Genen­tech gains clin­i­cal de­vel­op­ment ex­ec by ap­point­ing Lil­li Petruzzel­li; Paul Sekhri caps event­ful week, nam­ing Sap­na Sri­vas­ta­va CFO of eGe­n­e­sis

Lil­li Petruzzel­li

→ A spokesper­son for Genen­tech tells End­points News that Lil­li Petruzzel­li has forged a new path this week as SVP, ear­ly clin­i­cal de­vel­op­ment at Genen­tech’s Re­search and Ear­ly De­vel­op­ment or­ga­ni­za­tion (gRED). Petruzzel­li just spent two years as In­cyte’s group VP, ear­ly clin­i­cal de­vel­op­ment, and pri­or to In­cyte, she was glob­al head, trans­la­tion­al clin­i­cal on­col­o­gy at the No­var­tis In­sti­tutes of Bio­med­ical Re­search.

“Lil­li Petruzzel­li has a re­mark­able track record of part­ner­ing close­ly with dis­cov­ery teams to ad­vance in­no­v­a­tive mol­e­cules from re­search in­to first-in-hu­man stud­ies and through proof-of-con­cept,” Genen­tech’s head of gRED Aviv Regev said in a state­ment.

Sap­na Sri­vas­ta­va

→ Just a cou­ple days af­ter their Se­ries C haul, Paul Sekhri-led eGe­n­e­sis has a new CFO in place with Sap­na Sri­vas­ta­va. She’s pre­vi­ous­ly been chief fi­nan­cial and strat­e­gy of­fi­cer at Abide Ther­a­peu­tics, which Lund­beck ac­quired in the spring of 2019 in a $250 mil­lion deal. The ex-Mor­gan Stan­ley and Gold­man Sachs biotech an­a­lyst was al­so chief fi­nan­cial and strat­e­gy of­fi­cer at In­tel­lia Ther­a­peu­tics. Since its de­but three years ago this month, eGe­n­e­sis — which has been ge­net­i­cal­ly al­ter­ing pigs for the pur­pose of or­gan trans­plan­ta­tion in hu­mans — has to­taled more than $263 mil­lion in fund­ing, with al­most half ($125 mil­lion) com­ing in this week’s megaround.

→ While you or some­one you know have re­ceived their mR­NA vac­cine, Mod­er­na is fill­ing out lead­er­ship po­si­tions re­lat­ed to oth­er tar­gets, with Jonathan Hog­gatt locked in as di­rec­tor of hema­tol­ogy. Hog­gatt is a prin­ci­pal fac­ul­ty mem­ber at the Har­vard Stem Cell In­sti­tute and runs the Hog­gatt Lab, which con­cen­trates on tis­sue re­gen­er­a­tion and stem cell bi­ol­o­gy. He’s al­so a sci­en­tif­ic co-founder of Ma­gen­ta Ther­a­peu­tics.

Christophe Bour­don

Or­p­hazyme had been bask­ing in the glow of the FDA ac­cept­ing its NDA a few months ear­li­er for ari­mo­clo­mol in Nie­mann-Pick dis­ease type C pa­tients, and now Christophe Bour­don is helm­ing the Dan­ish biotech af­ter Kim Strat­ton de­cid­ed to re­sign Dec. 20. Bour­don takes con­trol at the Copen­hagen com­pa­ny af­ter his sec­ond stint at Am­gen, where he was SVP and GM of the US on­col­o­gy busi­ness. The Sanofi vet was Alex­ion’s SVP of Eu­rope, Mid­dle East, Africa, and Cana­da (EMEAC) in a two-year stay that was sand­wiched in be­tween his time at Am­gen.

