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.”

“Working with the CMT Research Foundation has felt like a partnership from day one.  By leveraging Shift’s core technology and experience with therapeutic development with the CMTRF’s deep understanding of core science related to CMT (through their staff and extensive network of advisors), we have collaboratively created an excellent research plan to move molecules towards the clinic for CMT,” says Steve O’Connor, CEO Shift Pharmaceuticals.

Shift Pharmaceuticals, a privately held Missouri-based company, develops Antisense Oligonucleotides as drug candidates for a variety of genetic disorders. Shift was co-founded by serial entrepreneur Dr. Steve O’Connor and world leading SMA research expert Dr. Chris Lorson. The company has licensed the core intellectual property from The University of Missouri, where the initial discovery and development was performed by Dr. Lorson.

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.