First Human Receives Gene Therapy to Rejuvenate Eye in United States

In June 2026 a Boston‑based biotech firm, Life Biosciences, administered the experimental gene therapy ER‑100 to the first human patient. The therapy, delivered by a modified virus injected directly into the eye, introduces three Yamanaka factors (OCT4, SOX2, KLF4) to reprogram retinal cells and potentially reverse age‑related damage associated with glaucoma.

The volunteer, an older man who had lost vision in one eye due to NAION, is part of a Phase 1 safety trial. The treatment includes a built‑in safety switch that deactivates the virus unless a second drug is taken, aiming to limit risks such as uncontrolled cell growth—an issue observed in earlier mouse studies. Researchers caution that efficacy is still uncertain and that tumors could develop, but preliminary animal data showed restored vision. The trial marks the first human use of this approach, with results expected later in the year.