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Life Biosciences ER-100 eye gene therapy trial

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What changed

2026-07-30 22:49 UTC → 2026-08-02 10:16 UTC · added removed

The Phase‑1 safety study of Life Biosciences’ ER‑100 eye‑gene therapy moved from its inaugural dose on 9 June 2026 to active enrollment by mid‑July, targeting up to 18 adults with open‑angle glaucoma or non‑arteritic anterior ischemic optic neuropathy (NAION). The trial, cleared by the U.S. Food and Drug Administration in January, uses a doxycycline‑controlled viral vector to deliver three Yamanaka reprogramming factors (OCT4, SOX2, KLF4) to damaged retinal ganglion cells, avoiding the oncogenic c‑MYC factor. A patient received On 9 June 2026 the first human administration participant was treated in June, a milestone highlighted by Nature as Boston, Massachusetts, marking the world’s first human use of a significant step rejuvenation‑focused gene therapy for biological rejuvenation research. an eye disease. The study will monitor participants enroll an initial cohort of 12 patients with open‑angle glaucoma, followed by a second cohort of up to six patients with NAION. Participants will be monitored for at least five years, focusing on with primary endpoints of safety, tolerability and early signs of retinal ganglion‑cell regeneration. Co‑founder David Sinclair notes the trial tests whether restoring youthful epigenetic instructions can mitigate age‑related optic‑nerve damage; no efficacy data have been released yet.

Versions

  1. 2026-08-02 10:16 UTC Life Biosciences ER-100 eye gene therapy trial
  2. 2026-07-30 22:49 UTC Life Biosciences ER-100 eye gene therapy trial

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