Verbrinacogene setparvovec (FLT-180a)
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Gene therapy
Preliminary data
Hemophilia B
- B-AMAZE: Chowdary P, Shapiro S, Makris M, Evans G, Boyce S, Talks K, Dolan G, Reiss U, Phillips M, Riddell A, Peralta MR, Quaye M, Patch DW, Tuddenham E, Dane A, Watissée M, Long A, Nathwani A. Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B. N Engl J Med. 2022 Jul 21;387(3):237-247. link to original article PubMed NCT03369444; NCT03641703