Ambys Medicines to Present New Data on Liver Cell Replacement Therapy Platform at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting

Data Highlighting Universal Human Hepatocyte Program in a Metabolic Liver Disease Model Will Be Featured in an Oral Presentation

Ambys Medicines, a company pioneering cell-replacement therapies for patients with liver failure, today announced that new data from its novel hepatocyte cell therapy platform and universal hepatocyte program will be highlighted in three presentations at the American Society of Gene and Cell Therapy (ASGCT) 25^th Annual Meeting, which will be held on May 16-19, 2022, in Washington, D.C. Ambys aims to unlock the full potential of hepatocyte replacement therapy for patients with acute and chronic liver failure and genetic liver diseases.

Ambys will present the first preclinical data on two of its genetically engineered hepatocyte programs—a universal hepatocyte program in development for chronic acquired and genetic diseases and a genetically engineered hyperfunctional hepatocyte program in development for specific genetic diseases. Additionally, the company will present new preclinical data on its proprietary in vivo bioreactor platform demonstrating superior levels of functionality versus hepatocyte-like cells generated from other sources.

“Hepatocyte replacement therapy represents a promising treatment approach for patients with liver failure, both from acquired and genetic disease, to restore lost hepatic function,” said Ronald Park, M.D., Chief Executive Officer of Ambys Medicines. “We’ve made significant progress advancing our pipeline of next-generation, modified human hepatocytes for a range of addressable patient populations, with the goal of providing a breakthrough treatment for patients with liver failure. We look forward to presenting the first data on our genetically engineered universal hepatocyte and hyperfunctional programs and new research on our proprietary in vivo bioreactor production platform at the upcoming ASGCT annual meeting.”

Oral Presentation

Title: Primary Human Hepatocytes, Genetically Engineered ex vivo to be Hypoimmunogenic, Can Rescue a Model of Metabolic Liver Disease

Session: Engineered Cell Therapies

Date and Time: Wednesday, May 18, 2022, at 3:45 p.m. ET

Abstract: 854

Room: 206

Poster Presentations

Title: Ex-vivo Genetically Engineered Hyperfunctional Primary Human Hepatocytes Can Produce Clinically-Relevant Levels of Therapeutic Factor IX in vivo

Session: Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases I

Date and Time: Monday, May 16, 2022, at 5:30 p.m. ET

Abstract: 243

Poster Number: M-124

Room: Hall D

Title: Human Hepatocytes Expanded in a Novel Rat Bioreactor Maintain Full Functionality in vitro and in vivo

Session: Cell Therapies I

Date and Time: Monday, May 16, 2022, at 5:30 p.m. ET

Abstract: 386

Poster Number: M-267

Room: Hall D

About Ambys Medicines

Ambys Medicines is focused on pioneering cell replacement therapies for patients with liver failure. Ambys’s proprietary platform enables the company to be the first and only company able to develop and manufacture functional human hepatocytes at scale. Our scientific approach has the potential to fundamentally transform the treatment paradigm for patients with acute and chronic liver failure and genetic diseases of the liver. Our lead program, AMI-918, is a hepatocyte replacement cell therapy in development to restore lost hepatic function. Beyond AMI-918, we are building a pipeline of next-generation modified hepatocytes that will rapidly expand the range of treatable patient populations. Learn more at ambys.com and follow us on Twitter, LinkedIn, and Instagram.

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Ambys Medicines to present new data on liver cell replacement therapy platform at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting.