To keep the CMT pipeline robust and diversified, CMTRF held 63 meetings in 2025 with biotech companies that are either new to CMT or expanding their focus in the field. These scouting conversations introduce potential partners to the science, the patient community, and specific opportunities where their platforms can make a difference—and they are a major reason the CMT drug development pipeline has doubled in recent years, with more than two dozen drug developers now actively engaged in CMT programs.

Velocity is CMTRF’s three‑year campaign, launched in late 2024 with a leadership gift, to raise $20 million dedicated exclusively to expanding, advancing, and accelerating treatments and cures for all types of CMT. Donors have already committed $8 million—40% of our goal—and Velocity funds are already being deployed to critical projects across the pipeline, from gene therapies and novel small molecules to tools that make future clinical trials faster, smarter, and more likely to succeed.

There are now 7 active CMT clinical trials led by CMTRF partners or in disease areas where CMTRF has played a catalytic role, including gene therapies and small molecule approaches that are entering or preparing for Phase 1/2 studies.
Each new trial represents years of
foundational research which was seeded or de-risked by philanthropy and gives patients real opportunities to participate in testing emerging therapies.

In 2025, CMTRF invested $2.6 million in 10 new research projects. These projects span multiple modalities—from small molecules to gene therapy and next‑generation delivery technologies—and are designed to move quickly toward clinical candidates and first‑in‑human trials.

Read more about our research portfolio.

More than 400 people took part in the 2025 Global CMT Research Convention, including patients, caregivers, scientists, drug developers, and regulators. Over three days, they shared new data, tackled problems together, and created direct dialogue between those living with CMT and those working on treatments— solidifying the Convention as the leading annual meeting for CMT drug development and a key driver of new clinical programs, biomarkers, and strategies to remove remaining barriers to approved therapies.