Sickle cell disease after Casgevy: seven companies to watch in 2026

Casgevy’s approval changed the sickle cell disease landscape. Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, the therapy showed that the disease could be tackled at its genetic root, using CRISPR/Cas9 to edit a patient’s own blood stem cells and increase the production of fetal hemoglobin. 

But Casgevy also made clear that curing sickle cell disease is not the same as making a cure broadly accessible. The therapy remains an ex vivo, autologous cell therapy; stem cells must be collected, edited outside the body, and reinfused after myeloablative conditioning. That makes treatment complex, infrastructure-heavy, and costly, with a U.S. price tag of $2.2 million.