Genes as drugs for glaucoma: latest advances

Abstract

Purpose of review To provide the latest advances on the future use of gene therapy for the treatment of glaucoma. Recent findings In preclinical studies, a number of genes have been shown to be able to reduce elevated intraocular pressure (IOP), and to exert neuroprotection of the retinal ganglion cells. These genes target various mechanisms of action and include among others: MMP3, PLAT, IκB, GLIS, SIRT, Tie-2, AQP1. Some of these as well as some previously identified genes (MMP3, PLAT, BDNF, C3, TGFβ, MYOC, ANGPTL7) are starting to move onto drug development. At the same time, progress has been made in the methods to deliver and control gene therapeutics (advances in these areas are not covered in this review). Summary While preclinical efforts continue in several laboratories, an increasing number of start-up and large pharmaceutical companies are working on developing gene therapeutics for glaucoma (Sylentis, Quetera/Astellas, Exhaura, Ikarovec, Genentech, Regeneron, Isarna, Diorasis Therapeutics). Despite the presence of generic medications to treat glaucoma, given the size of the potential world-wide market (∼$7B), it is likely that the number of companies developing glaucoma gene therapies will increase further in the near future.