One of the first research teams the CMT Research Foundation (CMTRF) funded was DTx Pharma, Inc., and the project focused on delivery of genetic medicine to the myelinating Schwann cells of the peripheral nerves – a major scientific challenge to effectively treating most forms of CMT. At the time, DTx was was using their delivery technology – called “FALCON” for fatty acid ligand conjugated oligonucleotides – to develop therapeutics for a genetic eye disease. Then, senior executives of CMTRF approached DTx and explained how their technology could potentially solve this major challenge in gene therapy for CMT. Once a proposal was developed and vetted by our Scientific Advisory Board, CMTRF offered to fund the project.
In short order, DTx has realized significant success in testing both the delivery system and a variety of novel RNA-based compounds that silence PMP22, the gene that causes CMT1A when overexpressed.
In this video, DTx CEO Arthur Suckow describes the company’s technology and gene therapy approach and announces that they expect to be in clinical trials next year and plan to complete a proof-of-concept trial in 2024!  We are pleased to have played a fundamental role in making this happen.