1 Global Alliances & External Research , Santen Incorporated, Emeryville, California.
2 Department of Pharmaceutical Sciences, Texas Southern University , Houston, Texas.
J Ocul Pharmacol Ther. 2018 Jan/Feb;34(1-2):7-39. doi: 10.1089/jop.2017.0125. Epub 2018 Jan 11.
Discovery ophthalmic research is centered around delineating the molecular and cellular basis of ocular diseases and finding and exploiting molecular and genetic pathways associated with them. From such studies it is possible to determine suitable intervention points to address the disease process and hopefully to discover therapeutics to treat them. An investigational new drug (IND) filing for a new small-molecule drug, peptide, antibody, genetic treatment, or a device with global health authorities requires a number of preclinical studies to provide necessary safety and efficacy data. Specific regulatory elements needed for such IND-enabling studies are beyond the scope of this article. However, to enhance the overall data packages for such entities and permit high-quality foundation-building publications for medical affairs, additional research and development studies are always desirable. This review aims to provide examples of some target localization/verification, ocular drug discovery processes, and mechanistic and portfolio-enhancing exploratory investigations for candidate drugs and devices for the treatment of ocular hypertension and glaucomatous optic neuropathy (neurodegeneration of retinal ganglion cells and their axons). Examples of compound screening assays, use of various technologies and techniques, deployment of animal models, and data obtained from such studies are also presented.
眼科研究的重点在于阐明眼部疾病的分子和细胞基础，并发现和利用与之相关的分子和遗传途径。通过这些研究，可以确定合适的干预点来解决疾病过程，并有望发现治疗这些疾病的疗法。向全球卫生当局提交新药临床试验（IND）申请，需要进行多项临床前研究，以提供必要的安全性和疗效数据。本文不涉及此类 IND 研究所需的特定监管要素。然而，为了增强此类实体的整体数据包，并为医学事务提供高质量的基础建设出版物，总是需要进行更多的研究和开发工作。本文旨在为治疗眼高压和青光眼视神经病变（视网膜神经节细胞及其轴突的神经退行性变）的候选药物和器械提供一些目标定位/验证、眼部药物发现过程以及机制和组合增强探索性研究的示例。还介绍了化合物筛选测定、各种技术和技术的使用、动物模型的应用以及从这些研究中获得的数据。
