Injection with epigenetic restoration shows preliminary signals in glaucoma

Life Biosciences released interim data from a Phase 1 trial of ER-100, administered as a single injection into the eye of patients with open-angle glaucoma (a common form in which slow pressure build-up gradually damages the optic nerve). The study was designed to evaluate safety and tolerability, not efficacy.

ER-100 delivers three transcription factors (proteins that control which genes are active in a cell), collectively called OSK, via a viral vector. Expression is then switched on for eight weeks using a daily oral antibiotic (doxycycline). The underlying idea is that OSK expression resets the epigenetic state of cells toward a younger profile, an approach known as epigenetic reprogramming.

What the data show

Three participants received the lowest dose. All three tolerated the treatment through day 56. No serious adverse events were directly attributed to ER-100. Reported side effects were related to the injection procedure, associated steroids, or pre-existing conditions.

In two of the three participants, preliminary signals of improvement were observed in visual field testing (the range a person can see without moving their eyes), scored in decibels. In each of these two participants, at least 28 percent of measured points in the visual field improved by more than 4 dB. The researchers stress that this is a Phase 1 study. The findings are therefore preliminary and are not intended to establish efficacy.

Next steps

Based on the safety data, the independent Data Safety Monitoring Board recommended escalating to the next dose level. The trial is ongoing. Whether epigenetic reprogramming will ultimately become a viable treatment for eye disease must be determined in larger, controlled studies.

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