GENE THERAPY
-
Four-year follow-up of Biomarin’s FVIII gene therapy
Dr. John Pasi, Centre Director at the Royal London Haemophilia Centre, presented 4-year follow-up data on Biomarin Pharmaceutical’s Phase 1 /2 gene therapy trial of...
-
My experience at the WFH Virtual Summit
I attended the WFH Virtual Summit and, although I would obviously have preferred to be there in person, I was impressed by the quality of...
-
Critical need for harmonized post-marketing surveillance in gene therapy
With regulatory approval of the first gene therapy for clinical use outside of clinical trials anticipated in mid-2020, there is an urgent need to collect...
-
Dangerously high FIX levels reported in hemophilia B gene therapy
Montreal – June 29, 2020 – During the question-and-answer period of a session on gene therapy during the World Federation of Hemophilia’s virtual summit (June...
-
Biomarin’s gene therapy for hemophilia A to be called Roctavian
Montreal, May 14, 2020 – Biomarin announced on May 11 its gene therapy currently awaiting approval by the U.S. Food and Drug Administration will be...