Molecular Therapeutics Research Program

摘要：分子治疗学 分子治疗学（MT）项目致力于评估癌症靶点，开发新的 治疗，并通过高度集成的基础科学和转化设计更有效的输送系统， 活动该计划由综合化学生物学中心主任Stephen Frye领导， 药物发现（CICBDD）和弗雷德Eshelman杰出教授，和加里约翰逊，凯南 药理学系杰出教授。MT计划由42名成员组成， 与四所学校和16个部门有联系。在上一个资助期内，项目成员 发表了675篇癌症相关文章。MT是高度合作的。这些论文中有16%是程序内的 33%是跨计划的（43%是合作的）。在2019年，我们的计划成员持有的赠款总额 2090万美元（直接成本）用于癌症相关的校外资助，包括来自NCI的640万美元（直接成本）， 1300万美元的其他同行资金。MT计划由在五大领域具有专业知识的调查人员组成： 化学和结构生物学;药物发现和开发;药物输送和纳米技术; 系统药理学和致癌信号。该项目中的许多研究人员都积极参与 与其他LCCC计划合作，使用LCCC的共享资源并为其提供指导。这些 相互作用使许多科学步骤所需的发现和发展有前途的 治疗在2015年，MT被评为“杰出”，指出“该计划的小弱点是，相对于 一些有前途的治疗方法已经进入了LCCC的研究人员发起的临床试验。由于这 综述，基于MT的发现，在FDA和其他机构已经启动了多项临床研究， 正如我们对先前批评的回应中所概述的那样。未来的计划建立在独特的翻译资源上 由于Eshelman创新研究所和Pinnacle Hill的成立， 百万美元用于将基于UNC的发现进展到患者身上。细胞和生物疗法将是一个领域， 通过扩大临床免疫治疗计划的GMP设施，MT成员的未来重点。 通过这些努力，MT将继续加速发现新的癌症治疗方法， 临床研究和乳腺癌计划，设计和执行转化和治疗试验，在我们的 患者

ABSTRACT: MOLECULAR THERAPEUTICS PROGRAM The Molecular Therapeutics (MT) program is dedicated to evaluating cancer targets, developing novel therapies, and devising more effective delivery systems via highly integrated basic science and translational activities. The Program is led by Stephen Frye, Director of the Center for Integrative Chemical Biology and Drug Discovery (CICBDD) and Fred Eshelman Distinguished Professor, and Gary Johnson, Kenan Distinguished Professor in the Department of Pharmacology. The MT Program consists of 42 members who are associated with four schools and 16 departments. During the last funding period, program members have published 675 cancer-related articles. MT is highly collaborative. 16% of these papers are intra-programmatic and 33% are inter-programmatic (43% collaborative). In 2019, our program members held grants totaling $20.9M (direct cost) in cancer-relevant extramural funding, including $6.4M (direct costs) from the NCI and $13.0M other peer funding. The MT program is comprised of investigators with expertise in five broad areas: Chemical and Structural Biology; Drug Discovery and Development; Drug Delivery and Nanotechnology; Systems Pharmacology; and Oncogenic Signaling. Many investigators in the program have active collaborations with other LCCC programs, using and providing direction for LCCC's shared resources. These interactions enable many of the scientific steps needed for the discovery and development of promising therapies. In 2015, MT was rated “outstanding” stating “the minor weakness of this program is that, relatively few of the promising therapeutics have moved into investigator initiated clinical trials at the LCCC.” Since this review, multiple clinical studies have been initiated at UNC and other institutions based on discoveries in MT, as outlined in our response to the prior critique. Future plans build on unique translational resources available due to the creation of the Eshelman Institute for Innovation and Pinnacle Hill, which bring more than $100 million to progress UNC-based discoveries into patients. Cellular and biologic therapies will be an area of future focus for MT members enabled by the expansion of the Clinical Immunotherapy Program's GMP facility. Through these efforts, MT will continue to accelerate discovery of new cancer therapeutics and, with the Clinical Research and Breast Cancer Programs, design and execute translational and therapeutic trials in our patients.