Scientists have created a highly effective gene therapy to address glaucoma, a serious eye disorder that may result in total vision loss and affects approximately 80 million individuals globally. Previously, the same team demonstrated that their therapy showed potential in treating dry age-related macular degeneration (AMD).
Researchers at Trinity College Dublin have formulated a very promising gene therapy to combat glaucoma—an eye condition that severely impairs vision and affects around 80 million people worldwide. This team has previously indicated that their therapy holds potential for treating dry age-related macular degeneration (AMD).
The researchers recently shared their findings in the International Journal of Molecular Genetics. Their study illustrates how the gene therapy significantly benefited animal models and human cells extracted from glaucoma patients.
Particularly, this therapy safeguarded critical “retinal ganglion cells” (RGCs), which play a vital role in vision, and enhanced their functionality in an animal model with glaucoma. In human retinal cells, the application of the gene therapy led to heightened oxygen use and ATP (energy) production, suggesting improved cell effectiveness.
The lead author of the article, Dr. Sophia Millington-Ward, a Research Fellow at Trinity’s School of Genetics and Microbiology, stated: “Glaucoma encompasses a complex range of optic neuropathies and is a major contributor to blindness. In Europe, about 1 in 30 individuals aged 40 to 80 have glaucoma, and the ratio increases to 1 in 10 for those over 90, indicating a widespread necessity for new treatment alternatives.”
“This condition has multiple contributing factors, making it challenging to treat. Current methods for glaucoma management include topical eye drops, surgery, or laser treatment, but results can vary significantly, with some patients not responding or experiencing serious side effects.”
“The urgent requirement for improved treatment options has driven us to pursue the development of gene therapies, and we are thrilled by the potential it is demonstrating.”
This innovative gene therapy employs an authorized virus to introduce an advanced gene (eNdi1) crafted by the Trinity team. The therapy aims to enhance mitochondrial activity (the “energy factories” of cells responsible for producing ATP) and diminish harmful reactive oxygen species.
Jane Farrar, a Research Professor in Trinity’s School of Genetics and Microbiology and the senior author of the study, commented:
“Creating widely applicable gene therapies for a large patient base is crucial, given the high costs associated with developing each therapy. Our findings highlight the real potential of this therapy to improve mitochondrial function in glaucoma cases.”
The next step involves translating these studies into clinical settings and patient applications, which will require several additional steps.
Building on these foundational successes, the Trinity team, alongside Loretto Callaghan, has recently established Vzarii Therapeutics to hasten the development of gene therapies for both dry AMD and glaucoma towards human clinical trials.
