Cut­ting loose from Mer­ck, Mike Nal­ly is 'run­ning to­ward a great op­por­tu­ni­ty'; No­var­tis part­ner­ship in hand, BeiGene pro­motes Ju­lia Wang to CFO

When Mike Nal­ly was an un­der­grad at Mid­dle­bury Col­lege in Ver­mont, he thought in­vest­ment bank­ing and fi­nance was his “path to sal­va­tion.”

He grad­u­at­ed in 1998 with his bach­e­lor’s in eco­nom­ics, then took a job at Don­ald­son, Lufkin & Jen­rette, an in­vest­ment bank lat­er bought by Cred­it Su­isse. He was tasked with do­ing re­search and analy­sis in the life sci­ences in­dus­try, but what he found sur­prised him.

Mike Nal­ly

“That re­al­ly fu­eled a pas­sion for the sci­ence, a pas­sion for the abil­i­ty to pos­i­tive­ly im­pact hu­man­i­ty, but al­so this ori­en­ta­tion to­ward a sense of pur­pose with­in my work,” Nal­ly said.

He went on to Har­vard Uni­ver­si­ty for his MBA, then spent 18 years in the ranks at Mer­ck, end­ing up as chief mar­ket­ing of­fi­cer of the phar­ma’s Hu­man Health unit. And now, amid a C-suite shake­up, Nal­ly is once again pack­ing his bags for Cam­bridge, MA, where he’ll be­come Flag­ship Pi­o­neer­ing’s lat­est CEO-part­ner just miles from his al­ma mater.

“We’re ex­cit­ed to come back af­ter 18 years around the world,” he said. “It’s a place that’s near and dear to our hearts.”

Ken Fra­zier

Nal­ly is tak­ing the helm at Gen­er­ate Bio­med­i­cines, an up­start lever­ag­ing ma­chine learn­ing and com­pu­ta­tion­al pow­er to learn the rules by which amino acid se­quences en­code for pro­tein struc­ture and func­tion. By do­ing so, Nal­ly be­lieves the 50-per­son team can gen­er­ate pro­teins that have “nev­er been seen on the plan­et be­fore.”

“We think the ap­proach has ap­plic­a­bil­i­ty across a range of modal­i­ties, whether that be an­ti­bod­ies, pep­tides, cy­tokines, and then more far-out places like mir­ror pro­tein cell and gene ther­a­py,” Nal­ly said. “There’s a wide range of ap­plic­a­bil­i­ty, and giv­en the in sil­i­co na­ture of dis­cov­ery, it al­lows you to dis­cov­er these pro­teins at a pace which is un­prece­dent­ed rel­a­tive to any­thing I’ve seen with­in in­dus­try.”

Nal­ly’s big move comes as Mer­ck’s chief ex­ec­u­tive Ken Fra­zier pre­pares to step down, and R&D MVP Roger Perl­mut­ter has hand­ed the reins over to Dean Li. Nal­ly, for­mer head of Mer­ck’s vac­cines busi­ness, was re­port­ed­ly in the run­ning to re­place Fra­zier, but the com­pa­ny in­stead went with CFO Robert Davis, a four-year Mer­ck vet­er­an.

While Nal­ly wouldn’t con­firm whether he was in con­sid­er­a­tion for the top spot, he says Davis’ ap­point­ment had noth­ing to do with his de­ci­sion to leave Mer­ck.

