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RegenxBio MPS therapy gets rare pediatric disease designation

December 31, 2015 1:33 AM UTC

RegenxBio Inc. (NASDAQ:RGNX) said FDA granted rare pediatric disease designation to RGX-111, a gene therapy in preclinical testing to treat mucopolysaccharidosis I (MPS I, Hurler syndrome). The designation is among the criteria that would make RegenxBio eligible to receive a Priority Review voucher under FDA's rare pediatric disease voucher program, if the therapy is approved.

In 1H16, RegenxBio intends to submit an IND for the adeno-associated virus serotype 9 (AAV9) vector delivering the human alpha-L-iduronidase ( IDUA) gene. In October, FDA granted RGX-111 Orphan Drug designation. ...