The epigenome, the system that helps control which genes are turned on and off in our cells, can become disrupted during aging and disease. Epigenetic restoration is based on the idea that some of those changes may be reversible, and that restoring the epigenome could help cells return to a healthier state to treat a variety of age-related diseases. Life Biosciences (Life Bio) is testing that idea in humans with an epigenetic restoration therapy called ER-100.
Today, at the American Academy of Ophthalmology Annual Meeting, the company announced the first results from its Phase I safety and tolerability trial: all three patients with glaucoma tolerated the treatment without adverse events or problems related to ER-100, and two of the three showed improvements in vision.
ER-100 is a gene therapy that delivers three genes, Oct4, Sox2, and Klf4 (OSK), to the eye. OSK has been shown to slow or reverse features of aging, injury, and disease in laboratory models. Most important to this conversation is that OSK improves vision in mice with glaucoma, a finding that was first published by the laboratory of David Sinclair in Nature, 2020. The work was extended in non-human primates by work from Dr. Bruce Ksander, also at Harvard Medical School, and Dr. Sharon Rosenzweig-Lipson, Chief Scientific Officer of Life Bio, who reported positive data in 2024. Today’s announcement represents the first results applying this approach in humans.
Glaucoma affects over 10% of people over 80 globally. Although treatments for glaucoma exist, current therapies primarily prevent or slow further vision loss rather than restore vision already lost. In Life Bio’s trial, three patients with glaucoma received an injection of ER-100 directly into the eye, a procedure known as an intravitreal injection. They then took a daily medicine called doxycycline to activate the treatment.
The first question in any Phase I clinical trial is whether a treatment is about safety. Does it cause harm? Can patients tolerate it? To begin answering those questions, the study started with a low dose of ER-100. Two hundred billion viral genomes may sound like a lot, but in the context of gene therapy it’s a relatively low dose. The patients who received low-dose ER-100 did not have any dose-limiting adverse events during the 56 days of treatment. Any medical problems that were reported were related to the injection itself, pre-existing conditions, or treatment with prednisolone, a steroid treatment which is commonly used in gene therapy trials to suppress overreactions of the immune system. Researchers do not believe ER-100 caused any of these problems.
So far, the treatment appears to be safe. But did it do anything useful? That is a more difficult question to answer. With only three patients, a low dose, and no control group, it is important to keep expectations in check. This study was designed to test safety, not to determine whether ER-100 could improve vision.
That is what makes the visual results particularly encouraging. Two of the three patients showed improvement on the Humphrey Visual Field test, an eye exam that measures your ability to detect light at multiple points across their field of vision. In those two patients, detection improved at more than 28% of the tested points in the visual field.
In an interview with Lifespan Magazine, Dr. David Sinclair said, “This news represents an encouraging first step in epigenetic age restoration. I look forward to seeing additional data as the safety trial progresses.”
It is important to put these results in context. This was an extremely small study, and the patients were not compared with a control group. The dose was also lower than the dose researchers plan to test in the next group of patients. We cannot directly measure how much ER-100 was active in the cells of these patients’ eyes, because those eyes are still in use. No one should walk away from this announcement thinking that blindness has been cured.
Even so, this is an important moment and big step for the fields of epigenetic restoration, gene therapy, and eye disease. The results offer a small window into an extraordinary possibility: that restoring the epigenome may one day become a viable way to treat human disease.
“We are encouraged by the Data Safety Monitoring Board’s recommendation to escalate to Dose Level 2, a decision that allows us to keep moving this trial forward on behalf of people living with optic neuropathies like glaucoma, a disease where vision loss is often considered irreversible,” said Dr. Rosenzweig-Lipson. “While this Phase I study is designed to evaluate safety and tolerability, the preliminary observations of visual field improvements in two participants are encouraging as we continue to advance the ER-100 program. This milestone marks an important step in Life Bio’s progress translating epigenetic restoration into a new class of medicines for age-related diseases.”
The next group of patients will receive 600 billion viral genomes, three times the dose used in the first group. That trial is ongoing, and the next round of results should help show whether the early safety findings and vision-related improvements hold up at the higher dose. Stay tuned.
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Last Updated: Oct 8, 2026 · 4 minute read
