The investigational gene therapy is Regenxbio’s RGX-314 for treating wet age-related macular degeneration (wet AMD), diabetic retinopathy (DR) and other chronic eye diseases. Wet AMD is characterized by loss of vision due to the formation of new and leaky blood vessels in the retina, while DR is a complication of diabetes patients whose blood vessels in the eye are damaged, causing vision problems.

The one-time treatment, which is currently being evaluated in separate trials for both wet AMD and DR, consists of the NAV AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina.

Regenxbio will be responsible for completing the ongoing trials, while both firms will collaborate and share costs on any further trials. AbbVie will lead clinical development and commercialization of RGX-314 worldwide, while Regenxbio will participate in US commercialization efforts under a mutually agreed plan.

Under the terms of the deal, AbbVie will pay Regenxbio an upfront sum of $370 million, with the latter firm eligible to receive up to $1.38 billion in additional development, regulatory and commercial milestone payments. They will share profits equally from net sales of RGX-314 in the US, with AbbVie paying Regenxbio tiered royalties on net sales elsewhere.

Author: Elaine Burridge, Freelance Journalist