IGF::OT::IGF - Gene Therapy Resource Program (GTRP) AAV Vector Production Core La

IGF::OT::IGF - 基因治疗资源计划 (GTRP) AAV 载体生产核心 La

• 批准号: 8755786
• 负责人: DANA LOTSPEICH
• 金额: $ 32.5万
• 依托单位: CHILDREN'S HOSP OF PHILADELPHIA
• 依托单位国家: 美国
• 财政年份: 2012
• 资助国家: 美国
• 起止时间: 2012-06-15 至 2017-06-14
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8755786
• 关键词: Adenoviruses; Certification; Charge; Clinical; Clinical Trials; Collaborations; Complex; Contractor; Data; Databases; Ensure; Equipment and supply inventories; Funding; Guidelines; Human; Laboratories; Manuals; Mission; Monitor; National Heart, Lung, and Blood Institute; Patients; Procedures; Process; Production; Quality Control; Recommendation; Records; Regulation; Research; Research Personnel; Resources; Running; Secure; Services; Shipping; Ships; Site Visit; Surveys; Teleconferences; Testing; Translational Research; Update; Work; experience; gene therapy; gene therapy clinical trial; meetings; pre-clinical; programs; satisfaction; scale up; vector; web site; working group

In June 2005 the NHLBI convened an expert Working Group to better understand the impediments to clinical progress in gene therapy and to advise the NHLBI how to best direct its limited resources to facilitate translational research in the field of gene therapy. The NHLBI responded to these recommendations by establishing the NHLBI Gene Therapy Resource Program (GTRP) in March 2007. The GTRP consists of a Clinical Coordinating Center (CCC) and four Core Laboratories. The Adenovirus-Associated Vector (AAV) Production Core Laboratory is charged with supplying preclinical and clinical-grade products to investigators in a manner that is compliant with all relevant regulatory requirements. The laboratory works very closely with investigators who request services from the GTRP, the Core Laboratories, and the NHLBI to ensure that all aspects of the GTRP run smoothly and that the work performed is within with the scope of the GTRP and the Mission of the NHBLI. During the initial funding period the AAV laboratory has conducted approximately 3-4 campaigns per year for 10-20 patient clinical trials. Two GMP-grade AAV vectors have been produced, and scale-up may be required if the products are approved for larger trials. Fulfilling the various service requests requires extensive interactions with all parties involved and the process can take months to years, particularly those aspects related to human clinical trials. Therefore, it is critical that the AAV laboratory have demonstrated experience in the management of complex research programs and human clinical trials.

2005年6月，NHLBI召集了一个专家工作组，以更好地了解基因治疗临床进展的障碍，并建议NHLBI如何最好地利用其有限的资源促进基因治疗领域的转化研究。2007年3月，NHLBI通过建立NHLBI基因治疗资源计划（GTRP）回应了这些建议。GTRP由一个临床协调中心（CCC）和四个核心实验室组成。腺病毒相关载体（AAV）生产核心实验室负责以符合所有相关法规要求的方式向研究者提供临床前和临床级产品。实验室与要求GTRP、核心实验室和NHLBI提供服务的研究者密切合作，以确保GTRP的所有方面顺利运行，并且所执行的工作符合GTRP和NHLBI的使命范围。在最初的资助期间，AAV实验室每年进行大约3-4次活动，用于10-20名患者的临床试验。已经生产了两种GMP级AAV载体，如果产品被批准用于更大的试验，可能需要扩大规模。满足各种服务要求需要与所有相关方进行广泛的互动，该过程可能需要数月至数年，特别是与人体临床试验相关的方面。因此，至关重要的是，AAV实验室在管理复杂的研究项目和人体临床试验方面具有丰富的经验。