NATIONAL INSTITUTE OF HEALTH (NIH), BESPOKE GENE THERAPY CONSORTIUM (BGTC)P3 BCC TO# 009 NCATS/ORDR
NATIONAL INSTITUTE OF HEALTH (NIH), BESPOKE GENE THERAPY CONSORTIUM (BGTC)P3 BCC TO# 009 NCATS/ORDR
批准号:
10722593
负责人:
HALEY ESSER
金额:
$22.9万
依托单位:
--
依托单位国家:
美国
项目类别:
财政年份:
2022
资助国家:
美国
项目状态:
已结题
起止时间:
2022-09-05 至 2023-09-04
关键词:
AffectAnimal Disease ModelsBasic ScienceBiologicalBiologyBooksCellsClinical ResearchClinical TrialsCommunitiesCustomDependovirusDevelopmentFoundationsFundingGene ActivationGene DeliveryGenesHumanMedicineMethodsNational Center for Advancing Translational SciencesPathway interactionsPersonsPreclinical TestingPrivatizationProcessProductionProtocols documentationRare DiseasesResearchResearch PersonnelScientistSpeedStandardizationTestingTherapeuticToxicologyUnited States Food and Drug AdministrationUnited States National Institutes of HealthViral Vectordelivery vehiclegene therapyimprovedprogramspublic-private partnershiprare conditionvector
中文摘要
加速药物伙伴关系(AMP)计划是美国国立卫生研究院(NIH),美国食品药品监督管理局(FDA)和多个公共和私人组织之间的公私合作伙伴关系。AMP项目由美国国立卫生研究院基金会(FNIH)管理,旨在提高我们对治疗相关生物学途径的理解,并验证可能与多种疗法开发相关的信息。
AMP定制基因治疗联盟(BGTC)旨在开发平台和标准,以加快定制或“定制”基因疗法的开发和交付,这些疗法可以治疗数百万受罕见疾病影响的人。
BGTC于2021年10月推出,是首个专注于罕见疾病的AMP计划,也是第六个AMP计划。这项工作旨在克服与开发基因疗法相关的主要障碍,并将创建一个基因疗法协议书,研究界可以使用该协议书来使罕见疾病的基因疗法开发过程更加有效。
英文摘要
The Accelerating Medicines Partnership® (AMP®) program is a public-private partnership between the National Institutes of Health (NIH), the U.S. Food and Drug Administration (FDA), and multiple public and private organizations. Managed through the Foundation for the NIH (FNIH), the AMP program aims to improve our understanding of therapeutically relevant biological pathways and validate information that could be relevant for the development of multiple therapeutics.
The AMP Bespoke Gene Therapy Consortium (BGTC) aims to develop platforms and standards that will speed the development and delivery of customized or ‘bespoke’ gene therapies that could treat the millions of people affected by rare diseases.
Launched in October 2021, BGTC is the first AMP initiative focused on rare diseases and the sixth AMP initiative overall. The effort aims to overcome major obstacles related to developing gene therapies and will create a gene therapy protocol book that the research community can use to make the process of developing gene therapies for rare conditions much more efficient.
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SERVICES (OCCS INFORMATION TECHNOLOGY AND APPLICATION
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批准号:2867722
-
项目类别:
-
资助金额:$27.67万
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财政年份:1998
-
负责人:HALEY ESSER
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依托单位:
SERVICES (OCCS INFORMATION TECHNOLOGY AND APPLICATION
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批准号:2884742
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项目类别:
-
资助金额:$10.0万
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财政年份:1998
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负责人:HALEY ESSER
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依托单位:
海外基金