GENV-HEM / GeneVentiv Therapeutics - LARVOL DELTA

A novel universal hemophilia AAV gene therapy with an improved vector expressing human activated coagulation factor V (ASGCT 2026) - "Indeed, using recombinant AAV8 expressing human FVa from a liver-specific promoter (AAV8.FVa), we demonstrated that modest increases in FVa levels are efficacious, durable, and safe in hemophilia mouse models, including in the presence of inhibitors...Conclusion In summary, this multi-step optimization strategy for AAV-mediated FVa expression identified novel combinations of regulatory elements and FVa cDNAs that safely and substantially reduced the minimum effective dose of the lead construct in HA mice, using a stringent injury model as the efficacy benchmark. This lead candidate will now advance to evaluation in hemophilia dogs, a clinically relevant animal model, supporting further development of a universal gene therapy approach for hemophilia."

Gene therapy • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases • F5