The Southwest Comprehensive Center for Drug Discovery and Development

描述(由申请人提供)：将新的治疗靶点推进到创新药物中是美国西南部的一个主要翻译瓶颈。为了解决这一迫切需求，我们的目标是为我们的区域药物发现科学界建立一个关键资源，填补主要的药物化学资源缺口。为此，我们的项目旨在部署西南药物发现和开发综合中心(SCCDD)，该中心将为将基本科学发现转化为临床提供一个有利的合作框架。根据设想，该中心将拥有资源丰富的跨学科能力，对药物化学采取工业化的方法，这更有可能对医疗保健产生重大影响。这一多重PI努力汇集了现有的跨学科科学家团队，他们拥有互补的技能集和重要的行业经验。为了确保SCCDD能够提供全面的服务，我们将利用TGen现有的药物发现和开发基础设施，并在战略上将化学创新联网到UA。该团队拥有公认的声誉和合作记录，并共享一个共同的重点使命：通过战略性地调整技术创新和获得标签资源，实现药物发现的现代化，共同促进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，以便在两年内达到中心运营状态。为了实现这一目标，我们将利用创新的使能化学、高通量筛选资源和专门的药物化学团队，建立一个亚利桑那州的化合物集合，全面建立一个有效的流程图，以加快化合物在价值链上的进展。四个单位将确保工艺流程如下。分子探针或HIT生成将由图书馆设计、开发和生产单位(单位1)与高通量/内容筛选单位(单位2)协同工作。这些能力将由一个专门的药物化学单元(单元3)完成，该单元以单独或批量方式运行，以促进化合物进展为具有临床评估潜力的有效分子。这三个科学单位将得到一个行政核心(单位4)的支持，该核心单位管理目标组合，为目标进展作出行政决定，跟踪项目，并与学术合作者或生物技术/制药公司发展战略伙伴关系。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，从而能够翻译来自多个治疗领域的靶点，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，通过在西南部建立翻译药物化学存在，SCCDD的基础设施和资源将广泛地使学术界和当地行业的功能性药物发现过程成为可能。 与公共健康相关：这项提议的共同目标是通过在亚利桑那大学建设药物化学基础设施和图书馆资源，利用TGen现有的高通量筛查设施，形成一个名为“西南药物发现和开发综合中心”的协作、跨学科网络。该中心将加快基本科学发现的转化，交付分子探测器，并在广泛的疾病领域将它们开发成更先进的分子。集体团队由来自政府、学术界、生物技术和制药部门的具有丰富经验的知识丰富的专家组成，他们分享了以最快的方式为患者发现新疗法的关键心态。

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.