GENV-002
Pompe Disease (Infantile-Onset and Late-Onset)
Pre-clinicalActive
Key Facts
Indication
Pompe Disease (Infantile-Onset and Late-Onset)
Phase
Pre-clinical
Status
Active
Company
About GeneVentiv Therapeutics
GeneVentiv Therapeutics is a private, pre-clinical stage biotech founded in 2019, specializing in next-generation gene therapies for rare diseases. Its pipeline features two first-in-class candidates: GENV-HEM for hemophilia (including inhibitor patients) and GENV-002, a gene editing therapy for Pompe disease designed to offer lifelong enzyme production. The company is led by an experienced CEO and CSO and is supported by a world-class scientific advisory board with deep expertise in hemophilia and AAV gene therapy. GeneVentiv operates a capital-efficient, in-licensing and development business model and is currently pre-revenue.
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