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：
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
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/10017132
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.

迫切需要针对阿尔茨海默氏病（AD）的新治疗和诊断靶标的分歧组合。为了朝着2025年对AD开发有效治疗的全国目标的进步，NIA制定了几项旨在确定新的AD药物目标的计划，包括AD AD的加速医学合作伙伴关系（AMP-AD）。尽管很有希望，但我们对大多数新兴的治疗假设和提名目标的理解尚不足够支持其整合到药物发现计划中。了解，支持将目标整合到药物发现管道中所需的证据量将远远超过任何一组的专业知识和能力，在这里，我们介绍了阿尔茨海默氏病（Open-AD）的开放药物发现中心。过去十年的活动最终表明，开放的科学方法可用于脱离风险的新治疗方式，例如从AMP-AD出现的一种方法来催化对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药物发现管道，使用来自整个研究社区的证据支持的良好支持的下一代AD目标组合，Open-AD将开发并公开传播资源（实验工具，问题，问题，知识，数据），这些资源可以支持各种独立评估的目标验证和药物发现。在AIM 1中，我们将开发一套优先的社区提名的治疗假设和目标。在AIM 2中，我们将开发目标启用包装，包括高质量，验证良好的试剂，用于目标验证和药物发现。在AIM 3中，我们将出现化学和生物学问题。在AIM 4中，我们将迅速并公开分发所有开放AD资产（包括问题），以通过任何有趣的学术和/或商业研究员对候选药物目标进行表征和实验验证。