The cell and gene therapy toolkit for junior faculty

项目总结/摘要 为了满足快速发展的癌症治疗领域对癌症研究人员的需求，我们 建议开设细胞和基因治疗科学和翻译的短期课程。我们的目标是培养一个干部 初级教师水平的国家癌症研究所资助的研究人员在关键技能有关的细胞和基因治疗，以加速 他们的工作。该课程涵盖基础、临床前工具、生物制造协议、知识 财产，法规遵从性，临床试验设计和协作科学的系统性挑战。的 课程按模块组织，为学习者提供了许多与教师1对1互动的机会 在群体设置中。贡献的教师是整个细胞和基因治疗领域的先驱 从细胞和基因治疗产品的发现到FDA首次批准的翻译谱，以及 一直负责在宾夕法尼亚大学，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，学院还包括翻译关键领域的专家和专家。 免疫生物学纳入临床试验，包括转化和临床研究专家，生物标志物 在细胞和基因治疗，监管科学和商业合作的表征。虽然 细胞和基因治疗的主题集中在癌症研究的一组特定主题上， 与癌症研究的许多方面有关，我们的初步数据表明，早期NCI- 资助的调查人员范围很广。这支持了我们的假设，即这些主题的研究培训至关重要 并且越来越多地与大多数癌症研究项目相关和普遍化。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。

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.