Gene Therapy for Sickle Cell Disease: New Recommendations

Clinical Takeaway

: The first consensus recommendations address patient selection, stem cell collection, myeloablative conditioning, and long-term follow-up for SCD gene therapy.

An overview of Gene Therapy for Sickle Cell Disease: Practice Recommendations from the American Society for Transplantation and Cellular Therapy and the International Society for Cell & Gene Therapy (2026)

Background

Two ex vivo autologous gene therapies — exagamglogene autotemcel (CRISPR-based gene editing) and lovotibeglogene autotemcel (lentiviral gene addition) — were approved in 2023 for patients ≥12 years of age with severe sickle cell disease (SCD) and a history of vaso-occlusive crises. Gene therapy involves a complex, multistep process, prompting the American Society for Transplantation and Cellular Therapy and the International Society for Cell & Gene Therapy to issue the first consensus recommendations for clinical implementation (…

Reviewing Author

Anjali A. Sharathkumar, M.B.B.S., M.D., M.S.

Disclosures

Consultant/Advisory BoardGenentech

Grant/Research SupportX4 Pharmaceuticals; Pfizer; Health Resources and Services Administration; American Thrombosis and Hemostasis Network/CDC; Carver College of Medicine

Editorial BoardsJournal of Thrombosis and Haemostasis

Leadership Positions in Professional SocietiesInternational Society on Thrombosis and Haemostasis (Finance Committee Member); American Society of Hematology Clinical Research Translational Institute