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.
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.
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