With Kathy High on board, AskBio makes next lead­er­ship play and se­lects Bay­er's Gu­ru Ra­ma­murthy as fi­nance chief; Myles O'Neill and Melis­sa Barnes set to re­tire from Eli Lil­ly

Gu­ru Ra­ma­murthy

→ The stars aligned to bring in Kathy High as pres­i­dent of ther­a­peu­tics, and Brain Neu­rother­a­py Bio founder Krystof Bankiewicz now leads its Parkin­son’s pro­gram. And in its first big lead­er­ship move since they signed on to the team, gene ther­a­py play­er AskBio has ap­point­ed Gu­ru Ra­ma­murthy CFO, a spokesper­son tells End­points News. Six months re­moved from strik­ing a deal to ac­quire AskBio, it should come as no sur­prise that Ra­ma­murthy is a 20-year Bay­er alum: More re­cent­ly he had been the Ger­man multi­na­tion­al’s glob­al fi­nance lead for the on­col­o­gy fran­chise, and pri­or to that, Ra­ma­murthy was CFO of the North Amer­i­can busi­ness at Bay­er Con­sumer Health.

Myles O’Neill

→ Long­time staffers Myles O’Neill (SVP and pres­i­dent of man­u­fac­tur­ing op­er­a­tions) and Melis­sa Barnes (SVP and chief ethics and com­pli­ance of­fi­cer) are re­tir­ing at Eli Lil­ly. The cur­tain clos­es for O’Neill at the In­di­anapo­lis phar­ma on May 2, while Barnes’ last day is June 27.

Melis­sa Barnes

O’Neill’s suc­ces­sor, Edgar­do Her­nan­dez, has been with Lil­ly since 2009 and will round out his tenure as SVP, glob­al par­enter­al drug prod­uct and de­vice man­u­fac­tur­ing. Alon­zo Weems, who is Lil­ly’s VP and deputy gen­er­al coun­sel, will re­place Barnes and has de­vot­ed near­ly a quar­ter-cen­tu­ry to the phar­ma gi­ant.

Alex Franzu­soff has re­tired as CEO of South San Fran­cis­co T cell biotech PACT Phar­ma, while the Scott Gar­land era of­fi­cial­ly be­gan Fri­day. Gar­land pre­vi­ous­ly helmed Por­to­la for two years un­til Alex­ion com­plet­ed its $1.4 bil­lion pur­chase of the com­pa­ny in Ju­ly 2020. Gar­land, who was with Genen­tech from 2002-11 and led sales and mar­ket­ing for Avastin and Rit­ux­an, then be­came CCO at Ex­elix­is and Re­lyp­sa, fin­ish­ing his tenure at the lat­ter as pres­i­dent of the US com­mer­cial launch of Veltas­sa.

Franzu­soff will stay on for a few more months at PACT Phar­ma in an ad­vi­so­ry ca­pac­i­ty.

Ty How­ton

Ty How­ton has re­unit­ed with CEO Bo Cum­bo to take on the roles of COO and gen­er­al coun­sel at Aa­van­tiBio. Cum­bo and How­ton’s tenures at Sarep­ta co­in­cid­ed with one an­oth­er when Cum­bo was chief com­mer­cial of­fi­cer and How­ton was a le­gal ex­ec, more re­cent­ly as the Duchenne biotech’s gen­er­al coun­sel. How­ton al­so brings ex­pe­ri­ence from Ver­tex as its chief le­gal of­fi­cer and Genen­tech as its chief health­care com­pli­ance of­fi­cer. His ap­point­ment quick­ly fol­lows that of Dou­glas Swirsky as CFO and trea­sur­er, which we had for you here last week.

John Schroer

Join­ing forces with Bris­tol My­ers Squibb on T cell ther­a­pies for sol­id tu­mors — with Bris­tol shelling out $70 mil­lion up­front — Ar­se­nal­Bio has tapped John Schroer as CFO and Tim Siri­choke as chief tech­ni­cal op­er­a­tions of­fi­cer. Schroer had been Trans­late Bio’s CFO since the spring of 2018, while Siri­choke is a No­var­tis vet who ar­rives at Ar­se­nal­Bio af­ter a stint at Genen­tech as VP of In­di­vid­u­al­ized Neo-Anti­gen Spe­cif­ic Ther­a­pies (IN­eST) man­u­fac­tur­ing.

