Ambys: Third Rock's Ambys closes a megaround with the clinic still a ways off for its regenerative cells for liver disease

When Ambys Medicines, a Third Rock Ventures-backed regenerative play, launched in 2018, it came with some serious fanfare for its liver disease-focused approach. Now, after years of quiet development, the company has a new trough of money to play with as it slowly approaches the clinic. Ambys closed $107 million in total Series A funding - with a $47 million extension add - Tuesday with a lead program creating healthy versions of liver cells still more than a year from the clinic, the biotech said. Ambys, which launched back in mid-2018 with a goal to rewrite the rules against severe liver disease using a broad range of modalities, says it's now ready to bring its first shot at that mission to the clinic by Q2 2023. The program, dubbed AMI-918, is an off-the-shelf cell therapy looking to replace damaged liver tissue with healthy cells, Ambys said. Meanwhile, the company is working on a follow-up program for which the details are still very slim. In a release, the company described the program this way: Ambys's second program is designed to bring the promise of its liver-cell platform to many more patients by providing extended durability of replacement cells and easier dosing and administration without the need for immunosuppressive therapy, which is intended to enable access to the broader population of patients with chronic liver disease. This follow-up program aims to slow disease progression and ultimately reverse disease in patients with decompensated liver disease. This advanced technology has the potential to benefit more than a half-million liver disease patients in the U.S. alone each year. Ambys launched with much fanfare and $140 million in backing from Third Rock Ventures as part of a regenerative plan of attack against liver disease. Third Rock has once again led this round of funding with participation from Takeda, Schroders Capital, Laurion Capital, Smilegate Investment, and Alexandria Venture Investments. Named after the salamander Ambystoma mexicanum famed for its regenerative abilities, the biotech promised to leverage both cell and gene therapy approaches in its pipeline. Markus Grompe In its years since launch, Ambys has erected a clinical-scale manufacturing facility for its cells the biotech says has allowed it to produce its off-the-shelf cells at scale - a significant logistical hurdle for the company's future chances. "When we started this effort, it was a moonshot, given the tremendous challenge of producing the types of cells needed at scale," CSO Markus Grompe said in a release. "With the opening of our production facility earlier this year, we are well on our way to overcoming that hurdle and will be able to produce high-quality, mature liver cells to support clinical development and early commercialization activities."