“I’m tremen­dous­ly ex­cit­ed about the op­por­tu­ni­ty at Flag­ship and with Gen­er­ate,” he said. “My move was not in re­sponse to any­thing oth­er than, you know, run­ning to­ward a great op­por­tu­ni­ty.” — Nicole De­Feud­is


Ju­lia Wang

→ Al­ready part­ner­ing with No­var­tis on tislelizum­ab, BeiGene had its eye on Boston Im­mune Tech­nolo­gies and Ther­a­peu­tics’ TN­FR2 an­tag­o­nist an­ti­bod­ies, with the two par­ties ar­rang­ing a de­vel­op­ment deal in Feb­ru­ary. With these col­lab­o­ra­tions in place, BeiGene has pro­mot­ed Ju­lia Wang to CFO, suc­ceed­ing the re­tir­ing Howard Liang, ef­fec­tive June 30. Wang came on board last year as SVP, en­ter­prise op­ti­miza­tion and deputy CFO for John Oyler’s folks. In her last stop at Alex­ion, she was SVP, glob­al busi­ness fi­nance and cor­po­rate plan­ning, and she al­so brings fi­nan­cial ex­pe­ri­ence from J&J and Pep­si­Co.

→ Ger­man CD­MO Rentschler Bio­phar­ma SE has plucked up Mar­tin Kessler as CEO of its US sub­sidiary Rentschler Bio­phar­ma Inc. Kessler comes from McK­in­sey & Com­pa­ny, where he com­plet­ed a 7-year stint as as­so­ciate part­ner. In ad­di­tion to the ti­tle of CEO, Kessler has been named SVP trans­for­ma­tion of Rentschler Bio­phar­ma SE.

Bri­an Alexan­der

→ It on­ly took three years for Bri­an Alexan­der to rise to the top of Roche sub­sidiary Foun­da­tion Med­i­cine, suc­ceed­ing Cindy Perettie as CEO of the ge­nom­ic pro­fil­ing com­pa­ny this com­ing Tues­day. Perettie, who be­came CEO in ear­ly 2019, an­nounced in Jan­u­ary that she would piv­ot to Roche Di­ag­nos­tics and take com­mand of the mol­e­c­u­lar lab so­lu­tions busi­ness. A ra­di­a­tion on­col­o­gist at Dana-Far­ber and Brigham and Women’s Hos­pi­tal, Alexan­der first ar­rived on the scene at Foun­da­tion Med­i­cine as SVP of clin­i­cal de­vel­op­ment in 2018 and was pro­mot­ed to CMO less than a year lat­er.

Car­olyn Star­rett

→ In an­oth­er Roche con­nec­tion, Flat­iron Health — which is an in­de­pen­dent af­fil­i­ate of the Roche Group — will be chang­ing CEOs, with Car­olyn Star­rett tak­ing over for co-founder Nat Turn­er start­ing April 16. On the same date, Flat­iron’s oth­er co-founder Zach Wein­berg, who was a Google staffer along with Turn­er, will al­so move on. Star­rett has led the com­mu­ni­ty on­col­o­gy busi­ness and got her start at Flat­iron in 2016 as VP, cus­tomer ex­pe­ri­ence and op­er­a­tions. Flat­iron was sold to Roche for $1.9 bil­lion back in Feb­ru­ary 2018.

→ Mak­ing its sec­ond sub­stan­tial hire in­volv­ing an Ab­b­Vie vet in as many weeks, No­var­tis has wooed Javier Boix from Rick Gon­za­lez’s crew to be­come glob­al head of com­mu­ni­ca­tions. Boix start­ed out at Ab­b­Vie be­fore the Ab­bott split in 2009 as a cor­po­rate and in­ter­nal com­mu­ni­ca­tions man­ag­er, work­ing his way up the lad­der to even­tu­al­ly lead the phar­ma gi­ant’s US com­mu­ni­ca­tions. Last week, No­var­tis ap­point­ed ex-Ab­b­Vie deputy gen­er­al coun­sel Karen Hale as chief le­gal of­fi­cer.

Pat­ty Allen

Vi­vid­ion now has Jeff Hat­field at the con­trols and $135 mil­lion to work with from a Se­ries C that in­clud­ed back­ing from an All-Star team of in­vestors. The next step this week is mak­ing Pat­ty Allen the per­ma­nent CFO af­ter two months with the in­ter­im tag. Allen’s most re­cent CFO gig was with Zaf­gen, and she’s been VP of fi­nance at Al­ny­lam as well as di­rec­tor of fi­nance at Alk­er­mes. In oth­er Vi­vid­ion news, Bri­an Koh has been giv­en the nod to lead trans­la­tion­al sci­ences and clin­i­cal de­vel­op­ment. Koh brings ex­pe­ri­ence from Gilead and Kite in clin­i­cal R&D in hema­tol­ogy and on­col­o­gy.

