Therapeutics Development Core (Core B)

治疗开发核心项目总结 治疗开发核心（核心B）的目标是建立一个有效的平台， 支持开发和验证化学、生物和细胞的管道， 用于治疗多种肾脏疾病的疗法。西北大学（NU） 一直处于药物发现的最前沿，用于各种疾病，最先进的基础设施 它们共同显著地扩展了我们对许多疾病的治疗手段 包括血液学、肿瘤学、神经学和流变学病症。然而，他们没有 优先考虑并针对肾脏特异性疗法的开发。我们建议填补这一空白， 在NU-GoKIDNEY中心内建立治疗开发核心。治疗剂 开发核心将作为临床前模型核心（核心）之间的有效转换管道 临床和翻译核心（Core C）。本核心建议提供“一站式”服务， 礼宾服务，为肾脏治疗开发提供访问和专家指导，最终目标是 临床翻译它将被建设为开发新产品提供集中咨询服务 肾脏疾病特异性治疗的NU-GoKIDNEY中心研究人员：（1）设计和测试 实验生物学和小分子疗法;（2）设计和测试细胞疗法;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将实施博士学位-水平导航系统，最大限度地提高服务效率， 促进创新合作。Core B还将使用即将推出的肾-HUB B来扩展 核心用户群，跟踪核心服务，增强用户交互，促进用户协作。核心B 董事和导航员共同拥有广泛的专业知识，包括新的细胞疗法 开发，虚拟药物和生物制剂设计，高通量筛选平台， 测定和基于纳米技术的精确药物靶向。核心B将正式化并捕获此 在同一屋檐下的集体专业知识，并为NU-GoKIDNEY中心的研究人员提供上级 客户服务与专家指导相结合，为新疗法选择最有效的途径 发展通过提供这些核心服务，我们预计治疗药物开发核心将：（1） 通过提供针对特定目标的咨询和导航，加速肾脏治疗的发展， 治疗药物的设计、开发、验证和生产过程;（2）提高肾脏疾病- 特定疗法发展到现有尖端专业知识和最先进技术的最前沿 基础设施的治疗发展在西北;（3）催化和协同创新 通过通用平台在NU-GoKIDNEY中心研究者的动态网络内进行协作 由Therapeutics Development Core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.