→ Woburn, MA-based Ax­i­al Ther­a­peu­tics has pro­mot­ed Stew­art Camp­bell to the po­si­tion of CEO, suc­ceed­ing David Don­abe­di­an. Camp­bell joined the com­pa­ny in 2016, serv­ing as SVP, R&D. Pri­or to hop­ping aboard the com­pa­ny, Camp­bell held roles at An­co­ra/Cor­den­Phar­ma, Sur­face Logix and In­smed.

→ To quote Tom Pet­ty, the wait­ing was the hard­est part for Aveo On­col­o­gy, but the wait is over af­ter the FDA gave its ap­proval to tivozanib, now mar­ket­ed as Fo­tiv­da, for re­lapsed or re­frac­to­ry ad­vanced re­nal cell car­ci­no­ma. Along with that news comes the pro­mo­tion of Mike Fer­ra­res­so to chief com­mer­cial of­fi­cer, who was pre­vi­ous­ly SVP, busi­ness an­a­lyt­ics and com­mer­cial op­er­a­tions. Tak­ing the same CCO post that CEO Michael Bai­ley held when he first joined AVEO, Fer­ra­res­so’s oth­er stops in­clude Ve­rastem and AM­AG Phar­ma­ceu­ti­cals.

Gre­go­ry Wein­hoff

Centes­sa Phar­ma­ceu­ti­cals — the group of 10 sub­sidiaries pulled to­geth­er by Medicxi’s Francesco De Ru­ber­tis that al­so hand­ed the CSO reins to ex-Op­er­a­tion Warp Speed chief Mon­cef Slaouihas tapped Gre­go­ry Wein­hoff as CFO. Wein­hoff co-found­ed and was CBO and CFO of Arvelle, which got the M&A wheels turn­ing in 2021 with An­geli­ni buy­ing out the com­pa­ny for $960 mil­lion. Ad­di­tion­al­ly, Wein­hoff was CFO at Ax­o­vant from 2015-19.

Centes­sa has al­so as­sem­bled a few lu­mi­nar­ies to serve on the board of di­rec­tors: Tesaro co-founder and ex-pres­i­dent Mary Lynne Hed­ley, for­mer FDA com­mis­sion­er Robert Califf and CRISPR Ther­a­peu­tics CEO Sam Kulka­rni were all named to the board Feb. 23.

Pe­ony Yu

→ Af­ter re­tir­ing from Fi­bro­Gen — which is once again in rox­adu­s­tat lim­bo fol­low­ing an­oth­er de­lay — in De­cem­ber, Pe­ony Yu is back on the CMO block with im­muno-on­col­o­gy play­er Apol­lomics start­ing this com­ing Tues­day. She re­places in­ter­im CMO Glenn Michel­son and spent 12 years with Fi­bro­Gen, get­ting pro­mot­ed to CMO in 2016. Else­where at Or­biMed-backed Apol­lomics, So­phie (Zhengjie) Sun has been named SVP, cor­po­rate de­vel­op­ment. Sun was VP and man­ag­ing di­rec­tor of Mer­ck KGaA’s In­no­va­tion Hub Chi­na and had served at the Ger­man phar­ma in var­i­ous posts since 2009.

In­ter­cept has sad­dled up to their reg­u­lar Peer Re­view barstool with the de­par­ture of CFO Sandip Ka­pa­dia. For those scor­ing at home, CEO Mark Pruzan­s­ki, CMO Ja­son Cam­pagna and now CFO Ka­pa­dia are all gone from the C-suite af­ter the FDA’s re­jec­tion of obeti­cholic acid in June, which prompt­ed a heat­ed re­sponse from Pruzan­s­ki at the time. The con­stant flux in In­ter­cept’s lead­er­ship con­tin­ued a few weeks ago with the ap­point­ment of chief com­mer­cial of­fi­cer Lin­da Richard­son, and now it’s Ka­pa­dia’s turn af­ter near­ly five years with the com­pa­ny. In the wake of Ka­pa­dia’s ex­it, In­ter­cept’s cor­po­rate con­troller Roc­co Venezia lands the dual roles of act­ing CFO and chief ac­count­ing of­fi­cer.

