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

Denali’s wholly owned program, DNL310 or tividenofusp alfa, is an Enzyme Transport Vehicle-enabled iduronate-2-sulfatase (IDS) replacement therapy in development for MPS II (Hunter syndrome).

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.

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 ...

DNL310 received Breakthrough Therapy Designation; BLA filing for Hunter syndrome is anticipated in early 2025. Denali's $1.28 billion cash runway supports expansion efforts, aiming for ...

We are grateful to the FDA for recognizing the potential of tividenofusp alfa as a meaningful treatment option for individuals with Hunter syndrome. We look forward to continued collaboration with ...

Actor Race Eberhardt is chasing after the bad guys and saving the day in a new movie called “Strait Undercover” — and he’s ...

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.

Hunter syndrome affects over 2,000 individuals, primarily males, in commercially accessible regions. With analysts maintaining a Strong Buy consensus and the company maintaining a healthy ...