PST-611 is a first in class non-viral vectorized therapy for the treatment of dry AMD/GA coding for human transferrin.
PulseSight Therapeutics SAS presented new data on its lead program PST-611, a DNA plasmid expressing human transferrin, in dry age-related macular degeneration (AMD) and geographic atrophy (GA) at the European Association for Vision and Eye Research 27th EVER Congress 2024.
PST-611 is a first in class non-viral vectorized therapy for the treatment of dry AMD/GA coding for human transferrin, a highly potent iron chelator, thus restoring normal iron homeostasis.1
During his presentation, Dr Thierry Bordet, CSO of PulseSight showed data from a retrospective study in a large cohort of dry AMD patients confirming higher level of free iron and increased transferrin saturation in AMD patients versus control patients. He presented data showing that transferrin supplementation in iRPE (human iPSC-derived retinal pigment epithelial) cells exposed to high concentration of iron, restores iron homeostasis and rescued RPE cells from oxidative stress, mitochondrial damage, inflammation, complement activation, and ferroptosis, preserving their integrity.1
In addition to this new candidate, Dr Karine Bigot, Head Pharmacology & Toxicology at PulseSight presented a poster demonstrating the safety of PST-611, administered to the ciliary muscle using a minimally invasive electrotransfection system.1
In the press release1 sharing these points, Thierry Bordet, CSO of PulseSight Therapeutics, said, “Excess iron is known to be highly toxic to retinal cells, leading to oxidative stress, inflammation, and ferroptosis. By restoring normal iron homeostasis, transferrin mitigates these toxic effects and protects retinal cells, offering the potential to slow GA lesions growth and improve patient’s visual function. As a novel non-viral vectorized gene therapy delivered through electroporation, PST-611 benefits from a very good safety profile. We are convinced that PST-611 could become a major treating option for dry AMD patients who are progressively losing sight and for which the medical need is huge. We look very much forward to demonstrating the value of our candidate in the soon to start clinical plan.”
PulseSight plans to submit a phase I clinical trial authorization (CTA) by the end of 2024, to be closely followed by a phase II proof-of-concept to demonstrate the efficacy and the safety of its drug candidate PST-611 by the end 2027.1
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