RegenXbio updates on macular degeneration, Duchenne therapies

 - Pivotal program for RGX-314, potential best-in-class, one-time gene therapy for the treatment of wet AMD, is active and expected to support BLA filing in 2024

-- Recently completed an End of Phase 2 meeting with FDA

-- First of two planned pivotal trials is active

-- Pivotal program expected to enroll a total of approximately 700 patients

-- Clear path for cGMP manufacturing process to support BLA

- Phase II trials for RGX-314 utilizing in-office, suprachoroidal delivery on-going; enrollment complete in first cohort of AAVIATE trial

- IND filing expected in mid-2021 for a novel, advanced microdystrophin gene therapy for the treatment of Duchenne Muscular Dystrophy

- Expects to end 2020 with between $515 million and $530 million in cash, cash equivalents and marketable securities

https://www.prnewswire.com/news-releases/regenxbio-announces-update-on-rgx-314-and-pivotal-program-for-the-treatment-of-wet-amd-and-new-gene-therapy-program-for-the-treatment-of-duchenne-muscular-dystrophy-301201378.html