Cell Rejuvenation Therapy for Age-related Macular Degeneration

Age-related macular degeneration (AMD) is a leading cause for blindness, resulting in loss of vision at the center of the visual field - the macula, due to damage to the retina.

The disease, usually affecting older adults (>55), was diagnosed in 2020 in 196 million people worldwide, with an estimated economic toll of $343 billion including $255 billion in direct healthcare costs.

In AMD, photoreceptors and retinal pigmented epithelium (RPE) cells which underlie the photoreceptors undergo degeneration.

The standard care is injections of anti-VEGF medicines such as ranibizumab (Lucentis), aflibercept (Eylea) and brolucizumab (Beovu) to the eye every 1-3 months. These delay disease progress but cannot restore lost vision, and have side effects such as bleeding in the eye.

During an ERC Starting project, the PI has discovered a new method that would allow to not only delay the disease but to also improve RPE cells function as a treatment for AMD. The proposed approach to rescue RPE cells is rejuvenation by a technique called partial reprogramming.

Our results suggest that the proposed technique is safe and efficient in rejuvenating mouse and human fibroblasts, and in improving their function.

In the current application, through in vivo studies, we will prepare for implementing our novel approach in our envisioned product I-SEE.

In I-SEE, we will aim to use our discovery of a unique combination of factors, that successfully reprogram cells, to drive aging RPE cells into rejuvenation and improved function.

We will calibrate the expression of the reprogramming factors in RPE cells, and test rejuvenation and improved function in AMD mouse models. IPR and partnering with clinician and industry will be developed concomitantly.

Our final product, I-SEE, is developed based on a PCT patent Application No. 63/210,030, as a cocktail of molecules of mRNA directly applied to the eye, to offer the first AMD treatment that could restore vision.