RDGT-101

RDGT-101 is an adeno-associated virus serotype 9 (AAV9)-based gene therapy developed for the treatment of mucopolysaccharidosis type IIIB (MPS IIIB), also known as Sanfilippo syndrome type B. MPS IIIB is a rare, progressive lysosomal storage disorder caused by mutations in the NAGLU gene, which leads to a deficiency in the enzyme N-acetyl-alpha-glucosaminidase and the subsequent toxic accumulation of heparan sulfate in the central nervous system. RDGT-101 is designed to deliver a functional copy of the NAGLU gene directly to the brain via intracerebroventricular injection, aiming to restore enzyme activity and mitigate the neurodegenerative symptoms associated with the disease. The therapy is currently being evaluated in clinical trials for safety, preliminary efficacy, and pharmacokinetics in pediatric patients with early-stage MPS IIIB.