Maintaining Momentum: CMTRF’s Vision for 2025 and Beyond

Jan 7, 2025 | Foundation News, Life with CMT

Greetings and Happy New Year to you all. When I began my tenure as CEO of the CMT Research Foundation in October 2024, I was immediately inspired and impressed by the work of the Foundation. I was equally moved by the dedication of the greater CMT community, especially our donors, who have driven this whole research field to new heights. CMTRF has made a tremendous amount of progress in such a short amount of time, and I am honored to serve as your CEO. I look forward to connecting with you personally to discuss your thoughts and goals for 2025 and beyond.

My service to the mission of CMTRF is deeply personal to me because I, too, am affected by Charcot-Marie-Tooth disease. CMT runs in my family and has affected multiple generations, including my father. My hope is that we will soon find treatments and cures for all types of CMT so that future generations — both in my family and in yours — won’t be affected by this devastating disease.

We will not rest until we discover effective treatments and cures. With the progress I see, I am confident that day will come. The truth is, future generations are depending on us to achieve our goal and stop CMT before it affects their quality of life.

The drug development market for CMT is buzzing with more companies working in CMT than ever before. There are two active clinical trials happening in the U.S. testing novel therapeutics for CMT — NMD Pharma (NMD670) and Applied Therapeutics (AT-007/SORD).

In 2024, CMTRF reviewed more than a dozen requests to fund research projects and added 5 new projects to our pipeline: ReviR Therapeutics (CMT1A), Dr. Charles Abrams, University of Illinois at Chicago (CMT1X), Dr. Stephan Zuchner, University of Miami, FL (CMT1J), Dr. Afrooz Rashnonejad, Nationwide Children’s Hospital (multiple CMT Types), and 82VS (CMT1A).

These projects aim to significantly advance the development of treatments and cures for CMT by testing existing drug candidates and commercially available drugs on CMT research models, developing and testing novel genetic medicines, optimizing drug delivery vehicles for improved activity in the nerve, and developing new mouse models to expand the toolbox for preclinical drug research.

2024 also wrapped up ENDGAME, our $10 million, three-year campaign. The goal of ENDGAME was to raise funds to deploy to the best research projects to find treatments and cures for CMT1A.

When ENDGAME began in September 2021, there were only four projects focused on the CMT1A subtype funded by CMTRF. Now there are 13.

Our work in CMT1A activated 9 new projects and 5 pre-clinical candidates: DTx/Novartis (DTx-1252), Oryzon Genomics (ORY-4001), Armatus Bio (ARM-101), Shift Pharmaceuticals (SHC1A-012), and Augustine Therapeutics (AGT100216). Additionally, we brought in additional companies that hadn’t worked in CMT before and activated their technologies to be applied to 1A.

CMTRF also received a $5 million gift from the Bentzin Family Foundations that will support the next phase for CMT research.

The Global CMT Research Convention continues to be the preeminent event of the year where over 200 CMT drug developers and experts from around the globe meet to exchange exciting new research data and ideas, as well as meet face-to-face with CMT patients.

Researcher Day was highlighted by Keynote Speaker Dr. Carsten Bönnemann, Senior Investigator at the National Institute of Neurological Disorders and Stroke and Chief of the Neuromuscular and Neurogenetic Disorders of Childhood Section. Dr. Bönnemann spoke about the development of a gene therapy for giant axonal neuropathy and the learnings that can be applied to CMT drug development.

Additionally, Dr. Arthur Suckow, former CEO of DTx Pharma, moderated a CEO panel which touched on pursuing drug programs and the challenges it presents, and Dr. Celia Witten, Deputy Director, Center for Biologics Evaluation and Research at the Food and Drug Administration, highlighted the FDA’s efforts in advancing medical product development for rare diseases.

Due to recommendations from the biotech community and our donors, the 2024 convention was expanded to three days, with the addition of the Young Researchers Innovation Forum, which set out to inspire young scientists to explore innovative ideas in Charcot-Marie-Tooth disease drug development. This year’s Convention will take place September 25-27, 2025, in Cambridge, MA.

We capped 2024 with the announcement of receiving a four-star rating from Charity Navigator, the highest possible rating, in our first year of evaluation. Charity Navigator’s impartial, third-party evaluation validates CMTRF’s effectiveness, financial transparency and reliability, and ensures our donors know that their contributions support an organization that is an exceptional steward of their funds, making every dollar count towards finding a cure for CMT.

This year would not have been possible without you — our donors, volunteers, researchers and patient community. We thank you for putting your faith in us. Your support fuels our progress and makes everything we do possible.

But we are not done yet.

In 2025, we are reaffirming our commitment to you by being available whenever you need us. We want to understand your CMT journey and the impact it has on your loved ones. We’re eager to hear your ideas on advancing research and to share in detail the projects we’re funding and the reasons behind our choices. Your voice matters, and we’re here to listen and engage with you every step of the way.

Please feel free to reach out to any one of us, anytime.

With gratitude,

Laura M. MacNeill, MBA
CEO, CMTRF