the idea drew some con­tro­ver­sy and the pro­gram was even­tu­al­ly shut down. But con­tin­u­ing to fight for the idea and the ra­tio­nale be­hind it, Roberts ul­ti­mate­ly felt vin­di­cat­ed with the Yescar­ta ap­proval, she said.

Keep­ing up the fight for what you be­lieve in takes a lot of courage, and Roberts said she aims to sur­round her­self with peo­ple who share that mind­set. It was one of the dri­ving mo­ti­va­tors be­hind build­ing her team at Kite.

“Courage is huge­ly im­por­tant, it takes guts to make a lot of the de­ci­sions that we do,” Roberts said. “Most of the time, you’re not nec­es­sar­i­ly know­ing — you don’t have a crys­tal ball — which sci­en­tif­ic di­rec­tion you’re go­ing, which di­rec­tion is the nec­es­sary one to take. And you’re go­ing to make mis­takes, you’re go­ing to make bad calls. It’s re­al­ly crit­i­cal that you se­lect the peo­ple who aren’t afraid of mak­ing the rapid de­ci­sion based on the avail­able in­for­ma­tion.”

Roberts’ biggest hope for the next gen­er­a­tion of women in bio­phar­ma is to con­tin­ue that courage. She says she didn’t have many women role mod­els com­ing up through the ranks and re­calls at­tend­ing the ear­ly days of the JP Mor­gan con­fer­ence in San Fran­cis­co where she’d walk in­to a room and find “a sea of men.”

One of the biggest signs things are chang­ing is how the phrase “I was able to com­mu­ni­cate this to my moth­er” has fall­en out of vogue in less than a gen­er­a­tion, Roberts says. She used to hear this say­ing rather fre­quent­ly in­tend­ed as a joke when sci­en­tists would present their find­ings, but when her son heard this at an IT con­fer­ence a few years ago he didn’t un­der­stand what it meant.

And as more women come up the ranks in biotech, Roberts wants them to see that hold­ing lead­er­ship po­si­tions is a goal that’s well with­in reach.

“One of the things I’ve grown up with is this con­cept, or where I’ve been ex­posed to it, where you can be — es­pe­cial­ly if you’re a man — a re­al bas­tard, frankly, and it’s OK, you can get away with it be­cause ‘Oh he’s bril­liant,’” Roberts said. “There’s still a lot that can be done there in terms of al­low­ing girls to re­al­ize that if that’s a nat­ur­al in­ter­est that they have, this is re­al­ly doable, it doesn’t re­quire a su­per­pow­er.” — Max Gel­man

‘Just say yes’: Lau­ra Shawver leads her fifth biotech with an all-fe­male C-suite

Lau­ra Shawver was walk­ing down a hall­way at Dav­en­port Cen­tral High School in Iowa when she was stopped by her AP chem­istry teacher, who asked about her plans for col­lege.

Maybe study nurs­ing, or ma­jor in mu­sic, Shawver replied. She was “al­ways kind of a nerd” — the type of grade school kid who loved mak­ing sci­ence fair projects.

The teacher looked at Shawver. “Well, why would you be a nurse, when you could be a doc­tor?” she pressed.

It was the ’70s, and un­til that mo­ment, it hadn’t dawned on Shawver: “I could be a doc­tor,” she thought.

So she did it. Shawver earned her bach­e­lor’s de­gree in mi­cro­bi­ol­o­gy from the Uni­ver­si­ty of Iowa, fell in love with re­search, then got her PhD in phar­ma­col­o­gy. And that AP chem teacher? She fol­lowed Shawver’s long ca­reer in drug de­vel­op­ment, which land­ed her as CEO of Sil­ver­back Ther­a­peu­tics, where she’s now lead­ing ef­forts to recon­cep­tu­al­ize an­ti­body-drug con­ju­gates for treat­ing can­cer.

“I think it’s im­por­tant … that as we en­ter in­to a field, as we grow in our con­fi­dence, as we think about ex­pand­ing our hori­zons, that we al­ways have some­body that is in some way say­ing, ‘Yeah you can do it, you can do it,’” Shawver said.

Af­ter fin­ish­ing grad­u­ate school, Shawver let cu­rios­i­ty guide her to a post­doc fel­low­ship at the Uni­ver­si­ty of Vir­ginia Can­cer Cen­ter in the mi­cro­bi­ol­o­gy de­part­ment. It was the mid-80s, and the sci­en­tif­ic com­mu­ni­ty was buzzing over the prospects of cloning.

