Dr. James Dahlman, Georgia Institute of Technology and Emory School of Medicine

This innovative research collaboration seeks to overcome the challenges of delivering CMT therapies to the peripheral nervous system by using lipid nanoparticles (LNPs), considered among the most advanced delivery systems for genetic therapies. Dr. James Dahlman is a world-renowned expert in the design of lipid nanoparticles and has developed a system to rapidly identify LNPs that can enter other cell types. Using this approach, he has designed nanoparticles that efficiently deliver gene therapy payloads outside the liver, and several have been licensed for clinical development.

With funding from the CMT Research Foundation, Dr. Dahlman and his research team will utilize this approach to identify LNPs that can deliver genetic therapies to Schwann cells — the specialized cells that protect the peripheral nerves and whose dysfunction causes disease in the most common forms of CMT. The Food and Drug Administration (FDA) and European Medical Association (EMA) have already approved LNPs to treat another genetic condition. Additionally, LNPs serve as the delivery vehicle for two of the COVID-19 vaccines that have received FDA emergency use authorization and are now being rolled out worldwide. However, LNPs have not yet been tried in CMT because of the difficulty in designing them to target tissues other than the liver.

If successful, findings from this study could ultimately result in the design of therapies for CMT patients that restore normal protein function.