FierceBiotech6d
Denali eyes approval after Hunter syndrome drug clears safety measures, reduces biomarkers in open-label study
After winning a breakthrough therapy designation for its Hunter syndrome enzyme replacement therapy, Denali Therapeutics is ...
pharmaphorum5d
Regenxbio expands MPS I gene therapy trial after first look at data
Data from the first patients enrolled into Regenxbio's trial of its gene therapy for rare inherited disease mucopolysaccharidosis type I (MPS I) – also known as Hurler syndrome – has shown the ...
FierceBiotech29d
RegenXBio sells Hunter syndrome gene therapy's U.S., Asia commercial rights for $110M upfront
That partner, Japan’s Nippon Shinyaku, is handing over $110 million upfront to lead on the commercialization of two gene therapies—RGX-121 for Hunter syndrome and RGX-111 for Hurler syndrome.