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- FDA Grants First Gene-Therapy Approval for Children 2+ with Sickle Cell DiseaseThe U.S. Food and Drug Administration issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients aged two years and older who have sickle cell disease with recurrent vaso-oc…
- BridgeBio gets funding for drug launch; United buys into cell therapyElsewhere, Otsuka’s kidney drug hit the goal of a confirmatory trial and Vertex’s CRISPR sickle cell drug secured expanded approval.BioPharma Dive · 1mo ago↗ source