Bo Shan

→ The Cel­gene con­nec­tions at An­ten­gene have been fur­ther re­in­forced by re­cruit­ing Kevin Lynch as CMO. Lynch was VP and head of clin­i­cal de­vel­op­ment and med­ical af­fairs in the Asia-Pa­cif­ic ter­ri­to­ry for the lat­ter part of his tenure at Cel­gene, which struck a li­cens­ing deal in 2017 and helped con­tribute to the Chi­nese can­cer biotech’s fi­nanc­ing be­fore Bris­tol My­ers Squibb bought out the com­pa­ny. Else­where at An­ten­gene, Bo Shan has been pro­mot­ed to CSO af­ter his turn as a cor­po­rate VP with dis­cov­ery, ear­ly de­vel­op­ment, and CMC. Pri­or to his ar­rival at An­ten­gene in 2018, Shan was VP in charge of CMC at As­cle­tis.

Rene Rus­so-helmed Xilio, which lined up a $95 Se­ries C in Feb­ru­ary for its an­ti-CT­LA-4 an­ti­body and IL-2 ag­o­nist, has hand­ed CFO du­ties to Sal­va­tore Giovine and made Chris Franken­field gen­er­al coun­sel. Giovine wraps up a 15-year run at J&J, with the last three as Janssen’s fi­nance di­rec­tor in US on­col­o­gy in charge of such drugs as Darza­lex and Im­bru­vi­ca. Franken­field comes to Xilio af­ter near­ly six years at Blue­print Med­i­cines which saw him move up to VP, cor­po­rate le­gal af­fairs.

Christo­pher Wright

Aa­van­tiBio CEO Bo Cum­bo has been grad­u­al­ly as­sem­bling his team with Dou­glas Swirsky and Ty How­ton hit­ting the scene as CFO and COO, re­spec­tive­ly, in the last month. CMO and Ver­tex vet Christo­pher Wright is the lat­est piece in the lead­er­ship puz­zle at Aa­van­tiBio. Wright was pre­vi­ous­ly a CMO with Iron­wood spin­out Cy­cle­ri­on and the chief de­vel­op­ment of­fi­cer at Iron­wood, both of which have fall­en on hard times. At the tail end of his sev­en-year run with Ver­tex, he served as SVP, glob­al med­i­cines de­vel­op­ment and af­fairs.

→ Not long af­ter get­ting the FDA’s seal of ap­proval for Kimyr­sa in acute bac­te­r­i­al skin and skin struc­ture in­fec­tions (AB­SS­SI), Melin­ta Ther­a­peu­tics has tapped John Har­low as chief com­mer­cial of­fi­cer. Har­low has been around the block in the 20 years lead­ing up to the Melin­ta nod; he’s jumped around to biotechs like No­var­tis, Sh­iono­gi and En­do Phar­ma­ceu­ti­cals in var­i­ous sales and mar­ket­ing po­si­tions be­fore fi­nal­ly get­ting his first C-suite break in 2019 as CCO of Bau­dax Bio.

William Kennedy

→ San Fran­cis­co-based Ex­ci­sion Bio­Ther­a­peu­tics, which launched in Feb­ru­ary with $60 mil­lion in to­tal fund­ing — our Ja­son Mast pro­filed the CRISPR work of co-founder and chief sci­en­tif­ic con­sul­tant Kamel Khalili at the time — is bring­ing William Kennedy in­to the fold as SVP of clin­i­cal de­vel­op­ment, start­ing to­day. Kennedy, a Big Phar­ma vet whose cred­its in­clude Janssen, Genen­tech and Mer­ck, comes to Ex­ci­sion from Cal­ci­lytix, where he was VP, de­vel­op­ment. Else­where, he was VP of clin­i­cal de­vel­op­ment at Au­dentes.

