Single-arm phase 1–2 trial of AAVS3 gene therapy in patients with Hemophilia B.
21 Jul, 2022 | 13:23h | UTCPhase 1–2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B – New England Journal of Medicine (link to abstract – $ for full-text)
News Release: Novel gene therapy could reduce bleeding risk for hemophilia patients – University College London
Commentary: Hemophilia B Gene Therapy Produces Durable Response for 90% of Participants – AJMC
Commentary on Twitter
Nine of 10 patients with hemophilia B who received a liver-directed adeno-associated viral vector containing a hyperactive variant of factor IX maintained factor IX activity for up to 42 months. Thrombosis developed in 1 patient. https://t.co/yId8HWS1u2 pic.twitter.com/CdDeizSMK6
— NEJM (@NEJM) July 20, 2022