December will serve as a milestone month for gene therapies.
There are two gene therapies for sickle cell disease with user fee deadlines at the end of 2023.
The Gene Therapy Wave Is Here
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The long-talked about wave of gene therapy approvals is beginning to reveal itself.
Learn what's happening at the US FDA. On the go.
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In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.
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Long-term follow-up requirements have taken a conservative approach but could be ripe for re-examination and global harmonization given the years of experience with the products, Kite Pharma executive director says; former FDA gene/cell therapy office head Wilson Bryan calls for elimination of the classwide REMS.
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Program for collaborative reviews of gene therapy applications is getting under way with the European Medicines Agency. After launch, the pilot could be expanded to earlier discussions around CMC and pharm/tox data requirements, FDA’s Nicole Verdun said.