Ambys Medicines, a cell and gene therapy company focused on transforming the treatment of severe liver disease by unlocking the full potential of hepatocyte transplantation, today announced their presentation highlighting the development of a first-of-its-kind bioreactor manufacturing platform. The data presented demonstrate Ambys' ability to expand mature, fully functional human hepatocytes while maintaining biological function. This is a critical step towards Ambys' goal of providing an off-the-shelf treatment capable of revolutionizing care for patients with severe liver disease. The data were presented at the IXA-CTRMS-2021 Virtual Congress on September 25, 2021.
The company also announced the opening of a state-of-the-art manufacturing facility in South San Francisco, Calif., which will be outfitted for industrial-scale expansion and production of hepatocytes under current Good Manufacturing Practices (cGMP). This unique facility will support the clinical development and early commercialization of Ambys' lead allogeneic program as well as the company's follow-on programs, enabling the first mature, human hepatocyte cell therapies for the treatment of severe liver disease. With Ambys' breakthrough technology, the promise of hepatocyte transplantation may finally be realized, extending the lives of those with no treatment options and reducing the need for liver transplants.
??"The concept of hepatocyte transplantation with donor cells has been explored in academic settings for over 20 years," said Ron Park, M.D., CEO of Ambys Medicines. "These past efforts showed that mature hepatocytes were active and that the approach was safe, but further progress has been limited by the inability to generate adequate quantities of high-quality hepatocytes sufficient to drive clinical benefit. We believe that Ambys' technology will facilitate broad use of hepatocyte transplantation to significantly alter the treatment paradigm for patients with severe liver diseases, and our data presented this week validate this strategy. Now, with the opening of our GMP facility, we can begin the process to scale hepatocyte expansion to the levels needed to support our clinical development efforts."
In the presentation, "Development of a novel rat bioreactor to facilitate the manufacture of human hepatocyte cell therapies for treating patients with severe liver diseases," Ambys demonstrated that donor human hepatocytes expanded in their proprietary in vivo bioreactor, robustly engrafted in diseased animal models, and restored liver function in those models. Moreover, these human cells maintained, or even enhanced, their hepatocyte phenotype and durability in in vitro testing.
"These data demonstrate our fundamental ability to produce high-quality, mature human hepatocytes at quantities needed to support our clinical development and early commercialization efforts," said Alan Smith, Ph.D., Chief Technology Officer. "Our lead program, AMI-918, an allogeneic, off-the-shelf investigational cell therapy aimed at providing functional liver support in the acute setting, is an important advancement in transforming the treatment of severe liver disease."
Liver diseases are one of the most underserved areas of medicine. More than 1.3 million people worldwide die each year from liver diseases, and the prevalence continues to grow. If successful, the company's platform of cell therapies can address a spectrum of unmet needs, including the more intractable long-term progression of severe liver disease and certain genetically driven liver diseases.
"Ambys' bioreactor is genetically modified to engraft and preferentially proliferate human hepatocytes," said Michael Holmes, Ph.D., Chief Scientific Officer. "This proprietary system amplifies the quantity of human hepatocytes and allows for greater consistency in the quality of cells. Industrializing this process will allow us to break through the barriers that have stalled hepatocyte transplantation and bring life-saving treatments to people affected by severe liver disease."
Ambys expects to start production in this manufacturing facility in mid-2022 and file its first investigational new drug application later that year.
Liver diseases remain one of the most underserved areas of modern medicine. Despite progress with a few diseases, severe liver diseases are not effectively addressed. Until now, the field has had to choose between promoting hepatocyte proliferation or function. Ambys' proprietary in vivo bioreactor has solved for both expansion and function, generating the first and only mature, off-the-shelf hepatocyte cell therapy that will be available at therapeutic doses. AMI-918 is an allogeneic, off-the-shelf hepatocyte cell therapy for the potential on-demand treatment of patients in acute settings, in conjunction with immunosuppression.
About Ambys Medicines
Ambys Medicines is a cell and gene therapy company focused on unlocking the full potential of hepatocyte transplantation and transforming the treatment of severe liver disease. Ambys' proprietary cell therapy platform potentially solves the supply constraints that have hindered progress, catalyzing the field and fundamentally transforming the approach to treating severe liver disease. Our lead program, AMI-918, and future engineered hepatocyte cell therapies are being developed across the spectrum of severe liver disease, including those of acquired and genetic origins. Visit us at ambys.com and follow us on Twitter, LinkedIn, and Instagram.
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