Life Biosciences reported interim Phase 1 results for ER-100, an intravitreal gene therapy delivering the OCT4, SOX2 and KLF4 reprogramming factors under doxycycline-inducible control, in three adults with open-angle glaucoma. A single Dose Level 1 administration (2×10^11 vg) was tolerated through Day 56 with no serious adverse events or dose-limiting toxicities, and two of three participants showed improvement on Humphrey visual field testing. The Data Safety Monitoring Board cleared escalation to Dose Level 2, and the trial continues.
Key Points
- Single intravitreal OSK gene therapy tolerated through Day 56
- Two of three glaucoma patients improved on visual field testing
- Safety board cleared escalation to higher dose level
Longevity Analysis
Partial epigenetic reprogramming has moved from rodent optic nerve models into human subjects, testing whether aged neural tissue retains the capacity to recover function rather than merely slow its decline. The eye serves as a contained, measurable proving ground for restoring tissue that the body does not ordinarily replace, with doxycycline-gated expression and a steroid taper used to constrain the risks of uncontrolled reprogramming and local inflammation. Three participants and 56 days of follow-up establish tolerability, not efficacy, and the durability question — whether restored function holds once the inducing agent is withdrawn — remains open.
Original published by LT Wire.