RGX-111

RGX-111 is an investigational, one-time gene therapy designed to treat mucopolysaccharidosis type I (MPS I), a rare neurodegenerative lysosomal storage disorder caused by deficiency of the α-l-iduronidase (IDUA) enzyme. The therapy uses a recombinant adeno-associated virus serotype 9 (AAV9) vector to deliver the IDUA gene directly to the central nervous system (CNS). By enabling CNS cells to produce and secrete functional IDUA enzyme, RGX-111 aims for long-term cross-correction of cells throughout the CNS, potentially preventing or slowing cognitive decline and other neurological symptoms associated with MPS I. The therapy is administered via intracisternal injection. Clinical trials have shown that RGX-111 is well-tolerated, with no drug-related serious adverse events reported; biomarker data indicate increased IDUA activity in cerebrospinal fluid and reductions in heparan sulfate levels post-treatment[1][3][5][6][8]. Developed using REGENXBIO’s NAV AAV9 platform, RGX-111 has received orphan drug product, rare pediatric disease, and Fast Track designations from the FDA[4][7].

Other names

AAV9-IDUAAAV-9-IDUAAAV 9-IDUAIDUA AAV9 gene therapy