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

SBIR 第一阶段：PolyRNA——治愈人体的根本性创新

• 批准号: 0810755
• 负责人: Lisa Kemp
• 金额: $ 10万
• 依托单位: Ablitech, Inc.
• 依托单位国家: 美国
• 项目类别: Standard Grant
• 财政年份: 2008
• 资助国家: 美国
• 起止时间: 2008-07-01 至 2008-12-31
• 项目状态: 已结题
• 来源: https://www.nsf.gov/awardsearch/showAward?AWD_ID=0810755&HistoricalAwards=false
• 关键词: SBIR; Phase; PolyRNA; Radical; Innovation

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.

这项小型企业创新研究（SBIR）I期项目将证明癌症治疗平台技术的可行性，该技术克服了其他尝试将siRNA传递给癌细胞的固有缺点。当前siRNA递送的困难是，体内的酶在达到目标之前会降解siRNA。市场上的大多数siRNA技术都是基于对RNA本身的永久修改以实现交付的。这些修改通常涉及siRNA的内部联系的变化，这通常会降低基因沉默能力。 Ablitech的聚-RNA系统涉及对siRNA进行可逆修改，仅用于我们的聚合物网络。 siRNA本身的骨干结构保持不变。这将允许提供最有效的siRNA单元，而不是牺牲酶稳定性的功效。我们计划专门针对胰腺癌，因为这种形式的癌症是最具侵略性的癌症之一，据估计，今年将有37,000多人被诊断出患有胰腺癌。胰腺癌的死亡率极高，5年生存率低于5％。转移性胰腺癌诊断后的平均预期寿命仅为3-6个月。通常，这项研究预计会导致一种可销售的平台技术，用于治疗各种疾病，例如癌症，HIV和其他疾病。 Ablitech的Poly-RNA的最初目标市场是癌症治疗市场，预计到2010年将产生55-7亿美元的销售额。目前，诸如默克公司，布里斯托尔 - 美尔斯·米尔斯·史基布（Bristol-Myers Squibb），辉瑞（Pfizer），辉瑞（Pfizer）以及其他拥有大型营销和分销渠道的大型制药公司占据了这一市场，并且近年来统治了该市场，并且近年来与小型技术公司获得了商业化的公司授权公司。 Ablitech试图遵循该模型进行多-RNA的商业化。