Preserving Cone Photoreceptors in Dry Age-Related Macular Degeneration

We are tackling the root cause of daylight color vision loss in dry AMD, cone photoreceptor degeneration, by developing a novel AAV-based gene therapy to promote cone survival in mouse models of dry AMD.

Dry Age-related macular degeneration (dAMD) is the leading cause of irreversible blindness in adults over 50; yet, there is no effective treatment to preserve or restore vision. The root cause of dAMD is the degeneration of cone photoreceptors, the cells that are essential for daylight color vision. We have recently discovered a novel genetic target, the Retinoic Acid signaling pathway, that, when activated, can promote cone survival. In this proposal, we aim to determine whether activation of this signaling pathway could also promote cone survival in models of dAMD.

We are one of the first groups to develop neuroprotective strategies for cone photoreceptors in dry AMD. We have the expertise and knowledge from studying cone degeneration in Inherited Retinal Degeneration that are applicable to protect cones from degeneration and atrophy in dry AMD. Furthermore, we have developed a novel AAV gene therapy vector that can promote cone survival in multiple retinal diseases.

If the aims are successful, we will have generated the pre-clinical data that our AAV vectors can promote cone survival in mouse models of dry AMD. After this study, we will then test this vector in large animal models for safety before potentially proceeding to a phase I clinical trial.