Orchard Therapeutics, a clinical-stage biotechnology company dedicated to transforming the lives of patients with rare disorders through innovative gene therapies, announced one of its scientific founders and scientific advisory board member, Professor Donald Kohn, has been recognised with the Pediatric Blood and Marrow Transplant Consortium (PBMTC) Lifetime Achievement Award.
The PBMTC Lifetime Achievement Award recognises outstanding individuals who have made major contributions to the field of paediatric haematopoietic stem cell transplantation.
The award highlights and honours the contributions and careers of individuals whose work exemplifies the PBMTC mission statement: “To support research and education to improve the availability, safety and efficacy of hematopoietic cell transplantation and other cellular therapeutics for children and adolescents.”
Professor Kohn has focused his career on the development of haematopoietic stem cell gene therapy for immunodeficiencies, haemoglobinopathies and inborn errors of metabolism. His work includes the development of tools for efficient gene addition or correction and persistent transgene expression, for the amelioration of genetic disease.
A formal presentation of the award was followed by a presentation of key achievements in Professor Kohn’s research. Professor Kohn described the 30-year journey developing ADA-SCID gene therapy, recognising the contributions of many in the field, leading to the development of Orchard’s lead product, OTL-101, for the treatment of ADA-SCID.
Professor Kohn said: “I am deeply honored to be recognized by the Pediatric Blood and Marrow Transplant Consortium with their Lifetime Achievement Award and look forward to continuing the development of transformative treatments for patients with severe and life-threatening diseases.”
Professor Bobby Gaspar, CSO of Orchard said: “This prestigious award recognizes the pioneering work of Professor Kohn in advancing cutting-edge research for hematopoietic stem cell gene therapy. At Orchard, we are honored to collaborate with professor Kohn and UCLA to bring transformative treatments to patients across the world. Dr Kohn’s dedication to patients, humility and team spirit are an inspiration for us all.”