Gene Therapy Resource Program Clinical Coordinating Center

基因治疗资源计划临床协调中心

• 批准号: 8860073
• 负责人: SUSAN SEPELAK
• 金额: $ 82.16万
• 依托单位: SOCIAL AND SCIENTIFIC SYSTEMS, INC.
• 依托单位国家: 美国
• 财政年份: 2012
• 资助国家: 美国
• 起止时间: 2012-06-01 至 2015-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8860073
• 关键词: Area; Basic Science; Clinical; Clinical Trials; Dependovirus; Funding; Heart Diseases; Hematological Disease; Laboratories; Logistics; Lung diseases; National Heart, Lung, and Blood Institute; Pharmacology and Toxicology; Production; Research Personnel; Resources; Services; Site Visit; Subfamily lentivirinae; Translations; clinical application; gene therapy; gene therapy clinical trial; meetings; pre-clinical; programs; symposium; vector

The objective of the NHLBI Gene Therapy Resource Program (GTRP) renewal is to continue supporting the translation of basic research in gene therapy to clinical application in the areas of heart, lung, and blood diseases. The GTRP renewal will continue to provide resources in the areas of preclinical and clinical-grade vector production in accordance with Good Manufacturing Practices (GMP), and in the conduct of pharmacology/toxicology studies. In addition, the GTRP renewal will provide funds and regulatory assistance to investigators wishing to conduct gene therapy clinical trials. The GTRP renewal will consist of the following five components: 1) a clinical coordinating center that will oversee and coordinate the logistics of the core laboratories and provide regulatory and funding assistance for clinical trials; 2) a preclinical-grade vector production core laboratory; 3) a clinical-grade vector production core laboratory for adeno-associated virus (AAV); 4) a clinical-grade vector production core laboratory for lentivirus; and 5) a pharmacology/toxicology core laboratory.

NHLBI基因治疗资源计划(GTRP)更新的目标是继续支持基因治疗的基础研究转化为心、肺和血液疾病领域的临床应用。GTRP更新将继续根据良好制造规范(GMP)在临床前和临床级媒介生产领域提供资源，并在进行药理学/毒理学研究方面提供资源。此外，GTRP的续签将为希望进行基因治疗临床试验的研究人员提供资金和监管援助。GTRP的更新将包括以下五个部分：1)临床协调中心，负责监督和协调核心实验室的后勤工作，并为临床试验提供监管和资金援助；2)临床级媒介生产核心实验室；3)临床级腺相关病毒(AAV)载体生产核心实验室；4)临床级慢病毒载体生产核心实验室；以及5)药理学/毒理学核心实验室。