About

About CMT Research Foundation The CMT Research Foundation is a patient-led, non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease. CMT affects one in 2,500 people (about the same prevalence as multiple sclerosis), including 150,000 Americans and more than 3 million people around the world. We have one single mission: to raise funds to invest in science that will lead to treatments and cures for CMT. Our impact goes beyond simply making investments. We identify key problems or gaps in progress to a cure, then aggressively find partners to attack the problems. We introduce CMT to the greater drug development community, something that has rapidly increased the number of experts working to solve the disease. We fund projects with the highest likelihood of leading to clinical trials and approved treatments in the near term. All investments are gated, meaning projects need to achieve milestones defined for each phase of work before receiving funding for the next. Every contract includes a return on the CMT Research Foundation’s initial investment which is then reinvested in new projects. About CMT Research Foundation The CMT Research Foundation is a patient-led, non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease. CMT affects one in 2,500 people (about the same prevalence as multiple sclerosis), including 150,000 Americans and more than 3 million people around the world. We have one single mission: to raise funds to invest in science that will lead to treatments and cures for CMT. Our impact goes beyond simply making investments. We identify key problems or gaps in progress to a cure, then aggressively find partners to attack the problems. We introduce CMT to the greater drug development community, something that has rapidly increased the number of experts working to solve the disease. We fund projects with the highest likelihood of leading to clinical trials and approved treatments in the near term. All investments are gated, meaning projects need to achieve milestones defined for each phase of work before receiving funding for the next. Every contract includes a return on the CMT Research Foundation’s initial investment which is then reinvested in new projects. The CMT Research Foundation is a non-profit patient advocacy group whose mission is to fund research that leads to the development of treatments and cures for CMT. Founded in 2018, the CMT Research Foundation has funded over 25 projects with biotechs and academic labs that support the critical science that overcomes barriers to therapeutics. We currently seek partnerships with companies who can deliver RNA therapies to the peripheral nervous system, help support the health of the PNS and develop gene therapies that address mutations that cause both loss of function and gain of function. Companies funded by the CMT Research Foundation include DTx Pharma (acquired by Novartis July 2023), Augustine Therapeutics (completed an $83m Series A March 2025), Alloy, a division of 82VS and AcuraStem ($580M licensing agreement with Takeda). CMT (Charcot-Marie-Tooth) is a monogenic peripheral neuropathy. Over 140 genes have been identified to cause CMT. Estimated prevalence of CMT is 3M people globally and 150,000 in the US. People with CMT live in fear as they watch their bodies deteriorate - some rapidly and others more slowly. Many people with CMT use wheelchairs, canes or leg braces to ambulate and have strong deficits in their fine motor skills. People with CMT have little hope as there are presently no approved treatments to slow, stop or reverse the progression of the disease, and only a few clinical trials underway or planned. The CMT Research Foundation is meeting this unmet need by understanding the key barriers to drug development and overcoming them through funding and partnerships with companies with novel technologies that address this underserved community. .