Open Drug Discovery Center for Alzheimer's Disease

阿尔茨海默病开放药物发现中心

• 批准号: 10017132
• 负责人: Gregory W Carter
• 金额: $ 742.01万
• 依托单位: EMORY UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-09-30 至 2024-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10017132
• 关键词: Affect; Alzheimer' s Disease; Alzheimer' s disease model; Amyloid; Animal Model; Antibodies; Biological; Biological Assay; Biology; Cell Line; Chemicals; Communities; Data; Development; Diamond; Disease; Disease Progression; Drug Targeting; Evaluation; Funding; Goals; Guide RNA; In Vitro; Industry; Industry Collaboration; Investigation; Knowledge; Learning; Legal patent; Medicine; Modality; Nature; Neurodegenerative Disorders; Nucleic Acid Probes; Outcome; Pathway interactions; Pharmaceutical Preparations; Pharmacologic Substance; Process; Proteins; Reagent; Records; Research; Research Personnel; Resources; Risk; Rivers; Scientist; Set protein; Structure; Testing; Therapeutic; Time; Validation; base; candidate validation; computerized tools; design; drug development; drug discovery; drug market; effective therapy; experience; high risk; in vivo; industry partner; interest; material transfer agreement; next generation; novel diagnostics; novel therapeutics; open data; pharmacokinetics and pharmacodynamics; pre-clinical; preclinical evaluation; programs; public repository; public-private partnership; research clinical testing; screening; structural genomics; targeted treatment; tau Proteins; therapeutic evaluation; tool

There is an urgent need for a diverse portfolio of new therapeutic and diagnostic targets for Alzheimer’s disease (AD). To hasten progress towards the national goal of developing an effective treatment for AD by 2025, the NIA has created several initiatives designed to identify new AD drug targets, including the Accelerating Medicine Partnership for AD (AMP-AD). Although promising, our understanding of most of the emerging therapeutic hypotheses and nominated targets is not yet sufficient to support their integration into drug discovery programs. Understanding that the amount of evidence required to support the integration of a target into a drug discovery pipeline would far surpass the expertise and capabilities of any one group, here we introduce the Open Drug Discovery Center for Alzheimer’s Disease (Open-AD). The past decade of activity has conclusively shown that the open science approach can be used to de-risk new therapeutic modalities, such as the ones emerging from AMP-AD, to catalyze biological validation of drug targets and to launch new commercial drug discovery programs. To reinvigorate the AD drug discovery pipeline with a diverse portfolio of well-supported next generation AD targets supported by evidence from across the research community, Open-AD will develop and openly disseminate resources (experimental tools, reagents, probes, knowledge, data) that can support target validation and drug discovery across a wide variety of independent evaluations. In Aim 1, we will develop a prioritized set of community-nominated therapeutic hypotheses and targets. In Aim 2, we will develop target enabling packages that include high-quality, well-validated reagents for use in target validation and drug discovery. In Aim 3, we will develop chemical and biological probes. In Aim 4, we will rapidly and openly distribute all Open-AD assets – including probes – to enable characterization and experimental validation of candidate drug targets by any interested academic and/or commercial investigator.

阿尔茨海默病 (AD) 迫切需要多种新的治疗和诊断靶点。为了加快实现到 2025 年开发有效 AD 治疗方法的国家目标，NIA 制定了多项旨在确定新 AD 药物靶点的举措，其中包括 Accelerated Medicine Partnership for AD (AMP-AD)。尽管前景广阔，但我们对大多数新兴治疗假设和指定靶点的理解还不足以支持它们融入药物发现计划。我们认识到支持将某个靶标整合到药物发现流程中所需的证据数量将远远超过任何一个团队的专业知识和能力，因此我们在此介绍阿尔茨海默病开放药物发现中心 (Open-AD)。过去十年的活动已经明确表明，开放科学方法可用于降低新治疗方式（例如 AMP-AD 中出现的治疗方式）的风险，以催化药物靶点的生物验证并启动新的商业药物发现计划。为了通过由整个研究界的证据支持的多种得到良好支持的下一代 AD 靶点组合来重振 AD 药物发现管道，Open-AD 将开发并公开传播资源（实验工具、试剂、探针、知识、数据），这些资源可以支持跨各种独立评估的靶点验证和药物发现。在目标 1 中，我们将制定一套社区提名的优先治疗假设和目标。在目标 2 中，我们将开发靶点支持包，其中包括用于靶点验证和药物发现的高质量、经过充分验证的试剂。在目标3中，我们将开发化学和生物探针。在目标 4 中，我们将快速公开地分发所有 Open-AD 资产（包括探针），以便任何感兴趣的学术和/或商业研究人员能够对候选药物靶标进行表征和实验验证。