Regenxbio’s RGX-111 gets FDA Rare Pediatric Disease designation

Monday, January 4, 2016

Regenxbio, a Rockville, Md.-based biotechnology company focused on the development, commercialization and licensing of recombinant adeno-associated virus (AAV) gene therapy, today announced that the FDA has granted Rare Pediatric Disease designation to RGX-111, the company’s investigational gene therapy product candidate for the treatment of mucopolysaccharidosis type I (MPS I).

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