Scientists at Trinity College Dublin have developed a very promising gene therapy to treat glaucoma, a debilitating eye condition that affects around 80 million people globally and can result in total visual loss. The group previously demonstrated the potential of its approach in the treatment of dry age-related macular degeneration (AMD).
The International Journal of Molecular Genetics just published the research that the scientists conducted. Their research demonstrates how gene therapy significantly improved glaucoma patient-derived human cells as well as glaucoma animal models. In an animal model of glaucoma, the medication specifically preserved crucial “retinal ganglion cells” (RGCs) and improved their function. The delivery of gene therapy resulted in greater generation of ATP (energy) and higher consumption of oxygen in human retinal cells, suggesting improved cell performance. The enhanced gene eNdi1, developed by the Trinity team, is delivered via an approved virus in the novel gene therapy. The next focus is on translating the studies to the clinic and patients, which involves numerous extra additional steps.
The Trinity team and Loretto Callaghan recently founded Vzarii Therapeutics to accelerate the development of dry AMD and glaucoma gene treatments towards human clinical trials, building on these and other core accomplishments.