Gre­go­ry Chow

Gre­go­ry Chow is tak­ing on an­oth­er CFO chal­lenge by head­ing to chemo­pro­teomics biotech Fron­tier Med­i­cines, which signed a pro­tein degra­da­tion deal with Ab­b­Vie for $55 mil­lion up­front in ear­ly De­cem­ber. Be­fore get­ting the call to join Chris Var­ma’s squad, Chow was EVP and CFO at Ap­tose Bio­sciences and al­so spent time at Wed­bush as man­ag­ing di­rec­tor and di­rec­tor of pri­vate place­ments.

→ Em­bold­ened by pos­i­tive da­ta from two Phase III tri­als of rof­lu­mi­last cream that would make an NDA a pos­si­bil­i­ty lat­er this year, Ar­cutis has filled three po­si­tions with Scott Bur­rows (CFO), Corey Padovano (VP of sales) and Sean Brug­ger (ex­ec­u­tive di­rec­tor of field med­ical af­fairs). Tak­ing over as CFO from John Smither, whose re­tire­ment is ef­fec­tive to­day, Bur­rows has been Ar­cutis’ VP of fi­nance since 2019 and held nu­mer­ous fi­nan­cial posts at Am­gen for 15 years be­fore a year with Shire lead­ing in­ter­na­tion­al in­vestor re­la­tions. Padovano had served at Gilead since 2013 and was the remde­sivir mak­er’s ex­ec­u­tive di­rec­tor, mar­ket­ing op­er­a­tions be­fore his new Ar­cutis gig. Brug­ger, an Am­gen vet in his own right, comes off al­most five years at Alex­ion and was re­cent­ly re­gion­al di­rec­tor of the meta­bol­ic dis­or­ders port­fo­lio.

Abi­gail Jenk­ins

→ Try­ing to put a lid on dai­ly pills, Wa­ter­town, MA-based Lyn­dra Ther­a­peu­tics has named Abi­gail Jenk­ins chief com­mer­cial and busi­ness of­fi­cer and Jacque­line Fer­nan­des VP of da­ta com­pli­ance and in­for­ma­tion tech­nol­o­gy. Jenk­ins be­gan her ca­reer at Pfiz­er in sales and mar­ket­ing, and her most re­cent post was with Emer­gent BioSo­lu­tions as SVP and busi­ness unit head of vac­cines. A 13-year Sunovion vet, Fer­nan­des is the lat­est to jump ship from Sarep­ta, where she served as se­nior di­rec­tor of da­ta com­pli­ance and chief pri­va­cy of­fi­cer.

→ Lon­don eye dis­ease biotech OKYO Phar­ma has called up­on Ra­jku­mar Patil to be CSO. For the last year, Patil had been VP of R&D with Ora (Oph­thalmic Re­search As­so­ci­ates). While he was with Al­con and lat­er the No­var­tis In­sti­tute of Bio­med­ical Re­search, Patil took on such roles as as­so­ciate di­rec­tor of re­search and head of mol­e­c­u­lar phar­ma­col­o­gy glau­co­ma and reti­na re­search.

Den­nis Giesing

→ Penn­syl­va­nia biotech Im­munome, which scored a De­part­ment of De­fense con­tract to de­vel­op biosyn­thet­ic con­va­les­cent plas­ma, has en­list­ed Den­nis Giesing as chief de­vel­op­ment of­fi­cer. Pri­or to Im­munome, Giesing was the long­time CSO at Taris Bio­med­ical un­til J&J bought the com­pa­ny in late 2019, at which point he tran­si­tioned to J&J’s on­col­o­gy team.

Mon­tis Bio­sciences has re­cruit­ed René Hoet as CSO. Hoet most re­cent­ly served as CSO at Im­check Ther­a­peu­tics and was pre­vi­ous­ly VP bi­o­log­ics re­search at Bay­er for 8 years. Pri­or to that, he was at Gen­mab.

