PARIS, France, May 11, 2026
PulseSight Therapeutics SAS announced positive results from its Phase 1 clinical trial (PST-611-CT1) evaluating PST-611, the company’s first-in-class non-viral gene therapy candidate for Geographic Atrophy (GA) secondary to dry Age-related Macular Degeneration (AMD). The findings were presented during a podium session at the ARVO 2026 Annual Meeting, highlighting encouraging safety, tolerability, and early efficacy signals for the investigational therapy in patients suffering from one of the leading causes of irreversible vision loss worldwide.
The study represents an important milestone for PulseSight’s innovative ophthalmology pipeline and reinforces growing industry interest in non-viral gene therapies designed to address retinal diseases with minimally invasive delivery technologies. Geographic Atrophy currently has limited treatment options globally, particularly in Europe, where the unmet need for effective long-term therapies remains substantial.
Phase 1 Trial Demonstrates Strong Safety and Early Efficacy Signals
The PST-611-CT1 study was designed as a first-in-human, single ascending dose clinical trial evaluating two dose levels of PST-611 across six patients diagnosed with late-stage dry AMD-related Geographic Atrophy. Conducted in Paris and Grenoble, the trial assessed the safety and tolerability of the therapy over a 16-week follow-up period. According to PulseSight, the trial successfully met both primary and secondary endpoints, with PST-611 demonstrating an excellent safety and tolerability profile at both low and high dose levels.
Most ocular adverse events observed during the study were mild, while two events were categorized as moderate. Importantly, researchers reported no intraocular inflammation, no treatment-emergent serious adverse events (SAEs), and no suspected unexpected serious adverse reactions (SUSARs). Best Corrected Visual Acuity (BCVA) remained stable throughout the entire follow-up period, further supporting the therapy’s favorable safety profile.
Advertisement
Although the trial was not primarily designed to evaluate efficacy, investigators observed encouraging anatomical and functional efficacy signals, including spontaneous reports from patients describing improvements in vision. Researchers also identified associated reductions in the progression of Geographic Atrophy lesions in certain patients. In one case, the therapeutic benefit appeared to extend beyond the official study follow-up period, generating additional optimism regarding the durability of the treatment response.
Innovative Non-Viral Gene Therapy Targets Iron Dysregulation in AMD
PST-611 is a novel gene therapy candidate encoding human transferrin, a naturally occurring protein responsible for regulating iron homeostasis within retinal tissues. Geographic Atrophy and dry AMD are increasingly associated with dysregulated iron metabolism, where excess free iron contributes to oxidative stress, inflammation, retinal degeneration, and progressive vision loss. PulseSight’s approach aims to restore physiological iron balance and prevent activation of downstream toxic pathways responsible for retinal damage.
Unlike traditional viral vector gene therapies, PulseSight utilizes a proprietary non-viral vectorized delivery platform involving DNA plasmids and electro-transfection technology. The system enables therapeutic proteins to be delivered through ciliary muscle cells, which act as biofactories capable of continuously producing therapeutic proteins that reach the retina over extended periods. Industry observers view non-viral ocular gene therapy technologies as a rapidly emerging area of innovation due to their potential safety advantages, scalability, and minimally invasive administration approach.
Preclinical studies conducted by PulseSight previously demonstrated protection of retinal pigment epithelium (RPE) cells and photoreceptors, along with preservation of visual function in animal models. The company believes these findings support the broader therapeutic potential of PST-611 in retinal degenerative diseases.
Phase 2a Trial Planned as Regulatory Review Continues
Following the positive Phase 1 results, PulseSight confirmed plans to initiate a repeat-dose Phase 2a clinical trial designed to further assess safety and explore efficacy across a larger patient population over a 52-week treatment period. The company has already submitted a Clinical Trial Authorization (CTA) application to France’s Agence Nationale de Sécurité du Médicament et des produits de santé (ANSM) for the upcoming study involving up to 20 patients across three clinical sites. Subject to regulatory approval, patient enrollment is expected to begin during the second half of 2026, with trial results anticipated in 2028.
The latest clinical findings further position PulseSight among emerging innovators in ophthalmic gene therapy while highlighting the increasing scientific momentum behind next-generation treatments targeting retinal degenerative diseases and chronic vision impairment.
Source: PulseSight Therapeutics press release
