June 09, 2026 —
Life Biosciences announced that the first participant has been dosed in a Phase 1 clinical trial of ER-100, an investigational AAV2-based gene therapy being developed for optic neuropathies, including open-angle glaucoma and non-arteritic anterior ischemic optic neuropathy, or NAION.
ER-100 is the first clinical candidate from Life Bio’s Epigenetic Restoration platform, which is designed to restore cellular function by resetting age-associated epigenetic patterns. The therapy uses an AAV2 vector to deliver controlled expression of three transcription factors — OCT4, SOX2, and KLF4, collectively known as OSK — to shift cells toward more youthful patterns of gene expression while preserving their identity and function.
The Phase 1 study will primarily evaluate the safety and tolerability of ER-100, with additional endpoints assessing visual function. The AAV2 therapy is designed to target retinal ganglion cells, the neurons that connect the eye to the brain and are damaged in optic neuropathies. Because retinal ganglion cells do not naturally regenerate, damage can lead to permanent vision loss.
Open-angle glaucoma is a chronic neurodegenerative eye disease and one of the leading causes of blindness in older adults. Existing treatments primarily focus on reducing intraocular pressure, but many patients continue to experience disease progression despite therapy. NAION is the most common acute optic neuropathy in adults over 50 and currently has no approved treatment.
Life Bio’s approach is based on the concept of partial cellular reprogramming, which uses a subset of Yamanaka factors to restore aspects of youthful cellular function without fully reverting cells to a stem-cell-like state. Earlier preclinical work led by David Sinclair and colleagues showed that OSK expression could promote optic nerve regeneration and vision recovery in aged and glaucoma-model mice.
A key feature of ER-100 is its controllable expression system. According to the company, the AAV2-delivered therapy is designed so that OSK expression can be activated by doxycycline, providing an additional safety mechanism to help regulate gene activity and reduce the risk of excessive expression.
The first-in-human dosing represents an important milestone for both AAV-based ocular gene therapy and the emerging field of cellular rejuvenation medicine. While the program remains early and safety will be a major focus, ER-100 is among the first clinical attempts to test whether AAV-delivered epigenetic restoration can be translated into a disease-modifying therapy for age-related human disease.
Beyond ER-100, Life Biosciences is developing additional applications of its Epigenetic Restoration platform across multiple organs and age-related diseases. For now, the company is focused on treating specific diseases of aging rather than whole-body rejuvenation.
