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.

In­side Track: Be­hind the Scenes of a Ma­jor Biotech SPAC

Dr. David Hung and Michelle Doig are no strangers to the SPAC phenomenon. As Founder and CEO of Nuvation Bio, a biotech company tackling some of the greatest unmet needs in oncology, Dr. Hung recently took the company public in one of this year’s biggest SPAC related deals. And as Partner at Omega Funds, Doig not only led and syndicated Nuvation Bio’s Series A, but is now also President of the newly formed, Omega-sponsored, Omega Alpha SPAC (Nasdaq: OMEG; oversubscribed $138m IPO priced January 6, 2021).

Someit Sidhu, JATT

An­oth­er life sci­ences SPAC has popped up from a small biotech CEO with the help of Take­da, No­var­tis vet­er­ans

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

The SPAC train has slowed down since the extraordinarily high levels from late 2020 into early this year, but Tuesday saw the filing of a new blank-check company targeting the life sciences industry.

Jatt Acquisition submitted its SEC paperwork Tuesday, penciling in an estimated raise of $120 million as it sets its sights on a reverse merger partner. The SPAC is run by Someit Sidhu, a co-founder of Pathios Therapeutics, as well as CEO of Akaza Bioscience and Izana Bioscience. Sidhu will control about 97% of the blank-check company’s shares, per the S-1.

Janet Woodcock, acting FDA commissioner (Al Drago/Bloomberg via Getty Images)

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In a letter to HHS Secretary Xavier Becerra on Wednesday, the group highlighted the “litany of flaws” in the FDA’s approval of the new drug, including the “unprecedented, inappropriately close” collaboration between the FDA and Biogen in the analysis of key trial data, basing approval on an unvalidated surrogate endpoint, not following the advice of its expert advisory committee (3 members of which have since resigned), and the wide label that the agency granted.

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CEO Harith Rajagopalan (Fractyl)

Af­ter a decade in the type 2 di­a­betes game, Fractyl Lab­o­ra­to­ries recharges with a fresh $100M and a new name

Harith Rajagopalan compared the way type 2 diabetes is managed to sticking your fingers in a dam that’s leaking from a number of places.

You can take drugs to lower your blood sugar, cholesterol, or blood pressure, but you’re not addressing what he says is the core issue — the metabolic abnormality that causes the disease.

“We’re so busy plugging the holes in the dam, we don’t have time to see that the whole infrastructure is at risk,” he said. “That infrastructure is a full-body systemic metabolic abnormality called metabolic syndrome, that we’re ignoring while we’re so busy trying to treat all of the individual symptoms of the condition.”

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Scientists have come up with the first evidence that Regeneron’s antibody cocktail, which has so far only been authorized for the outpatient setting, may reduce deaths of hospitalized Covid-19 patients — albeit only a subset.

The combination of casirivimab and imdevimab is the subject of the latest data cut from RECOVERY, the large-scale UK-based trial testing a variety of potential treatments. In total, 9,785 patients hospitalized with Covid-19 were enrolled in this arm of the study and were randomly assigned to receive either usual care plus the intravenous combo or usual care alone.

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It felt natural for Alain Maiore and Sebastian Amigorena to bring in Michel Sadelain as a co-founder of Mnemo Therapeutics. A CAR-T pioneer, Sadelain had been involved as an advisor since the early days — enthusiastic about Amigorena’s work in a genetic knockout that could enhance T cell memory and a new class of potential targets he’s discovered — and could introduce some well-known technologies to the toolbox. So they got the initial cash from Sofinnova Partners to plant roots in Paris and New York in early 2019; within a few months, they began to see more clearly just what the antigen discovery platform might unlock.

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Barry Greene, Sage CEO

UP­DAT­ED: Sage's sec­ond chance at de­pres­sion hits the PhI­II pri­ma­ry, but ques­tions re­main over dura­bil­i­ty, side ef­fects

Looking to make a comeback after a big Phase III flop, Sage Therapeutics revealed data they believe could change the entire depression treatment landscape, given the vast array of failures in the field. But some results are spooking investors, sending Sage $SAGE shares down early Tuesday.

First, the primary: Sage and Biogen reported Phase III data for once-daily zuranolone Tuesday morning, saying the experimental drug hit its primary endpoint by spurring a statistically significant change from baseline in the 17-item Hamilton Rating Scale for Depression total score. After 15 days, patients in the drug arm saw an average change of -14.1 points, compared to -12.3 on placebo.

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Bio­gen sig­nals a big PhI­II fail­ure as the lead gene ther­a­py in their $800M Night­star buy­out goes down in flames

That $800 million buyout of Nightstar has turned into a bust for Biogen as the lead therapy in the deal failed a pivotal study, signaling a severe setback for the biotech’s ambitions in gene therapies.

The big biotech put out the word after the market closed on Monday that the gene therapy they picked up in the deal for a degenerative blindness called choroideremia failed the Phase III study, just a month after their #2 drug in the deal also flopped in a mid-stage study.

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As Covid-19 shifts the world's at­ten­tion to biotech, Noubar Afeyan's Flag­ship builds $3.4B fund to fu­el new in­ven­tions. Here's the plan

A little more than a year ago, Flagship Pioneering rolled out a monster fund with $1.1 billion in it to bankroll the platform companies they were creating inside their own labs. But it turns out, that was just the prelude to a much, much larger raise, as both current investors — who’ve been reaping the rewards of some booming biotech stocks — join in with new investors betting on more in the years to come.

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