Ja­son Cam­pagna

→ One of those In­ter­cept de­fec­tors, Ja­son Cam­pagna, has of­fi­cial­ly moved on to be­come CMO of Q32 Bio, which net­ted $60 mil­lion in Se­ries B fund­ing in late Oc­to­ber as the biotech looks to strength­en its com­ple­ment drug pipeline. Cam­pagna, who start­ed out as In­ter­cept’s SVP and glob­al NASH lead in 2016 be­fore slid­ing in­to the CMO slot, was al­so an ex­ec with The Med­i­cines Com­pa­ny. Our Max Gel­man dis­cussed the cof­fee meet­ings Cam­pagna had with CEO Mike Brox­son lead­ing up to the ap­point­ment since they live in such close prox­im­i­ty — “We could prob­a­bly throw a rock at each oth­er’s hous­es,” Cam­pagna said.

Lu­ca Rastel­li has joined can­cer- and au­toim­mune dis­ease-fo­cused Ju­bi­lant Ther­a­peu­tics out of Bed­min­ster, NJ as CSO af­ter near­ly three years in the same po­si­tion at Kleo Phar­ma­ceu­ti­cals. The ex-EMD Serono staffer who led de­vel­op­ment of the ap­proved MET in­hibitor Tep­metko has al­so been VP, on­col­o­gy at BioX­cel.

→ Part­ing ways with Sanofi af­ter 19 years, Anne Da­gal­li­er has signed on to Paris-based mi­cro­bio­me biotech En­terome as CBO. Lead­ing up to this change in venue, Da­gal­li­er had been head of con­sumer health care al­liances and in-li­cens­es at Sanofi Glob­al Al­liances Man­age­ment. For a time she was al­so head of ex­ter­nal in­no­va­tion, in­fec­tious dis­eases in ad­di­tion to her var­ied strate­gic and BD roles at the phar­ma gi­ant.

Ed­uar­do Bruno Mar­tins

→ San Ma­teo, CA-based Sagimet Bio­sciences has reeled in Ed­uar­do Bruno Mar­tins as CMO. Most re­cent­ly, Mar­tins served as VP of clin­i­cal de­vel­op­ment at Ab­b­Vie. Pri­or to that, Mar­tins was VP of clin­i­cal de­vel­op­ment at Al­ler­gan. In ad­di­tion, Mar­tins has served in roles at Gilead, Genen­tech, Dy­navax Tech­nolo­gies and In­ter­mune among oth­ers.

→ Last week, Gainesville, FL biotech Cy­clo Ther­a­peu­tics reeled in ex-Ed­i­tas Med­i­cine CMO Ger­ald Cox as CMO. This week, the com­pa­ny has brought on Russ Belden as act­ing CCO. Belden jump-start­ed his ca­reer at Genen­tech’s SSF Com­mer­cial or­ga­ni­za­tion, spend­ing 16 years on board. Af­ter­wards, Belden be­came the founder, pres­i­dent and CEO of Bridge Con­sult­ing. In ad­di­tion, Belden cur­rent­ly serves as the iC­CO at three Phase III com­pa­nies.

Er­mi­um Ther­a­peu­tics — fo­cused on de­vel­op­ing ther­a­pies for au­to-im­mune dis­eases — has made a new ad­di­tion to its ex­ec­u­tive team with the ap­point­ment of An­negret Van der Aa as CSO and chief de­vel­op­ment of­fi­cer. Van der Aa brings ex­pe­ri­ence with her from her time at Gala­pa­gos, Inno­genet­ics, Ac­to­GeniX and OC­TIMET On­col­o­gy.

Teona John­son

→ Stock­holm-based Cal­lid­i­tas, “de­light­ed” with its strong Phase III da­ta for Ne­fe­con last fall to use CEO Renée Aguiar-Lu­can­der’s word, has hit a tri­fec­ta of ap­pointees for its US or­ga­ni­za­tion with War­ren Brooks (VP of US med­ical af­fairs), Teona John­son (head of US mar­ket­ing) and David Fer­raro (head of US sales). Brooks leaves Len Schleifer’s folks at Re­gen­eron af­ter 12 years at sev­er­al posts, most re­cent­ly as se­nior di­rec­tor, na­tion­al lead in im­munol­o­gy in med­ical af­fairs. Since 2010, John­son held mul­ti­ple roles at Pfiz­er, break­ing from the phar­ma gi­ant af­ter lead­ing the mar­ket­ing strat­e­gy for Ibrance as glob­al com­mer­cial di­rec­tor. And Fer­raro hails from Ky­owa Kirin, where he served as na­tion­al sales di­rec­tor for the on­col­o­gy/rare dis­ease busi­ness unit.

