Giroctocogene fitelparvovec, with the newest groundbreaking results coming out, prevents bleeding in men with severe hemophilia A for at least two years! Giroctocogene fitelparvovec (SB-525), is an investigational gene therapy produced by Sangamo Theraputics and is in collaboration with Pfizer, as a potential treatment for those with severe hemophilia A.
The groundbreaking study involved only one infusion given to 11 men, (one which was excluded because he did not complete the 2 years follow-up), with an annualized bleed rate (ABR), of 1.4 only occurring in the second year.
These patients were not given prophylaxis, and the hope of this treatment is to prevent bleeds and the need for prophylaxis for men with hemophilia A severe. This data was presented at the American Society of Hematology meeting.
A phase 3 study in patients with hemophilia A of giroctocogene fitelparvovec is now continuing the research.
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