P037: Design of a Multi-Center Randomized Phase 3 Clinical Trial (HURCULES) Evaluating OTL-203 in MPS-IH Vs Allogeneic Hematopoietic Stem Cell Transplantation

Mucopolysaccharidosis type I Hurler (MPS-IH) is an autosomal recessive lysosomal storage disorder characterized by deficiency of alpha-L-iduronidase (IDUA), which is responsible for the degradation of glycosaminoglycans (GAGs). The accumulation of GAGs throughout the body, including the brain, results in tissue damage and multi-organ dysfunction causing a wide range of musculoskeletal, cardiopulmonary, ophthalmic and auditory abnormalities, progressive neurologic disease, and early death. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the current standard of care (SoC) but is associated with significant complications and residual disease burden, having a limited effect on skeletal, ophthalmic, cardiac, and other manifestations of MPS-IH.