clemidisogene lanparvovec

Clemidisogene lanparvovec (RGX-121) is an adeno-associated virus serotype 9 (AAV9) vector-based gene therapy designed for the treatment of Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome. Developed by REGENXBIO in collaboration with NS Pharma, the therapy delivers a functional copy of the human iduronate-2-sulfatase (IDS) gene directly to the central nervous system. MPS II is a rare, X-linked lysosomal storage disorder caused by a deficiency in the IDS enzyme, which leads to the accumulation of glycosaminoglycans (GAGs) in cells, resulting in progressive neurodevelopmental decline and physical symptoms. By restoring IDS enzyme activity, clemidisogene lanparvovec aims to reduce GAG levels in the cerebrospinal fluid and stabilize or improve cognitive function. The therapy is administered via a single intracisternal injection. As of 2024, a Biologics License Application (BLA) has been initiated for the treatment of pediatric patients with MPS II.

Other names

AAV9-IDSAAV-9-IDSAAV 9-IDS