Research

Gene Replacement Therapy for CMT4C IND-Enabling Studies

With CMTA support, researchers at the University of Texas Southwestern in Dallas, TX, led by CMTA’s Strategy To Accelerate Research (CMTA-STAR) Advisory Board member Steven Gray, PhD, in collaboration with CMTA-STAR Advisory Board member Kleopas Kleopa, MD, at the Cyprus Institute of Neurology and Genetics, are advancing a potential genetic therapy for CMT4C toward human clinical trials. This project focuses on completing the necessary safety and regulatory steps required for clinical trial readiness.

The research team successfully held a Type B pre-IND (Investigational New Drug) meeting with the FDA (a formal consultation to review development plans and confirm required studies), receiving positive feedback that confirmed the next steps toward a full IND application. Preparations are underway for drug manufacturing and formal preclinical toxicology studies, both critical components of the IND submission process.

This project is part of CMTA’s donor-driven effort to accelerate the development of genetic therapies for CMT4C, bridging the gap between preclinical research and clinical application.

Project Foresee

Project Foresee is a CMTA-sponsored effort spearheaded by the Paulsen family to accelerate this CMT4C genetic therapy into the clinic. Building on years of CMTA-supported research led by Dr. Kleopa and now advanced in partnership with Dr. Gray, Project Foresee is focused on completing the steps required for an FDA IND application and initiating the first patient treatments.

Joined by the Vorpahl family and the Monique Manzella and JP Ross family, Project Foresee is designed as a three-year pathway with milestones that include FDA consultation, toxicology studies, and drug manufacturing. With a successful pre-IND meeting now behind us, the team is moving forward toward the ultimate goal of bringing this therapy to patients and paving the way for wider access in the future.

February 2026 Update

Following the previously held Type B pre-IND meeting with the FDA, the team received feedback outlining the remaining steps needed to support clinical trial initiation. Based on this guidance, the team is working with CMTA to begin drug manufacturing activities and conduct formal preclinical toxicology studies. Both are required components of an IND submission. These efforts represent critical steps toward securing regulatory clearance to begin first-in-human testing of this potential gene therapy for CMT4C.

The team expects drug manufacturing activities to begin in April–May 2026 and formal preclinical toxicology studies to initiate in the second half of 2026.