Life Biosciences has dosed the first participant in a Phase 1 clinical trial of ER-100, its lead epigenetic restoration therapy for optic neuropathies including open-angle glaucoma and non-arteritic anterior ischemic optic neuropathy (NAION).
The Boston-based biotechnology company said the trial will evaluate the safety and tolerability of ER-100, with additional endpoints assessing visual function. ER-100 is the first clinical candidate from Life Biosciences’ Epigenetic Restoration platform, which uses controlled expression of three transcription factors – OCT4, SOX2, and KLF4 (OSK) – to reset cellular gene expression patterns associated with younger, healthier function.
The company is initially targeting optic neuropathies, a group of disorders involving damage to retinal ganglion cells, the neurons that connect the eye to the brain. Because these cells do not naturally regenerate, damage can lead to permanent vision loss.
Current treatments for open-angle glaucoma primarily focus on reducing risk factors such as intraocular pressure, but do not directly address retinal ganglion cell damage. NAION, the most common acute optic neuropathy in adults over 50, currently has no approved treatments.
David Sinclair, co-founder of Life Biosciences and professor of genetics at Harvard Medical School, said the study marks the first opportunity to test whether restoring epigenetic information can affect human disease. “This is an important moment for Life Bio and for the field of aging biology,” he added. “Our research has suggested that aging is driven in large part by the loss of epigenetic information, not irreversible damage.”
Chief Scientific Officer Sharon Rosenzweig-Lipson said preclinical studies have shown that controlled OSK expression can reset epigenetic patterns, improve tissue performance, and restore visual function in animal models. She added: “Advancing ER‑100 into the clinic is an important step toward translating epigenetic restoration into a new class of medicines for age-related diseases.”
Beyond ER-100, Life Biosciences is exploring its epigenetic restoration platform across additional age-related diseases.
Source: Global Newswire.
