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rAAV2-hFIX
- Development stage
- Preclinical
- Lead developer
- Vector Gene Technology
- Modality
- Gene Silencing → Gene Therapies, Engineered AAV → AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies
- Administration
- Intravenous
01
Overview
rAAV2-hFIX is a gene therapy candidate developed by Vector Gene Technology for the treatment of Hemophilia B. It utilizes a recombinant adeno-associated virus serotype 2 (AAV2) vector to deliver a functional copy of the human Factor IX (hFIX) gene into the patient's cells. The therapeutic goal is to restore the endogenous production of the Factor IX protein, a critical blood-clotting factor that is deficient or dysfunctional in individuals with Hemophilia B. By providing a steady supply of Factor IX, the therapy aims to prevent spontaneous bleeding episodes and reduce or eliminate the need for frequent exogenous factor replacement infusions.
- Other names
- recombinant adeno-associated virus serotype 2 encoding human factor IXrecombinant AAV-2-hFIX
02
Targets
HSPG (Basement membrane-specific heparan sulfate proteoglycan core protein (perlecan))
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