BOSTON — October 8, 2026
Life Biosciences announced interim first-in-human data from its ongoing Phase 1 clinical trial of ER-100, an investigational gene therapy for optic neuropathies, including open-angle glaucoma (OAG). In the initial evaluation, a single intravitreal administration of ER-100, combined with daily oral doxycycline, was well tolerated through Day 56 in three participants with OAG. The Data Safety Monitoring Board recommended escalating to the next dose level.
Safety and Tolerability Findings
At the initial dose level of 2 × 10¹¹ vector genomes, no dose-limiting toxicities, adverse events of special interest, or serious adverse events were reported among the three participants. Reported adverse events were attributed to the procedure, steroid treatment, or pre-existing conditions rather than directly to ER-100. The ongoing study will evaluate a higher dose of 6 × 10¹¹ vector genomes in the next dose level.
Preliminary Visual Field Observations
Visual field testing showed preliminary improvements in two of the three participants at Day 56. In each participant, at least 28% of measured visual field points demonstrated an improvement greater than 4 decibels. These findings are early exploratory observations from a small, open-label Phase 1 study designed primarily to assess safety and tolerability; they do not establish clinical efficacy or confirm sustained vision improvement.
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Epigenetic Restoration Approach
ER-100 uses Life Biosciences’ Epigenetic Restoration platform, delivering the transcription factors OCT4, SOX2, and KLF4 (OSK) through an intravitreal injection, with expression induced by oral doxycycline for eight weeks. The investigational therapy is being developed for optic neuropathies, including OAG and non-arteritic anterior ischemic optic neuropathy (NAION). The Phase 1 trial (NCT07290244) is ongoing, and further clinical evaluation will be needed to determine the therapy’s safety and potential benefit.
Source::Life Biosciences,, press release
