NeuroGT, Inc. is an investigator-initiated biotech startup founded, with the mission of developing curative gene therapy products for the treatment of devastating rare neurogenetic diseases, towards clinical application in humans and commercialization. The goal is to cure the disease and improve the quality of life for not only the patients but also their families. Disease Target: Our current targets are a group of rare neuropathic lysosomal storage diseases, including mucopolysaccharidosis (MPS) I, MPS II, MPS IIIA, MPS IIIB, MPS IIIC and MPS IIID, for which no treatments are available. Key technology: Our key technology is the platform gene replacement therapy targeting the root cause, using trans-blood-brain-barrier adeno-associated virus serotype 9 (AAV9) vector, via systemic and direct intrathecal delivery to the CNS. Our products for MPS I, MPS II and MPS IIIB are at mid to late preclinical stages. Our pre-clinical data demonstrate that each of these products may offer the cure if administered early.