The cell and gene therapy toolkit for junior faculty

点击翻译按钮获取中文摘要

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.