ONL Therapeutics (ONL) is a clinical-stage biopharmaceutical company committed to protecting and improving the vision of patients with a range of retinal disease and conditions.
By advancing a breakthrough technology designed to prevent the death of key retinal cells caused by the activation of the natural Fas pathway, ONL is pioneering an entirely new approach to preserving sight. It is the first and only company focused on preventing Fas-mediated death of key retinal cells, which is a root cause of vision loss, a leading cause of blindness and a completely unaddressed medical need.
ONL is pioneering an entirely new approach to preserving sight.
Based on its industry-leading expertise in retinal neuroprotection, ONL is developing breakthrough, first-in-class treatments for serious, vision-threatening retinal diseases and conditions. Preclinical in vivo data, along with a growing body of literature, support potential application of the company’s technology platform in retinal detachment, age-related macular degeneration (AMD), glaucoma and non-infectious uveitis, among other chronic retinal diseases.
ONL’s lead therapeutic candidate, ONL1204, is a first-in-class small peptide Fas inhibitor designed to protect the retina from cell death via both direct and inflammatory signaling in a range of retinal disease. ONL1204 is initially being developed for the treatment of retinal detachment, a condition for which the company has been granted orphan drug designation by the FDA. The company has initiated a Phase I clinical trial for ONL1204 in retinal detachment. In addition to retinal detachment, scientific literature also demonstrates that Fas signaling is relevant to AMD and glaucoma and ONL is exploring these indications with additional preclinical research activities.
ONL believes that retinal detachment alone represents a >$500M global market opportunity, with several potential follow-on indications each representing multibillion-dollar markets. The company possesses a strong and wide-ranging intellectual property portfolio around both the Fas pathway and composition of its drug candidates, as well as strong management and expert advisory teams with decades of experience in drug development and retinal disease.
ONL1204 has been granted orphan drug designation for the treatment of retinal detachment.