Kriya Therapeutics, Inc. (“Kriya”), a biotechnology company committed to building durable medicines that can scale, today announced the publication of preclinical data demonstrating that a single intramuscular administration of an AAV vector expressing native FGF21 extended both health and lifespan in naturally aged mice. The study, titled AAV-mediated FGF21 gene therapy promotes health span extension by whole-body tissue-specific adaptations, was led by Professor Fátima Bosch in the Center for Animal Biotechnology and Gene Therapy, at the Universitat Autònoma de Barcelona (UAB) and appears in Molecular Therapy, the founding journal of the American Society of Gene & Cell Therapy (ASGCT).
“These data show the remarkable breadth of FGF21 biology, with a single administration driving durable benefits across organs well beyond the liver,” said Shankar Ramaswamy, MD, Co-Founder and CEO of Kriya Therapeutics. “These whole-body benefits in aged animals reinforce our conviction in native FGF21 gene therapy, and we remain focused on advancing KRIYA-497, our one-time gene therapy candidate expressing human native FGF21, for people living with steatotic liver disease and other conditions FGF21 is well positioned to address.”
Key findings
- A single intramuscular dose of AAV1-FGF21 in aged mice increased life expectancy by more than 20%, extending mouse median lifespan by nearly six months;
- Treated animals normalized body weight and adiposity, improved insulin sensitivity and glucose control, and increased energy expenditure;
- Liver, kidney, and heart function were preserved, with age-related kidney damage, fibrosis, and amyloidosis prevented;
- Muscle strength, endurance, and memory were maintained at levels equivalent to much younger animals;
- Transcriptomic analyses linked these gains to tissue-specific adaptations, including enhanced mitochondrial function, restored protein synthesis, enhanced hepatic detoxification, and reduced inflammation and fibrosis.
These findings extend a body of work showing that AAV-FGF21 gene therapy can counteract established metabolic dysfunction-associated steatohepatitis (MASH), type 2 diabetes, and obesity in preclinical models.1
Fátima Bosch, Ph.D., Professor in Biochemistry and Molecular Biology at UAB and Kriya Scientific Advisory Board Member, who led the study, added, “FGF21 acts as a systemic signal that keeps energy metabolism in balance across muscle, liver, fat, and brain. In aged mice, a single administration preserved mitochondrial function and proteostasis while limiting inflammation and fibrosis. These findings point to the potential of AAV-FGF21 gene therapy as a strategy to promote healthy aging.”
KRIYA-497 is currently being evaluated in a clinical trial for patients with MASH. It is investigational and has not been approved as safe or effective by any regulatory authority.
1Jimenez V, et al. Mol Ther. 2024;32. doi:10.1016/j.ymthe.2024.10.023
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 clinical-stage biopharmaceutical company developing gene therapies to address chronic diseases affecting millions of people around the world. Our pipeline includes potentially transformational medicines in multiple therapeutic areas—including in ophthalmology, metabolic disease and neurology—built on our fully-integrated proprietary manufacturing and engineering platform. For more information, visit https://kriyatx.com/, and follow us on LinkedIn and X.
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