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.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Big week for Alzheimer’s da­ta; As­traZeneca buys cell ther­a­py start­up; Dig­i­tal ther­a­peu­tics hits a pay­er wall; and more

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Am­gen, years be­hind ri­vals, says PhI obe­si­ty drug shows dura­bil­i­ty signs

While NBC ran “The Biggest Loser” for 17 seasons, deemed toxic by critics for the reality show’s punishing exercise and diet upheavals, researchers in pharmaceutical labs have been attempting to create prescription drugs that induce weight loss — and one pharma betting it can require less frequent dosing is out with a new crop of data.

Amgen was relatively late to the game compared to its approved competitor Novo Nordisk and green light-approaching rival Eli Lilly. But early data suggested Amgen’s AMG 133 led to a 14.5% weight reduction in the first few months of dosing, buoying shares earlier this fall, and now the California pharma is out with its first batch of durability data showing that figure fell slightly to 11.2% about 150 days after the last dose. Amgen presented at the 20th World Congress on Insulin Resistance, Diabetes & Cardiovascular Disease on Saturday afternoon.

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Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls

Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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Chris Kim, Liminatus Pharma CEO

A fledg­ling biotech goes SPAC route to bankroll can­cer vac­cine, CAR-T and CD47

A relatively unknown clinical-stage biotech — backed by a Korean lighting company and focused on a cancer vaccine out of a Thomas Jefferson University lab — is headed to Nasdaq via the blank check route.

Liminatus Pharma will get about $316 million in proceeds from the SPAC combination to fund its ongoing Phase IIa study of a cancer vaccine, bring a CAR-T therapy into the clinic and prep a CD47 immune checkpoint inhibitor for human trials, the company said this week.

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SQZ Biotech slash­es head­count by 60% as founder/CEO hits ex­it — while Syn­log­ic lays off 25%

It’s a tough time for early-stage companies developing highly promising, but largely unproven, new technologies.

Just ask SQZ Biotechnologies and Synlogic. The former is bidding farewell to its founder and CEO and slashing the headcount by 60% as it pivots from its original cell therapy platform to a next-gen approach; the latter — a synthetic biology play founded by MIT’s Jim Collins and Tim Lu — is similarly “optimizing” the company to focus on lead programs. The resulting realignment means 25% of the staffers will be laid off.

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Uğur Şahin, BioNTech CEO (ddp images/Sipa USA/Sipa via AP Images)

BioN­Tech bets on dif­fi­cult STING field via small mol­e­cule pact with a Pol­ish biotech

BioNTech is beefing up its relatively thin small molecule pipeline by adding weight to a clinically difficult corner of oncology R&D: STING agonists. To do so, BioNTech is teaming up with a 15-year-old Polish biotech and doling out €40 million, about $41.5 million, to start.

The deal is broken into two parts: First, BioNTech obtains an exclusive global license to develop and market Ryvu Therapeutics’ STING agonist portfolio as small molecules, whether alone or in combination with other agents.

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