GeneVentiv Therapeutics Awarded $2.5 Million SBIR Grant to Advance Gene Therapy for All Hemophilias, with or without Inhibitors
Summary
RALEIGH, N.C., April 16, 2024 /PRNewswire/ -- GeneVentiv Therapeutics, developer of GENV-HEM (AAV8.FVa) for all hemophilias, announced today it has been awarded a Direct to Phase II Small Business Innovation Research (SBIR) grant for approximately $2,500,000 from the National Heart Lung Blood Institute (NHLBI) at the National Institutes of Health (NIH). The award for "Development of a universal gene therapy for hemophilia A or B with or without inhibitors" enables a large animal study and assay development to support IND-enabling biodistribution and toxicology studies.GENV-HEM is serotype 8 Adeno-associated virus (AAV) expressing the active form of coagulation Factor V (FVa). The grant also supports assay development for an IND-enabling biodistribution and toxicology study to be funded by our Series A," commented Paris Margaritis, CSO of GeneVentiv Therapeutics. Our lead program, GENV-HEM (AAV8.FVa), is the only single infusion, universal, AAV-based gene therapy able to treat all types of hemophilia with or without inhibitors. 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.