For the first time in medical history, a human patient has received a treatment explicitly designed to reverse cellular aging. Scientists at Boston-based Life Biosciences have launched a Phase 1 clinical trial for ER-100 (AAV2-OSK), a novel gene therapy targeting optic neuropathies.
The therapy utilizes three specific proteins-Oct4, Sox2, and Klf4, collectively known as OSK factors-to perform partial epigenetic reprogramming. While a person’s DNA sequence remains stable throughout life, the epigenetic code controlling gene expression degrades due to aging, lifestyle, and disease. The OSK factors act as a biological reset button, restoring harmful epigenetic changes to a younger state without altering the underlying genetic code.
This approach builds on the Nobel Prize-winning discovery by Sir John B. Gurdon and Shinya Yamanaka, who demonstrated that mature cells could be reverted to stem cells. Life Biosciences aims to apply this mechanism safely to restore function in damaged tissues.
"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."
The initial study focuses on patients with open-angle glaucoma (OAG) and non-arteritic anterior ischemic optic neuropathy (NAION). OAG involves progressive vision loss from fluid buildup, while NAION is akin to a mini-stroke of the optic nerve causing sudden vision loss.
Beyond ocular conditions, Life Biosciences is developing therapies for liver disease and other organ systems. The sector is seeing increased investment, with competitors like Retro Biosciences, backed by Sam Altman, and UK-based Shift Bioscience also exploring OSK mechanisms to extend healthy human lifespan.