Sickle cell disease (SCD) gene therapy products, namely Vertex Pharmaceuticals' and CRISPR Therapeutics’ gene editing therapy exagamglogene autotemcel (exa-cel; marketed as Casgevy) and bluebird bio’s gene addition therapy lovotibeglogene autotemcel (lovo-cel; marketed as Lyfgenia), have been commercially approved in the United States since late 2023. As more clinics adopt these products for standard use, they need to take careful steps to insure appropriate infrastructure and protocols are in place for safe and effective administration to patients.
CGTLive® recently spoke to Robin E. Miller, MD, the director of the Sickle Cell Disease Program at Nemours Children’s Hospital, at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, held December 6 to 9, 2025, in Orlando, Florida. During the course of the discussion, Miller went over the various practical considerations for implementation of SCD gene therapy into clinical practice, including how to talk to patients about the therapies, and the need for long-term follow-up.