Open Drug Discovery Center for Alzheimer's Disease

阿尔茨海默病开放药物发现中心

基本信息

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
项目类别：
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&apos s Disease Alzheimer&apos 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

项目摘要

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标，为了加快实现到 2025 年开发有效的 AD 治疗方法的国家目标，NIA 制定了多项旨在识别新 AD 的举措。药物靶点，包括加速 AD 医学合作伙伴关系 (AMP-AD)，尽管我们对大多数新兴治疗假设和提名靶点的了解还不足以支持将其纳入药物发现计划。需要支持集成将一个目标纳入药物发现管道将远远超出任何一个团队的专业知识和能力，在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，催化药物靶标的生物验证并启动新的商业药物发现计划，通过多样化的组合重振 AD 药物发现管道。 - 支持下一代AD 目标得到整个研究界证据的支持，Open-AD 将开发并公开传播资源（实验工具、试剂、探针、知识、数据），以支持各种独立评估中的目标验证和药物发现。 1，我们将制定一套优先的社区提名的治疗假设和目标在目标 2 中，我们将开发目标支持包，其中包括用于目标验证和药物发现的高质量、经过充分验证的试剂。目标 3，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。