Mark Sten­house

Pen­cil­ing in a $125 mil­lion IPO last month af­ter scor­ing $130 mil­lion in fund­ing back in No­vem­ber, Mark McKen­na-led Prometheus Bio­sciences is hand­ing the role of COO to Mark Sten­house. He’s an Ab­b­Vie vet of al­most 30 years (1990-2018) who was the phar­ma gi­ant’s VP of US im­munol­o­gy and, be­fore that, VP and GM of the gas­troen­terol­o­gy fran­chise. In all, he spent 10 years with Ab­b­Vie in IBD, which is San Diego-based Prometheus’ fo­cus. Af­ter Ab­b­Vie, Sten­house was GM, screen­ing busi­ness at Ex­act Sci­ences, lead­ing com­mer­cial du­ties with the col­orec­tal can­cer de­tec­tion test Co­lo­guard.

Joel Schnei­der

Sol­id Bio’s clin­i­cal hold for its Duchenne mus­cu­lar dy­s­tro­phy drug lift­ed last Oc­to­ber, fend­ing off re­peat­ed slings and ar­rows from the FDA. Now Sol­id has pro­mot­ed Joel Schnei­der to COO and Erin Pow­ers Bren­nan is on board as chief le­gal of­fi­cer. Schnei­der — who was chief tech­ni­cal of­fi­cer and head of ex­plorato­ry R&D be­fore set­tling in his new gig on Tues­day — has spent sev­en years at Sol­id over­all, while Bren­nan leaves Cov­etrus, where she was SVP, gen­er­al coun­sel & sec­re­tary. Else­where at Sol­id, for­mer Glax­o­smithK­line and Pfiz­er reg­u­la­to­ry ex­ec Clare Kahn and ex-Take­da COO of R&D Geor­gia Ker­esty have been waved in to the Cam­bridge biotech’s board of di­rec­tors.

Si­mon Coop­er has suc­ceed­ed John Ryan as CMO of Kad­mon as Ryan piv­ots to the new role of ex­ec­u­tive med­ical di­rec­tor, clin­i­cal and reg­u­la­to­ry de­vel­op­ment. Coop­er has cer­tain­ly made the Big Phar­ma rounds as a clin­i­cal sci­ence leader at Roche, a glob­al pro­gram di­rec­tor at No­var­tis, the glob­al project head for im­munol­o­gy and in­flam­ma­tion at Sanofi, and as­set strat­e­gy di­rec­tor for Skyrizi with Ab­b­Vie. Af­ter his work with those heavy hit­ters, Coop­er took his first CMO gig with Anokion in 2019 be­fore of­fi­cial­ly start­ing at Kad­mon on Mon­day.

Sean Zhang

→ Bei­jing-based can­cer biotech In­no­Care, which racked up $289 mil­lion in its Hong Kong IPO in March 2020, has brought on Sean Zhang as CMO, leav­ing his CEO post at Hen­grui Ther­a­peu­tics. Be­fore get­ting pro­mot­ed to the top spot, Zhang was Hen­grui’s CMO and a board mem­ber. He too has hop­scotched around Big Phar­ma most of his ca­reer, first as a re­search sci­en­tist in Mer­ck and then as a clin­i­cal leader at J&J. He al­so spent four years in dis­cov­ery med­i­cine and clin­i­cal phar­ma­col­o­gy at Bris­tol My­ers Squibb and an­oth­er three years in clin­i­cal de­vel­op­ment at Glax­o­SmithK­line.

Aa­van­tiBioBo Cum­bo’s post-Sarep­ta gene ther­a­py en­deav­or — has en­trust­ed the CFO and trea­sur­er du­ties to Dou­glas Swirsky sev­er­al months af­ter tak­ing the tarp off with a meaty $107 mil­lion launch round. Swirsky, who had been in a con­sult­ing role at Aa­van­tiBio since Feb­ru­ary, helmed Rex­ahn Phar­ma­ceu­ti­cals for two years and has al­so been pres­i­dent and CEO of Gen­Vec. He al­so chairs the board of di­rec­tors at Cel­lec­tar Bio­sciences and Neu­roBo Phar­ma­ceu­ti­cals.