“Of course now any high school stu­dent can clone, but back then it took years and it was very dif­fi­cult, and I want­ed that op­por­tu­ni­ty to learn mol­e­c­u­lar and cell bi­ol­o­gy,” she said.

Shawver re­called her first sem­i­nar at UVA. Some­one was pre­sent­ing on some onco­gene, but it was like they were speak­ing a new lan­guage. Shawver knew phar­ma­col­o­gy, but mol­e­c­u­lar bi­ol­o­gy was a dif­fer­ent beast.

“I re­mem­ber leav­ing work that day, think­ing to my­self: ‘Oh my God, what have I got­ten my­self in­to? I’m nev­er go­ing to un­der­stand this.’” (Spoil­er alert: With time and ef­fort, she did.)

In 1989, Shawver start­ed a job at can­cer-fo­cused Tri­ton Bio­sciences, which inked a deal in 1990 to sell off its phar­ma­ceu­ti­cals busi­ness to Berlin-based Scher­ing. Two years lat­er, she hopped over to Sug­en.

“I grew up with that com­pa­ny,” she said.

As di­rec­tor of pre­clin­i­cal de­vel­op­ment, Shawver helped put five drugs in­to the clin­ic, and saw two pop out the oth­er side: Su­tent, which is ap­proved for kid­ney can­cer and oth­er gas­troin­testi­nal stro­mal tu­mors, and Pal­la­dia, which is ap­proved to treat mast cell tu­mors in dogs. She jumped from di­rec­tor to se­nior di­rec­tor to vice pres­i­dent to se­nior vice pres­i­dent to pres­i­dent, as the com­pa­ny went pub­lic, was ac­quired and merged.

“Al­most every­thing that hap­pens in a biotech­nol­o­gy com­pa­ny hap­pened in one com­pa­ny at Sug­en in that 10 years,” Shawver said.

She served as CEO of Phe­nomix from 2002 to 2010, then CEO of Cleave Bio­sciences from 2011 to 2017, then pres­i­dent and CEO of Syn­thorx — a biotech built around the in­ven­tion of a new DNA base pair — from 2017 to this year.

“There’s been so many, I’ve lost track now,” she said with a laugh.

In 2006, while work­ing at Phe­nomix, Shawver was di­ag­nosed with ovar­i­an can­cer. She had al­ready been mak­ing on­col­o­gy drugs for 15 years, but sud­den­ly, her per­spec­tive changed.

As a drug de­vel­op­er, she had looked for im­prove­ments that were sta­tis­ti­cal­ly sig­nif­i­cant enough to get a prod­uct to mar­ket. “We look at an over­all im­prove­ment in sur­vival of 6 months or a 50% re­sponse rate as be­ing phe­nom­e­nal,” she said. “What I re­al­ized when I … was di­ag­nosed my­self with ovar­i­an can­cer: That’s not phe­nom­e­nal at all.”

“I mean, you’re miss­ing half the pop­u­la­tion, you’re on­ly buy­ing a lit­tle bit more time, you’re not ac­tu­al­ly do­ing any­thing to fun­da­men­tal­ly change the out­come of the can­cer. The can­cer typ­i­cal­ly wins,” she added.

Shawver re­cov­ered with surgery and chemother­a­py treat­ment, and she did two things: First, she launched the Clear­i­ty Foun­da­tion in 2008 to help ovar­i­an can­cer pa­tients get ac­cess to mol­e­c­u­lar pro­fil­ing to in­form their treat­ment de­ci­sions. Then, she got think­ing about cures.

That’s what drew her to Sil­ver­back, which is fo­cused on us­ing AD­Cs to dri­ve an im­mune re­sponse to give “more and more peo­ple with sol­id tu­mors their cures,” she said. And her C-suite is all-fe­male, in­clud­ing CSO and pres­i­dent Va­lerie Ode­gard and CMO Nao­mi Hun­der.

“It’s re­al­ly im­por­tant for women when they are pre­sent­ed with an op­por­tu­ni­ty to just say yes,” Shawver said. “Whether you have the ex­pe­ri­ence or not, whether you have the con­fi­dence or not, just say, ‘Yes I can do that…’ When you see that there’s a gap, raise your hand…”

“Go for it. Do it. You’ll nev­er have such an ex­cit­ing ca­reer,” she said. — Nicole De­Feud­is 

Kim­ber­ly Smith goes in­side the in­dus­try to build a new kind of HIV biotech ex­ec­u­tive

Be­fore Vi­iV R&D chief Kim­ber­ly Smith was a biotech ex­ec­u­tive, she was a doc­tor for 22 years at the HIV ward of Chica­go’s Rush Uni­ver­si­ty Hos­pi­tal. And be­fore that, she was a 24-year-old stu­dent protest­ing the pres­i­dent of the Uni­ver­si­ty of Michi­gan, de­mand­ing they rec­og­nize MLK Day and the racism stu­dents were fac­ing on cam­pus.

