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.

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.

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.