→ Dig­i­tal health com­pa­ny Genome Med­ical has brought on Mar­co Bar­cel­la as chief prod­uct of­fi­cer and Bran­don Poe as CFO. Bar­cel­la most re­cent­ly served at Tia­tros and IBM Wat­son Health. Mean­while, Poe comes from Il­lu­mi­na, where he served as VP of fi­nance.

Clau­dia Nar­di­ni

En­thera Phar­ma­ceu­ti­cals has ap­point­ed Clau­dia Nar­di­ni as VP of re­search and de­vel­op­ment. Nar­di­ni had been head of glob­al pu­rifi­ca­tion & virus clear­ance prod­uct man­age­ment at Sar­to­rius Ste­d­im Biotech GmbH, and be­fore that she served at Bay­er and Kedri­on Bio­phar­ma.

Janux Ther­a­peu­tics, the small biotech that Mer­ck inked a $1 bil­lion deal with — has named Charles Win­ter as SVP of chem­istry, man­u­fac­tur­ing, and con­trols. Win­ter brings to the ta­ble a wealth of ex­pe­ri­ence from com­pa­nies like Gilead, De­nali, Anap­tys­Bio and Syn­thorx (now a Sanofi com­pa­ny). Win­ter launched his ca­reer at Genen­tech, where he spent 17 years.

Evonetix has ap­point­ed Michael Daniels as head of prod­uct man­age­ment. Daniels brings ex­pe­ri­ence from his times at Ar­cis Biotech­nol­o­gy, Can­cer Re­search UK, Ther­mo Fish­er and GE Health­care Life Sci­ences.

Mace Rothen­berg

Mace Rothen­berg’s post-Pfiz­er plans con­tin­ue to evolve by tak­ing on an­oth­er board ap­point­ment, this time with Au­los Bio­science, a biotech con­cen­trat­ing on (IL-2)-bind­ing mon­o­clon­al an­ti­bod­ies that Michael Ehlers co-found­ed. Just a week ago, the ex-Pfiz­er CMO joined the board of di­rec­tors at Tan­go Ther­a­peu­tics.

Michael Var­ney has now made his way to the ad­vi­so­ry board of Phoenix Mol­e­c­u­lar De­signs, which fo­cus­es on RSK ki­nase in­hibitors to fight triple neg­a­tive breast can­cer. Life af­ter re­tire­ment at Genen­tech has been kind to Var­ney, who is cur­rent­ly chair­man of R&D at Eras­ca and is al­so a se­nior ad­vi­sor at Fra­zier Health­care Part­ners.

Julie Ham­ble­ton

→ Ex-Bris­tol My­ers Squibb and Five Prime ex­ec Julie Ham­ble­ton has se­cured a spot on the board of di­rec­tors at Jonathan Lim-led can­cer biotech Eras­ca, along with Bi­hua Chen. Ham­ble­ton, who was al­so in clin­i­cal de­vel­op­ment at Genen­tech, is Arch On­col­o­gy’s in­ter­im pres­i­dent and CEO. Chen, who co-found­ed Cor­morant As­set Man­age­ment, fol­lowed the SPAC herd last Oc­to­ber with He­lix Ac­qui­si­tion.

→ With the FDA sign­ing off last month on Kro­nos Bio’s Phase III tri­al of en­tosple­tinib — one of the drugs Nor­bert Bischof­berg­er plucked from Gilead — the biotech has made Taiyin Yang a mem­ber of the board of di­rec­tors. Yang is a 28-year Gilead vet who is cur­rent­ly EVP of phar­ma­ceu­ti­cal de­vel­op­ment and man­u­fac­tur­ing for Dan O’Day’s crew.

