JCR Pharmaceuticals Co., Ltd. (TSE 4552; "JCR") announced today that it will present at the 21st Annual WORLDSymposiumtm 2025, held February 3-7, 2025, in San Diego, Calif. The poster presentations ...

and RGX-111 for Mucopolysaccharidosis I (MPS I), also known as Hurler syndrome. "This partnership with Nippon Shinyaku is exciting in that it maximizes our collective strengths and enables access ...

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.

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 ...

The collaboration focuses on treatments for Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome, and Mucopolysaccharidosis I (MPS I), or Hurler syndrome ... a Phase 1/2 study.

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 ...

two gene therapies to treat Mucopolysaccharidosis I & II (MPS). These diseases, also known as Hunter and Hurler syndrome, are lysosomal storage deficiencies characterized by the buildup of heparan ...

The other treatment, RGX-111, treats another type of mucopolysaccharidosis called Hurler syndrome. It’s earlier in clinical testing ... was replaced by current chief Curran Simpson on July 1.

and RGX-111 for Mucopolysaccharidosis I (MPS I), also known as Hurler syndrome. Under the terms of the agreement, Regenxbio will receive $110 million at closing and up to an additional $700 million if ...

RGX-121 is poised to be the first gene therapy for MPS II with potential FDA approval as early as late 2025, and RGX-111 has demonstrated very promising results in Phase 1 ... syndrome, and RGX-111 ...