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)/geographic atrophy (GA) at the European Association for Vision and Eye Research (EVER) 27th Congress in Valencia, Spain.
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, the company stated in a press release. PulseSight's chief scientific officer, Thierry Bordet, PhD, 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 vs control patients. He also 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.
The development of novel treatments for retinal diseases is often hindered by the challenge of delivering the drug to the back of the eye, PulseSight explained in its press release. To address this issue, Dr Karine Bigot, Head Pharmacology & Toxicology at the company presented a poster demonstrating the safety of PST-611, administered to the ciliary muscle using a minimally invasive electrotransfection system.
PulseSight plans to submit a phase I clinical trial authorization by year's end, followed by a phase II proof-of-concept to demonstrate the efficacy and the safety of PST-611 by the end 2027.