US biotech firm tests gene therapy to reverse cellular aging

A U.S. biotechnology company, Life Biosciences, has administered a gene therapy designed to reverse cellular aging in a human patient for the first time. The experimental treatment, known as ER-100 (AAV2-OSK), utilizes three proteins called OSK factors—Oct4, Sox2, and Klf4—to perform partial epigenetic reprogramming, aiming to reset cellular age and restore function.

This approach targets changes in the epigenetic code, which controls gene activity and is known to alter with age, lifestyle, disease, and injury. These alterations can contribute to various health issues, including cancer and neurological disorders. The ER-100 therapy delivers genetic instructions to switch these harmful changes off, effectively rejuvenating cells.

After successful testing in animal models, the therapy has entered Phase 1 clinical trials. The initial focus is on assessing safety in individuals with serious eye diseases such as open-angle glaucoma (OAG) and non-arteritic anterior ischemic optic neuropathy (NAION). David Sinclair, a co-founder of Life Biosciences, stated that research suggests aging is largely due to the loss of epigenetic information rather than irreversible damage, and this study is the first to test if restoring this information can treat human disease.

Life Biosciences is also developing similar cell-resetting therapies for liver disease and other organs. The company is not alone in this field; Retro Biosciences, backed by Sam Altman, and the UK-based Shift Bioscience are also exploring the use of Yamanaka factors to extend lifespan and combat age-related diseases.