Damon R. Race, President & CEO
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).
“GENV-HEM successfully treats hemophilia A or B with or without inhibitors in mice. The NIH award funds the testing of GENV-HEM in a canine model of hemophilia. 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.
“Non-dilutive grant funding and our recent manufacturing collaboration substantially address GENV-HEM production and assay development costs, ensuring that investors’ funds are maximally allocated to value increasing IND-enabling studies,” said Damon Race, CEO of GeneVentiv Therapeutics.
“GENV-HEM has the potential for lifetime treatment of hemophilia patients with or without inhibitors with a single infusion. This grant funding from NIH moves GENV-HEM one step closer to IND submission.”
About GeneVentiv Therapeutics:
GeneVentiv Therapeutics is a pre-clinical gene therapy company. 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. There are approximately 150,000 hemophilia patients in the developed world. 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. For additional information about GeneVentiv, please visit www.geneventiv.com.
SOURCE GeneVentiv Therapeutics, Inc.