PulseSight Therapeutics reported completion of the last patient, last visit in a phase 1 clinical trial of PST-611, a nonviral gene therapy candidate for dry age-related macular degeneration and geographic atrophy. Results are scheduled for presentation May 7 at the Association for Research in Vision and Ophthalmology Annual Meeting.

The first-in-human study enrolled 6 patients across 2 dose cohorts to evaluate safety and tolerability. The trial was conducted in Paris and Grenoble by investigators including Francine Behar-Cohen, MD, PhD, and Christophe Chiquet, MD, PhD. Professor Behar-Cohen will present the data.

PST-611 is designed to express transferrin, a protein involved in iron regulation. Dysregulation of iron homeostasis has been implicated in geographic atrophy, contributing to oxidative stress and retinal cell death. The therapy uses a nonviral plasmid delivered to the ciliary muscle, with the goal of sustained protein expression.

The study’s primary objective was safety. No efficacy outcomes have been reported to date. Preclinical data have suggested protection of photoreceptors and retinal pigment epithelium.

The company stated that results from this trial will inform a planned phase 2a study. Geographic atrophy remains an area of unmet need, with limited options to slow progression. RP