A biotechnology company has launched a first-of-its-kind human trial for a drug designed to turn back aspects of aging at the cellular level.
The experimental therapy, known as ER-100, comes from Boston-based Life Biosciences. In June 2026, the company said the first participant had been dosed in its Phase 1 study, after the U.S. Food and Drug Administration cleared the investigational new drug application earlier in the year. It is the first cellular rejuvenation therapy using partial epigenetic reprogramming to reach human clinical trials.
Roughly 18 adults are expected to take part in the study over the coming year, with the initial trial focused on whether the drug can be given safely and without severe side effects, along with early signals about visual function.
Earlier animal research, according to the company, showed restored vision in monkeys. The next step is finding out whether those results can translate to human patients.
The basic idea is this: cells contain instructions that regulate how they function, and the pattern of which instructions are switched on or off changes with age.
Life Biosciences argues that aging is driven in large part by losing access to that cellular information, rather than by irreversible harm to DNA itself.
ER-100 uses three proteins collectively called OSK in an effort to restore those earlier instructions, effectively pushing older cells toward a more youthful and functional condition.
David Sinclair, the Harvard Medical School genetics professor who co-founded Life Biosciences, said the study is a major milestone for the field.
“Our research has suggested that aging is driven in large part by the loss of epigenetic information, not irreversible damage,” he said.
“This clinical study represents the first opportunity to test whether restoring that information can ameliorate human disease.”
The first target for the treatment is the eye.
Researchers are enrolling patients with glaucoma and a rare disorder known as NAION, or non-arteritic anterior ischemic optic neuropathy. Both conditions damage retinal ganglion cells, the nerve cells at the back of the eye that send visual signals to the brain. When those cells are lost, the resulting vision damage is often permanent because they do not naturally regenerate. ER-100 is intended to revive those cells rather than simply slow further decline.
Life Biosciences CEO Jerry McLaughlin explained why the company chose eye disease as its starting point.
“We started with optic neuropathies because the biology is clear and the need is real,” he said, pointing out that current glaucoma treatments only manage risk factors like eye pressure, while NAION has no approved treatment at all.
Still, the company sees potential well beyond vision loss.
McLaughlin said researchers are already investigating whether the same approach could be used in liver disease, and added that the platform may one day have uses “across a variety of organs, because the biology of aging is relevant in many tissues”.
He also stressed that any broader rollout will depend on evidence from the trial. “Each next step must be earned with data rather than promised ahead of it,” he said.
That caution is notable in a sector already filled with big claims. McLaughlin said Life Biosciences wants the treatment assessed on research outcomes rather than publicity. “We’re not making claims about lifespan or selling supplements,” he said.
“We’re targeting a root cause of age-related diseases at the epigenetic level and asking to be judged on clinical data.”
Some scientists remain cautious about how well the risks are understood at this stage.
Speaking to Nature, Matt Kaeberlein, co-founder of longevity-focused company Optispan, said the science is still ‘really early’ and warned that ‘the potential for catastrophic side effects is high’ if the approach isn’t proven safe in humans, though he agreed the eye was a sensible place to start given the lower risk of life-threatening complications.
Pete Williams, a translational neurobiologist at the Centre for Eye Research Australia, went further, telling Nature that even if the trial succeeds, it wouldn’t necessarily prove the treated cells are truly ‘younger’ in any meaningful sense. He also flagged concerns about the level of public hype surrounding the treatment, saying, ‘if this goes catastrophically wrong, it might screw us all in the future’.
For now, attention is fixed on the outcome of this first human study. If ER-100 performs in people the way it reportedly did in monkeys, it may become the first strong sign that reversing aspects of aging in humans is moving out of theory and into reality.