Is it possible to rejuvenate damaged human cells? US biotech firm tests new gene therapy
For the first time, a human being has received a treatment designed to reverse cellular ageing. Scientists at Boston-based American biotechnology company Life Biosciences have developed a gene therapy aimed...
For the first time, a human being has received a treatment designed to reverse cellular ageing.
Scientists at Boston-based American biotechnology company Life Biosciences have developed a gene therapy aimed at restoring old or damaged cells in human patients.
Their therapy, known as ER-100 (AAV2-OSK), aims to target conditions characterised by damage in the optic nerve, known as optic neuropathies.
They use the so-called OSK factors or three proteins — Oct4, Sox2, Klf4 — to perform partial epigenetic reprogramming, resetting cellular age and restoring function.
After being tested in rodents and primates, the company announced that the first human has now received the treatment.
“This is the first-ever epigenetic restoration candidate approved for clinical trials, and if successful, treatment with ER-100 would be the first time cells have been rejuvenated in humans,” the company said in a press release.
How does the therapy work?
The three proteins used in the OSK gene therapy act as a reset button on cells, restoring them to a younger state.
A person’s DNA sequence remains mostly stable throughout life, even as the body ages. However, the epigenetic code — which controls which genes are turned on or off — changes over time.
These alterations can stem from lifestyle factors such as smoking or alcohol consumption, ageing, disease and injury — and can lead to harmful outcomes including cancer or neurological disorders.
The Life Biosciences therapy targets these changes by delivering a set of genetic instructions directly, with the three OSK proteins working as an “on” switch for the targeted cells and resetting the harmful changes built up over time.
This approach has been used in biology to turn ordinary cells back into stem cells, a discovery that earned Sir John B. Gurdon and Shinya Yamanaka the 2012 Nobel Prize in Physiology or Medicine.
What conditions does it target?
The therapy has now entered the first phase of clinical trials, in which scientists assess whether it is safe for humans.
“This is an important moment for Life Bio and for the field of ageing biology,” said David Sinclair, co‑founder of Life Biosciences and professor of genetics at Harvard Medical School.
“Our research has suggested that ageing is driven in large part by the loss of epigenetic information, not irreversible damage. This clinical study represents the first opportunity to test whether restoring that information can ameliorate human disease.”
The Phase 1 first-in-human study will enroll individuals with open-angle glaucoma (OAG) and non-arteritic anterior ischemic optic neuropathy (NAION), two serious eye diseases in which patients lose sight.
Open-angle glaucoma is a chronic, progressive eye disease in which the eye's drainage system clogs, leading to fluid buildup and raised eye pressure. Vision loss typically begins with subtle changes in side vision and progresses slowly over time.
NAION is often described as a mini-stroke of the optic nerve. It is a sudden condition caused by reduced blood flow to the back of the eye. Vision loss usually occurs quickly, is often noticed upon waking, and typically affects one eye.
What are the next steps?
Beyond ER‑100, Life Biosciences said it is developing applications for multiple indications in a variety of organs, reflecting the broad therapeutic potential. The company is already testing a second therapy for liver disease and is working to deliver its cell-resetting approach to other organs in the body.
Life Biosciences is not the only company exploring so-called Yamanaka factors to extend life expectancy, reverse ageing or address disease.
While no therapeutic uses have yet been cleared for these therapies, there is increased interest and investment in the field.
Retro Biosciences, an American company backed by OpenAI’s Sam Altman, is working towards a clear mission: adding ten healthy years to the human lifespan by developing therapies to reverse age-related diseases.
The biotechnology company Shift Bioscience, based in Cambridge in the United Kingdom, is also using the same OSK mechanism to target the underlying cause of age-driven diseases.