Nagesh Ma­han­thap­pa

→ De­but­ing in De­cem­ber with a $25 mil­lion round based on work from Har­vard’s David Liu and Alan Saghatelian, Exo Ther­a­peu­tics has elect­ed Nagesh Ma­han­thap­pa to the board of di­rec­tors. Ma­han­thap­pa, who found­ed and helmed Schol­ar Rock, is al­so a board mem­ber at Cas­ma Ther­a­peu­tics. Exo has al­so named Charles Roberts from St. Jude Chil­dren’s Re­search Hos­pi­tal to the sci­en­tif­ic ad­vi­so­ry board.

PYC Ther­a­peu­tics has se­lect­ed Ja­son Had­dock to be a part of its board of di­rec­tors. This isn’t Had­dock’s first board ap­point­ment, as he al­so cur­rent­ly sits on the board of Co­di­ak Bio­sciences. Pre­vi­ous­ly, Had­dock served as CFO of Ar­ray Bio­phar­ma, and pri­or to that, he was at Bris­tol My­ers Squibb and ArcherDX.

→ Tel Aviv, Is­rael-based Chemomab Ther­a­peu­tics has ex­pand­ed its board of di­rec­tors with four new ap­point­ments. The new hires are Alan Moses, Claude Nicaise, Joel Maryles and Neil Co­hen.

Claude Nicaise

Moses comes with a 14-year stint at No­vo Nordisk un­der his belt, where he served as SVP and glob­al chief med­ical of­fi­cer. Mean­while, Nicaise was SVP of strate­gic de­vel­op­ment and glob­al reg­u­la­to­ry af­fairs at Alex­ion af­ter a 21 year gig at Bris­tol My­ers Squibb.

Maryles hops aboard af­ter ca­reers at Our­Crowd and Cit­i­group In­vest­ment Bank­ing. On the oth­er hand, Co­hen — for­mer­ly the in­ter­im CEO of An­chi­ano Ther­a­peu­tics — comes along af­ter stints at Cas­tel Part­ners and Is­rael Seed Part­ners.

COM­PASS Path­ways has wel­comed Wayne Ri­ley to its board of di­rec­tors. Ri­ley is pres­i­dent of the State Uni­ver­si­ty of New York Down­state Health Sci­ences Uni­ver­si­ty, Brook­lyn and was pre­vi­ous­ly a di­rec­tor at Ver­tex.

Catharine Smith

→ The non-prof­it Ter­meer Foun­da­tion has brought on Catharine Smith as the or­ga­ni­za­tion’s ex­ec­u­tive di­rec­tor. Smith hails from the Clin­ton Foun­da­tion, where she serves as CEO, Clin­ton Health Mat­ters Ini­tia­tive. Pri­or to that, she held var­i­ous lead­er­ship roles at Har­vard Med­ical School.

→ San Fran­cis­co-based Bet­ter Ther­a­peu­tics has signed on An­drew Ar­mani­no and Ge­of­frey Park­er to its board of di­rec­tors. Ar­mani­no re­cent­ly re­tired as man­ag­ing part­ner and CEO at CPA firm Ar­mani­no. Mean­while, Park­er cur­rent­ly serves as COO, CFO and EVP of Tri­ci­da. Pre­vi­ous­ly, Park­er was CFO of Ana­cor Phar­ma­ceu­ti­cals and a man­ag­ing di­rec­tor at Gold­man Sachs.

Ma­ri­na Bozilenko has joined Acel­Rx’s board of di­rec­tors. Bozilenko, a Bear Stearns alum, is present­ly a strate­gic ad­vi­sor for William Blair af­ter 11 years as the in­vest­ment firm’s head of biotech & phar­ma and man­ag­ing di­rec­tor.

BY­OD Best Prac­tices: How Mo­bile De­vice Strat­e­gy Leads to More Pa­tient-Cen­tric Clin­i­cal Tri­als

Some of the most time- and cost-consuming components of clinical research center on gathering, analyzing, and reporting data. To improve efficiency, many clinical trial sponsors have shifted to electronic clinical outcome assessments (eCOA), including electronic patient-reported outcome (ePRO) tools.

