OGeneVentiv's lead program, GENV-HEM (AAV8.FVa), is the only single infusion, universal, AAV-based gene therapy able to treat all types of hemophilia. Unlike other AAV-based hemophilia gene therapies, GENV-HEM is the only gene therapy to utilize neutralizing antibodies to address missing clotting factor.
GENV-HEM has received Orphan Drug Designation from the FDA for Hemophilia A and B with or without inhibitors and a Letter of Support from the National Bleeding Disorders Foundation.
Copyright © The Pharma Letter 2024 | Headless Content Management with Blaze