# FDA Approves First Gene Therapy for Young Children with Sickle Cell Disease (Casgevy Supplemental Approval)

> fda · REPORTED · 2026-07-01

On July 1, 2026, the FDA issued a supplemental approval for Casgevy (exagamglogene autotemcel), the CRISPR-based gene therapy for sickle cell disease, expanding its label to cover patients as young as 2 years old, extending a landmark gene-editing treatment to the pediatric population.

About FDA / Vertex & CRISPR Therapeutics: Casgevy is a one-time CRISPR/Cas9 gene-editing therapy co-developed by Vertex Pharmaceuticals and CRISPR Therapeutics that functionally cures sickle cell disease by reactivating fetal hemoglobin production in patients' own stem cells.

- Company: FDA / Vertex & CRISPR Therapeutics
- Sector: therapeutics
- Primary source: https://www.globenewswire.com/news-release/2026/07/17/3329256/0/en/FDA-Approves-First-Oral-PCSK9-Inhibitor-to-Lower-LDL-Cholesterol-in-Adults-with-High-Cholesterol.html

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Source: MedFuel Intel news monitor. Verification tier: REPORTED. Informational only, not investment advice. Corrections: media@medfuelinc.com.