The CMT Research Foundation is pleased to announce that DTx Pharma, Inc. (DTx) has successfully completed their CMT Research Foundation-funded RNA-based therapy project.

With funding from the CMT Research Foundation, DTx designed and tested a library of novel RNA-based compounds called small interfering RNA (siRNA) which target PMP22, the gene that when over-expressed causes CMT1A. The siRNAs are designed to interfere with the body’s ability to produce PMP22 mRNA, thus lowering levels of PMP22 protein to normal. Importantly, the siRNAs are attached to DTx’s proprietary fatty acid motifs, which are thought to improve the delivery of the siRNAs into the Schwann cells of the peripheral nerves, where high PMP22 levels cause the nerve damage that lead to the symptoms of CMT. The most active compounds identified in initial studies in cultured Schwann cells were further evaluated for their ability to reduce PMP22 in a rodent model of CMT1A that carries several copies of the human PMP22 gene.

Based on the success of the initial research funded by the CMT Research Foundation, DTx is now in the ‘lead identification’ stage of developing a therapeutic candidate for CMT1A. This means that scientists at DTx are making chemical and design optimizations to select the most promising version of the therapeutic candidate to take forward. The next stage will be ‘IND-enabling’ research, which refers to a comprehensive suite of studies that are carried out in animals 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. DTx recently raised $100M to take their pipeline of therapeutic candidates, including a therapeutic candidate for CMT1A, forward toward clinical trials.

“At DTx, our entire team is deeply committed to the CMT1A program and to the patients for whom there is no current therapy,” said Denise Bevers, Chief Operating Officer of DTx Pharma, Inc.  “We are grateful to the CMT Research Foundation for its early funding of our program, as well as its ongoing commitment to opening doors for us with both patients and clinicians.”

The CMT Research Foundation is proud to have identified this company whose technology may solve the delivery problem in CMT therapeutics. Understanding the life-changing impact of bringing the first effective treatment to market, we acted rapidly to bring them into the CMT field and fund their initial CMT studies. The CMT Research Foundation continues to advise and support DTx as they move forward with their CMT program.