The EURORDIS Company Award for Innovation recognises Orchard Therapeutics’ leadership in harnessing the potential of hematopoietic stem cell gene therapy to change the course of severe inherited disorders.
EURORDIS particularly acknowledges the company for developing gene therapy for early onset metachromatic leukodystrophy (MLD), which addresses the urgent need of young patients and their families to treat a disease for which no approved therapeutic option previously existed.