Car­o­line Kurtz

Syn­log­ic has made some changes in its lead­er­ship team with the pro­mo­tions of Car­o­line Kurtz to chief de­vel­op­ment of­fi­cer and pro­mo­tion of Daniel Rosan to SVP and head of fi­nance. Kurtz joined Syn­log­ic in Oc­to­ber 2016. Pri­or to hop­ping aboard the com­pa­ny, Kurtz was at Iron­wood Phar­ma­ceu­ti­cals. Mean­while, Rosan joined Syn­log­ic from Bio­gen, where he held a six-year stint.

In ad­di­tion to the two pro­mo­tions, the Cam­bridge, MA-based com­pa­ny has ap­point­ed Jamie Austin as VP and in­com­ing head of reg­u­la­to­ry af­fairs. Austin comes aboard with ex­pe­ri­ence from her time at As­traZeneca, Ab­b­Vie and blue­bird bio.

→ For­mer as­sis­tant White House coun­sel Alexan­der Nguyen has stepped in as gen­er­al coun­sel at Uni­ty Biotech­nol­o­gy, which has seen its share of trou­bles and high-pro­file ex­its. Nguyen, who was on the White House le­gal team from 2010-11, has ex­pe­ri­ence at three sep­a­rate Vants to his cred­it: first as head of le­gal at Ax­o­vant, then as head of com­pli­ance at Roivant, and fi­nal­ly as gen­er­al coun­sel at Aly­vant.

As­cen­sion Health­care — fo­cused on ther­a­pies for he­mo­phil­ia and os­teoarthri­tis — has named Ben Ver­tannes as head of glob­al mar­ket­ing. Ver­tannes joins the com­pa­ny with ex­pe­ri­ence from his roles at Bay­er, No­var­tis, Wella and Pfiz­er.

Bar­bara Kosacz

Bar­bara Kosacz has made her way to the board of di­rec­tors at Leen Kawas-led Athi­ra Phar­ma not long af­ter be­com­ing a board mem­ber at Ar­se­nal­Bio. Peer Re­view cov­ered Kosacz’s ap­point­ment as COO and gen­er­al coun­sel of Kro­nos Bio last sum­mer.

→ Stan­ford Uni­ver­si­ty School of Med­i­cine pro­fes­sor (and not­ed Eliz­a­beth Holmes skep­tic) Phyl­lis Gard­ner has jumped on to the board of di­rec­tors at MiMedx. Since 2006, Gard­ner has al­so been a board mem­ber at Re­vance.

Ner­is­sa Kre­her

→ Sil­i­con Val­ley-based Re­zo­lute, work­ing on its lead drug RZ358 to treat con­gen­i­tal hy­per­in­sulin­ism, has se­lect­ed Vlad Hogen­huis and Ner­is­sa Kre­her for its board of di­rec­tors. Hogen­huis, a GSK vet, is the ex-COO of Ul­tragenyx, while Kre­her was just ap­point­ed CMO of En­tra­da Ther­a­peu­tics a few months ago.

Su­san Wind­ham-Ban­nis­ter

→ On clin­i­cal hold with its knee car­ti­lage re­gen­er­a­tion ther­a­py, His­to­gen out of San Diego has ap­point­ed Su­san Wind­ham-Ban­nis­ter to the board of di­rec­tors. Wind­ham-Ban­nis­ter, the pres­i­dent and CEO of Bio­med­ical Growth Strate­gies, is al­so the found­ing pres­i­dent and CEO of the Mass­a­chu­setts Life Sci­ences Cen­ter.

→ Lon­don-based Free­line Ther­a­peu­tics, which hit the pause but­ton last month on its gene ther­a­py tri­al, has brought in Col­in Love to be a mem­ber of the board of di­rec­tors. The Am­gen vet co-found­ed and is COO of Replimune.

Eliz­a­beth Garo­fa­lo

Tim Wal­bert-chaired gene ex­pres­sion biotech Ex­i­cure has wel­comed Eliz­a­beth Garo­fa­lo and An­drew Sas­sine to the board of di­rec­tors fol­low­ing the ap­point­ment of James Su­lat last month. Garo­fa­lo — who’s on the board at Aca­dia — was No­var­tis’ SVP and glob­al head of clin­i­cal de­vel­op­ment, and Sas­sine is CFO at Arc­turus.

