NGT-104

NGT-104 is an investigational adeno-associated virus serotype 9 (AAV9) vector-based gene replacement therapy designed for the treatment of Mucopolysaccharidosis Type IIIB (MPS IIIB, also known as Sanfilippo syndrome type B). Developed by NeuroGT, NGT-104 is administered as a one-time intravenous infusion to deliver a functional copy of the *NAGLU* gene, which encodes the lysosomal enzyme alpha-N-acetylglucosaminidase. MPS IIIB is a rare, progressive, and devastating neurodegenerative lysosomal storage disorder caused by mutations in the *NAGLU* gene, leading to a deficiency in alpha-N-acetylglucosaminidase and the toxic accumulation of heparan sulfate. By restoring functional enzyme expression, NGT-104 aims to halt or slow disease progression. In August 2026, the FDA cleared the Investigational New Drug (IND) application for NGT-104, allowing NeuroGT to initiate a Phase I/II clinical trial.