The first CRISPR medicine is approved: gene editing as a sickle-cell therapy

In December 2023 the FDA approved Casgevy (exagamglogene autotemcel), one of the first two cell-based gene therapies for sickle cell disease in patients 12 and older - and the first medicine built on CRISPR/Cas9 gene editing. Europe followed fast: EMA recommended approval of the first CRISPR medicine, with an EU-wide conditional marketing authorisation from February 2024. Edited stem cells restart fetal hemoglobin production, offering a potential one-time functional cure.

Casgevy made history as the first CRISPR-based medicine ever approved - FDA green light Dec 2023, EU authorization Feb 2024 - a one-time therapy for sickle cell disease.

The receipts

• U.S. FDA: FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease
• European Medicines Agency: First gene editing therapy to treat beta thalassemia and severe sickle cell disease