Regenxbio Dosed First Patient with RGX-111 for MPS I
Regenxbio (RGNX) has dosed the first patient with RGX-111 in the Phase I/II trial for the treatment of Mucopolysaccharidosis Type I (MPS I).
About Regenxbio Product RGX-111
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.
RGX-111 is a product candidate for the treatment of MPS I also known as Hurler syndrome. RGX-111 uses the AAV9 vector to deliver the α-l-iduronidase (IDUA) gene. Delivery of the enzyme that is deficient within cells in the CNS is expected to provide a permanent source of secreted IDUA beyond the blood-brain barrier, allowing for long-term cross-correction of cells throughout the CNS. This strategy could also provide rapid IDUA delivery to the brain, potentially preventing the progression of cognitive deficits that otherwise occurs in MPS I patients. RGX-111 has received orphan drug products, rare pediatric disease and Fast Track designations from the U.S. Food and Drug Administration (FDA).