Age-reversal gene therapy moves from mouse eyes to human trial

Yuancheng (Ryan) Lu, a geneticist at the Whitehead Institute, is focused on gene therapies that could prevent age-related vision loss. His interest is personal: age-related blindness runs in his family, and a 23andMe test showed he carries a mutation linked to macular degeneration.

While earning his PhD at Harvard Medical School in 2018, Lu used a reprogramming technique to repair crushed optic nerves in mice. The method relied on three genes, OSK, adapted from the broader OSKM process first demonstrated by Japanese researchers in 2006. By omitting Myc, the gene most associated with dangerous changes such as cancer, Lu sought to make the rejuvenation effect more controlled and suitable for testing in the eye.

Life Biosciences, cofounded by longevity scientist David Sinclair, has now moved a closely related therapy, ER-100, into human testing. On June 9, the company said it had injected the treatment into the eye of a person with glaucoma, marking a major step for a technology that began as Lu’s student work.

Lu remains cautious about the broader promise of age reversal. He sees OSK as a proof of concept rather than a cure-all, noting that the treatment can be toxic to many cell types and that aging appears to be driven by different factors in different tissues.