ONL1204, ONL’s lead therapeutic candidate, is a first-in-class small molecule peptide designed to protect key retinal cells, including photoreceptors, against the apoptosis (programmed cell death) that occurs in a range of retinal diseases and conditions. It is this death of these retinal cells that is the root cause of vision loss and the leading cause of blindness.

Researchers have shown that ONL1204 effectively inhibits the Fas pathway; one of the body’s primary mechanisms for inducing programmed cell death (apoptosis). Specifically, the compound’s activity inhibits the Fas receptor, blocks the activation of the Fas pathway, and prevents the apoptosis cascade which results in the death of key retinal cells, including photoreceptor.

While initial development efforts for ONL1204 are focused on retinal detachment, preclinical in vivo data, along with a growing body of literature, support potential application in age-related macular degeneration (AMD) and other chronic retinal diseases. Combined, the estimated market for the initial indications that ONL plans to target is >$12 billion globally.

Based on its ability to inhibit the Fas pathway,ONL believes that ONL1204 has therapeutic applications in retinal detachment and both the wet and dry forms of AMD.

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