Shift Pharmaceuticals Awarded $3 Million for Spinal Muscular Atrophy Treatment

Shift Pharmaceuticals, a Kansas City company, has been awarded $3 Million from the National Institutes of Health (NIH) and Congressionally Directed Medical Research Programs (CDMRP) to support research on Spinal Muscular Atrophy (SMA). These funds will be used to launch preclinical studies and advance research that will lead to clinical trials in humans.

SMA is the number one genetic cause of death in infants and young children affecting approximately 1 in 11,000 babies. Nearly 80% of patients with SMA die before the age of two and currently, there is no cure. SMA affects the part of the nervous system that controls voluntary muscle movement, taking away the ability to walk, eat, or breathe. The earlier the age of onset, the greater the impact on motor function.

Steve O’Connor, CEO of Shift Pharmaceuticals, and a team of regional researchers have designed a genetic drug that will increase the expression of the Survival Motor Neuron 2 protein (SMN2), a protein that is required for survival. Children with SMA have a deficiency of this protein. This drug will treat SMA patients with the goal of improving the quality and longevity of life.

“These funds are important for Shift Pharmaceuticals because we have a well-developed technology coming out of the University of Missouri and this will accelerate our preclinical phase that will eventually lead to clinical trials in humans,” said Dr. O’Connor. “The Kansas City region shows great promise in the opportunity for collaboration and because of this we will do most of our research in this area. There are several contract research organizations (CRO), labs, and researchers that have expertise in clinical trials, specifically molecule development.”

The NIH Small Business Innovation Research program funds early stage small businesses that are seeking to commercialize innovative biomedical technologies. This competitive program helps small businesses participate in federal research and development, develop life-saving technologies, and create jobs.

The CDMRP fills research gaps by funding high impact, high risk and high gain projects that other agencies may not venture to fund. While individual programs are unique in their focus, all the programs managed by the CDMRP share the common goal of advancing paradigm shifting research solutions that will lead to cures or improvements in patient care, or breakthrough technologies and resources for clinical benefit. The CDMRP strives to transform healthcare for Service Members and the American public through innovative and impactful research.

Shift Pharmaceuticals is a start-up drug development company with a platform technology for utilizing Morpholino based Antisense Oligos (MOs) for the treatment of Spinal Muscular Atrophy (SMA). The core platform targeting SMA has been validated by our academic partners at The University of Missouri and a patent has been filed on the intellectual property, which is in the process of being exclusively licensed to Shift Pharmaceuticals.  For more information contact

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