PulseSight Therapeutics has completed last patient, last visit in its phase 1 trial of PST-611, a non-viral gene therapy candidate for geographic atrophy (GA), with full results set to be presented at ARVO 2026.

The first-in-human study evaluated safety and tolerability of PST-611 in six patients with dry age-related macular degeneration (AMD)/GA across two dose cohorts. Conducted in Paris and Grenoble, the trial represents an early clinical step for the company’s minimally invasive, non-viral gene delivery platform.

Findings will be presented by Professor Francine Behar-Cohen at the ARVO Annual Meeting in Denver (May 7), marking a key milestone for the program as it progresses toward phase 2a development.

PST-611 is designed to express transferrin, a protein involved in iron regulation. Dysregulated iron homeostasis is increasingly recognized in dry AMD, contributing to oxidative stress, inflammation, and retinal cell death. Preclinical data suggest the therapy may protect photoreceptors and retinal pigment epithelium (RPE) cells while preserving visual function.

PulseSight’s approach uses electro-transfection to deliver DNA plasmids into the ciliary muscle, which then acts as a “biofactory” to produce therapeutic proteins capable of reaching the retina. The company believes this could offer a less invasive and potentially longer-acting alternative to current treatment strategies.

Dry AMD, particularly its advanced form GA, remains an area of significant unmet need, with limited options to slow disease progression. While the small sample size reflects the early-stage nature of the study, the upcoming ARVO presentation will be closely watched for initial safety signals and early indications of biological activity.

PulseSight says it plans to build on these findings in a phase 2a study, as interest in gene-based approaches to retinal disease continues to grow.

Source: PulseSight Therapeutics.