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.

“This sort of hap­pened so quick­ly, and we al­so went through a name change re­cent­ly — we were called some­thing else up un­til a month ago,” CEO Stephen Squin­to told End­points News. “We’ve en­gaged with a firm to de­vel­op mes­sag­ing and a new lo­go; we should have one some­time in the next three or four weeks.”

The com­pa­ny was co-found­ed in 2020 by Squin­to, Yale pro­fes­sors Pe­te Glaz­er, Elias Qui­jano and Bruce Turn­er af­ter it re­ceived fund­ing from the Blavat­nik Fund for In­no­va­tion at Yale, named for Len Blava­tinik, a bil­lion­aire busi­ness­man who is a chair­man for Ac­cess In­dus­tries. With the $40 mil­lion, Gen­nao will have some lab space. For now, the com­pa­ny op­er­ates out of a lab on Yale’s cam­pus.

Jim Wil­son

Gen­nao thinks its tech li­censed from Yale can ob­vi­ate the need for vi­ral vec­tor trans­fer com­mon to gene ther­a­pies, which can be dif­fi­cult to make and come with some well-es­tab­lished safe­ty red flags. Gen­nao was in­spired, in part, by the suc­cess of Pas­sage Bio and con­ver­sa­tion with that firm’s co-founder Jim Wil­son.

Pe­te Glaz­er

Wil­son told Glaz­er and Squin­to that even­tu­al­ly the field would have to shift away from the use of virus­es in ther­a­pies. The com­pa­ny has had some suc­cess in pre­clin­i­cal tri­als in mice so far, and if suc­cess­ful, would help rev­o­lu­tion­ize the gene ther­a­py land­scape. That is what drew Squin­to to the role af­ter six years away from run­ning a biotech.

The 25-year vet­er­an who co-found­ed Alex­ion served the com­pa­ny for 23 years. Pri­or to that, he was one of Re­gen­eron’s first em­ploy­ees, and he held a joint aca­d­e­m­ic po­si­tion at both the Tu­lane Uni­ver­si­ty and LSU Med­ical Schools. He has a his­to­ry with biotech star­tups, and though he was ready to step away from the dai­ly grind when he left Alex­ion in 2015, he was rein­vig­o­rat­ed when he stepped in­to an in­ter­im CEO role at Pas­sage Bio. His plan is to get the ball rolling at Gen­nao un­til the com­pa­ny is ready for some­one else to take the reins.

“I wasn’t look­ing to jump back in­to an op­er­at­ing role, but the sto­ry here was so com­pelling, I had to jump back in here,” Squin­to said.

This is the sec­ond gene ther­a­py com­pa­ny from Glaz­er’s lab. In 2019, Tru­code Gene Re­pair launched with am­bi­tious plans of tak­ing on CRISPR with a new, sim­i­lar form of gene edit­ing. Af­ter re­brand­ing to Ve­ra Ther­a­peu­tics, the com­pa­ny switched gears en­tire­ly, though they de­clined to elab­o­rate on the rea­sons be­hind the change, ex­cept to say the tech­nol­o­gy hadn’t failed.

Though the com­pa­ny on­ly has a hand­ful of em­ploy­ees, the funds from Se­ries A will help boost the staff size to to around 20 or 30, he said. Fund­ing will al­so help pro­vide sup­port to se­cure a bat­tery of pre­clin­i­cal da­ta.

The fi­nanc­ing was co-led by Or­biMed, where Squin­to is an ex­ec­u­tive part­ner, and Chau Khuong, an­oth­er part­ner at Or­biMed, will join the board on the round.

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

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Robert Califf (Pablo Martinez Monsivais, AP Images, File)

As buzz on Califf FDA nom heats up, in­dus­try and agency in­sid­ers of­fer a strong nod for the ‘per­fect’ choice

For once in this long, dramatic road to finding a new FDA commissioner, there’s been some continuity. Both CNN and Politico reported this weekend that Rob Califf met with President Biden to discuss the permanent commish role, following earlier news broken by the Washington Post that all signs point to Califf.

Although there may be a few Democrats who continue to grandstand about the dangers of COI (Califf has worked for Verily, sits on the board of Centessa Pharmaceuticals, and has other ties to industry research), with the pandemic ongoing and the need for some kind of continuity at FDA mounting, Califf is likely to meet the same fate as when he first won Senate confirmation in 2016, by a vote of 89-4 — Bernie Sanders and 6 others didn’t vote.

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AstraZeneca CEO Pascal Soriot (Raphael Lafargue/Abaca/Sipa USA)

A com­bo of As­traZeneca's Imfinzi and chemo wins where oth­ers have failed in piv­otal bil­iary tract test

Looking to run with the big dogs in the PD-(L)1 class, AstraZeneca’s Imfinzi has a tall hill to climb to compete in an increasingly bustling market. An aggressive combo strategy for the drug has paid off so far, and now AstraZeneca is adding another notch to its belt.

A combo of Imfinzi (durvalumab) and chemotherapy significantly extended the lives of first-line patients with advanced biliary tract cancer over chemo alone, according to topline results from the Phase III TOPAZ-1 study revealed Monday.

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Sean Ianchulev, Eyenovia CEO and CMO

Re­cent court de­ci­sion push­es FDA to re­ject and re­clas­si­fy drug-de­vice com­bo, crush­ing shares

Back in April, the FDA lost a crucial court case in which its broad discretion of regulating medical products that might satisfy the legal definitions of either “drug” and/or “medical device” was sharply curtailed.

In addition to the appeals court ruling that Genus Medical Technologies’ contrast agent barium sulfate (aka Vanilla SilQ) should not be considered a drug, as the FDA had initially ruled, but as a medical device, the agency also was forced to spell out which drugs would transition to devices as a result of the ruling.

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Peter Greenleaf, Aurinia CEO

Af­ter pass­ing on Ac­celeron, Bris­tol My­ers eyes bolt-on ac­qui­si­tion of au­toim­mune spe­cial­ist — re­port

Bristol Myers Squibb is looking to beef up its autoimmune portfolio by scooping up Aurinia Pharmaceuticals, Bloomberg reported.

The recent overtures to Aurinia, relayed by anonymous insiders, came just as Bristol Myers turned down buyout talks with partners at Acceleron — which Merck ultimately struck a deal to acquire for $11.5 billion. Bristol Myers has reportedly decided to cash out on its minority stake, likely bagging $1.3 billion in the process, while keeping the royalty deals on two of Acceleron’s blood disorder drugs.

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So — that pig-to-hu­man trans­plant; Po­ten­tial di­a­betes cure reach­es pa­tient; Ac­cused MIT sci­en­tist lash­es back; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

We’re incredibly excited to welcome Beth Bulik, seasoned pharma marketing reporter, to the team. You can find much of her work in our new Marketing channel — and in her weekly newsletter, Endpoints PharmaRx, which will launch in early November. Add it to your subscriptions here.

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NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

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