Jul 14, 2022

Endogena Launches Clinical Trial of Therapy to Activate Stem Cells in RP Patients’ Retinas

Eye On the Cure Research News

The emerging treatment is gene-agnostic.

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Endogena Therapeutics has dosed the first patient in its Phase 1/2 clinical trial for E-2353, its small molecule designed to activate dormant retinal stem cells in the eyes of patients with retinitis pigmentosa (RP) to produce photoreceptors, the retinal cells that make vision possible, but degenerate in RP. The endogenous retinal stem cells migrate from the ciliary epithelium, located just behind the iris, to the retina, where they develop into photoreceptors. The treatment is designed to work regardless of the gene causing the RP.

The 14-participant trial will take place at six sites in the US. Participants will receive the treatment through an injection into the vitreous, the soft gel in the middle of the eye. Each patient will have one eye treated.

To participate in the trial, participants must have a clinical and genetic diagnosis of RP — that is, they must have a mutation(s) identified in one of the 80-plus genes that cause RP. Vision in the treated eye must be between 20/50 and the ability to count fingers. 

Principal Investigator, Mark Pennesi, MD, PhD, professor of ophthalmology at the Casey Eye Institute in Oregon, said: "This is very exciting, and I am hopeful that this new treatment has the potential to be a major game-changer for patients with RP who currently have no other options."

A youtube video from Endogena provides additional information about E-2353.

Endogena is also conducting preclinical studies of a small molecule for activating retinal pigment epithelial (RPE) stem cells to develop into mature RPE for the treatment of geographic atrophy, the advanced form of dry age-related macular degeneration.