In most cases, patients enter data using apps installed on provisioned devices. At a time when 81% of Americans own a smartphone, why not use the device they rely on every day?

Voting in the 2020 election (AP Images)

The right to vote is fun­da­men­tal — a let­ter from biotech­nol­o­gy in­dus­try lead­ers

Biotech Voices is a collection of exclusive opinion editorials from some of the leading voices in biopharma on the biggest industry questions today. Think you have a voice that should be heard? Reach out to senior editors Kyle Blankenship and Amber Tong.

We oppose all attempts to introduce laws that reduce the rights of US citizens to vote or that restrict them from exercising that right. The right to vote is fundamental to democracy. States that have enacted, or are proposing to enact, legislation to restrict voting are undermining our democracy and posing a threat to our nation. As leaders of the life sciences industry, we stand for what we believe is right for our country, our enterprises, our employees and those who benefit from our work. We join the first groups of business leaders who have challenged these laws and will continue to make our collective voices heard on this matter.

Pascal Soriot (AstraZeneca via YouTube)

Af­ter be­ing goad­ed to sell the com­pa­ny, Alex­ion's CEO set some am­bi­tious new goals for in­vestors. Then Pas­cal So­ri­ot came call­ing

Back in the spring of 2020, Alexion $ALXN CEO Ludwig Hantson was under considerable pressure to perform and had been for months. Elliott Advisers had been applying some high public heat on the biotech’s numbers. And in reaching out to some major stockholders, one thread of advice came through loud and clear: Sell the company or do something dramatic to change the narrative.

In the words of the rather dry SEC filing that offers a detailed backgrounder on the buyout deal, Alexion stated: ‘During the summer and fall of 2020, Alexion also continued to engage with its stockholders, and in these interactions, several stockholders encouraged the company to explore strategic alternatives.’

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Near­ly a year af­ter Au­den­tes' gene ther­a­py deaths, the tri­al con­tin­ues. What hap­pened re­mains a mys­tery

Natalie Holles was five months into her tenure as Audentes CEO and working to smooth out a $3 billion merger when the world crashed in.

Holles and her team received word on the morning of May 5 that, hours before, a patient died in a trial for their lead gene therapy. They went into triage mode, alerting the FDA, calling trial investigators to begin to understand what happened, and, the next day, writing a letter to alert the patient community so they would be the first to know. “We wanted to be as forthright and transparent as possible,” Holles told me late last month.

The brief letter noted two other patients also suffered severe reactions after receiving a high dose of the therapy and were undergoing treatment. One died a month and a half later, at which point news of the deaths became public, jolting an emergent gene therapy field and raising questions about the safety of the high doses Audentes and others were now using. The third patient died in August.

“It was deeply saddening,” Holles said. “But I was — we were — resolute and determined to understand what happened and learn from it and get back on track.”

Eleven months have now passed since the first death and the therapy, a potential cure for a rare and fatal muscle-wasting disease called X-linked myotubular myopathy, is back on track, the FDA having cleared the company to resume dosing at a lower level. Audentes itself is no more; last month, Japanese pharma giant Astellas announced it had completed working out the kinks of the $3 billion merger and had restructured and rebranded the subsidiary as Astellas Gene Therapies. Holles, having successfully steered both efforts, departed.

Still, questions about precisely what led to the deaths of the 3 boys still linger. Trial investigators released key details about the case last August and December, pointing to a biological landmine that Audentes could not have seen coming — a moment of profound medical misfortune. In an emerging field that’s promised cures for devastating diseases but also seen its share of safety setbacks, the cases provided a cautionary tale.

Audentes “contributed in a positive way by giving a painful but important example for others to look at and learn from,” Terry Flotte, dean of the UMass School of Medicine and editor of the journal Human Gene Therapy, told me. “I can’t see anything they did wrong.”

