NHR-01

NHR-01 is an investigational adeno-associated virus (AAV) gene therapy designed for the treatment of GM2 gangliosidoses, a group of hereditary neurodegenerative lysosomal storage diseases that includes Tay-Sachs disease and Sandhoff disease. Developed by the New Hope Research Foundation in partnership with Forge Biologics, NHR-01 utilizes a novel AAV vector to deliver a corrective gene to the central nervous system. The therapy aims to restore the function of the hexosaminidase enzymes (Hex A and Hex B), which are deficient in these conditions, thereby preventing the toxic accumulation of GM2 gangliosides in neurons. It is currently being evaluated in Phase 1/2 clinical trials and utilizes proprietary minimally invasive delivery methods to target the central nervous system.