Clinical stage biotech company, Luxa Biotechnology, has announced “the largest vision improvement to date” in clinical trials of retinal pigment epithelial cell therapies for dry age-related macular degeneration (AMD).
The encouraging results are from the first low-dose cohort of Luxa’s ongoing Phase 1/2a clinical trial evaluating RPESC-RPE-4W.
RPESC-RPE-4W is an investigational RPE stem cell-derived therapy, designed to replace damaged or lost RPE cells in patients with dry AMD, a progressive retinal disease and leading cause of irreversible vision loss among older adults.
patients with severe vision loss gained an average of +21.67 ETDRS letters maintained through 12 months
In cohort one of the clinical trials, six participants received a subretinal injection of 50,000 RPESC-RPE-4W cells. Over the follow-up period, patients with severe vision loss gained an average of +21.67 ETDRS letters maintained through 12 months, the company said in a news release.
Patients with better baseline vision showed more modest gains (+3.0 letters at six months). No tumour formation, severe inflammation, or therapy-related serious adverse events were observed, the company noted.
“The results… demonstrate the potential of RPESC-RPE-4W not only to restore vision in patients with severe vision loss, but also to benefit those at earlier stages of disease,” said Luxa CEO Dr Keith Dionne.
“We are excited to continue advancing our clinical program, which recently received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA (the United States Food and Drug Administration), as a potential new regenerative therapy for dry AMD, a leading cause of blindness with no currently approved vision-improving treatments.”
The findings were published in Cell Stem Cell.2
References
- Luxa Biotechnology LLC, Luxa Biotechnology announces publication in cell stem cell describing positive clinical results for first-in-human clinical trial for dry AMD therapy (news release 17 Sept 2025) available at: businesswire.com/news/home/20250917979431/en/Luxa-Biotechnology-Announces-Publication-in-Cell-Stem-Cell-Describing-Positive-Clinical-Results-for-First-in-Human-Clinical-Trial-for-Dry-AMD-Therapy [accessed Sept 2025].
- Rao RC, Arduini BL, Stern J, et al. Safety and tolerability of RPESC-RPE transplantation in patients with dry age-related macular degeneration: Low-dose clinical outcomes. Cell Stem Cell. 2025 Sep 16:S1934-5909(25)00304-2. doi: 10.1016/j.stem.2025.08.012. Epub ahead of print.
