SBIR Phase I: PolyRNA - A Radical Innovation for Healing the Human Body

This Small Business Innovation Research (SBIR) Phase I project will demonstrate the feasibility of a platform technology for cancer therapy which overcomes the inherent shortcomings of other attempts to delivery siRNA to cancer cells. The difficulty with current siRNA delivery is that the enzymes in the body degrade the siRNA before it can reach its target. Most current siRNA technologies on the market are based on permanent modifications to the RNA itself to enable delivery. These modifications typically involve changes to the internal linkages of the siRNA which often decreases the gene silencing ability. Ablitech's Poly-RNA system involves making reversible modifications to the siRNA solely for attachment to our polymer network; the backbone structure of the siRNA itself is left unchanged. This will allow for the delivery of the most effective siRNA units rather than sacrificing efficacy for enzymatic stability. We plan to specifically target pancreatic cancer as this form of cancer is one of the most aggressive, and it is estimated that over 37,000 people will be diagnosed with pancreatic cancer this year. Pancreatic cancer has an extremely high mortality rate, with a 5-year survival rate of less than 5%. The average life expectancy after diagnosis with metastatic pancreatic cancer is only 3-6 months. Commercially, this research is anticipated to result in a marketable platform technology for gene therapy for the treatment of a variety of diseases such as cancer, HIV, and other diseases resulting from genetic abnormalities. The initial target market for Ablitech's Poly-RNA is the cancer therapy market, which is expected to generate $55-$70 billion in sales by 2010. Currently, large pharmaceutical companies such as Merck, Bristol-Myers Squibb, Pfizer, and others that possess large marketing and distribution channels dominate this market and in recent years have commercialized technologies licensed from small, innovative companies. Ablitech seeks to follow this model for the commercialization of Poly-RNA.