CMT Research Foundation Funds Shift Pharmaceuticals

Shift to Develop and Analyze Novel Series of Drugs Designed to Control Expression of PMP22 Gene The CMT Research Foundation (CMTRF), a nonprofit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease, today announced it has partnered with Shift Pharmaceuticals to fund research towards treatments for CMT1A, the most common form of CMT. Charcot-Marie-Tooth affects one in 2,500 people (about the same number as Multiple Sclerosis), including 150,000 Americans and nearly 3 million people around the world. CMT1A is caused by the overexpression of a protein which makes myelin, the insulating coating on the nerves. When too much protein is expressed, it results in a progressive deterioration of the nerves that can cripple those with CMT1A. While too much of PMP22 causes CMT1A, too little of PMP22 is also a problem as it causes HNPP, another form of peripheral neuropathy. Any therapeutic strategy must ensure proper amounts of PMP22 are being expressed and cannot simply knock-out the expression of the important gene. In this project, Shift Pharmaceuticals will develop and analyze a novel series of morpholino-based antisense oligonucleotide drugs (ASOs) that are designed to control and reduce (but not completely knock-out) expression of PMP22. Shift Pharmaceuticals believes that a morpholine-based approach provides the specificity and fine-tuning capabilities necessary to achieve this important balance. Morpholinos have been approved as a treatment for some mutations known to cause Duchenne Muscular Dystrophy. “This collaboration with Shift Pharmaceuticals in another strong example of how the CMT Research Foundation is applying proven technologies to CMT,” says Susan Ruediger, CEO. “We are pleased to have Shift Pharmaceuticals as part of the CMTRF family of initiatives aimed directly at finding a cure for this destructive disease.”