clemidsogene lanparvovec

Clemidsogene lanparvovec (RGX-121) is an adeno-associated virus (AAV) gene therapy being developed for the treatment of Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome. MPS II is a rare, X-linked recessive lysosomal storage disorder caused by a deficiency in the enzyme iduronate-2-sulfatase (I2S), which leads to the accumulation of glycosaminoglycans (GAGs) in tissues and the central nervous system (CNS). RGX-121 utilizes the NAV AAV9 vector to deliver a functional human iduronate-2-sulfatase (IDS) gene directly to the CNS via intracisternal administration. This approach is designed to provide long-term cross-correction of cells throughout the CNS, potentially halting or reversing the neurocognitive decline associated with the severe form of the disease. The therapy has received multiple regulatory designations, including Orphan Drug, Fast Track, and RMAT from the FDA.

Brand names

RGX-121RGX121RGX 121

Other names

RGX-121RGX121RGX 121