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- FDA Approves First Gene Therapy for Young Children with Sickle Cell DiseaseThe U.S. Food and Drug Administration today issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients aged 2 years and older with either sickle cell disease (SCD) with recu…fda press · 29d ago↗ source
- 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 · 29d ago↗ source
- Ipsen to buy Swiss biotech Memo Therapeutics in potential $800M dealThe purchase hands Ipsen an experimental medicine in late-stage testing for a virus that often affects kidney transplant patients.BioPharma Dive