ID-17503-FGF21

ID-17503-FGF21 is an adeno-associated virus (AAV) gene therapy candidate designed for the treatment of metabolic dysfunction-associated steatohepatitis (MASH). Developed by Taxell Therapeutics and Cure Genetics, the therapy utilizes a novel, liver-specific AAV capsid (ID-17503) identified through directed evolution in non-human primates. This capsid exhibits high liver tropism and reduced off-target effects compared to standard vectors like AAV5 or LK03. Upon a single intravenous administration, ID-17503-FGF21 delivers the gene encoding wild-type fibroblast growth factor 21 (FGF21) to hepatocytes, which then secrete the protein into the peripheral circulation. Preclinical studies in MASH models have demonstrated improvements in liver function, lipid levels, and fibrosis phenotypes, positioning it as a potential "one injection for life" therapeutic strategy.