Wildon Far­well

→ With its oligonu­cleotide ther­a­py pipeline for rare mus­cle dis­eases in de­vel­op­ment, 2020 IPO boom par­tic­i­pant Dyne Ther­a­peu­tics has cor­ralled Wildon Far­well as CMO soon af­ter Ash Dugar was ap­point­ed glob­al head of med­ical af­fairs. Far­well spent a decade at Bio­gen, lead­ing the de­vel­op­ment of oligonu­cleotide Spin­raza and hold­ing nu­mer­ous posts that cul­mi­nat­ed in his role as VP, glob­al head of neu­ro­mus­cu­lar dis­eases, med­ical af­fairs.

Jes­si­ca Stitt

→ Fresh off sparkling da­ta for AMD gene ther­a­py GT005, Lon­don-based Gy­ro­scope Ther­a­peu­tics has wel­comed Jes­si­ca Stitt as CFO. Stitt, who’s al­so held fi­nance po­si­tions at Alk­er­mes and Ther­a­vance, was pre­vi­ous­ly VP of fi­nance and op­er­a­tions at MyoKar­dia, which Bris­tol My­ers Squibb swooped in to buy for more than $13 bil­lion back in Oc­to­ber.

→ New York-based An­ti­body Ra­di­a­tion-Con­ju­gate (ARC) phar­ma Ac­tini­um ap­point­ed two clin­i­cal de­vel­op­ment ex­ecs (Avinash De­sai and Mary Mei Chen) in a two-week span late last fall, and now Mark Ku­bik has ac­cept­ed the role of Ac­tini­um’s CBO. A for­mer busi­ness de­vel­op­ment di­rec­tor at such biotechs as Macro­Gen­ics and Seat­tle Ge­net­ics (now Seagen), Ku­bik makes the tran­si­tion to Ac­tini­um af­ter a brief stay as CBO at On­coIm­mune, which Mer­ck bought for $425 mil­lion in a deal that was bro­kered by then-Op­er­a­tion Warp Speed chief Mon­cef Slaoui.

Mary Kay Fen­ton

→ Long­time Achillion CFO Mary Kay Fen­ton will take on the same re­spon­si­bil­i­ty at Ta­laris Ther­a­peu­tics, which land­ed a $115 mil­lion Se­ries B in its quest to help make or­gan trans­plan­ta­tion safer for pa­tients. Fen­ton was VP of strate­gic op­er­a­tions at Ver­tex’s cell and ge­net­ic ther­a­pies unit, and had been Sem­ma’s CFO and COO be­fore Ver­tex bought the com­pa­ny for $950 mil­lion in 2019.

→ Six months af­ter Sven Rohmann grabbed hold of the reins as CEO, Swedish can­cer biotech Im­mu­nicum AB has tapped Jeroen Rovers to be CMO. Rovers hails from DCprime, an­oth­er den­drit­ic cell ther­a­py out­fit which merged with Im­mu­nicum to­ward the end of last year, where he had been CMO since 2018. The ex-CMO at Kiadis and Ceron­co Bio­sciences re­places Pe­ter Sue­naert, who sticks with Im­mu­nicum as a clin­i­cal ad­vi­sor. Rovers’ ap­point­ment fol­lows that of Lot­ta Ferm as in­ter­im CFO and comes af­ter Im­mu­nicum’s or­phan drug des­ig­na­tion for il­ix­aden­cel to treat he­pa­to­cel­lu­lar car­ci­no­ma.

Al­i­son Schecter

Flag­ship-backed Ax­cel­la, which has lead drug can­di­dates for NASH and he­pat­ic en­cephalopa­thy in its pipeline, has made Al­i­son Schecter pres­i­dent of R&D af­ter com­plet­ing a year as CMO of Se­lec­ta Bio­sciences. Just be­fore that, Schecter was Sanofi Gen­zyme’s glob­al project head, rare dis­eases and the glob­al pro­gram head for he­mo­phil­ia at Bax­al­ta. She al­so brings ex­pe­ri­ence from the North­east J&J In­no­va­tion Cen­ter and the No­var­tis In­sti­tutes of Bio­med­ical Re­search.

