PulseSight Therapeutics announced that its Chief Scientific Officer, Thierry Bordet, PhD, will present new clinical and preclinical findings on the role of iron dysregulation and ferroptosis in age-related macular degeneration (AMD) at the 25th Euretina Congress in Paris, September 4–7.
According to PulseSight, emerging evidence highlights iron overload as a key driver of AMD progression, contributing to oxidative stress, inflammation, and ferroptosis—a form of iron-dependent cell death now recognized as a major factor in retinal degeneration. Transferrin, a naturally occurring glycoprotein, plays a critical role in iron regulation by binding and transporting iron in a non-toxic form, thereby preventing harmful accumulation.
In collaboration with Inserm and Cochin Hospital (Paris), PulseSight analyzed one of the largest aqueous humor datasets from patients with dry AMD/GA compared to age-matched controls. The study confirmed:
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Elevated iron levels in aqueous humor of AMD patients
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Increased transferrin saturation, reinforcing iron imbalance as a hallmark of disease pathology
These findings provide clinical confirmation of iron dysregulation in AMD, supporting iron modulation as a therapeutic strategy in GA—an area that remains underexplored.
Dr. Bordet will also share topline data from a mechanistic study investigating transferrin’s role in preventing ferroptosis. Results demonstrate transferrin’s protective effect against iron-induced cell death, offering deeper biological rationale for therapeutic approaches targeting ferroptosis. The full study has been submitted for peer-reviewed publication.
Advancing PST-611: A First-in-Class Gene Therapy Candidate
PulseSight’s lead investigational therapy, PST-611, is a non-viral vectorized therapy encoding transferrin designed to restore iron balance and preserve retinal structure and function in dry AMD/GA. PST-611 entered a Phase 1 clinical trial (PST-611-CT1) earlier this year.
“Ferroptosis is now a well-recognized target in AMD pathology. At PulseSight, we’ve designed a translational strategy to restore iron homeostasis and prevent ferroptosis," said Dr. Bordet said. "With PST-611, we combine mechanistic relevance, long-lasting efficacy, and a de-risked delivery platform. We’re excited to advance this first-in-class therapy for patients with dry AMD/GA.”
Euretina 2025 Presentation Details
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Abstract Number: RETINA25-1768
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Session: Vectorized Transferrin as a Novel Therapeutic Strategy for Dry Age-related Macular Degeneration/Geographic Atrophy: Preclinical and Clinical
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Date & Time: September 5, 13:06–13:12