ROCKVILLE, Md., Dec. 1, 2020 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV® Technology Platform, today announced that the first patient has been dosed in the Phase I/II trial of RGX-111 for the treatment of Mucopolysaccharidosis Type I (MPS I). RGX-111 is an investigational one-time gene therapy designed to deliver the α-l-iduronidase (IDUA) gene directly to the central nervous system (CNS) using the NAV AAV9 vector.
MPS I is a rare, autosomal recessive genetic disease caused by deficiency of IDUA, an enzyme required for the breakdown of the polysaccharides in lysosomes. These polysaccharides, called glycosaminoglycans (GAGs), accumulate in tissues of MPS I patients, resulting in characteristic storage lesions and diverse clinical signs and symptoms including in the central nervous system (CNS), which can include excessive accumulation of fluid in the brain, spinal cord compression, and cognitive impairment.