Light-Activated Eye Drops Restore Vision in Blind Mice — SkimNews

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- Prosthe6 compounds designed by an IBEC-led consortium restored saccadic eye movements in blinded zebrafish larvae and innate light-avoidance behavior in mouse models of age-related macular degeneration and retinitis pigmentosa, according to a paper in the Journal of the American Chemical Society.
- Pau Gorostiza's team at IBEC showed two lead compounds — prosthe6-12 and prosthe6-15 — worked both via intraocular injection and as topical eye drops, producing restored visual behavior under ordinary indoor and overcast-day illumination.
- The compounds target ON-bipolar neurons via the mGlu6 protein, functioning as "molecular prostheses" that re-activate surviving retinal circuitry downstream of dead photoreceptors without genetic modification or implanted hardware.
- Pedro de la Villa's group at the University of Alcalá ran the mouse behavioral experiments, with additional collaborators from IQAC-CSIC, the University of Barcelona, UAB, IRYCIS, and Fundació Eduard Soler.
- Gorostiza emphasized the approach works independent of the specific genetic mutation driving photoreceptor loss — a contrast with gene therapy, which the source notes is suitable for only a small fraction of patients.
- Eyelumina, a spin-off now being formed, will pursue translational development; the prosthe6 technology is already patented, with safety and formulation studies underway.
- The findings follow the first clinical trial of a photopharmacological vision-restoration drug (targeting an unrelated protein), signaling the field's gradual shift toward human testing.
Why it matters: AMD and retinitis pigmentosa together affect roughly 200 million people worldwide, most of whom cannot benefit from existing gene therapy because their mutations don't match approved treatments. A mutation-independent, drop-form therapy that works in ambient light could reach a far broader patient population than current options — though human trials remain years away and the compounds still require safety validation. The researchers themselves stress the approach does not reverse photoreceptor degeneration, only substitutes for lost function.
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