Cell Rejuvenation Therapy for Age-related Macular Degeneration

Introduction

Age-related macular degeneration (AMD) is a leading cause of 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.

Disease Mechanism

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

Current Treatments

The standard care involves injections of anti-VEGF medicines such as:

• Ranibizumab (Lucentis)
• Aflibercept (Eylea)
• Brolucizumab (Beovu)

These treatments are administered to the eye every 1-3 months. While they delay disease progression, they cannot restore lost vision and have side effects such as bleeding in the eye.

Research Background

During an ERC Starting project, the Principal Investigator (PI) discovered a new method that would allow for not only delaying the disease but also improving RPE cell function as a treatment for AMD.

Proposed Approach

The proposed approach to rescue RPE cells is rejuvenation by a technique called partial reprogramming. Our results suggest that this technique is safe and efficient in rejuvenating mouse and human fibroblasts, and in improving their function.

Implementation Plan

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

Goals of “I-SEE”

In “I-SEE”, we aim to:

1. Use our discovery of a unique combination of factors that successfully reprogram cells.
2. 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.

Collaboration and Development

Intellectual Property Rights (IPR) and partnerships with clinicians and industry will be developed concomitantly.

Final Product

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.