Regenx Biosciences

FDA grants Rare Pediatric Disease Designation to Regenxbio’s RGX-121 Gene Therapy

Wednesday, August 3, 2016

REGENXBIO, a biotechnology company focused on the development, commercialization and licensing of recombinant adeno-associated virus (AAV) gene therapy based on its proprietary NAV Technology Platform, has announced that the FDA has granted Rare Pediatric Disease Designation to RGX-121, REGENXBIO’s investigational gene therapy product candidate for the treatment of Mucopolysaccharidosis Type II (MPS II).

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