Topical gene delivery for Retinal Degenerative Diseases based on smart non-viral vectors
Chronic retinal disease treatment has been historically limited by several factors. Many drugs cannot pass through the cornea/sclera when administered topically, and the eye is protected from the bloodstream by the blood-retina barrier. We plan to overcome many of these issues by developing new technologies and innovative paradigms for the delivery of genes to the eye. We will create the world’s first topical gene therapy for inherited retinal diseases, opening the door to future clinical trials and the eventual widespread application of these technologies. This approach is challenging and clearly foundational, as this need has thus far been totally unmet.
The strength of this proposal resides in the multidisciplinary collaboration of clinically oriented researchers and clinicians with considerable experience in this field who have come together to promote a paradigm of changing innovation. We hope that our efforts can lead to rapid post-project translation.
The primary objective is to develop new topical formulations based on smart non-viral vectors as therapy for retinal degenerations for which there are currently no curative treatments, such as Retinitis Pigmentosa (RP), which affects millions of people worldwide, and Stargardt’s disease (SD).
The new treatments will use non-viral vectors for the delivery of specific genes (such as the gene ABCA4 for SD) to the damaged retinas, opening the door to future clinical trials and the eventual widespread application of these technologies. Hence, this proposal at once attempts to address a biological goal, active lines of research and a clearly unmet clinical need.