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

这个小型企业创新研究(SBIR)第一阶段项目将展示癌症治疗平台技术的可行性，该平台技术克服了将siRNA输送到癌细胞的其他尝试的固有缺点。目前siRNA传递的困难在于体内的酶在siRNA到达目标之前就将其降解。目前市场上的大多数siRNA技术都是基于对RNA本身的永久性修改，以实现交付。这些修饰通常涉及siRNA内部连接的改变，这往往会降低基因沉默的能力。Ablitech的Poly-RNA系统仅为连接到我们的聚合物网络而对siRNA进行可逆修改；siRNA本身的主干结构保持不变。这将允许提供最有效的siRNA单位，而不是为了酶的稳定性而牺牲效率。我们计划专门针对胰腺癌，因为这种癌症是最具侵袭性的癌症之一，预计今年将有超过3.7万人被诊断为胰腺癌。胰腺癌死亡率极高，5年生存率不到5%。被诊断为转移性胰腺癌后的平均预期寿命只有3-6个月。在商业上，这项研究有望产生一种市场上的平台技术，用于治疗各种疾病，如癌症、艾滋病毒和其他由基因异常引起的疾病。Ablitech的Poly-RNA最初的目标市场是癌症治疗市场，预计到2010年，该市场的销售额将达到550亿至700亿美元。目前，默克、百时美施贵宝、辉瑞等拥有大型营销和分销渠道的大型制药公司主导着这一市场，近年来，它们将从小型创新公司获得授权的技术商业化。Ablitech寻求遵循这种模式来实现Poly-RNA的商业化。

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.