Armed with $42.5M, Gem­i­ni em­braces the Goldilocks strat­e­gy in bring­ing pre­ci­sion med to AMD

For most biotech star­tups, the big chal­lenge lies in de­vel­op­ing one or two new drugs that promise to do a bet­ter job of tar­get­ing a par­tic­u­lar dis­ease.

But Gem­i­ni Ther­a­peu­tics — the lat­est in a long line­up of Cam­bridge, MA biotechs to roll out this year — isn’t like most up­starts.

James McLaugh­lin

CEO James McLaugh­lin has set out to divvy up the large pop­u­la­tion of pa­tients at high risk of dry age-re­lat­ed mac­u­lar de­gen­er­a­tion — AMD — in­to small, ge­net­i­cal­ly de­fined buck­ets of pa­tients, and then find the right drug most like­ly to help them. And that’s big enough to even­tu­al­ly in­volve a whole pipeline of ther­a­peu­tics.

“There are ge­net­ic vari­ants that in­crease the risk of dis­ease by 20 times or more. In­ter­est­ing tar­gets that were nev­er pur­sued; that is what got the pro­gram start­ed. Could we de­vel­op ther­a­peu­tics that were fea­si­ble and tractable for the eye?” says McLaugh­lin, the for­mer op­er­a­tions chief at gene ther­a­py pi­o­neer Voy­ager.

That process be­gan in stealth two and a half years ago, says McLaugh­lin, fol­lowed by the first seed fund­ing in 2016 as Gem­i­ni start­ed work­ing on the R&D side of the busi­ness. Now McLaugh­lin has a $42.5 mil­lion launch round co-led by At­las Ven­ture, Light­stone Ven­tures and Or­biMed. And the grow­ing 7-mem­ber team at Gem­i­ni will be start­ing out with three ba­sic tech­nolo­gies: re­com­bi­nant pro­teins, mon­o­clon­al an­ti­bod­ies and gene ther­a­py.

Find the right pop­u­la­tion first, he says, and you can de­ter­mine the right ther­a­peu­tic ap­proach sec­ond.

“It’s def­i­nite­ly am­bi­tious,” says the CEO, re­quir­ing dif­fer­ent teams to break down the tech and the pre­ci­sion med­i­cine they’re pur­su­ing. So there’s a team of gene ther­a­py ex­perts, a team on tar­get bi­ol­o­gy and a team on reti­nal dis­ease and AMD. “A lot of the con­ver­sa­tion is bridg­ing the gap be­tween the dis­ci­plines,” he adds, with ad­vis­ers and founders from around the globe.

The Uni­ver­si­ty of Ed­in­burgh’s Paul Bar­low, Andy Her­bert and Tufts Uni­ver­si­ty’s Jo­han­na Sed­don are the sci­en­tif­ic founders.

Gem­i­ni’s sci­en­tif­ic ad­vi­so­ry board in­cludes:

  • Reti­nal dis­ease and AMD ex­perts Alan Wright from the Uni­ver­si­ty of Ed­in­burgh, Dean Bok from UCLA and Bruce Jaf­fee.
  • Tar­get bi­ol­o­gy ex­perts in­clude John Atkin­son from Wash­ing­ton Uni­ver­si­ty in St. Louis, Michael Ehrmann from the Uni­ver­si­ty of Es­sen-Duis­berg, Kevin March­bank and Claire Har­ris from New­cas­tle Uni­ver­si­ty, and Si­mon Clark from the Uni­ver­si­ty of Man­ches­ter.
  • Gene ther­a­py ex­perts in­clude Shan­non Boye from Uni­ver­si­ty of Flori­da, Ar­avind Asokan from the Uni­ver­si­ty of North Car­oli­na, and Chris Mueller and Clau­dio Pun­zo from the Uni­ver­si­ty of Mass­a­chu­setts Med­ical School.

At this stage, Gem­i­ni has iden­ti­fied hun­dreds of ge­net­ic mu­ta­tions that play a role in the eye dis­ease. They’re start­ing out with the “Goldilocks zone,” small groups of 1% to 2% or so of the pa­tients like­ly to ben­e­fit the most from one of their tar­get­ed ap­proach­es. The big­ger groups can wait un­til lat­er, af­ter the biotech has shown what it can do with pa­tient pop­u­la­tions that re­sem­ble those found in rare dis­eases, where small tri­als can tell you a lot.

The re­cent up­beat re­view of Spark’s ground­break­ing gene ther­a­py for the eye fo­cused con­sid­er­able at­ten­tion on just how at­trac­tive the eye is as a drug tar­get. It’s large­ly con­tained, al­low­ing in­ves­ti­ga­tors to take a high­ly fo­cused ap­proach that avoids sys­temic threats. And it can be more straight­for­ward in as­sess­ing a drug’s im­pact.

For now, it’s Gem­i­ni’s en­tire world.

No­var­tis buys a new gene ther­a­py for vi­sion loss, and this is one pre­clin­i­cal ven­ture that did­n't come cheap

Cyrus Mozayeni got excited when he began to explore the academic work of Ehud Isacoff and John G. Flannery at UC Berkeley.

Together, they were engaged in finding a gene therapy approach to pan-genotypic vision restoration in patients with photoreceptor-based blindness, potentially restoring the vision of a broad group of patients. And they did it by using a vector to deliver the genetic sequence for light sensing proteins.

