Plasmid-based uveitis therapy proceeding to phase I trial

Biotech company Eyevensys has received approval from the French national regulatory agency ANSM to advance its nonviral gene therapy platform to clinical development.

The platform, EyeCET, is the first gene expression technology using plasmids to induce sustained production of therapeutic proteins for diseases of the eye. The lead product, EYS606, is a noninfectious uveitis treatment candidate that uses a plasmid encoding for a tumor necrosis factor (TNF)-α inhibitor. EYS606 was granted orphan drug designation by the European Medicines Agency (EMA) in February 2016.

EyeCET uses a proprietary electrotransfection injection system to deliver the plasmids in a rapid, minimally invasive procedure. First, the device inserts the plasmids into the ciliary muscle through a small needle, and then it emits a series of short electrical pulses to induce uptake of the plasmids by the muscle cells. The entire procedure is completed in less than 2 minutes.

Early research has shown that after treatment, the ciliary muscle cells will continuously produce the protein of interest at steady, clinically relevant levels for several months. The technology avoids several potential complications faced by viral vector therapies, including patient discomfort from subretinal injections and persistence and spread of viral particles in surrounding tissue.

"This clearance from the ANSM is a landmark achievement for Eyevensys given the unique features of EYS606, which combines an anti-TNFα plasmid with a novel and unique medical device, designed to carry out an electroporation procedure,” said Eyevensys CEO Raffy Kazandjian. “We are now in a position to demonstrate that our EyeCET technology can provide ophthalmology patients with much needed and improved treatment options. We plan to start our first phase I trial with EYS606 shortly."