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 ...
or MPS II, and RGX-111 for Hurler syndrome, or MPS I. Under the deal, REGENXBIO (NASDAQ:RGNX) will receive $110M upfront and up to $700M in potential milestone payments, plus double-digit ...
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.