Patient from Kelowna is first to be cured of rare immune disease

An 18-year-old from Kelowna, Ty Sperle, has been reported as the first person cured of chronic granulomatous disease after receiving an experimental prime editing gene therapy; his immune function remained durable six months after treatment.

An 18-year-old patient from Kelowna has been reported as the first person cured of chronic granulomatous disease (CGD) after receiving a gene modification treatment known as prime editing. He was diagnosed with CGD at age five and experienced frequent skin infections and an invasive bacterial lung infection. The case is described in the New England Journal of Medicine and involved treatment carried out at CHU Sainte-Justine in Montreal. Medical teams drew and enriched the patient’s blood stem cells, treated them with a gene editing product to correct the mutation, and the patient was discharged after 24 days in hospital care. Key facts: - The patient is identified as Ty Sperle, now 18, who was diagnosed with CGD at age five. - CGD is an inherited immune disorder reported to affect about one in 200,000 children and reduces the body’s ability to fight infections and inflammation. - The report describes this as the first recorded cure of CGD using a gene modification technique called prime editing. - The trial was set up by US-based Prime Medicine and run at CHU Sainte-Justine; the treating team included Dr. Élie Haddad and BC clinicians such as Dr. Stuart Turvey. - Sperle was discharged after 24 days in hospital care and, according to the report, his immune system’s antimicrobial activity remained durable six months after the procedure. Summary: The report documents a single case in which experimental prime editing of blood stem cells was followed by durable immune recovery six months after treatment, drawing attention from clinicians and health officials. Undetermined at this time.