FDA Approves Two Gene Therapies for Sickle Cell Disease.

The article focuses on the U.S. Food and Drug Administration's recent approval of two gene therapies, Casgevy and Lyfgenia, for treating severe sickle cell disease in patients aged 12 and older. Topics discussed include the CRISPR technology in Casgevy, the one-time treatments' safety and efficacy, their potential economic impact, and the cautious optimism expressed by experts regarding these groundbreaking therapies for a historically underserved patient community.