Early data from the phase 3 HOPE-B trial in adults with severe or moderate-severe Haemophilia B supports the promise of gene therapy in protecting patients against spontaneous bleeds and eliminating the need for continuous prophylaxis. Speaking in the late-breaking session at the ASH 2020 Annual Meeting, Professor Steven Pipe said the goal of gene therapy ...
Gene therapy trial provides HOPE for etranacogene dezaparvovec in haemophilia B
By Mardi Chapman
10 Dec 2020