The cell and gene therapy toolkit for junior faculty

初级教师的细胞和基因治疗工具包

基本信息

批准号：
10380059
负责人：
STEPHAN A. GRUPP
金额：
$ 33.87万
依托单位：
UNIVERSITY OF PENNSYLVANIA
依托单位国家：
美国
项目类别：
财政年份：
2020
资助国家：
美国
起止时间：
2020-04-01 至 2024-03-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/10380059
关键词：
Address Area Basic Science Biological Markers Biomanufacturing Blindness Cancer Center Cancer Research Project Cell Therapy Childhood Leukemia Clinic Clinical Clinical Research Clinical Trials Clinical Trials Design Collaborations Complex Data Development Educational Curriculum Environment Ethics Event FDA approved Faculty Foundations Funding Future Gene Therapy Agent Genes Goals Immunobiology Immunotherapy Industrialization Inherited Intellectual Property Investigation Knowledge Laboratories Leadership Malignant Neoplasms Methods Pediatric Hospitals Pennsylvania Perception Persons Philadelphia Problem Solving Program Development Protocols documentation Public Relations Recording of previous events Regulation Request for Applications Research Research Personnel Research Training Science Scientist Specialist Technology Testing Therapeutic Time Training Translating Translational Research Translations Universities Work Workforce Development anticancer research base cancer therapy career commercial application commercialization design early-career faculty education research experience gene therapy improved interest meetings next generation novel pre-clinical preclinical safety programs recruit response skill acquisition skills success therapeutic development therapy development tool translational cancer research translational pipeline vector virtual

项目摘要

Project Summary/Abstract To meet the need for a cancer research workforce in rapidly emerging areas of cancer therapeutics, we propose a short course in the science and translation of cell and gene therapy. Our objective is to train a cadre of junior faculty level NCI-funded investigators in critical skills related to cell and gene therapy to accelerate their work. The course covers foundations, preclinical tools, protocols for biomanufacturing, intellectual property, regulatory compliance, clinical trial design and systemic challenges in collaborative science. The program is organized by module and provides learners numerous opportunities to interact with faculty 1-on-1 and in group settings. The contributing faculty are pioneers in the field of cell and gene therapy across the translational spectrum from discovery to the first FDA approvals of both cell and gene therapy products, and have been responsible for major advances at Penn, CHOP, the Abramson Cancer Center and globally. In addition to basic scientists, the faculty includes experts and specialists in areas key to translating immunobiology into clinical trials, including specialists in translational and clinical research, biomarker characterization for cell and gene therapy, regulatory science, and commercial collaboration. Although focused on a specific set of topics in cancer research, the theme of cell and gene therapy is increasingly relevant to many aspects of cancer research, and our preliminary data suggests that interest among early NCI- funded investigators is broad. This supports our hypothesis that research training in these topics is essential and increasingly relevant and generalizable to most cancer research programs. Overall, this program will provide NCI-funded junior investigators with critical tools to harness the latest advances in the field of cell and gene therapy to translate their own findings into successful cancer therapies.

项目概要/摘要为了满足快速新兴的癌症治疗领域对癌症研究人员的需求，我们提出细胞和基因疗法的科学和转化短期课程。我们的目标是培养干部初级教师级别的 NCI 资助的研究人员掌握与细胞和基因治疗相关的关键技能，以加速他们的工作。该课程涵盖基础知识、临床前工具、生物制造协议、智力属性、监管合规性、临床试验设计和协作科学中的系统性挑战。这课程按模块组织，为学习者提供大量与教师一对一互动的机会以及在群组设置中。做出贡献的教师是整个细胞和基因治疗领域的先驱细胞和基因治疗产品从发现到 FDA 首次批准的转化谱，以及负责宾夕法尼亚大学、CHOP、艾布拉姆森癌症中心和全球的重大进展。在除了基础科学家外，教师队伍还包括翻译关键领域的专家和专家免疫生物学进入临床试验，包括转化和临床研究、生物标志物方面的专家细胞和基因治疗、监管科学和商业合作的表征。虽然专注于癌症研究中的一组特定主题，细胞和基因治疗的主题越来越多与癌症研究的许多方面相关，我们的初步数据表明早期 NCI 的兴趣资助的调查人员范围广泛。这支持了我们的假设，即这些主题的研究培训至关重要并且与大多数癌症研究项目越来越相关和普遍化。总体而言，该计划将为 NCI 资助的初级研究人员提供关键工具，以利用细胞和细胞领域的最新进展基因疗法将他们自己的发现转化为成功的癌症疗法。