June 05, 2026 NeuroGT, a mission-driven, clinical-stage gene therapy company, announced U.S. Food and Drug Administration (FDA) clearance of its Investigational New Drug Application (IND) for NGT-104, an AAV9 gene replacement product to treat MPS IIIB. The approved Phase I/II human clinical trial is expected to start enrolling patients in January 2027.
MPS IIIB (Sanfilippo Syndrome Type B) is a rare, fatal genetic disorder caused by mutations in the NAGLU gene. Children with the disorder often display few symptoms until they reach 1-2 years of age. As heparan sulfate accumulates, causing progressive damage to the brain and peripheral organs, patients gradually lose developmental milestones. Common symptoms include cognitive delays, behavioral challenges, hyperactivity, poor sleep, seizures, and hearing and vision loss. While individual symptoms vary, MPS IIIB is always fatal, typically by the mid to late teens. There are currently no FDA approved treatments.
NeuroGT’s AAV9 treatment for MPS IIIB is engineered with proprietary codon optimization, maximizing homogeneous distribution of the gene product and therapeutic impact after crossing the blood-brain barrier. Delivered by a single intravenous infusion, it is expected to be a one-time treatment that prevents further disease progression, improving the quality of life for affected children and their families.
NeuroGT is a clinical-stage gene therapy Public Benefit Corporation committed to using its breakthrough AAV9 gene therapy platform to develop and commercialize one-time treatment cures which will prevent or halt disease progression in ultra-rare pediatric neurodegenerative diseases, including Hurler, Scheie, and Hurler-Scheie
Syndromes (MPS I), Hunter Syndrome (MPS II) and Sanfilippo Syndromes A, B, C, and D (MPS IIIA, B, C, D), giving these children and their families the greatest chance of living full, healthy and happy lives.
To enable successful gene therapy treatments for these diseases, NeuroGT has developed several breakthrough gene therapy enabling technologies that are widely applicable to almost all gene therapy treatments, and to support its mission financially NeuroGT is actively seeking gene therapy company partners to commercialize these breakthrough enabling technologies for other gene therapy disease targets.
These enabling technologies include: a low-cost, high-yield rAAV producing cell line; a highly effective antibody depletion technology enabling AAV gene therapy treatment or retreatment for patients with pre-existing antibodies to AAV; and an Extracellular Vesicle Cargo technology for GTs with non-secreted proteins which
enables the bystander effect, significantly reducing dose size and potential risks of toxicity.
