Drug Discovery and Delivery Core

摘要 有一个重大的未得到满足的医学需求，即开发治疗听力障碍的药物疗法。然而，几乎没有人 世界各地的学术中心都致力于这些努力。将重点放在药物治疗上 我们的药物发现和交付核心(DDDC)翻译听力中心(THC)的此类障碍旨在 建立最先进的药物开发渠道，为THC的个别研究项目提供便利。我们会 利用研究项目负责人(RPL)和联合中心调查人员开发的项目 推进听力障碍药物治疗领域。我们的DDDC将指导和培训下一位 一代听力研究人员并吸引新的研究人员为这一快速发展的领域做出贡献。在.期间 第一阶段，DDDC将：1)在硅胶、体外和体内开发高通量筛查，以发现和验证 听力障碍的新疗法；2)建立药物化学管道，优化化学实体 在研究项目中使用或在1或在RPL中发现)；3)建立药物传递和 药代动力学/药效学(PK/PD)方法；4)为DDDC服务制定可持续性计划。 来自Creighton大学和邻近机构(Boys town国家实验室)的坚定承诺 和内布拉斯加大学医学中心)，DDDC已经建立了几乎所有必要的设备 和专家人员。DDDC主任和联席主任在药物开发方面拥有丰富的经验 用于听觉障碍、药物化学和计算结构研究。经济衰退带来的持续影响 DDDC将建立一个最先进的和高度协作的中心，以便增加外部赠款和 赞助项目将逐步覆盖DDDC的主要费用。我们还将强调教育 组件，以便新的研究人员将熟悉这一快速发展的耳治疗学和 在未来几年保持这一势头。

ABSTRACT There is a significant unmet medical need to develop pharmacotherapeutics for auditory disorders. Yet, few academic centers in the world are dedicated to these efforts. With a strong focus on pharmacotherapeutics for such disorders in the Translational Hearing Center (THC), our Drug Discovery & Delivery Core (DDDC) aims to establish a state-of-the-art drug development pipeline to facilitate individual research projects in THC. We will leverage projects developed by Research Project Leaders (RPLs) and allied Center Investigators to significantly advance the field of pharmacotherapeutics for auditory disorders. Our DDDC will mentor and train the next generation of hearing researchers and attract new investigators to contribute to this fast advancing field. During Phase 1, DDDC will: 1) develop in silico, in vitro and in vivo high-throughput screens to discover and validate novel therapeutics for auditory disorders; 2) establish a medicinal chemistry pipeline to optimize chemical entities used in research projects or discovered in 1 or in RPLs); 3) establish drug delivery and pharmacokinetic/pharmacodynamic (PK/PD) methodologies; 4) develop a sustainability plan for DDDC services. With strong commitment from Creighton University and neighboring institutes (Boys Town National Laboratory and University of Nebraska Medical Center), the DDDC has already established nearly all necessary equipment and expert personnel. The Director and Co-Directors of DDDC have extensive experience in drug development for auditory disorders, medicinal chemistry and computational structural studies. The sustained impact of the DDDC will be to build a state-of-the-art and highly collaborative center so that additional extramural grants and sponsored programs will gradually cover the main cost of DDDC. We will also emphasize the educational component so that new investigators will become familiar with this fast-moving field of ototherapeutics and maintain the momentum for years to come.