Eric Kelsic, Dyno Therapeutics CEO

Dyno's Er­ic Kel­sic fills the tank in his quest for bet­ter AAV with a group of big-name sup­port­ers on board

Ade­no-as­so­ci­at­ed virus­es (AAV) for gene ther­a­py have re­ceived a ton of scruti­ny through­out the field’s his­to­ry af­ter a smat­ter­ing of safe­ty scares and their lim­it­ed ther­a­peu­tic range. Hop­ing to crack the field wide open through a cap­sid de­sign rev­o­lu­tion, Er­ic Kel­sic and his team at Dyno have drummed up im­mense ex­cite­ment — and now a hefty war chest.

Dyno Ther­a­peu­tics has bagged a $100 mil­lion Se­ries A with back­ing from the likes of round leader An­dreessen Horowitz and new in­vestor Cas­din Cap­i­tal in its quest to use AI to de­sign bet­ter AAV cap­sids for gene ther­a­py, the com­pa­ny said Thurs­day.

As the gene ther­a­py in­dus­try has run in­to a tech­no­log­i­cal road­block with a nar­row range of AAVs used to de­liv­er those drugs, Kel­sic’s team is us­ing deep learn­ing to comb through mil­lions of en­gi­neered cap­sid de­signs to find safer and eas­i­er-to-man­u­fac­ture vi­ral vec­tors for a range of tis­sues.

So far, that mis­sion has earned the sup­port of high-lev­el run­ning mates in No­var­tis, Roche/Genen­tech and Sarep­ta in deals worth more than $4 bil­lion cu­mu­la­tive­ly — and now Dyno is ready to add even more part­ner­ships to the fold. The newest round of fund­ing will give Dyno the space to keep de­vel­op­ing its cap­sid de­sign plat­form as well as hire up its team and start tak­ing on new part­ners as part of its busi­ness de­vel­op­ment plan mov­ing ahead.

Kel­sic, a Cal­Tech grad and for­mer George Church acolyte at Har­vard, told End­points News his plan is to ex­pand the team about three-fold from its cur­rent 50-em­ploy­ee ros­ter but left the door open for even more ex­pan­sion in that time frame. Mean­while, the biotech looks to widen its plat­form to add three new tar­get tis­sues to its grow­ing fold — the lung, heart and kid­ney. Those join ex­ist­ing tar­get ar­eas in liv­er, eye, mus­cle and CNS.

As part of the fi­nanc­ing, An­dreessen Horowitz gen­er­al part­ner Jorge Conde will join the board. In a blog post pub­lished in tan­dem with the news, Conde point­ed to Dyno’s de­ci­sion not to build a pipeline of its gene ther­a­pies and in­stead in­vest in part­ner­ships as a big draw for his firm:

“From its in­cep­tion, Dyno chose to es­chew the tra­di­tion­al ap­proach of in­vest­ing in its own prod­uct pipeline (i.e. de­vel­op­ing its own gene ther­a­pies) and in­stead de­cid­ed to in­vest in the plat­form that will serve as the hor­i­zon­tal ‘in­fra­struc­ture’ lay­er to sup­ply nov­el AAVs to pow­er all gene ther­a­pies.”

In terms of where Dyno is look­ing for its next big part­ner­ship deals, Kel­sic said the fo­cus was all on com­pa­nies com­mit­ted to us­ing the tech to im­prove pa­tient care — and that doesn’t nec­es­sar­i­ly mean just big names.

“Our mis­sion is to max­i­mize im­pact on pa­tients so the most im­por­tant thing is we want to work with part­ners who can ac­cel­er­ate the progress of cur­ing dis­eases,” he said. “We would love to work with more ex­pe­ri­enced play­ers … but in time, we re­al­ly want to open up whole new fron­tiers, which means work­ing with a whole new va­ri­ety of dif­fer­ent types of part­ners.”

With its ma­jor round in hand and a syn­di­cate of in­vestors back­ing it, it’s no ques­tion that Dyno has thought — and been asked — about an IPO giv­en the pace of the mar­ket and its A-list sup­port. For now, Kel­sic said, the com­pa­ny is stay­ing pat but nev­er say­ing nev­er.

“I would say we have a lot of op­tions for how we fund the com­pa­ny, which is the best pos­si­ble way to be,” Kel­sic said. “We have had a lot of in­ter­est from in­vestors at all stages as well as bankers in­ter­est­ed in IPO, but for now we’re in re­al­ly good shape with this Se­ries A, and we still have growth to come.”

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).

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.”

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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Michel Sade­lain puts his name and new cell en­gi­neer­ing tech be­hind 'ag­nos­tic' CAR-T start­up chas­ing epi­ge­net­ic anti­gens

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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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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Hal Barron, GSK R&D chief (Endpoints News)

Hal Bar­ron gam­bles $625M cash on high-wire TIG­IT act, throw­ing Glax­o­SmithK­line in­to heat­ed race and com­plet­ing next-gen I/O trin­i­ty

Count Hal Barron and GlaxoSmithKline in for the TIGIT fight.

The stakes are as high as the risks: While a growing pack of Big Pharma rivals is lending credence to the hypothesis that TIGIT will be the next big immune checkpoint and cancer drug target, the first clinical trials have shown response rates that can be described as modest at best. But Barron’s bet is on the whole “axis” that the receptor sits on, with an eye on testing its new anti-TIGIT antibody not just in combo with PD-1 but also in triplets.

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Andrew Hopkins, Exscientia CEO

Ex­sci­en­tia spends Soft­Bank's cash in bid to edge out AI ri­vals

Exscientia is sprinting to win the great AI biotech race.

The UK company, having long labored on small discovery deals with large pharmas, raised up to $525 million in a Series D led by the infamous Japanese conglomerate SoftBank in April and followed it up less than a month later with a Bristol Myers Squibb deal that paid $50 million cash and $1.2 billion in milestones.

Now, the Oxford spinout is splurging on a shiny new tool. On Monday they announced they purchased the three-year-old molecule-screening biotech Allcyte, a longtime collaborator, for $60.6 million in cash and stock.

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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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Alexander Vos, VectorY CEO

Start­ing fresh in man­u­fac­tur­ing, For­bion start­up re­fu­els to steer next-gen gene ther­a­py ap­proach for ALS, Alzheimer's in­to clin­ic

Forbion laid out its case for a next-generation gene therapy approach when it took the wraps off VectorY Therapeutics and its vectorized antibody tech in February. Now, the Dutch VC has tapped an experienced hand at cell and gene therapy manufacturing to steer the ship — and pulled a marquee syndicate for a €31 million ($37.6 million) seed round.

Alexander Vos, the new CEO, is a venture partner at BioGeneration Ventures and jumps immediately from VarmX, a BGV portfolio company developing an anticoagulant. But before that, he had led Dutch CDMO PharmaCell for eight years until it was bought out by Lonza.