The path from clin­ic to cor­po­rate is well-trod­den, less so the path there from protest. It’s one of a few things that make Smith dif­fer­ent than the av­er­age head of R&D.

HIV biotechs have al­ways ex­ist­ed in un­easy ten­sion with ac­tivists, per­haps more so than drug de­vel­op­ers for any oth­er dis­ease: In 1987, Bur­roughs Well­come set the cost of the first HIV drug, AZT, so high it led to TV-grab­bing protests that turned pub­lic opin­ion. Ac­tivists em­ploy some of the same tac­tics to­day to protest Gilead and the long-ris­ing, now $23,000-per-year cost of Tru­va­da.

Smith was at Michi­gan when the protests over AZT be­gan, and she watched with sup­port as ac­tivists fought to change how drug re­search was done. Al­though a nat­u­ral­ly cu­ri­ous thinker who dis­sect­ed frogs from the creek near her house at age 12 and pur­sued in­fec­tious dis­ease in part be­cause in­fec­tious dis­ease physi­cians are med­i­cine’s de­tec­tives, she would spend as much of her ca­reer ex­ca­vat­ing the bi­ol­o­gy of HIV as she would fight­ing for re­search that was fair, di­verse and in­clu­sive. In nu­mer­ous ar­ti­cles and one book, she doc­u­ment­ed the treat­ment needs of women and peo­ple of col­or and how of­ten they were left out of tri­als.

And had it not been for a sur­prise call from a for­mer men­tor, she like­ly would have stayed in Chica­go, work­ing to change the sys­tem from the out­side. In­stead, she’s cut the mold of a new kind of HIV biotech ex­ec­u­tive, one that, with­in the con­straints of a Big Phar­ma-owned com­pa­ny, is more at­tuned to pric­ing, to ac­tivist de­mands and more com­mit­ted to di­ver­si­ty in their re­search.

“Pow­er con­cedes noth­ing with­out de­mand,” she told me when I asked about ac­tivist cri­tiques of in­dus­try, quot­ing Fred­er­ick Dou­glass. “Of course there should be a chal­lenge to phar­ma­ceu­ti­cal com­pa­nies.”

Vi­iV spun out of GSK in 2009 and Smith joined in 2013. She takes pride in the fact that the Fair Pric­ing Coali­tion gave Vi­iV, as she puts it, “a soft thumbs up” on pric­ing. A 2019 FPC state­ment says the price the com­pa­ny put on their Dova­to dai­ly pill “bucks the trend of run­away HIV drug pric­ing”: “FPC asked and Vi­iV lis­tened,” chair Tim Horn added.

In 2013, the year Smith joined, Vi­iV be­came the on­ly ma­jor com­pa­ny to put some of its HIV drugs in a med­i­cine patents pool set up by the UN, prompt­ing Pub­lic Cit­i­zen to send let­ters to six oth­er ma­jor drug com­pa­nies ask­ing why they didn’t do the same.

She al­so takes pride in her com­pa­ny’s di­ver­si­ty, both of her em­ploy­ees and of the re­search they con­duct. It’s been an is­sue for Smith since her med school days, when she watched HIV af­flict the worst of so­ci­ety and much of the med­ical world turn their back on the peo­ple they were sup­posed to help. “HIV was fas­ci­nat­ing to me, sci­en­tif­i­cal­ly and med­ical­ly … But there was al­so the so­cial-po­lit­i­cal as­pect of it, be­cause back in those days, peo­ple with HIV — the stig­ma still ex­ists, but the stig­ma then was hor­rif­ic,” Smith said. “I was a po­lit­i­cal per­son, I want­ed to be part of that.”

At the time, 1993, the first an­ti­retro­vi­rals were reach­ing the mar­ket, of­fer­ing hope of chang­ing the course of the dis­ease. Still, they weren’t yet a cure and Smith pro­nounced one or two pa­tients dead per night. “It was heart­break­ing,” she said. “And these were young peo­ple, young men who have sex with men and in many cas­es young women, it was all of Amer­i­ca.”

Smith pulled through, buoyed in part by the fam­i­lies who re­lied on her. She be­gan study­ing how the new drugs af­fect­ed the im­mune sys­tem. She tracked the newest ad­vances with ex­cite­ment but al­so grow­ing frus­tra­tion. The pub­lic face of HIV at the time was white, male and gay and those were of­ten the faces of drug tri­al par­tic­i­pants. But that im­age didn’t re­flect the faces Smith tend­ed to at Rush, nor the ac­tu­al na­tion­al sta­tis­tics.

