We are pleased to announce that The Office of Technology Commercialization at The Research Institute at Nationwide Childrens Hospital has just awarded The Kaspar Laboratory a grant to explore a novel platform for producing AAV gene therapeutics, the same gene therapy used in our SMA clinical trial as well as in our muscular dystrophy, Rett syndrome, and ALS research. AAV gene therapeutics (schematic in our picture) are 20 nanometers in diameter with precise DNA coding elements, such as the SMN gene placed inside. Think of these particles as tiny bullets with DNA stuffed on the inside that enter into cells, delivering the therapeutic payload. It takes an Electron microscope just to see these particles! Dr. Kaspar and Dr. Likhite was thrown into The Shark Tank to present the new concept to a group of entrepreneurs and esteemed business individuals and was chosen as the concept to make an initial high risk investment in. If we are successful with this new approach, AAV production for clinical trials will no longer be limited and can likely speed the production process. We are thankful for this support and Please wish us Luck and Success.
Posted on: Sat, 27 Dec 2014 11:49:18 +0000
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