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: 10250427
• 负责人: Gregory W Carter
• 金额: $ 731.2万
• 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/10250427
• 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.

迫切需要一系列新的阿尔茨海默病(AD)治疗和诊断靶点。为了加快实现到2025年开发有效治疗AD的国家目标的进展，NIA创建了几项旨在确定新的AD药物靶点的倡议，包括AD加速医学伙伴关系(AMP-AD)。尽管前景看好，但我们对大多数新兴的治疗假说和被提名的靶点的理解还不足以支持它们整合到药物发现计划中。考虑到支持将靶点整合到药物发现管道中所需的证据数量将远远超过任何一个小组的专业知识和能力，这里我们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确凿地表明，开放科学方法可以用来降低新的治疗方式的风险，例如从AMP-AD出现的那些，催化药物靶标的生物学验证，并启动新的商业药物发现计划。为了重振AD药物发现渠道，通过来自整个研究界的证据支持的、得到良好支持的下一代AD目标的多样化组合，Open-AD将开发和公开传播资源(实验工具、试剂、探测器、知识、数据)，这些资源可以在各种独立评估中支持目标验证和药物发现。在目标1中，我们将制定一套优先的社区提名的治疗假说和靶点。在目标2中，我们将开发目标启用包，其中包括用于靶标验证和药物发现的高质量、经过良好验证的试剂。在目标3中，我们将开发化学和生物探针。在目标4中，我们将快速和公开地分发所有Open-AD资产-包括探测器-以使任何感兴趣的学术和/或商业研究人员能够表征和实验验证候选药物靶标。