Cocktail for endogenous regeneration of retinal ganglion cells and retinal pigment epithelium and methods of use

A chemical cocktail reprograms Muller cells into RGCs and RPEs, overcoming the inefficiencies of current treatments by enhancing their regenerative potential, thereby restoring vision in conditions like glaucoma and AMD.

WO2026128698A1 Publication Date: 2026-06-18CHILDRENS HOSPITAL OF LOS ANGELES

Patent Information

Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
CHILDRENS HOSPITAL OF LOS ANGELES
Filing Date
2025-12-11
Publication Date
2026-06-18

AI Technical Summary

Technical Problem

Current treatments for vision loss due to retinal ganglion cell (RGC) and retinal pigment epithelium (RPE) degeneration, such as in glaucoma and AMD, are inadequate, as they rely on cumbersome methods like pluripotent stem cell differentiation and viral transplants, which are time-consuming and variable in quality, and lack effective strategies for enhancing Muller cell regenerative potential.

Method used

A chemical cocktail comprising a histone deacetylase inhibitor, GSK-3 inhibitor, TGFpRl inhibitor, compound increasing cAMP levels, Wnt/p-catenin signaling pathway modulator, and optionally a7 nicotinic acetylcholine receptor agonist, is used to reprogram Muller cells into RGCs and RPEs, providing a more efficient and controlled method for regeneration.

🎯Benefits of technology

The chemical cocktail effectively differentiates Muller cells into functional RGCs and RPEs, restoring vision by enhancing their regenerative capacity and addressing the limitations of existing treatments.

✦ Generated by Eureka AI based on patent content.

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Abstract

Provided by the inventive concept is a composition / chemical cocktail including small molecules and cell signaling / growth factors. The composition / chemical cocktail may be used to: differentiate endogenous glial cells into retinal ganglion cells (RGCs) and / or retinal pigment epithelia (RPE); regenerate RGCs and / or RPE; treating optic neuropathies, including glaucoma; and treating macular degeneration, such as age-related macular degeneration (AMD).
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