Hurler syndrome (mucopolysaccharidosis type I) is a lysosomal storage disorder characterized by deficiency of a specific enzyme, alpha-L-iduronidase, responsible for the degradation of glycosaminglycans (GAGs), and results in excessive buildup of GAGs throughout the body, leading to symptoms also in the central nervous system (CNS). JCR has developed technology to penetrate the blood-brain barrier (J-Brain Cargo) and, based on successful mouse experiments, has initiated the development of a recombinant alpha-L iduronidase (JR-171) as a drug candidate to treat Hurler syndrome patients, reducing GAG accumulation in the brain.

JCR news release, October 27, 2017