avigbagene parvec

Avigbagene parvec (FLT201) is an investigational gene therapy for Gaucher disease type 1. It consists of a recombinant adeno-associated viral (AAV) vector (serotype S3) containing a codon-optimized expression cassette for human β-glucocerebrosidase variant 85 (GCase85), a rationally engineered enzyme with extended half-life designed to enhance substrate degradation compared to wild-type GCase delivered by enzyme replacement therapy (ERT). The therapy delivers genetic material encoding GCase85 to liver cells, enabling the body to produce the enzyme endogenously, thereby reducing toxic substrate buildup and potentially ameliorating or reversing disease progression with a single administration. Developed and manufactured by Spur Therapeutics, avigbagene parvec is currently in Phase 1/2 clinical studies and is being prepared for Phase 3 trials in Gaucher disease type 1.

Brand names

avigbagene parvec

Other names

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