Boston biotech begins first human trial of gene therapy aimed at reversing cellular aging in glaucoma patients

Life Biosciences, a Boston-based biotechnology company, administered the first dose of its experimental gene therapy ER-100 to a patient with open-angle glaucoma on June 9. The Phase 1 trial seeks to restore function to the optic nerve by delivering three genes—OCT4, SOX2 and KLF4 (collectively OSK)—that can revert adult cells to a stem-like state. The approach builds on earlier animal research that showed partial visual improvement but also highlighted a risk of tumor formation if the genes are over-expressed.

"Our research has suggested that aging is driven in large part by the loss of epigenetic information, not irreversible damage," said David Sinclair, a genetics professor at Harvard Medical School and co-founder of Life Biosciences, in a press release.

"Advancing ER-100 into the clinic is an important step toward translating epigenetic restoration into a new class of medicines for age-related diseases," added Sharon Rosenzweig-Lipson, chief scientific officer at the company.

Current glaucoma treatments can only halt further vision loss; they do not restore sight. By targeting the eye, which is not essential for survival, researchers hope to evaluate the therapy’s safety before considering applications to other tissues. The trial will closely monitor participants for signs of uncontrolled cell growth, a known concern from prior mouse studies.

If successful, the therapy could represent a new strategy for treating age-related ocular diseases, though experts caution that extensive testing is required to confirm both efficacy and safety.