If you’ve ever been frustrated by the slow pace of research progress or questioned how a single donation makes a difference, the collaboration between the CMT Research Foundation and DTx Pharma proves that one simple action, one bold move and one donation is all it takes to change the future of Charcot-Marie-Tooth disease as we know it.

Safely and effectively delivering therapies to the peripheral nervous system is one of the primary barriers to treating and curing all CMT types. Evidence suggests that the science and technology may already exist to deliver gene therapy treatments for diseases like CMT, but applying those resources requires creative and unconventional strategies to advance drug discovery for this disease. Unsatisfied with the slow pace of CMT research progress, that’s precisely why the CMT Research Foundation exists.

Thinking Differently About Gene Therapies for CMT

In June 2019, the CMT Research Foundation met with DTx Pharma at the BIO (Biotechnology Innovation Organization) International Convention. DTx Pharma is a privately held biotechnology company creating novel RNA-based therapeutics to treat the genetic drivers of disease. Although DTx Pharma had never previously worked in the CMT field, CMT Research Foundation experts believed their technology had promising potential to deliver gene therapies for CMT. Six months after that first meeting, the CMT Research Foundation and DTx Pharma launched a partnership focused on CMT1A, with a research grant of $128,000 from the Foundation to fund their entire program.

Early data from DTx Pharma’s study indicated that its technology effectively reduced PMP22 in cellular models. Their promising findings and innovative partnership with the CMT Research Foundation enabled DTx Pharma to receive a $385,000 grant from the National Institutes of Health to continue its CMT1A program. In ongoing results, treated mice showed improvements in symptoms related to and indicative of CMT1A. Nerve conduction testing in the mice confirmed this data.

The Road to Clinical Trials

On March 1, 2021, DTx Pharma announced that it raised $100 million to pursue its leading two programs, including its CMT1A research program initially funded by the CMT Research Foundation. That first $128,000 grant and the resulting research progress have attracted the investment funds necessary to translate findings into potential treatments for patients and families.

“The DTx Pharma team has uncovered new ways to leverage fatty acids to overcome some of the challenges that have greatly limited RNA therapeutics as a class,” said Arthur T. Suckow, Ph.D., chief executive officer of DTx Pharma. “These funds will allow us to utilize our technology to bring disease-modifying drugs to CMT patients with serious unmet medical needs who deserve treatments to improve their lives.”

DTx Pharma will use proceeds from the financing to advance its pipeline utilizing its novel FALCON (Fatty Acid Ligand Conjugated OligoNucleotide) platform technology and accelerate the march toward clinical trials.

In less than two years since being introduced to CMT by the CMT Research Foundation, DTx Pharma is working to map a path to clinical trials for CMT1A. The CMT Research Foundation recently convened a group of CMT1A patients to meet with DTx Pharma to increase their understanding of the impact of CMT on people’s lives, how to measure meaningful outcomes in a clinical trial, and the kinds of procedures that would be acceptable to patients in a clinical trial. With this community feedback and the new $100 million in funding, DTx could potentially have their therapy in clinical trials as early as 2023.

By thinking differently, acting boldly and investing intentionally in the most promising research, this is how a single donation becomes the catalyst for potential treatments and cures.

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Continue the Momentum in CMT Discovery

Your donation to the CMT Research Foundation will fund cutting-edge research that’s solely focused on drug development, continue moving promising projects forward, and attract new biotech and pharma companies to work on CMT. Together, we can change the course of CMT during our lifetime — and for every future generation.