A gene therapy alliance in X-linked chronic granulomatous disease with Orchard Therapeutics

Orchard Therapeutics (“Orchard”), a clinical-stage biotechnology company dedicated to transforming the lives of patients with rare disorders through innovative gene therapies announces today a strategic alliance with Généthon, to develop gene therapy for X-linked chronic granulomatous disease (“X-CGD”). Généthon is a non-profit research and development organization whose mission is to develop gene therapies for orphan genetic diseases from research to clinical validation.
X-linked CGD is a primary immune deficiency resulting from a mutation on the X-chromosome and affecting mainly boys. Patients suffering from this disease are susceptible to severe life-threatening bacterial and fungal infections and excessive inflammation characterized by granuloma formation in any organ, for instance, the gastrointestinal and genitourinary tract. Repeated episodes of infection and inflammation severely reduce the life expectancy and quality of life of individuals.