Dedicated to Research Committed to a Cure

Investing in innovative research to find treatments and cures for Charcot-Marie-Tooth disease.

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ASGCT

NMD Pharma Recruiting CMT Type 2 Patients for Phase 2 Study

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ASGCT

Live Webinar: The Story and The Science Behind Augustine Therapeutics’ $85M Raise: April 3, 2025: 7-8 pm ET

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Dr. Jerry Mendell, a leading gene therapy pioneer, will speak at "OHio, What a Night" on April 10, in Columbus

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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 5 therapeutics that are approaching clinical trials

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CMTRF Case Studies

We funded DTx Pharma (Novartis). Now their therapeutic treatment for CMT1A is going to clinical trials.

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. With our help, DTx was able to use successful results to get additional funding from NIH. Now, DTx Pharma was purchased by Novartis for $1 billion and the therapeutic treatment for CMT1A is going to clinical trials.

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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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CMTRF underwrites Augustine Therapeutics’ HDAC6 inhibitor. Now, the Belgian Pharmaceutical company is moving to phase ½ 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 raised $18.5 Million in a Series A First Closing, with proceeds from the Series A being used to advance Augustine’s first clinical candidate AGT100216 into a Phase 1/2 clinical trial in 2025.

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