MPS III (Sanfilippo Syndrome) Program

About NeuroGT

NeuroGT is a private, preclinical-stage biotech founded in 2021 and based in Cambridge, USA, developing transformative gene therapies for rare neurogenetic disorders. The company's core strength lies in its integrated technology platform, which includes an AAV9 delivery system, an antibody-clearing technology to enable re-dosing, and novel manufacturing capabilities. Led by a founder with a strong translational track record in AAV gene therapy for MPS disorders, NeuroGT is targeting high-unmet-need pediatric conditions with the goal of providing durable, one-time treatments. The company is pre-revenue and appears to be seeking partnerships and investment to advance its pipeline.

Therapeutic Areas