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The FDA’s approval of Casgevy for children as young as two promises to shift pediatric sickle cell care from lifelong management to a one-time curative intervention, but raises urgent questions about cost, access, and health equity.
The FDA's expansion of Casgevy to children aged 2–11 solidifies Vertex and CRISPR Therapeutics' lead over bluebird bio’s Lyfgenia, opens a lucrative new patient segment, and validates the safety of CRISPR gene editing in very young patients.
While gene therapies offer unprecedented curative potential for rare and chronic diseases, high costs and logistical hurdles are creating a significant access gap in the U.S. healthcare system. This briefing examines the systemic challenges preventing widespread adoption and the emerging financial models designed to bridge the divide.