Scientists Develop Novel Gene Therapy for Glaucoma

Trinity College Dublin scientists, in collaboration with biotechnology company Exhaura Ltd., have made a significant development towards a new therapeutic treatment for glaucoma.

The Smurfit Institute of Genetics team has demonstrated that a gene therapy-based approach can lower intraocular pressure in pre-clinical models of glaucoma. The study, published in the journal Science Advances, found that a single injection of a viral vector could increase aqueous fluid flow from the eye's front, resulting in decreased eye pressure. The viral vector was engineered to instruct cells to produce an enzyme matrix (metalloproteinase-3, or MMP-3) that kickstarts the fluid flow process.

"This exciting project allowed us to bridge the gap between academia and industry and work very closely with a gene therapy company to develop a cutting edge therapy that we believe holds immense promise for patients in the future," said Professor Matthew Campbell, Professor in Genetics at Trinity.

It is noteworthy that the research incorporated numerous disease models and utilized human donor eyes to assess the effectiveness of the gene therapy approach. This comprehensive approach lends even more promise to the remarkable findings.

From Rare to Common Diseases

In recent years, gene therapy has made remarkable progress, resulting in multiple drugs receiving approval from both the FDA and EMA. However, all of the approved gene therapies to date have been for rare or ultra-rare conditions. With the increasing advancement in our knowledge of the underlying mechanisms of common diseases, the potential for gene therapy to treat these conditions is now becoming feasible.

Dr. Jeffrey O'Callaghan, the postdoctoral research fellow at Trinity and the lead author of the study, commented, "Our team's innovative approach to treating glaucoma with gene therapy is the culmination of over seven years of intensive research. We are optimistic that this therapy will serve as a foundation for the development of treatments for other types of debilitating eye diseases."

The study involved a multidisciplinary team of geneticists, ophthalmologists, and translational biologists. Additionally, the team collaborated closely with Exhaura Ltd to ensure that all experimental outputs were focused on the translation of findings into a regulatory-approved clinical program.

Reference:

Jeffrey O'Callaghan et al, Matrix metalloproteinase-3 (MMP-3) mediated gene therapy for glaucoma, Science Advances (2023). DOI: 10.1126/sciadv.adf6537. www.science.org/doi/10.1126/sciadv.adf6537