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Ambys Medicines is a South San Francisco, California-based biotechnology company developing regenerative cell replacement therapies to treat severe acquired and genetic liver diseases. The enterprise focuses on engineered hepatocyte treatments, including its lead preclinical candidate AMI-918, designed to restore liver function and provide an alternative to traditional organ transplantation. The pre-clinical firm has secured $187 million in total known funding, which includes a $140 million initial financing package and a subsequent $47 million Series A equity extension. Ambys is backed by lead venture capital investor Third Rock Ventures and maintains a strategic pharmaceutical partnership with Takeda Pharmaceutical. Following the opening of a dedicated manufacturing facility, the company's intellectual property assets were recently acquired by the biotechnology firm Cytotheryx. Ambys Medicines was founded in 2018 by Markus Grompe, Martin Burke, Juan Carlos Izpisua Belmonte, and Holger Willenbring.
Ambys Medicines has raised $107.0M across 2 funding rounds.
Ambys Medicines has raised $107.0M in total across 2 funding rounds.
Ambys Medicines is a biopharmaceutical company developing cell replacement therapies for patients with liver failure, including acute, chronic, and genetic liver diseases.[1][2] Its proprietary platform enables scalable manufacturing of functional human hepatocytes—the first and only such capability—powering its lead program, AMI-918, a hepatocyte replacement therapy to restore lost hepatic function, alongside a pipeline of next-generation modified hepatocytes.[1][2] The company serves patients lacking effective treatment options, addressing unmet needs in liver disease by transforming the treatment paradigm through cell therapy.[1][2]
Ambys Medicines was founded by Markus Grompe, M.D., a pioneering researcher in liver stem cell biology and hepatocyte transplantation, who serves as Chief Scientific Officer.[1] The idea emerged from Grompe's expertise in advancing cell-based solutions for severe liver conditions, leading to Ambys's unique platform for scalable hepatocyte production.[1][2] Key milestones include forming clinical and scientific advisory boards in 2022 with experts in liver disease and cell/gene therapies to guide AMI-918 toward clinical trials and expand the pipeline, marking progress toward clinical-stage status.[1][2]
Ambys rides the surging wave of cell and gene therapies, particularly in regenerative medicine for organ failure, where liver disease affects millions globally with few curative options.[1][2][4] Timing aligns with advances in scalable cell manufacturing and CRISPR-like engineering, amplified by post-pandemic investment in biotech platforms.[1] Market forces like aging populations and rising liver failure incidence (from metabolic diseases and genetics) favor Ambys, positioning it to influence the ecosystem by pioneering hepatocyte therapies and inspiring similar approaches for other organs.[1][2][4]
Ambys is poised to enter clinical trials with AMI-918, leveraging its advisory expertise to accelerate toward becoming a clinical-stage biotech.[1][2] Trends in engineered cell therapies and AI-optimized manufacturing will shape its path, potentially expanding to more liver indications and partnerships. Its influence could grow by validating scalable hepatocytes, reshaping liver treatment and drawing investment into cell replacement platforms—echoing its founding mission to transform patient outcomes where none existed.
Ambys Medicines has raised $107.0M across 2 funding rounds. Most recently, it raised $47.0M Series A in December 2021.
| Date | Round | Lead Investors | Other Investors | Status |
|---|---|---|---|---|
| Dec 1, 2021 | $47M Series A | Third Rock Ventures | ARCH Venture Partners, Frazier Healthcare Partners, Matrix Capital Management Company, SignalFire, Alexandria Venture Investments, Laurion Capital Management, Erwin Boos, Smilegate Investment, Takeda | Announced |
| Aug 1, 2018 | $60M Series A | Third Rock Ventures, Asit Parikh | — | Announced |
Ambys Medicines has raised $107.0M in total across 2 funding rounds.
Ambys Medicines's investors include Third Rock Ventures, ARCH Venture Partners, Frazier Healthcare Partners, Matrix Capital Management Company, SignalFire, Alexandria Venture Investments, Laurion Capital Management, Erwin Boos, Smilegate Investment, Takeda, Asit Parikh.