Therapeutic Pipeline

RGX-111 for MPS I

RGX-111 is our product candidate for the treatment of Mucopolysaccharidosis Type I (MPS I).

RGX-111 is designed to use the NAV® AAV9 vector to deliver the human α-l-iduronidase (IDUA) gene to the central nervous system (CNS).

MPS I is a rare recessive genetic disease caused by deficiency of IDUA, an enzyme required for the breakdown of polysaccharides heparan sulfate and dermatan sulfate in the lysosomes of cells. Many patients develop symptoms related to glycosaminoglycan storage in the CNS, which can include excessive accumulation of fluid in the brain (hydrocephalus), spinal cord compression and cognitive impairment.

Current standard of care treatments leave a significant unmet need for a method to safely achieve long-term IDUA reconstitution in the CNS.

Delivery of the enzyme that is deficient within cells in the CNS could provide a permanent source of secreted IDUA beyond the blood-brain barrier, allowing for long-term cross correction of cells throughout the CNS.

RGX-111 has received Orphan Drug Product, Rare Pediatric Disease and Fast Track designations from the FDA.


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