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    Date submitted
  • 26-Oct-2017

Anti-sense Pharmaceutical Company


Shift Pharmaceuticals is a start-up drug development company with a platform technology for utilizing Morpholino based Antisense Oligos (MAOs) for the treatment of Spinal Muscular Atrophy (SMA). The core platform targeting SMA has been validated (with peer-reviewed publications) by our academic partners at The University of Missouri and a patent has been filed (and claims approved) on the intellectual property.

Additional Questions

Who is your customer?

Medical professionals and patients treating newborn patients born with Spinal Muscular Atrophy (SMA).

What problem does this idea/product solve or what market need does it serve?

SMA is the most common inherited motor neuron disease occurring in 1 out of every 11,000 live births with a carrier frequency of 1 in 40 worldwide. Sadly, most children born with SMA fail to reach their second birthday. Treating SMA should be a global priority due to the relatively high frequency for a “rare” disease combined with the often catastrophic impact on families and the pharmacoeconomic impact on the healthcare system. This devastating disease results from the loss of Survival Motor Neuron-1 (SMN1). There is a nearly identical backup copy of the gene called SMN2, however a small difference in the genetic sequence results in a protein that cannot inherently prevent disease formation.

What attributes will make this idea/product successful? Why do you believe that those features will create success?

Our published scientific data in the only viable animal model for SMA has demonstrated better efficacy than Spinraza (the only FDA approved drug for SMA) and Spinraza has already become a blockbuster product with $271M in sales in Q3 2017. We have additional unpublished data that has demonstrated even greater efficacy and lower toxicity than the current approved product. Thus, we believe this product can command a significant market share in this field.

Explain how you (your team) will execute to make this idea/product successful? What gives you (your team) an advantage over others already in the market or new to this market?

Steve O'Connor, PhD, is a successful serial entrepreneur in the life-sciences sector, Steve has been leading technology startups since 1992 in a number of sectors, including diagnostics, drug discovery, water treatment, etc. He brings to Shift Pharmaceuticals significant experience in technology development, intellectual property, fundraising, business partnering, and company building. Chris Lorson, PhD (our Chief Scientific Officer) is a world leader in studying SMA in genetically engineered mice. These animals are the only animal model for studying SMA and very few labs in the world are equipped to handle the breeding and growth of these compromised animals at the same level as the Lorson Lab. Paul Morcos, PhD, is a world leading scientist in the design, manufacturing and quality control of morpholino molecules, being a senior executive at GeneTools for ~ 18 years. GeneTools is the market leader is supplying morpholino oligos for research and pre-clinical purposes.