Several companies—including JCR Pharmaceuticals, Denali Therapeutics and Regenxbio— have products in the pipeline that could ...

Despite having gone through this before with her first son, Yusof, and despite the doctors explaining that this was a gene ...

Fifteen-year-old Dominic Henriquez from Prosper, Texas, is bringing hope to boys like him who live with Hunter syndrome, a rare genetic disorder that primarily affects males. Dominic’s journey ...

RegenxBio said Tuesday that Kyoto, Japan’s Nippon Shinyaku Co. Ltd. will develop and commercialize its one-time gene therapies for Hunter syndrome and Hurler syndrome, both affecting children.

With an FDA approval submission for RegenXBio’s Hunter syndrome gene therapy already underway, the biopharma has now found a commercialization partner for both the U.S. and Asian markets.