Drug intelligence / Profile preview

six-small molecule cocktail

Development stage
Preclinical
Lead developer
Children's Hospital Los Angeles
Modality
Small Molecules
Administration
Intravitreal
01

Overview

The six-small molecule cocktail (6C) is an experimental regenerative therapy designed for vision restoration in conditions such as glaucoma and other optic neuropathies. Developed by researchers at Children's Hospital Los Angeles and the University of Southern California, the cocktail is administered via intravitreal injection. Its mechanism of action involves the in vivo chemical reprogramming of Müller glia—the primary glial cells of the retina—into functional retinal ganglion cells (RGCs). This process bypasses the need for exogenous stem cell transplantation by stimulating endogenous glial cells to proliferate, migrate to the ganglion cell layer, and differentiate into neurons that extend axons to the optic nerve and establish connections with the brain's lateral geniculate nucleus. Preclinical studies in mouse models of ocular hypertension and NMDA-induced injury have demonstrated that the 6C cocktail can support sustained vision recovery lasting over three months.

Other names
6C cocktailsix-small-molecule cocktail

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