The CMT Research Foundation collaborated with DTx Pharma, Inc. (DTx) to design and test novel RNA-based compounds in an experimental model of CMT1A. This project was successful, and DTx is now in the ‘lead identification’ stage of developing a therapeutic candidate for the treatment of CMT1A. This means that scientists at DTx are making chemical and design optimizations to select the most promising version of the therapeutic candidate for further research and development. The next stage will be ‘IND-enabling’ research, which refers to a comprehensive suite of studies that are carried out in rodents and other model organisms to ensure the FDA and other regulatory bodies that the candidate therapeutic is ready for human clinical trials, which could begin as early as 2023.

This project built on previous proof-of-concept work demonstrating that antisense RNA therapeutics can decrease the levels of PMP22 in the peripheral nerves of mice. DTx’s proprietary delivery technology has the potential to render RNA-based therapeutics, such as antisense oligonucleotides (ASOs) and small interfering RNA (siRNA), more potent, safer and longer-acting.

The CMT Research Foundation introduced DTx Pharma to the CMT field and funded their first CMT research, with an initial investment of $128,000 for this project. DTx has leveraged successful results from this project to secure additional funding from the National Institutes of Health. Additionally, DTx recently raised $100M to take their pipeline of therapeutic candidates, including a therapeutic candidate for CMT1A, forward toward clinical trials.