Dr. Kleopas Kleopa, Cyprus Institute for Neurology and Genetics, and Dr. Scott Q. Harper, Nationwide Children’s Hospital

The CMT Research Foundation partnered with the Cyprus Institute of Neurology and Genetics and Nationwide Children’s Hospital to study a gene therapy approach to lower levels of PMP22, the protein whose overexpression causes CMT1A. The researchers showed that the therapy led to a substantial reduction in the PMP22 protein in the peripheral nerve tissues of mice with CMT1A, along with improvements in nerve myelination and the ability of the nerves to conduct electrical impulses. Most importantly, the therapy improved strength and coordination in the mice. The therapy provided these benefits whether the treatment was given at early or later stages of the neuropathy. Additional data from a project extension that funded a longer treatment period, an examination of the immune response to treatment, and an exploration of neurofilament light as a potential blood-based biomarker for the therapy also provided promising data.

Given the strength of these data, the asset is now in the process of being licensed by emerging biotechnology innovator Armatus Bio and will be known as ARM-101. This represents the second major success for the CMT Research Foundation. Just two and a half years after we began discussions about this project with Dr. Kleopa, the therapy is showing strong results consistent with a viable treatment for CMT1A and is now being adopted by industry to accelerate development for potential clinical trials.