giroctocogene fitelparvovec

Giroctocogene fitelparvovec is an investigational gene therapy for the treatment of moderately severe to severe hemophilia A. It consists of a recombinant adeno-associated virus serotype 6 (AAV6) vector that delivers a codon-optimized, B-domain–deleted human factor VIII (F8) gene to the patient's cells. The therapy is administered as a single intravenous infusion and aims to enable patients with hemophilia A to produce their own functional factor VIII protein for an extended period, thereby reducing or eliminating the need for routine prophylactic infusions and decreasing bleeding episodes. Clinical trials have shown that giroctocogene fitelparvovec can achieve clinically meaningful increases in circulating FVIII activity levels and significantly reduce annualized bleeding rates compared with standard FVIII replacement therapy. The drug was initially developed by Sangamo Therapeutics in collaboration with Pfizer; as of late 2024, Sangamo has regained full rights to its development[1][3][4][5][6][8].

Other names

giroctocogene fitelparvovecSB-525SB525SB 525PF-07055480PF07055480PF 07055480