The CMT Research Foundation is pleased to announce that our partner DTx Pharma just completed the second milestone in a study we’re funding which uses their proprietary technology to suppress the overexpression of PMP22, the protein that causes CMT1A. Their work has the potential to create more potent, safer and longer-acting RNA-based therapeutics, such as antisense oligonucleotides (ASO) and small interfering or silencing RNA (siRNA), and move this technology closer to the clinic for CMT1A patients.

During milestone 2, DTx Pharma identified an additional set of siRNAs that strongly decrease the expression of PMP22 in cultured cells. These new siRNAs expand the pool of candidates available for DTx Pharma to test in the next phase of the project and increase the chance of discovering a siRNA that works well to decrease the levels of PMP22 in a mouse model of CMT1A.

The CMT Research Foundation invested seed money to start this project with DTx Pharma. Since our original investment, DTx Pharma has invested three times as much into the project. Additionally, the company is seeking significant follow-on funding from government agencies and investment partners. This partnership exemplifies the CMT Research Foundation’s role to identify new technologies that can be applied to CMT, provide initial funding and scientific expertise to advance drug development, and partner with researchers and industry leaders to speed treatments to patients.

Learn more about our research priorities and see the other CMT1A research projects we’re currently funding.