Smith read pa­per af­ter pa­per and then looked with dis­may at the de­mo­graph­ics. She start­ed writ­ing about it and, as her pro­file grew, lob­by­ing the NIH to do some­thing. Even­tu­al­ly, they formed the un­der-rep­re­sent­ed pop­u­la­tion’s com­mit­tee of the AIDS clin­i­cal tri­al net­work and named Smith co-chair.

“It was like, ‘OK, Kim, we’re go­ing to shut you up and give you your own com­mit­tee, where you can fight this out,” Smith said.

Then in 2013, her phone rang. It was John Pot­tage. Pot­tage had been an at­tend­ing at Rush when Smith was a res­i­dent, be­fore he left to join Ver­tex, which was then fo­cused on an­tivi­rals. Pot­tage had kept up with Smith’s work, as a clin­i­cian and a clin­i­cal tri­al­ist, and want­ed to know if she might want to help run Vi­iV’s clin­i­cal re­search.

“She has this abil­i­ty, this tal­ent but she’s al­so able to com­mu­ni­cate it,” Pot­tage told me. “To get peo­ple to un­der­stand where she’s com­ing from and take peo­ple with her.”

Smith had nev­er con­sid­ered in­dus­try but Vi­iV was dif­fer­ent. It was all HIV, and it meant an op­por­tu­ni­ty to make a larg­er im­pact on the field that had be­come her life’s work. Af­ter work­ing un­der Pot­tage for 6 years and help­ing de­vel­op four dif­fer­ent HIV drugs, she was named to his old po­si­tion of head of R&D last year.

Now she tries to mold HIV re­search in a vi­sion she’s built over 3 decades. That means long-in­ject­ing that helps pa­tients avoid dai­ly pills and the stig­ma that, for some, can come with them. It al­so means tri­als that draw from pa­tients who are most af­fect­ed.

The con­trast was clear over the sum­mer. In a sig­nif­i­cant achieve­ment for Smith and the com­pa­ny, a tri­al they were run­ning com­par­ing once-every-8-week ex­per­i­men­tal in­jec­tion vs dai­ly PrEP was cut short in Ju­ly for over­whelm­ing ef­fi­ca­cy. The study in­clud­ed 4,600 peo­ple across 40 con­ti­nents, half of the par­tic­i­pants were black and 12% were trans­gen­der women.

At the same time, they await­ed re­sults for a study that was en­tire­ly com­posed of women. On No­vem­ber 9, that tri­al read out, show­ing their in­jectable was 89% more ef­fec­tive at pre­vent­ing HIV than dai­ly pills.

Gilead, mean­while, won ap­proval for De­scovy, a Tru­va­da fol­low-up, af­ter a tri­al that gen­er­at­ed wide­spread crit­i­cism for in­clud­ing ze­ro women.

“Ob­vi­ous­ly, I can’t com­plain to their faces any­more like I used to when I was their ad­vi­sor and I was not in in­dus­try,” Smith said of com­pa­nies that don’t in­clude di­verse par­tic­i­pants. “But what I do is make sure that we set an ex­am­ple of try­ing to do it bet­ter.” — Ja­son Mast 

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

UP­DAT­ED: Gink­go Bioworks re­sizes the de­f­i­n­i­tion of go­ing big in biotech, rais­ing $2.5B in a record SPAC deal that weighs in with a whop­ping $15B-plus val­u­a­tion

Ginkgo Bioworks execs always thought big. But today should redefine just how big an upstart biotech player can dream.

In the largest SPAC deal to clear the hurdles to Nasdaq, the biotech that envisioned everything from remaking synthetic meat to a whole new approach to developing drugs has joined forces with one of the biggest disruptors in biotech to slam the Richter scale on dealmaking.

Soon after becoming the darling of the VC crew and clearing the bar on a $4 billion valuation, Ginkgo — a synthetic biotech player out to reprogram cells with industrial efficiency — has now struck a deal to go public in the latest leviathan SPAC that sets its pre-money valuation at $15 billion. In one swift vault, Ginkgo will combine with Harry Sloan’s Soaring Eagle Acquisition Corp. and leap into the public markets.

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Stephen Squinto, Gennao Bio CEO (Gennao)

Alex­ion co-founder Stephen Squin­to is back in the game as CEO, this time for a small gene ther­a­py play­er

With his name already behind a rare disease success story in Alexion, Stephen Squinto was looking for a great story to drive him to jump back into the biotech game. He found that in a fledging non-viral gene therapy company, and now he’s got a few backers on board as well.

