Life Biosciences reports first human data for OSK gene therapy ER-100 in glaucoma: three participants

Life Biosciences, a Boston company developing partial-reprogramming therapies, has released the first human data from its phase 1 trial of ER-100. The company says a single injection, with a course of the antibiotic doxycycline to switch the therapy on, was well tolerated in three people with open-angle glaucoma. This is an early safety update on a handful of people, issued by the company itself, and it should be read as such.

What the researchers did

ER-100 is an investigational gene therapy. According to the company, it delivers three transcription factors (OCT4, SOX2 and KLF4, together called OSK) to retinal cells using a modified virus (AAV) injected into the eye. OSK production is switched on by doxycycline taken by mouth for 8 weeks (56 days), and an oral prednisolone course is tapered to limit inflammation in response to the viral vector. Life Bio describes its approach as “epigenetic restoration”.

The trial (ClinicalTrials.gov NCT07290244) is a first-in-human, open-label, non-randomized phase 1 study. Its registry entry lists a start date of March 2, 2026 and a planned enrollment of up to 18 adults aged 40 to 85: 12 with open-angle glaucoma and 6 with non-arteritic anterior ischemic optic neuropathy (NAION), a sudden form of optic nerve damage. The registered primary outcomes concern safety and tolerability. Participants are to be followed for up to five years.

The press release, dated October 8, 2026, reports interim data from the first three glaucoma participants, who received the lowest dose level (2×10¹¹ vector genomes).

What they found

All figures below are as reported by the company.

• Safety: the therapy plus daily doxycycline was “well-tolerated” through Day 56 in all three participants. No dose-limiting toxicities, adverse events of special interest or serious adverse events were reported. The company says all reported adverse events were related to the procedure, the steroid or pre-existing conditions, and not directly to ER-100.
• Dose escalation: the independent Data Safety Monitoring Board recommended moving to Dose Level 2 (6×10¹¹ vector genomes) in glaucoma participants, and the trial is ongoing.
• Vision test results: improvements on Humphrey visual field testing were seen in 2 of 3 participants at Day 56. In each of those two, at least 28% of tested points improved by more than 4 decibels.

The release does not give individual baseline values or a statistical analysis, and it does not describe the visual-field result for the third participant.

What this means

The registry describes this as the first administration of ER-100 in humans, following preclinical evaluation in laboratory models. The update is therefore an early look at how a reprogramming-style therapy behaves in people, and the company reports that the independent safety board recommended moving to a higher dose. Because the trial is designed to assess safety and tolerability, the visual-field observations are exploratory. The company itself describes them as “preliminary signals”.

Limits of the evidence

• Three people. The interim data cover three participants at one dose level.
• No control group. The trial is open-label and uncontrolled, so changes in vision tests cannot be attributed to ER-100. Participants and clinicians knew what was given.
• Short follow-up. The data run to Day 56 of a trial planned to follow people for up to five years.
• Company-reported and not peer-reviewed. We found no independent publication of these data. This is a press release from the trial sponsor, and we have not seen the underlying results.
• Early-stage investigational therapy. ER-100 is not approved for any use, and the registry lists four recruiting sites, all in the United States.
• Eye-specific. Results in the eye say little about reprogramming approaches in other tissues, or about aging in general.

Sources

• Life Biosciences. Life Biosciences Announces First-in-Human Data from Ongoing Phase 1 Trial Evaluating ER-100 in Optic Neuropathies. Press release, October 8, 2026 (opened for this article).
• Trial registry: ClinicalTrials.gov NCT07290244 (opened for this article).

This article is for information only and is not medical advice. Speak to a healthcare professional before changing medication or supplements.