Life Biosciences Doses First Patient in Phase 1 of ER‑100 for Optic Neuropathies, Aiming to Rejuvenate Aging Cells
The trial will assess the safety and tolerability of ER‑100, an epigenetic restoration therapy designed to restore retinal ganglion cell function, while also measuring visual outcomes.
Key Points
- Life Biosciences has dosed the first participant in a Phase 1 trial of ER‑100.
- The investigational therapy is being developed for open-angle glaucoma and non-arteritic anterior ischemic optic neuropathy.
- The trial will evaluate safety and tolerability, with additional endpoints assessing visual function.
Life Biosciences has dosed the first participant in a Phase 1 trial of ER‑100 for optic neuropathies. The investigational therapy is being developed for open-angle glaucoma (OAG) and non-arteritic anterior ischemic optic neuropathy (NAION).
What is ER-100?
ER‑100 is the first clinical candidate from the company’s Epigenetic Restoration platform. It uses controlled expression of three transcription factors, OCT4, SOX2 and KLF4, collectively known as OSK. The therapy is designed to reset cellular gene expression patterns and restore function in retinal ganglion cells.
Life Biosciences reports that controlled OSK expression improved tissue performance and restored visual function in animal models, but this will be the first test in humans.
“This is an important moment for Life Bio and for the field of aging biology,” said David Sinclair, Ph.D., Co‑founder of Life Biosciences and Professor of Genetics at Harvard Medical School. “Our research has suggested that aging is driven in large part by the loss of epigenetic information, not irreversible damage. This clinical study represents the first opportunity to test whether restoring that information can ameliorate human disease.”
Why Optic Neuropathies Are the Initial Target
Optic neuropathies damage retinal ganglion cells, the neurons that connect the eye to the brain. Because these cells do not naturally regenerate, the damage can cause permanent vision impairment.
Current glaucoma treatments primarily address risk factors such as intraocular pressure. They do not directly repair retinal ganglion cell damage, and vision loss can continue despite treatment. Life Biosciences is also developing potential applications of its Epigenetic Restoration platform for other organs and indications.
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