Yet some researchers say they’re still waiting on Astellas to release more data. The company has yet to publish a full paper detailing what happened, nor have they indicated that they will. In the meantime, it remains unclear what triggered the events and how to prevent them in the future.

“Since Audentes was the first one and we don’t have additional information, we’re kind of in a holding pattern, flying around, waiting to figure out how to land our vehicles,” said Jude Samulski, professor of pharmacology at UNC’s Gene Therapy Center and CSO of the gene therapy biotech AskBio, now a subsidiary of Bayer.

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Launched by MIT grads, a small start­up gets $20M to back a ro­bot­ics rev­o­lu­tion in cell ther­a­py man­u­fac­tur­ing

As co-director of an experimental cellular therapy process development and manufacturing group at UCSF specializing in T cell therapies for autoimmune conditions, Jonathan Esensten has learned a lot about the challenges involved when his group hand-fashions a cell therapy. Esensten — who was a postdoc in Wendell Lim’s lab and counts the legendary Jeffrey Bluestone as a mentor — gives them all high marks at being great at what they do, but time and again there are variations in the treatments they construct.

Anand Shah (FDA)

For­mer head of FDA’s med­ical and sci­en­tif­ic af­fairs on Covid: ‘FDA has nev­er been test­ed like this’

Anand Shah has served the American public in a unique way, crisscrossing over the last two administrations between serving as an attending radiation oncologist focused on prostate cancer at NIH, serving as CMO at the Center for Medicare and Medicaid Innovation, and most recently, leading the FDA’s operations on medical and scientific affairs from within the commissioner’s office.

Shah, who stepped down from the FDA in January, caught up with Endpoints News in a phone interview on Tuesday afternoon, offering his thoughts on the agency’s latest decision to pause the J&J vaccinations in the US, and reflecting on his time at an agency during this once-in-a-lifetime pandemic.

UP­DAT­ED: J&J paus­es vac­cine roll­out as feds probe rare cas­es of blood clots

The FDA and CDC have jointly decided to stop administering J&J’s Covid-19 vaccine after reviewing data involving six reported US cases of a rare and severe type of blood clot in individuals after receiving the vaccine.

CDC will convene a meeting of its Advisory Committee on Immunization Practices on Wednesday to further review these cases and assess their potential significance. “FDA will review that analysis as it also investigates these cases. Until that process is complete, we are recommending a pause in the use of this vaccine out of an abundance of caution,” Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research and Anne Schuchat, Principal Deputy Director of the CDC, said in a joint statement Tuesday morning.

Patrizia Cavazzoni, new CDER director

Pa­trizia Cavaz­zoni named per­ma­nent di­rec­tor of CDER, adding to ques­tions around where Wood­cock will end up

Patrizia Cavazzoni on Monday became the permanent director of the FDA’s Center for Drug Evaluation and Research, which puts to rest the idea that Janet Woodcock, Cavazzoni’s predecessor, might return to lead CDER if she isn’t made permanent commissioner.

Woodcock, who’s currently serving as acting commissioner and principal medical advisor to the commissioner, a position she was detailed to last year, may not make the move to permanent commissioner because of lingering questions from Senate Democrats. She previously served as director of CDER since 1994. Cavazzoni took over as acting director of CDER when Woodcock moved over to Operation Warp Speed to run the therapeutics side of the Trump-era program.

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Barbara Weber, Tango Therapeutics CEO (Tango)

It takes two to Tan­go: The biotech us­ing CRISPR to dis­cov­er new can­cer gene tar­gets rides a $353M SPAC deal to Nas­daq

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

The latest biotech-SPAC deal has arrived, and it’s dancing its way to Nasdaq to the tune of several hundred million dollars.

Tango Therapeutics and its CRISPR-focused search for new cancer genes is reverse merging with Boxer Capital’s blank-check company, the biotech announced Wednesday morning. With a spotlight on three lead programs, Tango expects total proceeds to equal about $353 million in the deal, which includes the roughly $167 million held in the SPAC and an additional $186 million in PIPE financing.