ABBV-RGX-314 for Retinal Diseases

REGENXBIO is developing surabgene lomparvovec (sura-vec,ABBV-RGX-314) in collaboration with AbbVie as a potential one-time treatment for wet AMD and diabetic retinopathy.

Current anti-VEGF therapies for wet AMD and diabetic retinopathy 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, and patients often experience vision loss with reduced frequency of treatment.

Sura-vec consists of  the NAV® AAV8 vector, which encodes  an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF) activity, modifying the pathway by which new, leaky blood vessels grow and contribute to retinal fluid accumulation.

Sura-vec is being evaluated using two routes of administration: subretinal delivery and suprachoroidal delivery. 

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.

ATMOSPHERE®, NCT04704921, and ASCENT™, NCT05407636, are two pivotal trials evaluating the subretinal delivery of ABBV-RGX-314 in patients with wet AMD. 

The Phase II AAVIATE® trial, NCT04514653, is evaluating sura-vec for the treatment of wet AMD using suprachoroidal delivery.

Diabetic retinopathy (DR) is the leading cause of vision loss in the working-age population 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. 

The Phase II ALTITUDE®, NCT04567550, is evaluating sura-vec for the treatment of DR using suprachoroidal delivery.