Schond Green­way is jump­ing on board as CFO of Cere­cor, suc­ceed­ing Chris Sul­li­van, who had been in the in­ter­im CFO slot for a year and will now be­come the Mary­land phar­ma’s chief ac­count­ing of­fi­cer. Green­way had pre­vi­ous­ly been Mesoblast’s VP, in­vestor re­la­tions and was al­so an ex­ec­u­tive di­rec­tor in in­vestor re­la­tions at Halozyme.

→ Strik­ing up a $10 mil­lion up­front pact with Sarep­ta last sum­mer to de­vel­op im­li­fi­dase as a pre-treat­ment for DMD and LGMD pa­tients, Hansa Bio­phar­ma out of Swe­den has giv­en the nod to Mag­nus Ko­rs­gren to be head of R&D. An As­traZeneca and No­var­tis vet, Ko­rs­gren has de­vot­ed the last six years to Fer­ring Phar­ma­ceu­ti­cals, more re­cent­ly as VP, head of trans­la­tion­al med­i­cine.

Ger­ald Cox

→ Ex-Ed­i­tas Med­i­cine CMO Ger­ald Cox has been named act­ing CMO at Gainesville, FL biotech Cy­clo Ther­a­peu­tics, which is work­ing on cy­clodex­trin-based drugs that tar­get Nie­mann-Pick dis­ease type C and Alzheimer’s. Cox, a long­time staff physi­cian at Boston Chil­dren’s Hos­pi­tal, had spent more than 15 years at Gen­zyme, leav­ing as VP of rare dis­ease clin­i­cal de­vel­op­ment be­fore his two years at Ed­i­tas.

Gary Sender has re­tired as CFO of Dublin-based Nabri­va Ther­a­peu­tics, and start­ing March 12, Daniel Dolan will take his place. The 12-year Shire vet had been Ra­dius Health’s VP of fi­nan­cial plan­ning & analy­sis since 2017. Nabri­va got side­tracked with a CRL in June 2020 for its an­tibi­ot­ic Con­tepo, with man­u­fac­tur­ing is­sues at Nabri­va’s Eu­ro­pean CMO site as the main cul­prit. Nabri­va pinned the lack of re­sponse to the FDA’s con­cerns on trav­el re­stric­tions.

Leonard Pao­lil­lo

→ Seat­tle-based Im­pel Neu­roPhar­ma, with its pri­ma­ry fo­cus on CNS dis­eases, has a pair of new faces with Leonard Pao­lil­lo as chief com­mer­cial of­fi­cer and Ger­ald Penn as VP, mar­ket ac­cess & trade. Pao­lil­lo makes the leap from Ky­owa Kirin’s North Amer­i­can af­fil­i­ate, where he was EVP and CCO, while Penn just held the same ti­tle at Es­pe­ri­on that he does at Im­pel Neu­roPhar­ma. The new lead­er­ship comes af­ter Im­pel Neu­roPhar­ma brought Amarin alum Rigo Canal in­to the fold as VP, com­mer­cial op­er­a­tions and an­a­lyt­ics.

→ British biotech 4D phar­ma got in on the SPAC ac­tion, merg­ing with blank check com­pa­ny Longevi­ty back in Oc­to­ber, and in an­tic­i­pa­tion of break­ing through on Nas­daq, the mi­cro­bio­me play­er has re­cruit­ed John Beck as CFO. Beck’s most re­cent CFO ex­pe­ri­ence was a two-year run at Rit­ter Phar­ma­ceu­ti­cals from 2018-20. Else­where at 4D phar­ma, ex-Lig­and and Se­quenom CFO Paul Maier has climbed on to the board as a non-ex­ec­u­tive di­rec­tor.