Un­learn, de­vel­op­er of the first ma­chine-learn­ing plat­form that cre­ates Dig­i­tal Twins used to en­able small­er, faster clin­i­cal tri­als, has pulled in Ann E. Tay­lor to its board of di­rec­tors. Tay­lor cur­rent­ly serves as CMO of As­traZeneca. Pri­or to her role at As­traZeneca, Tay­lor was VP, glob­al clin­i­cal de­vel­op­ment at Med­im­mune and had stints at No­var­tis and Pfiz­er.

An­drew Sin­clair now has a seat re­served on the board of di­rec­tors at Adicet Bio. Sin­clair is a part­ner and port­fo­lio man­ag­er at Abing­worth and is in his 13th year with the group.

→ De­vel­op­ing ther­a­pies to re­pair dam­aged tis­sues, Pitts­burgh-based Noveome Ther­a­peu­tics has cho­sen Christo­pher Velis to join its board of di­rec­tors. Velis is the co-founder and ex­ec­u­tive chair­man of Mi­ra­ki In­no­va­tion, BOA Bio­med­ical and Arc­tic Fox Bio­med­ical. In ad­di­tion, Velis is the co-founder of Au­ris Health.

Dana Rol­li­son

NanoS­tring Tech­nolo­gies has made two new board ap­point­ments with the ad­di­tions of Dana Rol­li­son and Janet George. Rol­li­son cur­rent­ly serves as VP, chief da­ta of­fi­cer and as­so­ciate cen­ter di­rec­tor of da­ta sci­ence for the Mof­fitt Can­cer Cen­ter. Mean­while, George is the VP of au­tonomous en­ter­prise, ad­vanced an­a­lyt­ics with ma­chine learn­ing and ar­ti­fi­cial in­tel­li­gence at Or­a­cle. Pri­or to Or­a­cle, George has held stints at San­disk, Ya­hoo, eBay and Ap­ple among oth­ers.

→ La Jol­la, CA-based Arte­lo Bio­sciences has added Tama­ra Sey­mour to its board of di­rec­tors. Sey­mour pre­vi­ous­ly served in roles at Im­mu­nic, Sig­nal Ge­net­ics, De­loitte and Bea­con Dis­cov­ery, among oth­ers.

→ Ad­vanced can­cers-fo­cused Pen­rose Ther­a­peuTx has added mi­to­chon­dr­i­al re­searcher Navdeep Chan­del to its sci­en­tif­ic ad­vi­so­ry board. Chan­del is pro­fes­sor of med­i­cine bio­chem­istry and mol­e­c­u­lar ge­net­ics at North­west­ern Uni­ver­si­ty’s Fein­berg School of Med­i­cine.

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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Sajith Wickramasekara, Benchling via YouTube

Meet Bench­ling, the lat­est uni­corn seek­ing to rev­o­lu­tion­ize the way sci­en­tists do work with the help of the cloud

There’s another unicorn in biotech land, as Benchling and its leading R&D cloud platform pull in a $200 million Series E to help scientists accelerate drug development. In doing so, the company hit a lofty $4 billion valuation — nearly five times what it was worth around this time last year, according to Forbes.

Despite the fact that drug development is becoming significantly more complex, the industry continues to run on paper, emails and spreadsheets, co-founder and CEO Sajith Wickramasekara said in a video on Benchling’s website. The MIT grad sought to change that by creating software that allows scientists to better track, model and forecast their work.

Covid-19 roundup: No­vavax shakes up lead­er­ship with two pro­mo­tions and de­par­ture of CFO; Mod­er­na, No­vavax shots added to mix-and-match study in the UK

Novavax has had a busy month, filled with supply chain issues and manufacturing deals that have affected the rollout of its Covid-19 vaccine. Tuesday, the company announced updates to its leadership team.

CFO Greg Covino will step down from that role after just five months for personal reasons, the release said, but take on a new role as executive advisor. John Trizzino, current chief commercial officer and chief business officer, will take the CFO role over in the interim.

Amy­lyx to move for­ward with ALS pro­gram in Eu­rope, but FDA wants an­oth­er look; Hu­ma­cyte adds $50M in debt fi­nanc­ing

Amylyx is one of several companies looking to break through in the tough ALS field, and Wednesday they announced they’re moving forward with regulatory plans.

The Cambridge, MA-based biotech said they’re submitting a marketing application to the EMA for their AMX0035 program by the end of 2021. Wednesday’s news comes a few weeks after they revealed similar plans to move forward with Canadian health regulators by June 30.

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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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.

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.