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Article

Phase 1–2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B

20 Jul 2022

Abstract excerpt

BACKGROUND: FLT180a (verbrinacogene setparvovec) is a liver-directed adeno-associated virus (AAV) gene therapy that uses a synthetic capsid and a gain-of-function protein to normalize factor IX levels in patients with hemophilia B. METHODS: In this multicenter, open-label, phase 1-2 trial, we assessed the safety and efficacy of varying doses of FLT180a in patients with severe or moderately severe hemophilia B...

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PMID: 35857660
DOI: 10.1056/nejmoa2119913

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