PAASP GmbH, Heidelberg, Germany.
Department of Experimental Neurology, Charité-Universitätsmedizin Berlin, Berlin, Germany.
Nat Rev Drug Discov. 2021 Jan;20(1):64-81. doi: 10.1038/s41573-020-0087-3. Epub 2020 Nov 16.
Academic research plays a key role in identifying new drug targets, including understanding target biology and links between targets and disease states. To lead to new drugs, however, research must progress from purely academic exploration to the initiation of efforts to identify and test a drug candidate in clinical trials, which are typically conducted by the biopharma industry. This transition can be facilitated by a timely focus on target assessment aspects such as target-related safety issues, druggability and assayability, as well as the potential for target modulation to achieve differentiation from established therapies. Here, we present recommendations from the GOT-IT working group, which have been designed to support academic scientists and funders of translational research in identifying and prioritizing target assessment activities and in defining a critical path to reach scientific goals as well as goals related to licensing, partnering with industry or initiating clinical development programmes. Based on sets of guiding questions for different areas of target assessment, the GOT-IT framework is intended to stimulate academic scientists' awareness of factors that make translational research more robust and efficient, and to facilitate academia-industry collaboration.
学术研究在确定新的药物靶点方面发挥着关键作用,包括了解靶点的生物学特性以及靶点与疾病状态之间的联系。然而,为了开发新药,研究必须从纯粹的学术探索推进到启动临床试验中候选药物的识别和测试工作,这通常由生物制药行业来进行。这一转变可以通过及时关注目标评估方面的问题来促进,例如与目标相关的安全性问题、可成药性和可检测性,以及目标调节的潜力,以实现与已确立的治疗方法的差异化。在这里,我们提出了 GOT-IT 工作组的建议,旨在支持学术科学家和转化研究的资助者确定和优先考虑目标评估活动,并定义一条关键路径,以实现科学目标以及与许可、与行业合作或启动临床开发计划相关的目标。基于针对目标评估不同领域的一系列指导问题,GOT-IT 框架旨在激发学术科学家对使转化研究更稳健和高效的因素的认识,并促进学术界与产业界的合作。