Paris, France, 2 June 2025 – PulseSight Therapeutics SAS, an ophthalmology biotech company developing disruptive non-viral vectorized therapies with minimally-invasive delivery technology, is pleased to announce it has received the Clinical Trial Authorization (CTA) from the Agence Nationale de Sécurité du Médicament et des produits de santé (ANSM) for a Phase I trial (PST-611-CT1) assessing PST-611 safety and tolerability in human.
AMD is the leading cause of central vision loss in the elderly, affecting 200 million people worldwide. AMD’s pathogenesis is complex, and the disease still represents a high unmet need. Dry AMD involves the dysregulation of iron homeostasis, leading to an excess of free iron causing highly toxic effects such as inflammation, oxidative stress, and ultimately retinal cells death.
PST-611 is a first-in-class non-viral vectorized therapy for the treatment of dry AMD/ GA, expressing human transferrin, a highly potent iron regulator, playing a central role to restore normal iron homeostasis.
PST-611-CT1 is a first-in-human single ascending dose trial aiming to confirm the favorable safety profile of the drug and validate the maximal dose for the Phase II proof-of-concept study, in six to a maximum of twelve dry age-related macular degeneration (AMD)/geographic atrophy (GA) patients.
The study will be conducted in Paris and Grenoble by Professor @Francine Behar-Cohen, MD, PhD at the Department of Ophthalmology, Cochin – Assistance Publique-Hôpitaux de Paris (AP-HP), the inventor of our technology, and Professor Christophe Chiquet, MD, PhD at the Department of Ophthalmogy, CHU Grenoble Alpes. It is expected to start later in the summer with a readout anticipated early 2026.
The PST-611-CT1 Phase I trial builds on PulseSight’s previous clinical demonstration of the favorable safety profile of its innovative delivery technology and benefits from a solid preclinical package demonstrating its potential to address 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.
Judith Greciet, CEO of PulseSight Therapeutics said, “Congratulations to our team and consultants for their excellent work to reach this first milestone to bring PST-611 to patients. We believe PST-611 holds the potential to become a major new treatment option for patients with dry AMD/GA. Our goal is to confirm the safety and the optimal dose of our drug candidate to then rapidly move into a phase II proof-of-concept study, to demonstrate transferrin’s ability to protect retinal cells from atrophy and preserve vision.”
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Media contact
Sue Charles, Charles Consultants
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E: sue@charles-consultants.com
About age-related macular degeneration (AMD)
AMD develops with aging. It is a disease with progressive, painless loss of central vision with a strong burden on patients’ everyday life, impacting their ability to read, recognize faces and see objects, and is ultimately leading to irreversible central vision loss. Dry AMD is the most common form of AMD, progressing through successive stages into the late form of dry AMD also called geographic atrophy (GA). Wet AMD is a less common type of late AMD causing faster vision loss. Any stage of dry AMD can turn into wet AMD. In all its forms, AMD represents a compelling unmet need for more effective and durable treatment options, with a large and growing market, estimated to reach $27.5 Billion by 2031.
About PST-611 for GA
PST-611 encodes the human transferrin protein, a crucial regulator of iron homeostasis and holds the potential to effectively address key pathological mechanisms in dry AMD/GA, whilst requiring re-treatment only every four to six months. This program is expected to enter the clinic by late summer 2025.
About PulseSight Therapeutics
PulseSight is clinical-stage biotech company committed to developing disruptive non-viral vectorized therapies with minimally-invasive delivery technology to protect and improve the vision of patients with retinal disease with a focus on age-related macular degeneration (AMD) including wet AMD and geographic atrophy (GA) secondary to dry AMD.
Already clinically validated for its safety and sustained activity, PulseSight’s technology platform delivers DNA plasmids encoding therapeutic proteins into the ciliary muscle using an electro-transfection system. The ciliary muscle cells act as biofactories, expressing therapeutic proteins that reach the retina with high distribution, providing a safe and long-lasting treatment for major eye diseases.
Based in Paris, France the PulseSight’s investors are Pureos Bioventures, ND Capital and Korea Investment Partners (KIP).
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