ATLANTA (December 20, 2019) The CMT Research Foundation (CMTRF), a nonprofit focused solely on delivering treatments and cures for Charcot-Marie-Tooth, today announced it has partnered with DTx Pharma, an RNA medicines company breaking open new therapeutic areas for oligonucleotide (short DNA or RNA molecules) drugs by overcoming the delivery challenge that has limited the utility of this promising new potential gene therapy for CMT1A.
CMT is one of the most common inherited neurological disorders, affecting people across the globe. CMT1A, which affects more than 125,000 Americans (between 50-65% of all CMT patients), is caused by a duplication of the peripheral myelin protein 22 (PMP22) gene. PMP22 is a major component of myelin and its overexpression causes degradation of the neuronal sheath resulting in loss of sensation and/or numbness in the legs and arms, muscle weakness, atrophy and foot deformities. Because CMT is progressive, patients continue to lose function as they age, creating a greater degree of disability. Currently, there is no cure or any effective treatment for CMT.
This project builds on previous proof of concept work demonstrating that antisense oligonucleotides can decrease the levels of PMP22 in the peripheral nervous system of mice However, because of poor drug delivery to the target tissue, the doses required were far too high to be safely used in humans. DTx Pharma’s proprietary technology has the potential to enhance the activity of oligonucleotide therapies, creating the more potent, safer and longer-acting molecules needed to move this technology toward the clinic for CMT1A patients. Funding will also support development of a small interfering RNA (siRNA) targeting PMP22 as an alternate approach to reduce PMP22 levels.
“We are pleased to partner with DTx Pharma. Their technology may provide a major breakthrough, renewing hope in patients that this type of gene therapy approach is possible to treat or even cure CMT1A,” says Susan Ruediger, CEO of CMTRF.
“Our technology is well-matched to the peripheral nervous system but we hadn’t considered CMT as a target until meeting with Susan and her team. Partnering with the CMT Research Foundation provides DTx Pharma with scientific and clinical experts who specialize in peripheral neuropathy, while connection with patients and families affected by CMT helps us on a personal level to understand the need to accelerate development of new therapies for CMT,” says Arthur T Suckow, CEO of DTx Pharma.
DTx Pharma is developing technology that will unleash RNA-based therapeutics as the modality of choice for personalized treatment across most therapeutic areas. DTx has developed proprietary technology based on lipidation, the direct conjugation of long chain fatty acids to siRNA, that enables delivery of siRNA therapeutics across multiple cell types. This technology enables oligonucleotide therapeutics to compete with small molecules and other biologics across most indications and additionally, enables access to drug targets and indications that small molecules and biologics could never be able to modulate.
The CMT Research Foundation (CMTRF) is focused solely on delivering treatments and cures for CMT. Founded by two patients who are driven to expedite drug delivery to people who live with CMT globally, the organization funds research focused on drug development. The 501(c)(3) federal tax-exempt organization is supported by personal and corporate financial gifts.