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Taking Aim at CMT1J with a genetic medicine

Taking Aim at CMT1J with a genetic medicine

Jul 16, 2026 |

The CMT Research Foundation, in partnership with the 1J Foundation, has funded a new study at the University of Miami led by Dr. Stephan Züchner to evaluate a newly-identified genetic variant linked to Charcot-Marie-Tooth disease type 1J and to test whether a targeted...

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CMT affects 1 in 2,500 people in the United States and more than 3 million people worldwide.

 

There is currently no cure for CMT.

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WHO WE ARE

Accelerating a cure through research funding.

The CMT Research Foundation is a patient-led non-profit 100% focused on delivering treatments and cures for Charcot-Marie-Tooth, which affects 3 million people worldwide.

Since 2018, our donors and scientists have put CMT front and center in the pharmaceutical, biotech and investor communities, making a cure for all types of CMT a not-too-distant reality.

We have funded 6 therapeutics that are in preclinical or active trials

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CMTRF Case Studies

Novartis' EKD060 compound for CMT1A is going to clinical trials

Novartis acquired CMTRF funed partner DTx Pharma in 2023 for $1 billion. DTx Pharma created a platform that delivers therapeutics to cells in a safe way. We introduced DTx Pharma to CMT and funded their first CMT research with a grant of $128,000. The results from the testing were positive and Novarts is now taking it to clincial trials.

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CMTRF underwrites Augustine Therapeutics’ HDAC6 inhibitor. Now, the Belgian Pharmaceutical company is in a Phase 1 clinical trial.

CMTRF helped Augustine Therapeutics underwrite the development of novel potent and subtype-selective small-molecule inhibitors of the cytosolic histone deacetylase 6 (HDAC6) enzyme to reverse and inhibit the pathophysiological changes associated with neuromuscular diseases. Now Augustine Therapeutics has completed their Series A financing round and raised $85 million, which will be used to advance their lead candidate, AGT-100216, through a Phase I clinical trial. In May 2025, Augustine announced the dosing of their first patient in a Phase 1 clinical trial.

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Armatus Bio licenses ARM-101 from CMTRF funded-partners Dr. Kleopa (Cyprus Institute of Neurology and Genetics) and Dr. Harper (Nationwide Children’s Hospital in Ohio) for clinical trials.

Drs. Kleopa and Harper developed a therapy that safely delivers miRNA, a PMP22 silencer, to the peripheral nervous system. Upon successful completion of their CMTRF-funded project, Armatus Bio licensed ARM-101 for further testing and now the therapeutic treatment is preparing for clinical trials.

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