Drug Discovery and Delivery Core

摘要 存在开发用于听觉障碍的药物治疗剂的显著未满足的医疗需求。然而， 世界上的学术中心都致力于这些努力。专注于药物治疗， 翻译听力中心（THC）中的此类疾病，我们的药物发现和交付核心（DDDC）旨在 建立最先进的药物开发渠道，以促进THC的个别研究项目。我们将 利用研究项目负责人（RPL）和相关中心研究者开发的项目， 推进听觉障碍药物治疗领域。我们的DDDC将指导和培训下一个 新一代的听力研究人员，并吸引新的研究人员为这个快速发展的领域做出贡献。期间 DDDC的第一阶段将：1）开发计算机模拟、体外和体内高通量筛选，以发现和验证 听觉障碍的新疗法; 2）建立药物化学管道以优化化学实体 用于研究项目或在1或RPL中发现）; 3）建立药物递送， 药代动力学/药效学（PK/PD）方法; 4）制定DDDC服务的可持续性计划。 随着来自克雷顿大学和邻近机构（男孩镇国家实验室）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的主任和联席主任在药物开发方面具有丰富的经验 用于听觉障碍药物化学和计算结构研究。持续影响 DDDC将建立一个国家的最先进的和高度合作的中心，使额外的校外赠款和 赞助的项目将逐步支付DDDC的主要费用。我们还将强调教育 组成部分，使新的研究人员将熟悉这个快速发展的领域的耳治疗， 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.