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Potential Gene Therapy GENV-HEM Earns Orphan Drug Status

Potential Gene Therapy GENV-HEM Earns Orphan Drug Status

by Damon Race | Nov 3, 2021 | In the News

Hemophilia News Today by Marta Figueiredo PhD | November 3, 2021 The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to GENV-HEM, GeneVentiv Therapeutics’ investigational gene therapy for hemophilia A and B patients, with or without...
GeneVentiv Receives Orphan Drug Designation (ODD) for GENV-HEM for the treatment of Hemophilia A or B with or without inhibitors

GeneVentiv Receives Orphan Drug Designation (ODD) for GENV-HEM for the treatment of Hemophilia A or B with or without inhibitors

by Damon Race | Nov 2, 2021 | Press

Preclinical data provide quantitative evidence of GENV-HEM’s ability to achieve phenotypic correction in animal models of hemophilia, independent of the presence of any clotting factor inhibitors. Estimated 95,000 patients in the U.S. and Europe represent...

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