GENE THERAPY
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Two deaths in gene therapy trial for rare neuromuscular disease
The CHS recently learned of two deaths of children under five years old in a clinical trial using AAV8-based vectors in another rare disease area,...
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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...
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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...
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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...
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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...