With an FDA approval submission for RegenXBio’s Hunter syndrome gene therapy already underway, the biopharma has now ...

Hunter syndrome results from the absence of an enzyme needed to break down cellular waste. Without it, waste builds up in the body, causing progressive damage to various systems.

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).

Nippon Shinyaku pays $110M upfront to Regenxbio for US/Asia rights to RGX-121 and RGX-111 gene therapies for ...

Due to Hunter’s specific diagnosis, one of his initial symptoms was fevers that would randomly spike upwards of 102 degrees ...

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