In CMT1A the body produces too much PMP22 protein in the peripheral nerves, and a major goal of drug development in CMT1A is to reduce the amount of PMP22 protein in nerve cells. Samsara Therapeutics has discovered a number of drug-like molecules called autophagy enhancers that stimulate the body’s natural ability to remove and recycle damaged or dysfunctional proteins. In an early preclinical study, they showed that this class of drugs improves outcomes in CMT1A model mice, reducing PMP22 protein levels and restoring measures of nerve structure and function. This partnership will allow Samsara to further develop these molecules and prepare for clinical trials. The project is divided into two broad sets of activities – compound optimization and IND-enabling studies. If the project is successful, it will result in the identification of a lead candidate and a data package to submit to the FDA ahead of potential clinical trials. The principal investigator for the project is John K. Blackwood, PhD, Vice President of Biology (pictured).