Therapeutics Development Core (Core B)

治疗学发展核心的项目总结 治疗开发核心(核心B)的目标是建立一个有效的平台 支持化学、生物和细胞管道的开发和验证 治疗多种肾脏疾病的治疗学。西北大学(NU) 一直站在治疗多种疾病的药物发现的前沿，最先进的基础设施 已经建成，共同极大地扩展了我们治疗多种疾病的医疗设备 包括血液病、肿瘤病、神经病和风湿病。然而，他们并没有 针对肾脏特定疗法的开发进行了优先考虑和量身定制。我们建议通过以下方式填补这一空白 在NU-GoKIDNEY中心内建立治疗开发核心。治疗学 开发核心将作为临床前模型核心(核心)之间的高效转换通道 A)和临床和翻译核心(核心C)。本中心建议为以下人士提供“一站式”服务 礼宾服务，以便获得肾脏治疗开发的机会和专家指导，最终目标是 临床翻译的一部分。它的建设将为小说开发提供集中咨询服务 为Nu-GoKidney中心的研究人员提供肾脏疾病特定疗法：(1)设计和测试 实验生物和小分子疗法；(2)细胞疗法的设计和测试；以及 (3)设计和测试基于纳米技术的治疗“有效载荷”的精确靶向 细胞和组织。核心B将实施博士级别的导航员系统，以最大限度地提高服务效率和 促进创新合作。Core B还将利用即将推出的Nephro-Hub来扩展 核心用户群，跟踪核心服务，增强用户互动，促进用户协作。核心B 导演和领航员共同拥有广泛的专业知识，包括新颖的细胞疗法 开发、虚拟药物和生物制剂设计、高通量筛选平台以提高效率 以及基于纳米技术的精确药物靶向。核心B将形式化并捕获这一点 在同一屋檐下集合专业知识，为努戈基德尼中心的调查人员提供更好的 客户服务与专家指导相结合，选择最有效的新疗法路线 发展。通过提供这些核心服务，我们预计治疗开发核心将：(1) 通过提供针对特定目标的咨询和导航，加快肾脏治疗的开发 治疗药物的设计、开发、验证和生产过程；(2)提升肾脏疾病- 将特定疗法发展到现有尖端专业知识和最新技术的前沿 西北大学治疗学发展中的基础设施；(3)催化和协同创新 通过公共平台在NU-GoKIDNEY中心调查人员的动态网络中进行协作 由治疗发展核心提供。

PROJECT SUMMARY TO THERAPEUTICS DEVELOPMENT CORE The goal of the Therapeutics Development Core (Core B) is to establish an efficient platform for supporting the development and validation of a pipeline of chemical, biological, and cellular therapeutics for the treatment of a wide spectrum of kidney diseases. Northwestern University (NU) has been at the forefront of drug discovery for a wide range of diseases, and state-of-the-art infrastructures have been built that collectively have significantly expanded our therapeutics armamentarium for many diseases including hematological, oncological, neurological, and rheumatological conditions. However, they have not been prioritized and tailored to kidney-specific therapeutics development. We propose to fill this gap by establishing a Therapeutics Development Core within the NU-GoKIDNEY Center. The Therapeutics Development Core will function as an efficient translational conduit between the Preclinical Models Core (Core A) and the Clinical and Translational Core (Core C). This Core proposes to provide a “one-stop” shop for concierge services for access and expert guidance to kidney therapeutics development, with the ultimate goal of clinical translation. It will be constructed to provide centralized consultation services for developing novel kidney disease-specific therapeutics to the NU-GoKIDNEY Center investigators for: (1) designing and testing experimental biological and small molecule therapeutics; (2) designing and testing cellular therapeutics; and (3) designing and testing nanotechnology-based precision targeting of therapeutic “payloads” to the desired cells and tissues. Core B will implement a Ph.D.-level Navigator system for maximizing service efficiency and promoting innovative collaborations. Core B will also use the soon-to-be-launched nephro-HUB to expand the core user base, track core services, enhance user interactions and promote user collaborations. Core B Directors and Navigators collectively possess extensive expertise encompassing novel cellular therapeutics development, virtual drug and biologics design, high-throughput screening platforms for efficiency determination, and nanotechnology-based precision drug targeting. Core B will formalize and capture this collective expertise under one roof, and provide investigators of the NU-GoKIDNEY Center with superior customer service combined with expert guidance in choosing the most efficient route for novel therapeutics development. By providing these core services, we anticipate that the Therapeutics Development Core will: (1) accelerate kidney therapeutics development by providing target-specific consultations and navigation through the processes of therapeutics design, development, validation and production; (2) elevate kidney disease- specific therapeutics development to the forefront of existing cutting-edge expertise and state-of-the-art infrastructures in therapeutics development at Northwestern; (3) catalyze and synergize innovative collaborations within a dynamic network of NU-GoKIDNEY Center investigators via a common platform provided by the Therapeutics Development Core.