Life Biosciences has dosed the first patient in a Phase 1 clinical trial for ER-100, an investigational gene therapy for optic neuropathies. The trial, registered as NCT07290244, will assess the safety and tolerability of this approach, which uses three Yamanaka factors to reset cellular gene expression patterns.
This video is an industry news summary and is not medical advice. The treatments it describes are not offered at this clinic.
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A gene therapy candidate called ER-100 has entered human testing. Here is exactly what has been confirmed so far. ER-100 comes from the company Life Biosciences. Its approach is called partial epigenetic reprogramming. It uses three of the four Yamanaka factors: OCT4, SOX2 and KLF4. The fourth one, c-Myc, is deliberately left out. On 28 January 2026, the U.S. FDA cleared the Investigational New Drug application for ER-100. The first participant was dosed on 9 June 2026. The study is registered as NCT07290244. The trial enrolls two eye conditions: open-angle glaucoma, and non-arteritic anterior ischemic optic neuropathy. This is a Phase 1 study. Phase 1 asks one question first: is it safe in people. It is not a measurement of benefit. ER-100 is not approved, is not available for purchase, and is not offered at this clinic. This is an industry news summary, not medical advice.
Why start with eye diseases?
The Phase 1 trial targets two specific optic neuropathies: open-angle glaucoma and non-arteritic anterior ischemic optic neuropathy (NAION). These conditions represent areas of significant unmet medical need where retinal ganglion cells are damaged.
How is it supposed to work?
ER-100 is designed to deliver three specific transcription factors—OCT4, SOX2, and KLF4 (collectively OSK)—into retinal ganglion cells using an AAV vector via an intravitreal injection. The design deliberately excludes the fourth Yamanaka factor, c-Myc. The therapy incorporates a doxycycline-inducible switch mechanism, where OSK expression is activated when a patient takes low-dose doxycycline and turns off when the antibiotic is stopped. The goal is to reset cellular gene expression patterns.
What stage is ER-100 at right now?
Regulatory clearance for the Phase 1 trial was granted on January 28, 2026 (see the company announcement in Sources). The first patient was dosed on June 9, 2026. This trial is primarily designed to evaluate the safety and tolerability of ER-100.
What does this mean for you?
Stage: Phase 1 · Where: the United States · Can you book it? No — this therapy is not offered at this clinic.
The trial is registered on the U.S. clinical trials database (NCT07290244). This does not mean the therapy is approved, proven effective, or available for booking, purchase, or use.
Sources
- Life Biosciences company announcement on first patient dosing
- ClinicalTrials.gov entry for NCT07290244
- Life Biosciences press releases (includes the IND clearance announcement, January 28, 2026)
This article is an industry news summary for general information. It is not medical advice, and the therapies described here are not offered at this clinic.
