Hemophilia: Gene Therapy

The February issue of CAP Today published an abstract describing some initial vector-mediated gene transfer therapy success using the vector AAV5-hFVIII-SQ. The work is published in Rangarajan S, Walsh L, Lester W, et al. AAV5–factor VIII gene transfer in severe hemophilia A [published online ahead of print December 9, 2017]. N Engl J Med. doi:10.1056/NEJMoa1708483.

The same group published their work with RNA interference (RNAi) therapy in the August 31 issue of NEJM. Tap or click here for the full article at Pasi KJ, Rangarajan S, Georgiev P, et al. Targeting of antithrombin in hemophilia A or B with RNAi therapy. N Engl J Med. 2017;377:819–28. doi: 10.1056/NEJMoa1616569.