CRISPR Therapeutics

Co-developed Casgevy (exa-cel) with Vertex Pharmaceuticals — the first CRISPR-based therapy approved by the FDA (December 2023) for sickle cell disease and transfusion-dependent beta-thalassemia. On July 1, 2026 the FDA granted supplemental approval extending Casgevy to sickle cell patients as young as 2 years old, down from the previous minimum age of 12.