ATLANTA (March 13, 2023) The CMT Research Foundation, a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease, has invested in a project at Vanderbilt University School of Medicine Basic Sciences that seeks to treat the problem of overproduction of PMP22, the primary genetic cause of CMT in patients with CMT1A. This project, to be run by Charles Sanders, PhD, Vice Dean and Professor of Biochemistry and Medicine, aims to restore PMP22 to healthy functioning levels.
“Vanderbilt has world-class expertise and capabilities to make breakthrough discoveries toward the development of novel therapeutics,” says Cleary Simpson, CEO of the CMTRF. “Recently, Dr. Sanders and his team developed new cell-based approaches to target the PMP22 protein and are now ready to direct these approaches to enable drug discovery. If this effort is successful, the project will be well positioned for further preclinical evaluation in mouse models.”
Dr. Sanders will be joined by Bruce Carter PhD, Professor of Biochemistry at Vanderbilt and member of the CMTRF Scientific Advisory Board, as co-principal investigator. Aspects of their research are supported by Ancora Innovation, a company wholly owned by affiliates of Deerfield Management that supports Vanderbilt’s life science research and leverages Deerfield’s expertise in accelerating drug development.