pharmaphorum2d
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 ...
FierceBiotech22d
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.
Nature3y
Leveraging learning in gene editing
By changing the messages encoded within DNA sequences, it creates new possibilities ... catalytic activity of cells from patients with Hurler Syndrome, an inherited condition that impairs a ...