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Ref 26 Know Your Numbers Emerging

ER-100 - first human data for epigenetic reprogramming

Life Biosciences reported interim Phase 1 data for ER-100, a one-time eye injection delivering the OSK reprogramming factors (Oct4, Sox2, Klf4), switched on by daily doxycycline, in three people with open-angle glaucoma. The low dose was tolerated over 56 days with no drug-related serious adverse events, and the safety board cleared escalation to a higher dose.

Key takeaways:

  • Safety was the endpoint: 3 patients, lowest dose, no serious drug-related adverse events at day 56
  • Early signal only: 2 of 3 patients improved on visual field testing; no placebo group and no direct evidence yet that cells became epigenetically younger
  • Next step is a higher-dose cohort (600 billion viral genomes); efficacy requires later controlled trials
Why it matters - Partial reprogramming has moved from mouse work into a first human safety readout - a genuine milestone for the field, and a clear test case for separating a milestone from a proven therapy.

Source: agingtoday.com

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