Overview
- Life Biosciences said on June 9 that it had injected the first volunteer in its early-phase trial testing a cellular reprogramming gene therapy for glaucoma.
- The therapy delivers three partial Yamanaka reprogramming genes to retinal ganglion cells using a viral vector and turns those genes on and off with the antibiotic doxycycline to limit exposure.
- The trial is designed as a safety and proof-of-concept study that will enroll about a dozen people with glaucoma and later include some with NAION to look for initial signs of benefit.
- Preclinical work in mice and nonhuman primates showed nerve regeneration and vision restoration in some models and sponsor-reported safety signals, but independent experts warn long-term risks such as cancer remain unresolved.
- The move follows large private investment in reprogramming startups and represents a cautious, disease-by-disease path to test whether partial reprogramming can be applied safely in people.