Steven Gray, PhD, an Associate Professor at the University of Texas Southwestern Medical Center has developed a gene therapy for one rare form of CMT called Giant Axonal Neuropathy, and aims to develop similar gene therapy treatments for other forms of CMT. Dr. Gray is a member of the CMTA’s STAR Advisory Board and his work is supported via CMTA sponsored research grants.
Dr. Gray’s lab has developed gene therapy treatments for three rare forms of CMT, Giant Axonal Neuropathy (GAN), CMT4J, and CMT4A. The treatment for GAN has been in human clinical trials since 2015, the work for CMT4J is moving toward a human clinical trial, and the work on CMT4A is in an early stage of development and testing. The lab’s general gene therapy development strategy is a “platform” approach. Six other similar treatments developed by the lab have already moved into human clinical trials for other rare diseases. Of the 100+ genes causing the different forms of CMT, several are amenable to treatment using this “platform” approach with current gene delivery technology. The other forms of CMT will likely require new innovations and technology to enable gene therapy treatments to advance – work that is being supported through separate CMTA funded projects.
Katherine Forsey, PhD, the CMTA’s Chief Research Officer said “The Gray laboratory’s innovative “platform” approach to the development of gene therapies for certain rare types of CMT has been very successful and the team has considerable experience advancing their work into human clinical trials. With support from the CMTA community, we will continue funding gene therapy advances and exploring additional CMT types that could benefit”.
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