Kriya Therapeutics, Inc., a biopharmaceutical company, has announced promising preclinical results for its investigational AAV-
FGF21 gene therapy. This new therapy aims to treat
metabolic dysfunction-associated steatohepatitis (MASH), a severe
liver condition affecting millions worldwide. The data, generated by researchers at the Universitat Autonoma de Barcelona (UAB), has been published in Molecular Therapy, a journal of the American Society of Gene & Cell Therapy (ASGCT).
KRIYA-497 is an investigational gene therapy designed to be administered intramuscularly to enable continuous expression of the native
fibroblast growth factor 21 (FGF21) protein. FGF21-based therapies have shown beneficial metabolic effects in various organs, including the liver. The new data indicates that KRIYA-497 can effectively reverse
fibrosis and protect against hepatocellular carcinoma in animal models of MASH.
Dr. Shankar Ramaswamy, Co-Founder and CEO of Kriya, expressed enthusiasm about the recent findings, highlighting the dire need for effective treatments for MASH. He emphasized that MASH is a significant chronic disease, and there is a desperate need for new, durable treatment options. The company is optimistic about advancing KRIYA-497 as a potential one-time gene therapy for this debilitating condition.
The UAB research team, in their article titled "Reversion of metabolic dysfunction-associated steatohepatitis by skeletal muscle-directed FGF21 gene therapy," reported several key findings:
1. A single intramuscular administration of AAV1-FGF21 resulted in sustained elevated levels of FGF21 in obese male and female mice.
2. The long-term treatment led to a complete reversal of hepatic fibrosis and prevented liver tumors in animals monitored for over nine months post-treatment.
3. AAV-FGF21 therapy also mitigated obesity, adiposity, and insulin resistance in the treated animals, which are major contributors to MASH.
4. Similar treatment in dogs demonstrated durable protein expression and biological activity in crucial metabolic tissues.
The study's lead author, Dr. Fátima Bosch, a professor at UAB and a member of Kriya's Scientific Advisory Board, stated that these findings strongly support the durability and efficacy of a one-time intramuscular AAV-FGF21-based gene therapy in animals. She expressed optimism about the potential impact of this therapy on human patients with MASH and its related complications.
MASH is characterized by excess fat accumulation in the liver, leading to inflammation, cell damage, and fibrosis. It is estimated that around 20 percent of individuals with MASH will progress to advanced liver disease, including cirrhosis and cancer. The condition affects approximately 40 million people in the United States and the European Union. Currently, there is only one FDA-approved treatment for MASH, leaving a significant unmet medical need.
Kriya Therapeutics is dedicated to revolutionizing medicine with the goal of eliminating human suffering and enabling people to live healthy lives free from disease. The company, with operations in Palo Alto, California, and Research Triangle Park, North Carolina, has raised over $600 million. This funding will support the development of their extensive pipeline of gene therapies targeting ophthalmology, metabolic diseases, and neurological conditions.
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