RALEIGH, NC – GeneVentiv Therapeutics, a pre-clinical gene therapy company focused on blood diseases and disorders, today announced that Sylvia Fong, Ph.D. has joined the Company’s Scientific Advisory Board as the Company prepares for large animal studies of GENV-HEM (AAV8.FVa) leading into an Investigational New Drug (IND) application. GENV-HEM is indicated for hemophilia A or B with or without inhibitors.
“We are honored to have Sylvia join our Scientific Advisory Board. Sylvia brings immense pre-clinical development experience in hemophilia gene therapy,” commented Damon Race, CEO of GeneVentiv Therapeutics. Sylvia Fong most recently served as Executive Director, Head of Hematology at BioMarin and led the pre-clinical development of RoctavianTM, a hemophilia A gene therapy.
“Having successfully advanced Roctavian™ through clinical trials to FDA approval, I am thrilled to support GeneVentiv in progressing GENV-HEM into the clinic,” said Sylvia Fong, a member of GeneVentiv’s Scientific Advisory Board. “Hemophilia A patients and especially inhibitor patients are still waiting for a lasting gene therapy treatment to come to market. The available pre-clinical data show promise that GENV-HEM can fulfill this need.”
Sylvia Fong is a globally recognized leader in AAV gene therapy for hemophilia and brings more than 25 years of translational research experience in developing therapies for rare genetic diseases. Sylvia spearheaded the development of the first AAV-based gene therapy for hemophilia A, successfully navigating from early research to regulatory approval. Sylvia has demonstrated expertise across diverse therapeutic areas, orchestrating collaborations with key stakeholders to drive strategic initiatives and establish an early-stage hematology R&D pipeline.
About GeneVentiv Therapeutics
GeneVentiv Therapeutics is a preclinical gene therapy company focused on identifying, in-licensing, and developing transformative genetic medicines for diseases with significant unmet needs. GeneVentiv is advancing two first-in-class programs. GENV-HEM is a liver-directed gene therapy designed to provide activated Factor V (FVa) for patients with hemophilia A, with or without FVIII inhibitors. GENV-002 is an in vivo gene-editing therapy for both infantile-onset (IOPD) and late-onset (LOPD) Pompe disease, designed to stably integrate a functional GAA transgene and establish the liver as a durable source of GAA enzyme.
Together, these programs are designed to address serious diseases where significant unmet needs remain.
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