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ER-100

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Investigational ocular gene therapy designed to restore retinal-cell function through controlled partial epigenetic reprogramming with OSK factors.

Modality
AAV-delivered, inducible OSK gene therapy
Availability (US)
Phase 1 clinical development
Biomarkers
safety and tolerability; visual function; retinal and optic-nerve measures
Hallmark targeted
epigenetic alterations, loss of regenerative capacity
Kind
investigational gene therapy
Other papers
Lu et al., Nature 2020; DOI 10.1038/s41586-020-2975-4
Clinical trials
NCT07290244; Phase 1 study in open-angle glaucoma and non-arteritic anterior ischemic optic neuropathy