Blinding diseases caused by photoreceptor degeneration, such as age-related macular degeneration and retinitis pigmentosa, affect 200 million people worldwide and are the leading causes of visual impairment and blindness. Now, a study published in the Journal of the American Chemical Society presents a new class of photoswitchable drugs capable of restoring key visual functions in animal models of blindness.
Two compounds, known as prosthe6-12 and prosthe6-15, have shown particularly promising results. The restored behaviours have been observed not only after intraocular injection but also following topical administration as eye drops.
"Our goal was to restore vision using a molecular mechanism that is as close as possible to how the healthy retina works," says Rosalba Sortino, co-first author of the article and recipient of the University of Barcelona's Extraordinary Doctoral Award for the 2023-2024 academic year for her thesis defended at the Faculty of Pharmacy and Food Science.
"Instead of bypassing retinal processing, we aimed to reactivate it right at the same level of the retinal circuit than the lost photoreceptor cells," explains Sortino, postdoctoral researcher on the Nanoprobes and Nanoswitches Group led by expert Pau Gorostiza, an ICREA research professor at the Institute for Bioengineering of Catalonia (IBEC) and co-leader of the study.
Gorostiza, also a member of the CIBER Area for Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN), says that "these molecules do not cure blindness, because they do not address the cause of photoreceptor degeneration. But they are remarkably effective at restoring sight, and they do so using a very simple and potentially patient-friendly approach".
