PulseSight Doses First Patient in PST-611 Trial

PulseSight Therapeutics has announced the dosing of the first patient in its phase 1 clinical trial (PST-611-CT1), marking a key milestone in the clinical development of its lead investigational therapy, PST-611, for dry age-related macular degeneration (AMD) and geographic atrophy (GA).

About PST-611: A First-in-Class Non-Viral Therapy

PST-611 is a first-in-class, non-viral vectorized therapy designed to treat dry AMD/GA by expressing human transferrin—a highly potent iron regulator that plays a central role in restoring normal iron homeostasis. This innovative approach aims to address the underlying mechanisms of disease progression by regulating excess iron, which can contribute to retinal degeneration.

Details of the Phase 1 Study

The PST-611-CT1 trial is a first-in-human, single-ascending dose study focused on assessing the safety and tolerability of PST-611. Conducted in Paris and Grenoble, the study plans to enroll between 6 and 12 patients with dry AMD/GA to establish the therapy’s safety profile and determine the maximal tolerated dose. These findings will inform the design of a subsequent phase 2a proof-of-concept trial. Preliminary results from the phase 1 study are expected in early 2026, subject to patient recruitment.

Clinical Leadership and Innovative Delivery Approach

The study is led by Professor Francine Behar-Cohen, MD, PhD, at the Department of Ophthalmology, Cochin – Assistance Publique-Hôpitaux de Paris (AP-HP), who is also the inventor of the technology, and by Professor Christophe Chiquet, MD, PhD, at the Department of Ophthalmology, CHU Grenoble Alpes.

Professor Behar-Cohen highlighted the significance of advancing this technology into human trials:

“Having pioneered the development of the electro-transfection technology that delivers DNA plasmids encoding therapeutic proteins into the ciliary muscle of the eye, I am very excited to move PST-611, expressing transferrin, into its first clinical trial. Late-stage dry AMD/GA is a progressing disease that leads to vision loss and for which we have no therapeutic options for our patients. Based on its mechanism of action and thanks to the innovative delivery technology, PST-611 has potential to become a major treatment option for these patients.”

Company Perspective and Outlook

Judith Greciet, CEO of PulseSight Therapeutics, underscored the importance of this milestone:

“The dosing of the first patient in our PST-611-CT1 trial is a very exciting milestone for the company. Supported by the previous clinical demonstration of the safety profile of our innovative delivery technology and a solid preclinical package, we believe PST-611 holds the potential to improve both anatomical and functional features of dry AMD/GA. Moreover, the sustained and long-lasting expression of transferrin should help reduce the need for frequent reinjections, strongly improving patients’ compliance to the treatment. Once the safety and the maximal dose are confirmed, our goal is to swiftly move into a phase 2a proof-of-concept study, to demonstrate the ability of transferrin to protect retinal cells from atrophy and preserve vision.”