FDA Greenlights First Human Trial of Epigenetic ‘Rejuvenation’ Therapy for Vision Loss

The U.S. Food and Drug Administration has cleared Life Biosciences’ Investigational New Drug application for ER-100, allowing the Boston-based biotechnology company to begin a Phase 1 clinical trial in patients with open-angle glaucoma and non-arteritic anterior ischemic optic neuropathy (NAION).

Life Biosciences, co-founded by Harvard geneticist David Sinclair, announced the clearance today, marking the first time a therapy based on partial epigenetic reprogramming has advanced into human testing.

The upcoming study will evaluate the safety, tolerability, immune response and preliminary visual outcomes of ER-100, an investigational therapy designed to restore aged or damaged retinal cells to a more youthful functional state. The trial will enroll patients with two optic neuropathies characterized by the irreversible loss of retinal ganglion cells, which connect the eye to the brain.

ER-100 is based on research stemming from Nobel Prize-winning discoveries that showed mature cells can be reprogrammed toward a younger state. Rather than fully reverting cells into stem cells, the therapy enables controlled expression of three Yamanaka transcription factors (OCT4, SOX2, and KLF4) to partially reset epigenetic markers associated with aging.

The company’s platform was developed through research refined in academic laboratories, including work associated with Harvard. In preclinical studies, including nonhuman primate models, Life Biosciences reported restoration of youthful DNA methylation patterns and improvements in visual function following intravitreal injection.

The decision to begin in ophthalmology is deliberate. The eye provides a contained environment for gene delivery and allows for quantifiable measures of function, offering a defined clinical setting before potential expansion into additional age-related diseases.

Open-angle glaucoma is a leading cause of blindness worldwide and can progress despite pressure-lowering treatment. NAION, often referred to as a “stroke of the eye,” causes sudden vision loss and currently has no approved therapies.

The Phase 1 trial represents an early-stage evaluation, but it moves cellular rejuvenation from preclinical research into regulated human testing, a step long anticipated in the longevity research field.