U.S. biotech firm begins first human test of gene therapy for cellular ageing
A Boston-based biotechnology company has started the first clinical trial of a gene therapy intended to reverse cellular ageing. The treatment, called ER-100, targets optic nerve damage and has moved into Phase 1 safety testing.
EuronewsA U.S. biotechnology company has administered a gene therapy designed to reverse cellular ageing to a human patient for the first time. The therapy, known as ER-100 (AAV2-OSK), uses three proteins—Oct4, Sox2, and Klf4—to perform partial epigenetic reprogramming.
It aims to reset cells affected by optic neuropathies, conditions that damage the optic nerve. The company said the treatment had previously been tested in rodents and primates before receiving approval for human trials.
Background on the approach The three proteins act on the epigenetic code that controls which genes are active. This code changes over time due to ageing, disease, injury, or lifestyle factors such as smoking and alcohol use. The therapy delivers genetic instructions that turn the proteins on in targeted cells, aiming to restore a younger state without altering the underlying DNA sequence.
The same OSK factors were used in earlier laboratory work that earned the 2012 Nobel Prize in Physiology or Medicine.
Trial details and next steps The Phase 1 study will enroll patients with open-angle glaucoma and non-arteritic anterior ischemic optic neuropathy. Open-angle glaucoma involves gradual fluid buildup and vision loss, while the second condition causes sudden vision loss from reduced blood flow to the optic nerve.
The company stated that ER-100 is the first epigenetic restoration candidate approved for clinical trials. Beyond the eye, the company is developing applications for other organs and is already testing a second therapy for liver disease. Other firms are also exploring similar reprogramming methods, though no therapeutic uses have been cleared.
