Third Rock Ventures is debuting Ambys Medicines, which will develop cell and gene therapies as well as drugs for chronic liver diseases. Ambys starts life with $140 million in committed capital, including $100 million from Takeda, part of which came in the startup’s $60 million series A round.
Specifically, Ambys is working on cell therapy for hepatocyte transplantation, gene therapy for liver regeneration and drug therapy to replace lost protein function. The Bay Area biotech has chosen to focus on liver disease because of the “staggering” number of patients with chronic liver disease and end-stage liver failure for whom the only therapeutic option is liver transplant.
The substantial funding will keep Ambys going for at least four to five years, allowing it to develop its three platforms in parallel, said Jeff Tong, a venture partner at Third Rock and Ambys’ interim CEO.
“All too often, you see young companies with exciting technologies that, at the first sign one of the approaches is working, direct all their resources to that approach [which] basically starves other approaches of resources,” Tong said. Ambys wants to fully resource all of its methods, he said.
While Ambys doesn’t have a timeline for when each piece of its pipeline will reach the clinic, it’s going as fast as it can. And Takeda will help with that. Under the deal, the Japanese pharma has the option to the ex-U.S. rights for the first four products to reach IND. If it takes this option, Takeda will take over half of the development costs and be on the hook for development and regulatory milestones down the line—all of which will extend Ambys’ resources.
Ambys will hold onto the U.S. rights for its programs, surmounting the challenge in early-stage partnerships of creating a deal that allows the biotech to build long-term value, Tong said.
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Ambys’ headcount is about 20, with 10 full-time staffers, a “handful of us from Third Rock” and contractors. The startup will be “hiring aggressively,” Tong said, aiming to reach 30 employees by the end of the year.
Tong is keeping the CEO seat warm for now, while Third Rock’s Jeffery Finer and Glenn Pierce are serving as interim chief technology officer and interim chief medical officer respectively. Stanley Hollenbach is Ambys’ pharmacology head and, in case you’re wondering what happened to Michael Holmes after he left Sangamo last month, he’s joining the gang too, as chief scientific officer.
As for Takeda, it has historically focused on gastroenterology to include all organ systems involved—“The upper [gastrointestinal tract], lower GI, inflammatory diseases of the gut, how the esophagus and stomach work, and of course, the liver and pancreas,” said Asit Parikh, Takeda’s head of gastroenterology.
The Ambys partnership gives Takeda a foothold in chronic liver disease, a section of gastroenterology that Takeda had not yet entered, but would need to be “the world’s best,” Parikh said.