Regenxbio Partners with Nippon Shinyaku for MPS Disease Treatments

Regenxbio Inc., a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy, announced the closing of its previously announced strategic partnership with Nippon Shinyaku.

Under the terms of the agreement, Regenxbio and Nippon Shinyaku will develop and commercialize RGX-121 (clemidsogene lanparvovec) for Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome and RGX-111 for Mucopolysaccharidosis I (MPS I), also known as Hurler syndrome in the United States and Asia.

""RGX-121 and RGX-111 represent potentially transformative new medicines for patient populations in great need of new options,"" said Curran M. Simpson, president and CEO of Regenxbio. ""This partnership bolsters our ability to make important progress on these programs, and we look forward to serving the MPS community with Nippon Shinyaku.""

RGX-121 is on track to be the first gene therapy for MPS II, with potential FDA approval as early as late 2025.

RGX-121 is a potential one-time AAV therapeutic for the treatment of boys with MPS II. RGX-121 expressed protein is structurally identical to normal I2S. Delivery of the IDS gene within cells in the CNS could provide a permanent source of secreted I2S beyond the blood-brain barrier, allowing for long-term cross correction of cells throughout the CNS.

RGX-121 has received Orphan Drug Product, Rare Paediatric Disease, Fast Track and Regenerative Medicine Advanced Therapy designations from the US Food and Drug Administration and advanced therapy medicinal products (ATMP) classification from the European Medicines Agency.

RGX-111 is designed to use the AAV9 vector to deliver the a-l-iduronidase (IDUA) gene to the central nervous system (CNS). By providing rapid IDUA delivery to the brain, RGX-111 could potentially help prevent the progression of cognitive deficits that otherwise occurs in MPS I patients. Positive interim data from a phase I/II trial of RGX-111 were reported in February 2023. RGX-111 has received Orphan Drug Product, Rare Pediatric Disease and Fast Track designations from the US Food and Drug Administration.

Regenxbio is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Since its founding in 2009, Regenxbio has pioneered the development of AAV Therapeutics, an innovative class of gene therapy medicines.

Based on Nippon Shinyaku's business philosophy, ""Helping people lead healthier, happier lives,"" the company aim to be an organization trusted by the community through creating unique medicines that will bring hope to patients and families suffering from illness.

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