A New Approach to Protecting Vision

Age-related macular degeneration (AMD) is a leading cause of irreversible vision loss, and for a late-stage form called geographic atrophy (GA), current treatments offer limited benefits and require frequent injections. This creates a significant need for new, more effective, and longer-lasting therapies.

Our research focuses on a different approach to protecting the retina. We are developing a potential new therapeutic called ONL1204, a small peptide that inhibits a protein called the Fas receptor. When activated, the Fas receptor can cause cell death and inflammation in the eye, which are key drivers of AMD. By blocking this receptor, ONL1204 has shown promise in preventing this damage in preclinical models of retinal disease.

Our study aimed to answer two critical questions about ONL1204:

  • How long does it last in the eye? We found that a single injection of ONL1204 stayed in the eyes of rabbits and minipigs for a remarkably long time, with a half-life of over 100 days. This suggests it could be a long-lasting treatment, reducing the burden of frequent injections for patients.

  • Does it still work after all that time? We tested the drug's ability to protect the retina in a mouse model that mimics key features of dry AMD. The results showed that even with just two administrations, ONL1204 effectively preserved retinal health, reduced inflammation, and protected the retinal cells from a key marker of cell death.

This research highlights that ONL1204 not only remains in the eye for a long period but also continues to provide a protective benefit over time. These findings support its potential as a promising new therapeutic for chronic retinal diseases, including geographic atrophy.

For more information, please see the full publication here.