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FDA Expands Use of Gene Therapy for Treatment of Patients Ages 2+ with Sickle Cell ...
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Fusion42 · 21 July 2026 · Fusion42 review
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.
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If you build infrastructure for cell therapy manufacturing, conditioning, or logistics, you've just doubled your addressable patient population overnight. Vertex and CRISPR Therapeutics now need to scale production for a younger cohort with longer expected lifespans - that's volume and contract lock-in.
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1 source · 20 Jul 2026
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