IUSM Alzheimer's Disease Drug Discovery Center: PTC Software

项目摘要 总体：印第安纳大学医学院(IUSM)的战略目标 阿尔茨海默病药物发现(ADDD)中心是将尖端技术 早期发现药物的能力，并有助于更广泛地研究新兴药物 阿尔茨海默病的目标假设，目标是产生新的类别 潜在的治疗方法。发展与发展中心将把自己确立为一个战略和 NIA AMP-AD和Model-AD计划的运营合作伙伴。经过设计，这将是 提供药物发现功能，将靶向发现工作(AMP-AD)与 新发现的铅分子在基于人类的AD动物模型中的特征 病理学、遗传学和翻译生物标记物(模型-AD)。 为了完成ADDD中心的使命，我们组建了一支 来自印第安纳大学、普渡大学和印第安纳临床与研究中心的科学家 翻译科学研究所(CTSI)。这个团队在以下方面有相当的专业知识 阿尔茨海默病生物学，中枢神经系统药物发现和开发， 以及在将推动ADDD的核心发现技术方面的卓越科学 中心的贡献和交付成果。关键优势和差异化优势 拟议的ADDD中心的主要科学协调和 通过IUSM进行管理。具体目标是： 1.创建具有特征的AD药物发现目标组合，这些目标代表 新的干预假说，利用对 基础研究和对新出现疾病的了解。 2.执行高级临床前目标验证和启用研究，以 优先考虑治疗发现的最佳机会。 3.为达到以下目标的目标创造高质量且具有良好特性的铅分子 这是一个里程碑式的标准，是进一步翻译投资的机会。 4.打造灵活的数据共享平台，为全球科研人员提供数据 和Target Enablement包，以扩展ADDD中心的工作。

Project Summary Overall: The strategic goal of the Indiana University School of Medicine (IUSM) Alzheimer’s Disease Drug Discovery (ADDD) Center is to integrate sophisticated capability for early drug discovery and contribute to a broader study of emerging Alzheimer’s Disease target hypotheses with the goal of generating new classes of potential therapeutics. The ADDD CENTER will establish itself as a strategic and operational partner for the NIA AMP-AD and MODEL-AD initiatives. By design, this will provide drug discovery capability to bridge the work in target discovery (AMP-AD) with newly discovered lead molecules characterized in AD animal models based on human pathology, genetics and translational biomarkers (MODEL-AD). To accomplish the mission of the ADDD CENTER, we have assembled a team of scientists from Indiana University, Purdue University, and the Indiana Clinical and Translational Sciences Institute (CTSI). This team has considerable expertise in Alzheimer’s disease biology, CNS pharmaceutical drug discovery and development, and scientific excellence in the core discovery technologies that will drive the ADDD CENTER’s contributions and deliverables. A key advantage and differentiated strength of the proposed ADDD CENTER is the primary scientific coordination and administration through IUSM. Specific Aims are: 1. Create a portfolio of characterized AD drug discovery targets representing novel intervention hypotheses that capitalizes on significant investments in basic research and emerging disease understanding. 2. Perform advanced pre-clinical target validation and enablement studies to prioritize the best opportunities for therapeutic discovery. 3. Create quality and well characterized Lead molecules for targets that meet milestone criteria and are opportunities for further translational investment. 4. Create a flexible data sharing platform to enable global researchers with data and Target Enablement Packages to expand on the work of the ADDD CENTER.