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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
- 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
About this update from Regenxbio Inc.
[{"type":"text","content":"- 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\n -- Recently completed an End of Phase 2 meeting with FDA\n -- First of two planned pivotal trials is active\n -- Pivotal program expected to enroll a total of approximately 700 patients\n -- Clear path for cGMP manufacturing process to support BLA\n - Phase II trials for RGX-314 utilizing in-office, suprachoroidal delivery on-going; enrollment complete in first cohort of AAVIATE trial\n - IND filing expected in mid-2021 for a novel, advanced microdystrophin gene therapy for the treatment of Duchenne Muscular Dystrophy\n - Expects to end 2020 with between $515 million and $530 million in cash, cash equivalents and marketable securities\n\n\nROCKVILLE, Md., Jan. 5, 2021 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today provided an update on the RGX-314 programs, including the announcement that the pivotal program for RGX-314 for the treatment of wet age-related macular degeneration (wet AMD) is now active. In addition, REGENXBIO announced a new program, RGX-202, a novel, potentially best-in-class, one-time gene therapy for the treatment of Duchenne Muscular Dystrophy (DMD). \n\"2020 was a very productive year at REGENXBIO, and we are excited to move into 2021, which we expect to be another year of clinical execution. The initiation of our first pivotal program for RGX-314 for the treatment of wet AMD is a great step forward for the field as we look to broaden the applicability of gene therapy to larger patient populations. In addition, we are excited to announce RGX-202, a potential one-time gene therapy for the treatment of DMD. RGX-202 is the first gene therapy program in the REGENXBIO pipeline to be developed under the leadership of our Chief Scientific Officer, Olivier Danos. We look forward to filing an IND for this program later this year,\" said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. \"We continue to advance our pipeline of innovative therapies in the clinic as well as our manufacturing capabilities. I would also like to express my deep gratitude to our employees and clinical partners as well as patients and their families for their ongoing commitment and support despite the challenges posed by the global COVID-19 pandemic.\"\nPivo...