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Epigenetic Reprogramming Gene Therapy Shows Early Vision Gains in First Human Glaucoma TrialLongevity & Aging

Epigenetic Reprogramming Gene Therapy Shows Early Vision Gains in First Human Glaucoma Trial

Life Biosciences has released interim data from the first human trial of partial epigenetic reprogramming. The therapy, ER-100, is a single injection into the eye that delivers three reprogramming genes (OCT4, SOX2 and KLF4, known as OSK). Daily doxycycline pills switch the genes on for 56 days. Three people with open-angle glaucoma, a leading cause of blindness in older adults, received the lowest dose. The treatment was well tolerated, with no dose-limiting toxicities or serious adverse events. Two of the three participants showed improved visual field scores at Day 56. Safety reviewers cleared the trial to move to a higher dose. The trial is a small, open-label Phase 1 safety study, so the vision signals are preliminary and could reflect chance or other factors. Still, it is the first human test of an idea long studied in animals: resetting the age-related epigenetic changes that damage cells, potentially restoring function that was thought to be permanently lost.

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