Since 2018, CMTRF has had an outsized impact on inspiring drug hunters to work on developing therapeutics for CMT and establishing itself as a key stakeholder in the CMT drug development community. It was the Foundation’s early and continued support of DTx Pharma that led to its $1 billion acquisition by Novartis in July, accelerating its path to becoming the first RNA targeted therapeutic for CMT1A—this awakened industry to CMT.

In just five short years, five of our funded projects have resulted in pre-clinical candidates. Other projects have contributed invaluable learnings that will clarify the best pathways to attack and eradicate all types of CMT.

In the past 12 months, we reviewed over a dozen requests to fund research projects. We have active projects for CMT 1A, 1B, 1X, 2E and 4B. We continue to seek out multiple approaches to solve the delivery challenge to peripheral nerves, to understand the complexities and the opportunities to make gene therapy safe and effective for CMT patients, and to bring new technologies and scientific perspectives to the CMT field.

CMT Research Foundation has invested over $10 million in research. Eight of our 20 projects are exploring gene therapy treatments; others are focused on small molecules and drug screenings, and one project is developing a humanized mouse model of CMT1B, enabling researchers to study the disease more comprehensively.

This year, we established the CMTRF Research Advisory Committee (RAC), which is tasked with ensuring that our scientific strategy supports the Foundation’s singular mission to find treatments and cures for CMT. The RAC is made up of members Dr. Grace Pavlath (Solid Biosciences), Dr. William Motley (Rapport Therapeutics), Dr. Paul August (ReviR Therapeutics), Dr. Arthur Suckow (Co-founder and former CEO of DTx Pharma), and Dr. Charlotte Sumner (Johns Hopkins). Collectively, the RAC members represent decades of experience and accomplishments within the therapeutic and drug development space for CMT and other neurological diseases. They will work closely with the Scientific Advisory Board to fine-tune project selection and prioritization to fund the types of research that have the greatest potential for impact. Moreover, they will provide scientific guidance to the Board of Directors.

CMTRF’s Global CMT Research Convention in September continues to be the most impressive gathering of CMT drug development experts from around the world who come together to exchange ideas and strategies and meet face-to-face with patients. Some 450 scientists, pharma companies, investors and patients attended the two-day convention in Cambridge, MA. The 2024 convention will take place September 26-28 in Cambridge, MA.

In 2024, we will invest in new technologies that merit consideration for CMT. We are dedicated to generating scientific breakthroughs that propel the field forward by offering fresh perspectives and innovative findings, steering clear of duplicating the work done by others. We will work with key opinion leaders  and other organizations to foster collaboration that is critical to the work of drug development.

Thank you for your support of CMT research and your faith in our commitment to persist until we have treatments for CMT patients. Thanks to your donations, 2023 was a landmark year in CMT research. Together, we can accelerate the discovery of treatments.

Please consider kicking off 2024 with a significant donation to CMT research. This will enable us to identify and fund the best scientific projects now, without further delay.

With gratitude and optimism,

Cleary Simpson

CEO, CMTRF