Nicholas Hols­man

→ Ire­land’s Shorla Phar­ma, which pulled in $8.3 mil­lion in a mod­est Se­ries A in June 2020, has en­list­ed Nicholas Hols­man as chief com­mer­cial of­fi­cer. Be­fore tran­si­tion­ing to the on­col­o­gy play­er, Hols­man had led com­mer­cial op­er­a­tions since the sum­mer of 2019 at On­copep­tides, now helmed by for­mer Toca­gen CEO Mar­ty Du­vall.

→ With pro­grams in se­vere in­flam­ma­to­ry dis­eases (ABX464) and he­pa­to­cel­lu­lar car­ci­no­ma (ABX196), Paris-based Abi­vax has se­lect­ed So­phie Biguenet as CMO, re­plac­ing the re­tir­ing Jean-Marc Steens. Biguenet’s Big Phar­ma ex­pe­ri­ence traces back to Bio­gen and Bris­tol My­ers Squibb and she’s been Ab­b­Vie’s med­ical di­rec­tor and pe­di­atric lead for HCV, RSV and HIV glob­al pe­di­atric clin­i­cal de­vel­op­ment. Biguenet joins Abi­vax af­ter hold­ing her first CMO post at Swiss biotech Ver­san­tis.

→ Ich­nos Sci­ences has re­cruit­ed Michael D Price as CFO. Price for­mer­ly served as VP and CFO of Akcea Ther­a­peu­tics and held po­si­tions at Novleion Ther­a­peu­tics, Noven Phar­ma­ceu­ti­cals and Bent­ley Phar­ma­ceu­ti­cals.

Byung Ha Lee

T-cell-fo­cused NeoIm­muneTech has pro­mot­ed Byung Ha Lee to the po­si­tion of CSO. Lee joined the com­pa­ny in 2016 as prin­ci­pal sci­en­tist be­fore be­ing named di­rec­tor of R&D in 2019. Pri­or to his roles at NeoIm­muneTech, Lee was at Sam­sung Bio­med­ical Re­search In­sti­tute and at Genex­ine.

→ A fa­mil­iar face and a new staffer have shored up the C-suite at Bay Area biotech Cor­cept Ther­a­peu­tics, with Atabak Mokari as CFO and his pre­de­ces­sor Charles Robb step­ping in as CBO. Mokari was pre­vi­ous­ly the CFO for Bel­licum, while Robb slides in­to the CBO po­si­tion af­ter nine years as Cor­cept’s CFO. Cor­cept’s drug Ko­r­lym was ap­proved in 2012 for en­doge­nous Cush­ing’s syn­drome.

Richard Pye

Sen­syne Health has named Richard Pye as CFO and ex­ec­u­tive di­rec­tor. Pye pre­vi­ous­ly served as chief in­vest­ment of­fi­cer for the com­pa­ny and hopped aboard from Sum­mit Ther­a­peu­tics. In ad­di­tion, the com­pa­ny’s in­ter­im CFO Michael Nor­ris and Ge­off Race are join­ing the board of di­rec­tors as non-ex­ec­u­tive di­rec­tors. Nor­ris is cur­rent­ly di­rec­tor and CFO of Beck­ley Psytech, while Race is the co-founder, EVP, CFO and CBO of Min­er­va Neu­ro­sciences.

ERS Ge­nomics has ap­point­ed Jon Kra­tochvil as VP for busi­ness de­vel­op­ment & li­cens­ing for North Amer­i­ca. Kra­tochvil hops aboard from Mil­l­li­pore­Sig­ma, where he was di­rec­tor of busi­ness de­vel­op­ment and li­cens­ing. His oth­er roles in­clude stints at Wash­ing­ton Uni­ver­si­ty and Ab­bott Lab­o­ra­to­ries.

eTh­eR­NA has reeled in David Rick­etts as con­sul­tant di­rec­tor of busi­ness de­vel­op­ment for man­u­fac­tur­ing ser­vices. Rick­etts comes aboard with ex­pe­ri­ence as CEO of Phar­ma­Di­ag­nos­tics and Link Tech­nolo­gies.

