CureCMT

CureCMT4J is a patient-led biotechnology organization dedicated to developing a cure for Charcot-Marie-Tooth disease type 4J (CMT4J), a rare and aggressive neurodegenerative disorder. The organization focuses on advancing a gene therapy program that utilizes an adeno-associated virus (AAV9) vector to deliver a functional copy of the FIG4 gene. By funding research and collaborating with academic and clinical partners, CureCMT4J has successfully transitioned its lead candidate from preclinical development into first-in-human clinical trials. Their mission is to provide a life-saving treatment for patients affected by this ultra-rare condition while establishing a model for other rare disease drug development.