The Charcot-Marie-Tooth Association, through its Strategy To Accelerate Research (CMTA-STAR), has developed a preclinical toolbox of CMT animal and cell models to support therapeutic testing across multiple types of CMT. These resources enable fast, cost-effective evaluation of potential treatments.
Toolbox models are currently available for:
CMT1A, CMT1B, CMTX1 (also known as CMT1X or CMTX), CMT2A, CMT2E, CMT2Y, CMT4A, and CMT4J. Additional types are added as needed.
CMTA is the only patient advocacy organization offering a dedicated, preclinical CMT toolbox infrastructure to support therapeutic discovery and development.
KEY FEATURES
Access and Licensing
CMTA has created, licensed, or partnered toolbox models for therapeutic testing under favorable terms for CMTA-STAR partners in both commercial and academic settings. Created and licensed models are maintained at CMTA-affiliated CROs. Partnered models are housed in academic laboratories.
Animal Models
- CMT1A: Mouse and rat (licensed)
- CMT2A: Two rat models (created)
- CMT2E: Mouse model (licensed)
- CMTX1: Mouse model (licensed)
- CMT4A: Rat model (created)
- CMT1B: Four mouse models (partnered)
- CMT2J: Mouse model (partnered)
More information on CMT animal models can be found on the website of CMTA’s Alliance Partner, PsychoGenics.
In Vivo Drug Profiling
Evaluation includes drug tolerability, motor behavior, electrophysiology, biomarkers, histology, and peripheral nerve-specific pharmacokinetics (PK).
Stem Cell Lines
Over 20 patient-derived induced pluripotent stem cell (iPSC) lines for CMT1A, CMT1B, CMTX1, CMT2A, CMT2E, CMT2Y, and CMT4J. All lines are derived from clinically validated patients. Control lines are available.
More information on CMT cell lines can be found on the website of CMTA’s Alliance Partner, the New York Stem Cell Foundation (NYSCF).
In Vitro Drug Profiling
Assays include Schwann cell and motor neuron conversion from iPSCs, advanced axonal microscopy for CMT2A, and Schwann cell functional testing for CMT1A.
Project Planning
CMTA facilitates confidential study design with scientific experts and CMTA-STAR Advisory Board members. Drug identities do not need to be disclosed.
THERAPY TESTING ALLIANCES
- Comprehensive Testing: Conducted in partnership with CROs, specialized labs, and expert collaborators in the CMT research space.
- Efficiency Across Modalities: Toolbox infrastructure supports small molecules, genetic therapies, biologicals, and delivery technologies.
- Industry Reach: 40+ partners across pharma, biotech, and research services — spanning early discovery through clinical-stage development.
- De-risking Drug Development: Equips therapy developers with preclinical resources to establish proof of concept quickly.
- Clinical Translation: Works in parallel with CMTA’s clinical network and patient community to advance promising treatments.
CMTA-STAR TOOLBOX IMPACT
CMTA-STAR accelerates therapy development through a powerful combination of preclinical models, expert resources, and a global research network. The Therapy Testing Alliance connects drug developers with the tools, assays, and partnerships needed for rapid, cost-effective evaluation. This infrastructure has already supported programs advancing to clinical trials, including the fast-tracked development of treatments for CMT-SORD.
ACCESS THE TOOLBOX FOR YOUR CMT STUDIES
To explore the CMTA-STAR Preclinical Toolbox for your research, contact:
Steve Lin, PhD
Director of R&D Alliances
steve@cmtausa.org
