Life Biosciences just announced they gave their new therapy, called ER-100, to the first person in a Phase 1 clinical trial. The company is trying to treat eye conditions that cause permanent vision loss in aging individuals, like open-angle glaucoma and NAION.

This new drug addresses the problem with current treatments that mostly only manage risk factors. For example, glaucoma medicines target eye pressure and intend to lower the pressure. These medications don’t fix the damage to the retinal ganglion cells, they just manage it.

Retinal ganglion cells are the main neurons that connect your eye to your brain, and they don’t naturally regenerate. When these cells get damaged it leads to lost vision. Moreover, NAION, a condition that causes sudden vision loss from bad blood flow, has no approved treatments at all.

Reversing Age-Related Vision Loss

age related vision loss
The new drug targets age-related vision diseases like glaucoma; Photo: Toa55/Shutterstock

Life Bio’s new platform uses three specific transcription factors: OCT4, SOX2, and KLF4 (or OSK for short). The goal is to reset the cells’ gene expression patterns so they act young and functional again.

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“This is an important moment for Life Bio and for the field of aging biology,” said David Sinclair, Ph.D., Co‑founder of Life Biosciences and Professor of Genetics at Harvard Medical School. “Our research has suggested that aging is driven in large part by the loss of epigenetic information, not irreversible damage. This clinical study represents the first opportunity to test whether restoring that information can ameliorate human disease.”

All Eyes on the Trials

The Phase 1 trial will look at whether ER-100 is safe and how well patients tolerate it, while also measuring visual function.

“This milestone reflects years of rigorous scientific development and translational research,” said Sharon Rosenzweig‑Lipson, Ph.D., Chief Scientific Officer of Life Biosciences. “Our preclinical studies have demonstrated that controlled OSK expression can reset epigenetic patterns associated with healthy cellular function, improve tissue performance, and restore visual function in animal models.”

Rosenzweig‑Lipson added, “Advancing ER‑100 into the clinic is an important step toward translating epigenetic restoration into a new class of medicines for age-related diseases.”

Significantly, if this approach works for the eyes, Life Bio hopes to use the same platform to treat age-related diseases in other organs down the line. For now, researchers will have to wait and see what the clinical trial results show.