New Hope on the Horizon: PulseSight Therapeutics Enters the Fight Against Retinal Blindness
For individuals and families navigating the challenges of inherited retinal diseases (IRDs) and other severe retinal conditions, news of advancements in therapeutic development offers a beacon of hope. PulseSight Therapeutics, a new ophthalmology biotech company, has officially launched with a mission to develop non-viral gene therapies for severe retinal diseases, including age-related macular degeneration (AMD) and potentially other neurodegenerative retinal disorders like retinitis pigmentosa. This development is significant as it could lead to new treatment options that are less invasive and offer long-lasting benefits for those facing vision loss.
PulseSight Therapeutics, based in Paris, France, launched on February 28, 2024, with seed funding from Pureos Bioventures and ND Capital. The company aims to clinically validate its innovative delivery platform by advancing two late-stage preclinical drug candidates. Their approach utilizes a proprietary non-viral gene therapy ocular platform that employs an electro-transfection system. This system is designed to deliver DNA plasmids, which encode therapeutic proteins, into the ciliary muscle to treat major eye diseases.
One of the key advantages highlighted by PulseSight is its non-viral gene therapy platform, which is designed to provide long-lasting gene expression and favorable distribution within the retina through a minimally invasive delivery technology. The technology has already undergone validation in a Phase I/II clinical study, which reportedly demonstrated a good safety profile for both the plasmid and the delivery system, as well as a long-lasting clinical benefit of up to eight months in patients with chronic noninfectious uveitis.
PulseSight's pipeline includes two main programs: PST-809 and PST-611. PST-809 is their lead program, targeting wet AMD. This therapy involves a dual-gene plasmid encoding for an anti-VEGF agent, aflibercept, along with decorin, a protein with anti-angiogenic and anti-fibrotic effects. Preclinical studies have reportedly shown PST-809 to have superior efficacy compared to intravitreal aflibercept in reducing vascular leakage and promoting retinal pigment epithelium (RPE) wound healing, potentially reducing the need for frequent injections to as little as once every six months.
The second program, PST-611, is aimed at geographic atrophy (GA) in late-stage dry AMD. This therapy uses a plasmid that encodes the human transferrin protein, a natural iron transporter crucial for controlling iron levels in the eye. Preclinical experiments with PST-611 have indicated beneficial effects in removing iron, reducing oxidative stress, preserving RPE integrity, and preventing retinal degeneration and vision loss. Notably, PST-611 has been awarded orphan drug designation by the FDA and EMA for retinitis pigmentosa, suggesting its potential applicability to other neurodegenerative retinal disorders beyond AMD.
For patients and families in the IRD community, the launch of PulseSight Therapeutics represents a potential expansion of treatment options. The focus on non-viral gene therapies and minimally invasive delivery could mean less burdensome treatment regimens and potentially more sustained therapeutic effects. While the initial focus is on AMD, the orphan drug designation for PST-611 in retinitis pigmentosa highlights the broader implications this technology could have for the wider IRD community. The company is currently seeking Series A funding to advance its clinical development.
We will continue to monitor PulseSight Therapeutics' progress as they move towards clinical trials, bringing these promising non-viral gene therapies closer to patients.