RGX-314

A single treatment with the potential
for long-lasting results

diver

RGX-314 is being developed as a potential one-time treatment for wet AMD, diabetic retinopathy and other additional chronic retinal conditions treated with anti-VEGF.

RGX-314 is being developed as a novel, one-time subretinal treatment that includes the NAV AAV8 vector containing a gene encoding for a monoclonal antibody fragment. The expressed protein is designed to neutralize vascular endothelial growth factor (VEGF) activity, modifying the pathway for formation of new leaky blood vessels and retinal fluid accumulation.

Wet AMD is characterized by loss of vision due to excess blood vessel formation between two layers of cells in the retina. This excess blood vessel formation results in fluid leakage that can result in physical changes in the structure of the retina and changes in vision. As this process becomes more severe, blindness can result from scar formation due to hemorrhaging.

Current anti-VEGF therapies require repetitive and inconvenient intraocular injections, typically ranging from every four to eight weeks in frequency, to maintain efficacy. Due to a variety of factors, including inconvenience and discomfort associated with frequent injections in the eye, patient compliance is a significant concern with anti-VEGF therapies. Patients often experience vision loss with reduced frequency of treatment.

We plan to initiate a Phase IIb clinical trial for wet AMD in the first quarter of 2020.

Diabetic retinopathy (DR) is the leading cause of vision loss in the working-age population and affects approximately 8 million people in the United States. DR is a complication of diabetes and is a progressive retinopathy, the severity of which ranges from mild non-proliferative diabetic retinopathy to a more advanced proliferative diabetic retinopathy (PDR). The main causes of vision loss secondary to DR are the vision-threatening complications of PDR, marked by the growth of new abnormal blood vessels onto the surface of the retina and vitreous cavity causing severe vision loss and diabetic macular edema (DME) leading to visual impairment. DME can occur at any stage of DR as the blood vessels in the retina become increasingly fragile and leak fluid.

We plan to file an IND for a Phase II clinical trial for DR in first quarter of 2020.