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Eli Lilly CEO David Ricks (Evan Vucci/AP Images)

A P val­ue of 0.38? NE­JM re­sults raise new ques­tions for Eli Lil­ly's vaunt­ed Covid an­ti­body

Generally, a P value of 0.38 means your drug failed and by a fair margin. Depending on the company, the compound and the trial, it might mean the end of the program. It could trigger layoffs.

For Eli Lilly, though, it was part of the key endpoint on a trial that landed them a $1.2 billion deal with the US government to supply up to nearly 1 million Covid-19 antibodies.

So what does one make of that? Was the endpoint not so important, as Lilly maintains? Or did the US government promise a princely sum for a pedestrian drug?

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Noubar Afeyan, Flagship founder and CEO (Victor Boyko/Getty Images)

UP­DAT­ED: Flag­ship launch­es Sen­da Bio­sciences with $88M in back­ing, look­ing to pi­o­neer the field of 'In­ter­sys­tems Bi­ol­o­gy'

Flagship Pioneering has a fresh company out this week, one that aims to lay the groundwork for a whole new discipline.

Senda Biosciences launched Wednesday with $88 million in Flagship cash. The goal? Gain insights into the molecular connections between people and coevolved nonhuman species like plants and bacteria, paving the way for “Intersystems Biology.”

Guillaume Pfefer has been tapped to run the show, a 25-year biotech veteran who comes from GSK after leading the development of the company’s shingles vaccine.

Daphne Koller, Getty

Bris­tol My­er­s' Richard Har­g­reaves pays $70M to launch a neu­rode­gen­er­a­tion al­liance with a star play­er in the ma­chine learn­ing world

Bristol Myers Squibb is turning to one of the star upstarts in the machine learning world to go back to the drawing board and come up with the disease models needed to find drugs that can work against two of the toughest targets in the neuro world.

Daphne Koller’s well-funded insitro is getting $70 million in cash and near-term milestones to use their machine learning platform to create induced pluripotent stem cell-derived disease models for ALS and frontotemporal dementia.

Eli Lilly CEO David Ricks at the Rose Garden, May 26, 2020 (Evan Vucci/AP Images)

Eli Lil­ly lines up a block­buster deal for Covid-19 an­ti­body, right af­ter it failed a NI­AID tri­al

Two days after Eli Lilly conceded that its antibody bamlanivimab was a flop in hospitalized Covid-19 patients, the US government is preparing to make it a blockbuster.

The pharma giant reported early Wednesday that it struck a deal to supply the feds with 300,000 vials of the drug at a cost of $375 million — once it gets an EUA stamp from the FDA. And once that 2-month supply deal is done, the government has an option on another 650,000 doses on the same terms — which could potentially add another $812 million.

Q32 Bio grabs $60M to kick off hu­man stud­ies for next-gen com­ple­ment drugs — with some Covid-19 tweaks along the way

For a company that launched in the early months of the pandemic, Q32 Bio had its fair share of run-ins with the new normals under Covid-19.

The original plan, for instance, was to conduct first-in-human studies of the IL-7 receptor antibody it licensed from Bristol Myers Squibb in the Netherlands. But they realized shortly after that while the country was beginning to open up clinical trials, there were additional restrictions on drugs that tampered with immunological mechanisms.

Jude Samulski, Marianne De Backer

Bay­er buys a biotech ‘race horse’ with a $4B deal — $2B in cash — aimed at go­ing big in­to gene ther­a­py

In the latest sign that Big Pharma wants a leading place in the push to develop a new generation of cell and gene therapies, Bayer is stepping up today with a $2 billion cash deal to buy out one of the fast-moving pioneers in the field, while adding up to $2 billion more in milestones if the new pharma subsidiary can deliver the goods.

As part of a continuing series of deals engineered by Bayer BD chief Marianne De Backer, the pharma player has snapped up Asklepios, more commonly referred to in more casual fashion as AskBio. And they are paying top dollar for a Research Triangle Park-based company that raised $225 million a little more than a year ago to back the brainchild of Jude Samulski, the gene therapy pioneer out of the University of North Carolina Gene Therapy Center.

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Konstantin Poukalov

Per­cep­tive re­cruits A-list in­vestors to back its in-house Chi­na start­up with a mam­moth $310M raise

It took two years for Perceptive Advisors to conceive and boot up LianBio, its big bet on a new kind of in-licensing model for China, seeding it with enough cash to set up two anchoring deals with MyoKardia and BridgeBio. The result was a startup that was all ready to go, reaping $310 million just a little over two months after official launch.

Homegrown Chinese biotechs — many of them boasting of US ties and execs with overseas credentials — have been raking in mega-venture rounds in 2020, both from influential local backers and overseas VC firms that have been loading up new cash. As with IPOs, the deal flow might be slower but the amounts are often more staggering. LianBio’s latest round, unusually, is branded both a Series A and crossover.

Ar­cus and As­traZeneca part­ner on a high stakes an­ti-TIG­IT/PD-L1 PhI­II can­cer study, look­ing to im­prove on a stan­dard of care

For AstraZeneca, the PACIFIC trial in Stage III non-small cell lung cancer remains one of the big triumphs for AstraZeneca’s oncology R&D group. It not only made their PD-L1 Imfinzi a franchise player with a solid advance in a large niche of the lung cancer market, the study — which continues to offer data on the long-range efficacy of their drug — also helped salve the vicious sting of the failure of the CTLA-4 combo in the MYSTIC study.