This is the twelfth patent issued for Odyssey Thera's PCA process.

The patent describes a method for detecting protein-protein interactions in living cells and animals. Protein interactions define the intricate biochemical networks in living cells, and are used at Odyssey Thera to monitor drug response. The invention makes it possible to monitor specific biochemical parameters, including the extent and kinetics of drug target modulation, in a live subject.

"This patent extends our broad estate in drug discovery technologies," said John K. Westwick, Ph.D., Odyssey Thera CEO and co-author on the patent. "One of the most challenging aspects of drug discovery is transitioning drug candidates from the test tube, to native targets in living cells, and from there to live subjects. With this invention, drug discovery and development are achieved with a single system that faithfully monitors the activity of a drug at each step." Dr. Westwick continues, "Whole animal studies using fluorescence or luminescence detection are widely used in the pharmaceutical industry, but strategies were needed to increase the scope of biochemical activities that can be tracked. The approaches described here will significantly increase the value of live subject imaging efforts."

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The researchers then used a sophisticated genetic technique to repair the mutated dystrophin gene in the isolated DMD CD133+ cells so that dystrophin synthesis was restored. Importantly, intramuscular or intra-arterial delivery of the genetically corrected muscle cell progenitors resulted in significant recovery of muscle morphology, function, and dystrophin expression in a mouse model of muscular dystrophy.

These data demonstrate that genetically engineered blood or muscle-derived CD133+ cells represent a possible tool for future stem cell-based autograft applications in humans with DMD, says Dr. Torrente. The authors caution that significant additional work needs to be done prior to using this technology in humans. Additional research will substantially enhance our understanding of the mechanisms underlying this effect and may lead to the improvement of gene and cell therapy strategies for DMD.

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