CTM4J

CTM4J is an investigational gene therapy being developed by **The Columbus Foundation** for the treatment of **CTM4J-related neurological disorder**, also known as **Charcot-Marie-Tooth disease type 4J (CMT4J)**. The therapy utilizes an adeno-associated virus (AAV) vector to deliver a functional copy of the *FIG4* gene to the nervous system. CMT4J is a rare, autosomal recessive, and rapidly progressive neurodegenerative disorder caused by mutations in the *FIG4* gene, which encodes a phosphoinositide 5-phosphatase essential for endolysosomal trafficking. By restoring the expression of the FIG4 protein, CTM4J aims to normalize cellular lipid metabolism and halt the progression of motor and sensory neuropathy. The program is currently in Phase 1/2 clinical development.

Other names

FIG4 gene therapyFIG-4 gene therapyFIG 4 gene therapyCMT4J gene therapyCMT-4J gene therapyCMT 4J gene therapy