The Southwest Comprehensive Center for Drug Discovery and Development

描述（由申请人提供）：将新的治疗靶点转化为创新药物是美国西南部的主要转化瓶颈。为了解决这一迫切需求，我们的目标是建立一个关键的资源，以弥补我们区域药物发现科学界的主要药物化学资源差距。为此，我们的项目旨在部署西南药物发现与开发综合中心（SCCDD），该中心将为将基础科学发现转化为临床提供有利的合作框架。通过扩展，预计该中心将拥有资源丰富的跨学科能力，采用工业化的药物化学方法，更有可能对医疗保健产生重大影响。这种多重PI努力将现有的跨学科科学家团队与互补的技能组合和重要的行业经验组合在一起。为了确保SCCDD能够提供全面的产品，我们将利用TGen现有的药物发现和开发基础设施以及UA的战略网络化学创新。该团队拥有良好的声誉和合作记录，并有一个共同的任务：通过战略性地调整技术创新和获取使能资源，使药物发现现代化，共同促进更有效地为有需要的患者提供更有效的治疗。这一“重大机遇”的目标是部署必要的基础设施和资源，以在两年的时间框架内达到中心的运行状态。为了实现这一目标，我们将利用创新的化学、高通量筛选资源和专门的药物化学团队，建立一个亚利桑那化合物收集库，总体上建立一个功能流程图，以快速加快沿着价值链的化合物进展。四个单元将确保工艺流程如下。分子探针或命中生成将由库设计、开发和生产单元（单元1）与高通量/内容筛选单元（单元2）协同工作。该能力将通过一个专门的药物化学单元（unit 3）来完成，该单元可以单药或批量操作，以促进化合物进展为具有临床评估潜力的有效分子。这三个科学单位将由管理核心（Unit 4）提供支持，管理目标组合，为目标进展做出行政决策，跟踪项目，并与学术合作者或生物技术/制药公司建立战略伙伴关系。SCCDD将利用创新技术，利用化合物合成、计算和筛选方面的协同效率，更有效地发现新的小分子，从而翻译多个治疗领域的靶标，包括但不限于肿瘤、代谢、心血管、免疫和神经疾病领域。简而言之，通过在西南地区建立一个转化药物化学中心，SCCDD的基础设施和资源将广泛地为学术界和当地工业提供功能药物发现过程。

DESCRIPTION (provided by applicant): Advancing new therapeutic targets into innovative drugs is a major translational bottleneck in the southwestern United States. To address this urgent need, we aim to build a critical resource closing major medicinal chemistry resource gaps for our regional drug discovery scientific community. Towards this end, our project aims to deploy the Southwest Comprehensive Center for Drug Discovery and Development (SCCDD) which will provide an enabling collaborative framework for translating basic scientific findings into the clinic. By extension it is envisioned the Center will have resourced interdisciplinary capabilities with an industrialized approach to medicinal chemistry that is more likely to have a significant impact on healthcare. This multiple PI effort assembles existing interdisciplinary teams of scientists with complementary skill sets and significant industry experience. To ensure that the SCCDD can provide a comprehensive offering, we will leverage TGen's existing drug discovery and development infrastructure and strategically network chemistry innovations at the UA. The team has an established reputation and track record of collaboration, and shares a common focused mission: to modernize drug discovery by strategically aligning technological innovations and access to enabeling resources that collectively facilitates more efficient delivery of more effective treatments for the patients in need. The objective of this "Grand Opportunity" is to deploy the necessary infrastructure and resources to reach Center operation status in a two year timeframe. To achieve this objective, we will build an Arizona compound collection, utilizing innovative enabling chemistry, high-throughput screening resources, and a dedicated medicinal chemistry team, overall establishing a functioning flowchart to rapidly expedite compound progression along the value chain. Four units will ensure the process flow as follows. Molecular probe or hit generation will be pursued by a library design, development, and production unit (Unit 1) working in concert with a high-throughput / content screening unit (Unit 2). The capabilities will be completed with a specialized medicinal chemistry unit (Unit 3) operating in either singleton or batch modalities to facilitate compound progression into efficacious molecules with potential for clinical evaluation. These three scientific Units will be supported by an administrative core (Unit 4) managing the target portfolio, making executive decisions for target progression, following projects, and developing strategic partnerships with academic collaborators or biotech/pharma. The SCCDD will leverage innovative technologies taking advantage of synergistic efficiencies in compound synthesis, computation, and screening, for more effective discovery of novel small molecules enabling the translation of targets from multiple therapeutic areas including - but not limited to - neoplastic, metabolic, cardiovascular, immunological, and neurological disease areas. In short, by creating a translational medicinal chemistry presence in the Southwest, the SCCDD infrastructure and resources will widely enable a functional drug discovery process in academic circles and local industry. PUBLIC HEALTH RELEVANCE: The common goal of this proposal is to leverage currently existing high-throughput screening facilities at TGen by building medicinal chemistry infrastructure and library resources at the University of Arizona, leading to the formation of a collaborative, cross-disciplinary network named "the Southwest Comprehensive Center for Drug Discovery and Development". The Center will accelerate the translation of basic scientific findings delivering molecular probes and developing them into more advanced molecules in a broad range of disease areas. The collective team consists of knowledgeable experts with experience ranging from government, academia, biotech and the pharmaceutical sector, sharing the critical mindset of discovering novel therapeutics for the patient in the most expeditious manner possible.