Back– Results demonstrate statistically significant fibrosis reversal and protection against hepatocellular carcinoma in animal models of MASH –
– Kriya plans to evaluate KRIYA-497, a one-time intramuscular gene therapy designed to enable continuous expression of native FGF21, in people with MASH –
PALO ALTO, Calif. and RESEARCH TRIANGLE PARK, NC — Nov. 13, 2024 — Kriya Therapeutics, Inc., (“Kriya”), a biopharmaceutical company developing gene therapies to address common diseases affecting millions of people around the world, today announced the publication of preclinical data for its investigational AAV-FGF21 gene therapy, generated by researchers at the Universitat Autonoma de Barcelona (UAB), Spain, in Molecular Therapy, the founding journal of the American Society of Gene & Cell Therapy (ASGCT). The results underscore the potential of KRIYA-497, an investigational, intramuscularly-administered AAV gene therapy designed to express native fibroblast growth factor 21 (FGF21) protein. Other FGF21-based therapies have clinically demonstrated beneficial metabolic effects across several target organs including the liver.
“We are thrilled with the latest published data on our FGF21 gene therapy program, which has demonstrated fibrosis reversal and protection against hepatocellular carcinoma in translatable animal models of MASH,” said Shankar Ramaswamy, M.D., Co-Founder and CEO of Kriya. “MASH represents one of the most significant chronic diseases of our time, and patients are in desperate need of additional safe, effective and durable treatment options. We are excited to advance a potential one-time gene therapy to address this debilitating disease.”
The UAB-based authors of the article, titled “Reversion of metabolic dysfunction-associated steatohepatitis by skeletal muscle-directed FGF21 gene therapy”, noted the following:
- One-time intramuscular administration of AAV1-FGF21 in obese male and female mice resulted in sustained increased circulating levels of FGF21;
- The long-term treatment resulted in complete reversal of hepatic fibrosis while halting the development of liver tumors in animals followed for over 9 months following gene therapy treatment;
- AAV-FGF21 treatment also counteracted obesity, adiposity and insulin resistance in animals, which are significant drivers of MASH; and
- The same treatment in dogs resulted in durable protein expression and biological activity in key metabolic tissues.
The Online First edition of the article can be found here.
“These results provide strong support for the durability and efficacy in animals of a one-time intramuscular AAV-FGF21-based gene therapy,” said Fátima Bosch, Ph.D., Professor in Biochemistry and Molecular Biology at UAB, Kriya Scientific Advisory Board Member, and director of the study. “Based on this research, we are very excited about the potential to impact the lives of people with MASH, and we are hopeful that this approach may someday be a major breakthrough in the treatment of MASH and its related comorbidities.”
About Metabolic Dysfunction-Associated Steatohepatitis (MASH)
MASH is a condition in which excess fat deposits in the liver cause inflammation, cell damage and fibrosis. Approximately 20 percent of people with MASH will progress to advanced liver disease and associated complications, including cirrhosis and cancer. MASH affects approximately 40 million people in the United States and European Union. There is currently only one FDA approved treatment for MASH, with significant unmet need remaining to be addressed.
About Kriya Therapeutics
Our mission is to revolutionize medicine, with the ultimate goal of eliminating human suffering and enabling people to live without the burden of disease. Kriya is a biopharmaceutical company developing gene therapies to address common diseases affecting millions of people around the world. With operations in Palo Alto, California and Research Triangle Park, North Carolina, Kriya has raised over $600 million, which will be used to advance a broad pipeline of gene therapies for ophthalmology, metabolic disease and neurology. For more information, please visit www.kriyatx.com/ and follow us on LinkedIn and X.
Media Contact:
Kelli Perkins