GS001

GS001 is an investigational gene therapy designed for the treatment of severe **hemophilia A**. It consists of a recombinant adeno-associated virus serotype 8 (**rAAV8**) vector engineered to deliver a codon-optimized transgene encoding human **B-domain-deleted coagulation factor VIII** (**BDD-hFVIII**) under the control of a liver-specific promoter. The therapy is administered as a one-time intravenous infusion to transduce hepatocytes, which then produce and secrete functional factor VIII into the systemic circulation. This approach aims to provide sustained endogenous factor VIII activity, thereby restoring blood clotting function and reducing or eliminating the need for prophylactic factor replacement. GS001 was developed by **Gritgen Therapeutics** in collaboration with the **Institute of Hematology & Blood Diseases Hospital** (IHBDH), Chinese Academy of Medical Sciences. Early clinical data from a Phase 1/2 pilot trial (NCT04728841) in Chinese patients have demonstrated its safety and ability to achieve clinically meaningful factor VIII expression levels.

Other names

AAV8-BDD-FVIII gene therapyAAV-8-BDD-FVIII gene therapyAAV 8-BDD-FVIII gene therapy