First-ever experimental gene therapy aims to restore vision by rejuvenating eye neurons

Life Biosciences is testing ER-100, an experimental gene therapy designed to restore vision by reviving damaged retinal neurons. Unlike existing treatments that slow disease, ER-100 uses epigenetic reprogramming to reset aging retinal cells, targeting optic neuropathies such as glaucoma and NAION, where vision loss is irreversible.

The therapy works by controlled expression of three Yamanaka transcription factors — OCT-4, SOX-2, and KLF-4 (OSK) — to rewind cellular aging signatures. In preclinical studies, ER-100 restored age-related methylation patterns and improved visual function in multiple animal models, including nonhuman primates. With FDA approval for its first human trial, researchers will now evaluate safety, tolerability, and early signs of benefit, opening the door to a disease-modifying approach that could transform treatment for neurodegenerative eye disorders.

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