Scientists from Trinity College Dublin have developed a highly promising gene therapy to treat glaucoma.
The team has published in the International Journal of Molecular Genetics1 showing how the gene therapy conferred significant benefit in animal models, and in human cells derived from people with glaucoma.
Specifically, the therapy protected key “retinal ganglion cells” (RGCs) that are important in vision and improved their function in an animal model of glaucoma.
The team previously showed its therapy offered promise in treating dry age-related macular degeneration (AMD).
“The need for better treatment options has inspired and motivated us to continue developing gene therapies, and we are delighted with the promise it is showing.”
In human retinal cells, delivery of the gene therapy increased oxygen consumption and ATP (energy) production, indicating enhanced cell performance, Trinity College said in a news release.2
First author, Dr Sophia Millington-Ward, said the team was “delighted with the promise” shown by the potential gene therapies.
“Glaucoma is a complex group of optic neuropathies and a leading cause of blindness… this is a really common condition that badly needs new treatment options.
“It is a multifactorial condition with many different risk factors, which adds to the complexity of treating it. Current glaucoma treatments focus on the use of topical eye drops, surgery, or laser therapy, however the outcomes are variable, with some patients not responding and/or suffering serious side effects.
“The need for better treatment options has inspired and motivated us to continue developing gene therapies, and we are delighted with the promise it is showing.”
The new gene therapy uses an approved virus to deliver an enhanced gene (eNdi1) developed by the Trinity team. The therapy was designed with the aim of boosting mitochondrial activity and reducing damaging reactive oxygen species.
Based on these and other foundational achievements, Vzarii Therapeutics has been founded to expedite future development of dry AMD and glaucoma gene therapies towards human clinical trials.
References
- Millington-Ward S, Palfi A, Farrar GJ et http://https//www.tcd.ie/news_events/articles/2024/trinity-teams-gene-therapy-offers-promise-for-treating-glaucoma–as-well-as-amd-/al. AAV-NDI1 therapy provides significant benefit to murine and cellular models of glaucoma Int. J. Mol. Sci. 2024, 25(16), 8876; doi: 10.3390/ijms25168876.
- Trinity College Dublin, Trinity team’s gene therapy offers promise for treating glaucoma – as well as AMD (news release, 9 Sept 2024) available at: tcd.ie/news_events/articles/2024/trinity-teams-gene-therapy-offers-promise-for-treating-glaucoma–as-well-as-amd-/ [accessed 18 Sept 2024].