On Tuesday, Gennao Bio launched with a $40 million Series A co-led by OrbiMed and Logos Capital with participation by Surveyor Capital. The biotech, which is looking to use its cell-penetrating antibody platform to deliver nucleic acid “payloads” during into the nucleus, had to rush for its initial series — and had a name change along the way.

FDA un­veils six ICH guide­lines ahead of meet­ing with Health Cana­da

A sign that the FDA’s non-Covid-related processes are beginning to normalize: The release of six guidelines from the International Council of Harmonisation.

Years in development, the ICH documents offer an international perspective on drug development, with these latest guidelines covering everything from recommendations to support the classification of drug substances, featured in the M9 guidance, to standards for nonclinical safety studies for pediatric medicines in the S11 guideline.

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Sanofi, Glax­o­SmithK­line, Boehringer ac­cused of play­ing games, de­stroy­ing emails re­lat­ed to law­suit over con­t­a­m­i­nat­ed Zan­tac

A recent court filing raises new questions about how major pharma companies like Sanofi, GlaxoSmithKline, and Boehringer Ingelheim have dealt with a lawsuit related to recalls of certain over-the-counter heartburn drugs due to the presence of a potentially cancer-causing substance found in them.

More than 70,000 people who took Sanofi’s Zantac and other heartburn drugs containing ranitidine, which have been recalled over the past two years, have sued the manufacturers, including generic drugmakers, and other retailers and distributors as part of a consolidated suit before US District Court Judge Robin Rosenberg in Florida.

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Luisa Salter-Cid, Pioneering Medicines CSO (Credit: Bristol Myers Squibb via Twitter)

Luisa Salter-Cid joins long­time Bris­tol My­ers col­lab­o­ra­tor Paul Bion­di at Flag­ship's new drug brew­ery

Paul Biondi is gaining a familiar research chief at his corner of Flagship Pioneering.

The ambitious VC — famous for bold platform plays such as Moderna — said Luisa Salter-Cid is joining Pioneering Medicines as CSO, with the mandate of leveraging the platforms of various Flagship portfolio companies to conceive new treatments.

The two had crossed paths at Bristol Myers Squibb, where Biondi brought in a series of new drugs throughout his prolific business development stretch while Salter-Cid worked her way up the ladder to become head of immunology, small molecule immuno-oncology and genomics discovery.

Chris Garabedian (Xontogeny)

Per­cep­tive Ad­vi­sors, Xon­toge­ny bring the band back and then some with a $515M sec­ond fund sniff­ing out lead com­pounds

When Perceptive Advisors and startup accelerator Xontogeny initially teamed up on an early-stage VC round in 2019, the partners hoped to prove their investments could be a force multiplier for early-stage companies. Now, with that proof of concept behind them, the pair have closed a second VC round worth more than double the money.

Dubbed PXV Fund II and headed by Xontogeny CEO and former Sarepta head Chris Garabedian, the $515 million fund will target 10 to 12 early-stage preclinical companies with Series A rounds in the $20 million to $40 million range with opportunities for Series B follow-ups. The oversubscribed fund is bringing the band back with initial investors from PXVI as well as new investors that include “top-tier” asset managers, endowments, foundations, family offices, and individual investors.

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A clos­er look at the FDA’s more than 700 pan­dem­ic-re­lat­ed record re­quests to re­place on­site in­spec­tions

As the pandemic constrained the FDA’s ability to travel for onsite manufacturing inspections, the agency increasingly turned to requesting records to fill the gap, even for hundreds of US-based facilities.

FDA explains in its guidance on manufacturing inspections during the pandemic that the agency can request records (not to be confused with the FDA’s remote interactive evaluations) directly from facilities “in advance of or in lieu of” certain onsite inspections. Companies are legally required to fulfill those requests because a denial may be considered limiting an inspection, which could lead to the FDA deeming a drug made at that site to be adulterated.

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George Golumbeski (Credit: ARCH Ventures)

George Golumbes­ki signs on at a Eu­ro­pean spe­cial­ist VC firm with $265M to bet on ge­net­ic dis­eases

Since decamping from Celgene during a management overhaul close to three years ago, George Golumbeski has been channeling years of connections and dealmaking experience to a select group of company boards as a self-styled independent biotech exec — while dabbling in a somewhat behind-the-scenes role in venture capital at places like ARCH and Vertex Ventures.

He’s now adding one more gig to that menu, this time to scout promising startup ideas for genetic diseases.

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