Gene therapy promises to revolutionise the treatment of inherited eye disease but to date has been applied only to rare diseases caused by single gene defects. Glaucoma is the leading cause of irreversible blindness worldwide but only a small minority of glaucoma cases have single gene defects. Glaucoma progression can often be slowed or stopped by lowering eye pressure, yet around 14% of glaucoma patients continue to go blind despite currently available treatments. We are currently exploring the potential use of gene therapy to treat glaucoma that is progressing despite effective treatment to lower the eye pressure. We have developed a novel AAV2 vector that modulates brain-derived neurotrophic factor signalling and is currently progressing towards human clinical trials. Results to date will be discussed.