For decades, the idea of a drug that could rewind aspects of human aging has belonged more to science fiction than hospital formularies. That boundary is starting to blur as a gene therapy called ER-100 moves from lab benches into a first human clinical trial, with scientists testing whether a carefully controlled “rejuvenation” signal can repair damaged eye tissue and, eventually, much more.
The experimental treatment, developed by Life Biosciences and rooted in a strategy known as Partial Epigenetic Reprogramming, is being positioned as a potential way to restore function in cells that have grown old or been injured. While the initial focus is on blinding optic neuropathies, the underlying approach is explicitly framed as a bid to reverse cellular aging itself rather than simply slow it.
From longevity theory to ER-100 in the clinic
The scientific story behind ER-100 starts with the idea that aging is, at least in part, an information problem inside our cells. Researchers at Life Biosciences, co-founded by Harvard scientist David Sinclair, have argued that cells accumulate epigenetic noise as we age, which disrupts how genes are switched on and off. Their answer is Partial Epigenetic Reprogramming, or PER, which uses specific factors to reset those epigenetic marks just enough to restore youthful function without erasing a cell’s identity. ER-100 originated from this PER platform, which is designed to restore aged or injured cells to a healthier state without altering the underlying DNA sequence.
That concept has now crossed a major regulatory threshold. In Jan, Life Bio secured clearance of an IND, or Investigational New Drug application, for ER-100 in a program targeting Optic Neuropathies, formally recorded as ER-100. A companion announcement described how Life Biosciences Announces FDA Clearance of IND Application for ER, positioning the therapy as a potential way to improve multiple visual assessments in patients with severe eye disease. The company and its scientific leadership have emphasized that this is not cosmetic “hyper-beautification” but an attempt to reverse the cellular hallmarks of Age in tissues where degeneration has devastating consequences.
What ER-100 actually does inside the eye
At its core, ER-100 is a gene therapy that delivers instructions into cells so they briefly express a set of reprogramming factors, then shut them off again. The approach uses a controlled form of epigenetic reprogramming that aims to rejuvenate retinal ganglion cells and other neurons affected by optic nerve damage, without pushing them all the way back to an embryonic state. According to Life Biosciences Secures, ER-100 is described as The First Cellular Rejuvenation Therapy Using Epigenetic Reprogramming To Enter clinical testing, with the goal of treating conditions that cause vision loss by restoring cellular resilience rather than simply protecting what remains.
To keep that rejuvenation signal from running out of control, the system is designed to be tightly regulated. Reporting on similar PER-based therapies has highlighted how the reprogramming cassette can be switched on only when patients receive a specific oral drug, and then turned off again once the desired effect is reached. One analysis of these age-reversal strategies noted that to stop the ageing reversal from going too far, the therapy is only activated when patients are given particular doses of a companion compound, a safeguard described in coverage of drugs that can. In the eye, that kind of on-off control is especially important, because over-reprogramming neurons could risk losing their specialized function and, paradoxically, worsening vision.
The first ER-100 patients and what the trial will test
The initial human test of ER-100 is deliberately narrow, focusing on people who already face serious vision loss. The Study Overview on the official registry describes a clinical trial whose goal is to evaluate the safety and tolerability of a single dose of ER-100 in adult participants with glaucoma or non-arteritic anterior ischemic optic neuropathy. That Study Overview frames the work as a Phase I clinical trial, with ER-100 administered once and patients followed closely for adverse events and early signals of benefit.
Additional reporting on the program explains that The Phase I trial will recruit participants who have been diagnosed with non-arteritic anterior ischaemic optic neuropathy and open-angle glaucoma, two conditions that damage the optic nerve and for which current treatments are limited. The same coverage notes that The Phase I trial will also explore how ER-100 might eventually help people affected by these conditions beyond simple symptom management, by looking at structural and functional measures of the optic nerve. Those details, drawn from The Phase, underscore that the first priority is safety, but the design is already probing whether a single exposure to this reprogramming signal can nudge damaged neurons toward recovery.
The people and platform behind the “age reversal” push
Behind ER-100 is a network of scientists and entrepreneurs who have spent years arguing that aging itself should be treated as a modifiable process. Harvard University researcher David Sinclair has been one of the most visible advocates of this view, and he has now tied that vision directly to the clinic. In Jan, he stated that his biotech startup, Life Biosciences, had received FDA clearance to begin the first human clinical trial of this rejuvenation approach, a milestone shared in a Harvard post that highlighted the FDA’s role in greenlighting the experiment. Separate coverage described how, earlier this year, a Harvard University researcher David Sinclair said aging can be reversed and that Now a startup is ready to try his idea in human volunteers, a framing captured in analysis of age reversal trials.
Life Biosciences itself has been steadily building the PER platform beyond the eye. Company materials describe Life Bio as a clinical-stage biotechnology company pioneering cellular rejuvenation therapies to reverse aging processes by resetting epigenetic marks without altering the underlying DNA sequence, a mission summarized in the corporate section titled About Life Biosciences. Earlier profiles have noted that the Boston headquarters of Life Biosciences anchor a broader push to use gene therapy to restore tissue function, with the company aiming to restore and prolong a patient’s health span by targeting fundamental aging mechanisms, as described in an overview of Using gene therapy to live longer, healthier lives. Another report on the Boston base of Life Biosciences emphasized that the company plans 2026 human trials for gene therapy that could reverse cellular aging and restore tissue function, situating ER-100 within a pipeline that includes other candidates and ambitions beyond ophthalmology, as detailed in coverage of Boston.
Beyond the eye: what ER-100 hints about future anti-aging drugs
Although ER-100 is the first PER-based therapy to reach human testing, it is not the only candidate in Life Biosciences’ rejuvenation arsenal. In preclinical work, the company has reported that another program, ER-300, significantly improved multiple aspects of liver health in a mouse model of metabolic dysfunction-associated steatohepatitis, often abbreviated as MASH, using a GAN DIO-MASH system. Those findings, presented as part of the company’s broader PER platform in liver and ocular diseases, were summarized in a corporate update that highlighted ER-300 and its Key data, including the specific reference to 300 and the MASH, GAN, DIO model. The implication is that the same reprogramming logic being tested in the eye could eventually be applied to organs like the liver, where age-related decline and chronic disease intersect.
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