Diseases of the eye are well suited to therapeutic intervention using sd-rxRNA compounds. Local administration to the eye is an accepted method of administration and generally avoids systemic exposure. RXi’s sd-rxRNA technology is particularly well-suited this therapeutic area, having improved cellular uptake compared to ‘standard’ siRNAs and potent silencing activity.
The cornea is the eye’s outermost layer. It is the clear, dome-shaped surface that covers the front of the eye. It provides the majority of the focusing capabilities of the eye and is a critical component for vision. Corneal scarring refers to an injury of the cornea of the eye that causes opacity and visual impairment. The effects of corneal scarring can vary from blurring to blindness in the eye.
RXI-109, a self-delivering RNAi (sd-rxRNA) compound is in development for the treatment to reduce subretinal fibrosis or retinal scaring. A clinical study, RXI-109-1501, is underway to block the formation of sub-retinal scarring in patients with wet AMD with the goal to preserve vision for a longer period of time than with anti-VEGF treatment alone.
In support of RXI-109-1501, samples from a pilot ocular toxicology study in non-human primates (shown below) clearly showed that a single dose of RXI-109 reduced CTGF in the retina and in the cornea.