Mar­tin Chavez

Mar­tin Chavez has been elect­ed chair­man of the board at Chris Gib­son-helmed Re­cur­sion, the Salt Lake City drug de­vel­op­er that raised a hefty $239 mil­lion Se­ries D last Sep­tem­ber and is part­ner­ing with Bay­er on fi­brot­ic dis­eases. Chavez, a mem­ber of Re­cur­sion’s board for al­most a year, was with Gold­man Sachs from 2005-20 in a num­ber of ca­pac­i­ties such as chief in­for­ma­tion of­fi­cer, CFO and glob­al co-head of the Se­cu­ri­ties Di­vi­sion.

→ Plant-based tech com­pa­ny Ca­lyxt has pulled Yves Ribeill as ex­ec­u­tive chair of the board of di­rec­tors as the com­pa­ny ini­ti­ates a search for a new CEO to suc­ceed ex­it­ing Jim Blome, who is pur­su­ing oth­er op­por­tu­ni­ties. Ribeill has served on the board of di­rec­tors for Ca­lyxt since Ju­ly 2018 and for a time served as the com­pa­ny’s in­ter­im CEO from Au­gust 2018 to Oc­to­ber 2018. Ribeill is the CEO of Ri­bo­gen­ics and a founder and for­mer CEO of Scynex­is. Pri­or to these roles, he served at Rhône-Poulenc and Aven­tis in France and the UK.

Don­nie Mc­Grath

→ Along with this week’s news of Ar­ti­zan Bio­sciences’ $11 mil­lion Se­ries A-2 round and a part­ner­ship with Bio­haven, New Haven, CT-based Ar­ti­zan Bio­sciences is al­so adding Don­nie Mc­Grath and Seth Rud­nick to the board of di­rec­tors. A Bris­tol My­ers Squibb alum, Mc­Grath is Bio­haven’s chief of cor­po­rate strat­e­gy and busi­ness de­vel­op­ment, and ex-Canaan part­ner Rud­nick was chair­man and CEO of Cy­toTher­a­peu­tics.

Robert Gagnon has joined the board of di­rec­tors at Is­raeli on­col­o­gy out­fit Pur­ple Biotech. The Bio­gen alum has been Ve­rastem’s chief busi­ness and fi­nan­cial of­fi­cer since 2018.

June Bray

BioX­cel has re­served a seat for Al­ler­gan vet June Bray on its board of di­rec­tors. Bray, who’s served as Al­ler­gan’s SVP, glob­al reg­u­la­to­ry af­fairs and med­ical writ­ing, has al­so been a reg­u­la­to­ry ex­ec at Organon.

→ Red­wood City, Cal­i­for­nia-based Am­Max Bio has en­list­ed John Var­i­an for its board of di­rec­tors as an in­de­pen­dent board mem­ber. Var­i­an joins with ex­pe­ri­ence from his time as CEO and di­rec­tor of XO­MA. Pri­or to XO­MA, Var­i­an was COO of Aryx Phar­ma­ceu­ti­cals and CFO of An­er­gen, Genset and Neurex. Var­i­an al­so sits on the board of Stel­las Life Sci­ences.

→ Gas­troen­terol­o­gy com­pa­ny 9 Me­ters Bio­phar­ma has reeled in Michael Rice to its board of di­rec­tors. Rice is the co-founder and pres­i­dent of LifeSci Ad­vi­sors and the co-founder of LifeSci Cap­i­tal. Rice’s oth­er stints in­clude roles at Canac­cord Adams, ThinkE­quity, Bank of Amer­i­ca and JP­Mor­gan/Ham­brecht & Quist.

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.