At the 2026 meeting of the Association for Research in Vision and Ophthalmology (ARVO), PulseSight Therapeutics presented phase 1 results for PST-611, an investigational gene therapy for geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD).

The first-in-human study enrolled 6 patients across 2 dose cohorts and evaluated the safety and tolerability of a single administration of PST-611 over 16 weeks. The trial was conducted in Paris and Grenoble under the leadership of Francine Behar-Cohen and Christophe Chiquet.

According to the company, the study met its primary and secondary endpoints. Most ocular adverse events were mild, with 2 reported as moderate. Investigators reported no intraocular inflammation, treatment-emergent serious adverse events, or suspected unexpected serious adverse reactions. Best-corrected visual acuity remained stable throughout follow-up.

Although the trial was not designed to evaluate efficacy, investigators described early functional and anatomical signals, including patient-reported vision improvements and changes in geographic atrophy lesion growth patterns. One patient reportedly demonstrated a sustained effect beyond the follow-up period.

PST-611 encodes transferrin, a protein involved in iron homeostasis, reflecting a proposed role of iron dysregulation in geographic atrophy.

PulseSight said it has submitted a clinical trial authorization application in France for a 52-week phase 2a repeat-dose study enrolling up to 20 patients at 3 sites. Enrollment is expected to begin in the second half of 2026, pending regulatory review. RP