Experimentation

Ageing: cell-rejuvenating therapy administered for the first time

Life Biosciences has announced that it has administered its partial epigenetic reprogramming therapy to the very first human

by Francesca Cerati

Young scientists conducting research investigations in a medical laboratory, a researcher in the foreground is using a microscope StockPhotoPro - stock.adobe.com

3' min read

Translated by AI
Versione italiana

Key points

  • Experts' warnings

3' min read

Translated by AI
Versione italiana

A patient with glaucoma walks into a clinic, receives an injection in the eye and starts taking an antibiotic. We do not know his name. We know that he has agreed to be the first human in history to receive a treatment designed to rejuvenate his cells and that, from this moment on, he carries in his eye three genes capable, at least in mice and monkeys, of reversing cellular ageing. Nothing extraordinary, on the surface. Everything extraordinary, in essence.

Although this is a Phase 1 clinical trial – registered with the US National Institutes of Health (NIH), with a precise protocol and measurable objectives – what is being tested touches on one of medicine’s oldest dreams: making old cells young again.

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The Boston-based biotechnology company Life Biosciences has treated the first participant in a clinical trial using a gene therapy – known as ER-100 – in an attempt to reverse the damage to the optic nerve caused by glaucoma. The mechanism is based on the activation of three genes that appear to be capable of ‘partially reprogramming’ aged cells, causing them to behave as if they were younger.

Medicina rigenerativa, "Corpo autoguarisce, risultati sorprendenti"

An idea that originated in *Nature* in 2020

The story of ER-100 began in a laboratory at Harvard Medical School. In 2020, the team led by Professor David Sinclair – co-founder of Life Biosciences – published a groundbreaking study in the journal *Nature*: by activating just three of the four so-called ‘Yamanaka factors’ (Oct4, Sox2 and Klf4) in mice with damaged optic nerves, the researchers succeeded in promoting neuronal regeneration and reversing vision loss. The study made the cover of Nature and sparked global interest in partial epigenetic reprogramming as a basis for new gene therapies.

Since that publication, Life Biosciences has spent years working to translate those findings into a drug suitable for human use. In 2024, the company presented new data at the annual congress of the American Association of Ophthalmology in Chicago, confirming the results in non-human primates and expanding knowledge on dosage and treatment timing. On 28 January 2026, the FDA authorised the start of clinical trials, paving the way for the only cell rejuvenation therapy using epigenetic reprogramming ever to reach human subjects.

How it works

The therapy uses a modified virus – an AAV vector, commonly used in gene therapies – to deliver the genetic instructions for producing the three proteins directly into the retinal ganglion cells, the cells whose extensions form the optic nerve. The virus has been engineered so that it cannot cause infectious diseases. Once injected, the genes remain dormant until the patient takes doxycycline orally for 56 days, which acts as a ‘switch’ to activate them. If the drug is stopped, the genes switch off. A control system designed specifically to minimise risks.

The decision to exclude Yamanaka’s fourth factor, c-Myc – which is associated with uncontrolled cell growth – is deliberate: it serves to reduce the risk of tumours, which has historically held back this line of research.

What is being tested and why the eye

The Phase 1 study will assess the safety and tolerability of a single dose of ER-100 in adults with open-angle glaucoma and NAION (non-arteritic anterior ischaemic optic neuropathy), a rarer, acute condition that also causes damage to the optic nerve. Up to 12 participants will be treated with two different dose levels and followed for 5 years. The primary objective is not, at least for now, to rejuvenate the entire body, but to demonstrate that the approach is safe and that the neurons of the optic nerve – which do not normally regenerate in adults – can be induced to do so. The eye was also chosen for strategic safety reasons: any adverse effects would remain localised, without potentially life-threatening systemic consequences.

Experts' warnings

Despite the enthusiasm, the scientific community is urging caution. “Gene editing has great potential if it can be used safely in humans,” says Matt Kaeberlein, co-founder of Optispan, a Seattle-based preventive medicine company focused on longevity. “But the technology is still in its infancy and the risk of catastrophic side effects is high.”

In fact, if this initial mechanism is confirmed to be safe, it could revolutionise the way medicine approaches ageing.

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