ER‑100 therapy tested for glaucoma, NAION

Life Biosciences is recruiting patients for the first human trial of ER-100, an experimental gene therapy for open-angle glaucoma and non-arteritic anterior ischemic optic neuropathy. (clinicaltrials.gov) (lifebiosciences.com) Both diseases damage retinal ganglion cells, the nerve cells that carry visual signals from the eye to the brain. In glaucoma that damage is often linked to eye pressure; in non-arteritic anterior ischemic optic neuropathy, or NAION, it follows a sudden loss of blood flow to the optic nerve. (lifebiosciences.com) (nature.com) ER-100 is designed to deliver genetic instructions for three proteins — OCT4, SOX2 and KLF4, or OSK — into retinal cells using a modified adeno-associated virus, a common gene-therapy delivery vehicle. The goal is to change chemical controls on genes without changing the underlying DNA sequence. (clinicaltrials.gov) (lifebiosciences.com) The ClinicalTrials.gov record says this is a Phase 1 safety study of a single ER-100 dose, followed by 56 days of systemic doxycycline to switch on OSK expression. Participants will undergo eye exams, lab tests, body-fluid sampling and long-term follow-up for up to five years. (clinicaltrials.gov) The trial is split into two sequential parts: dose escalation in people with open-angle glaucoma, then dose expansion in people with NAION. Life Biosciences said the study will assess safety, tolerability, immune responses and multiple visual assessments. (clinicaltrials.gov) (lifebiosciences.com) This is not a proof-of-efficacy readout yet. The company and the trial registry both describe ER-100 as a first-in-human study focused on whether the treatment can be given safely before any larger test of vision benefit. (clinicaltrials.gov) (nature.com) Life Biosciences said the Food and Drug Administration cleared its investigational new drug application on January 15, 2026, and the company announced that clearance publicly on January 28. Nature Biotechnology described ER-100 as the first cellular rejuvenation therapy to reach human clinical trials. (lifebiosciences.com 1) (lifebiosciences.com 2) (nature.com) The company’s pitch rests on animal data. Life Biosciences has reported that ER-100 restored visual function and increased axon survival in a nonhuman primate model designed to mimic NAION, and earlier work presented with academic collaborators linked OSK treatment to restored vision in mouse glaucoma models. (lifebiosciences.com) (biospace.com) Life Biosciences closed an $80 million Series D on April 8, 2026, and said the money would support the Phase 1 ER-100 trial and other programs from its partial epigenetic reprogramming platform. For now, the news is that a long-discussed lab concept has moved into a recruiting human eye study. (lifebiosciences.com)