Shashi Murthy, PhD, Nanite Inc., Boston, MA

The CMT Research Foundation has partnered with Nanite Inc., a Boston-based biotechnology company that specializes in advanced techniques to improve drug delivery. Developing safe, effective methods to deliver therapeutics to the peripheral nervous system is a critical step towards a cure for CMT. The cells of the peripheral nervous system are shielded by the blood-nerve barrier, which makes it difficult to achieve effective local concentrations of the therapeutic. This project will explore the delivery of genetic medicines to Schwann cells via polymer nanoparticles (PNPs), an emerging technology for drug delivery. PNPs will be optimized both for their ability to cross the blood-nerve barrier and their ability to deliver genetic therapies to Schwann cells. This project is targeting CMT1A, but if successful, the polymersome technology could be applied to other types of CMT.