FDA Expands Use of Gene Therapy for Treatment of Patients Ages 2+ with Sickle Cell ...

FDA expands approval of Exa-cel, a CRISPR gene therapy, to children ages 2 and up with sickle cell disease or transfusion-dependent beta thalassemia, down from the previous 12+ age limit. Clinical data from CHOP shows 100% of followed patients achieved sustained remission of transfusions or severe pain crises over 16+ months.

This Wire brief sits within Fusion42's coverage of Digital Health. Wire is Fusion42's founder-focused intelligence feed: each story is connected to the funds and startups it names — every one with a live profile on Raise or Scout — so founders can follow the capital and the momentum behind the headline rather than just the headline itself. Wire analysis is one of the live surfaces Arthur reasons over.