Gene therapy gives Manchester boy his life back

A 13-year-old from Oldham received Casgevy (exa-cel), a one-time CRISPR-based gene-editing treatment for beta-thalassaemia in November 2024 and has returned to Royal Manchester Children's Hospital to thank the staff who cared for him.

Adam Rehman, a 13-year-old from Oldham, returned to Royal Manchester Children's Hospital to thank the staff who treated him. He received a one-time CRISPR-based gene-editing therapy called Casgevy (also known as exa-cel) in November 2024 for beta-thalassaemia. The treatment edits a patient's own stem cells and was among the first licensed therapies to use CRISPR. Adam had relied on monthly blood transfusions since he was eight months old and had no suitable stem cell donor available. Key details: - Adam was one of the first children to receive Casgevy (exa-cel), a CRISPR-based gene-editing treatment, according to NHS Blood and Transplant (NHSBT). - The official list price for the treatment is reported as £1.65 million, though NHS and manufacturer Vertex agreed a confidential deal that is likely lower. - The therapy involves collecting a patient's stem cells, editing the faulty gene in a laboratory, and returning the edited cells so the body can produce healthy red blood cells. - Adam had monthly transfusions from infancy and his sister, who had the same condition, received a stem cell transplant in 2023; no match was found for Adam. - He reunited with the therapeutic apheresis specialist nurse Clair Baron at the hospital, and NHSBT staff including Teresa Baines commented on the significance of the treatment for patients. Summary: The CRISPR-based therapy has ended Adam's reliance on monthly transfusions and allowed him to return to school and everyday activities, according to NHS Blood and Transplant staff. He visited the hospital to thank the nurse who helped start his treatment. Undetermined at this time.