Latest news with #MPSII
Business Wire
a day ago
- Business
- Business Wire
JCR Pharmaceuticals Announces Launch of Mucopolysaccharidosis (MPS) Type II Disease Awareness Film Featured in
HYOGO, Japan--(BUSINESS WIRE)-- JCR Pharmaceuticals Co., Ltd. (TSE 4552; 'JCR'), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, announced the launch of a film featured in The Next Frontier, a global branded film series presented by the Biotechnology Innovation Organization (BIO) and produced by BBC StoryWorks Commercial Productions. The Next Frontier series explores how biotechnology can be used effectively to address urgent challenges the world faces. As part of the series, the JCR-sponsored film tells the story of a family affected by mucopolysaccharidosis type II (MPS II, or Hunter syndrome), a rare, progressive, life-threatening lysosomal storage disorder. 'This film demonstrates the hardships and challenges that people living with MPS II face on a daily basis from the perspective of a family affected by this devastating and life-limiting disease,' said Shin Ashida, Chairman, President and CEO of JCR Pharmaceuticals. 'We are honored to participate in this important video series to shine a spotlight on MPS II and increase awareness worldwide about this debilitating rare disease for which patients desperately need new treatment options. This impactful story is a great illustration of how collaborating with patient communities drives progress. We are committed to supporting the MPS II patient community, and we look forward to advancing our investigational therapy for people living with MPS II.' The Next Frontier series highlights biotechnology's potential to help create a healthier and more sustainable future for all. The series delves into how powerful storytelling can turn complexity into clarity and breakthrough science into emotional connection. The series debuted at the BIO International Convention, taking place from June 16-19, 2025. Watch the JCR film here, and view the full The Next Frontier series here. About The Next Frontier Series The Next Frontier is a global branded film series presented by Biotechnology Innovation Organization (BIO) and produced by BBC StoryWorks Commercial Productions, and it explores how biotechnology can be used effectively to address urgent challenges the world faces. The series highlights biotechnology's potential to help create a healthier and more sustainable future for all. The series delves into how powerful storytelling can turn complexity into clarity and breakthrough science into emotional connection and features the tools that help the public not just understand biotech but believe in its potential. If the future depends on science, it also depends on the stories we tell. About Biotechnology Innovation Organization (BIO) BIO is the world's largest biotechnology organization, providing advocacy, business development and communications services, worldwide. BIO members are at the forefront of researching and developing innovative biotechnologies across healthcare, agriculture, industry, and the environment. Corporate members range from entrepreneurial companies developing a first product to Fortune 500 multinationals. BIO is helping to drive a revolution that aims to cure patients, protect our climate, and nourish humanity. About Mucopolysaccharidosis Type II (Hunter Syndrome) Mucopolysaccharidosis type II (MPS II, or Hunter syndrome) is an X-linked recessive lysosomal storage disorder caused by a deficiency of iduronate-2-sulfatase, an enzyme that breaks down complex carbohydrates called glycosaminoglycans (GAGs, also known as mucopolysaccharides) in the body. Hunter syndrome, which affects an estimated 2,000-3,000 individuals worldwide (according to JCR research), gives rise to a wide range of somatic and neurological symptoms. The current standard of care for Hunter syndrome is enzyme replacement therapy. Central nervous system symptoms related to MPS II have been unmet medical needs so far. About JCR Pharmaceuticals Co., Ltd. JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceutical company that develops treatments that go beyond rare diseases to solve the world's most complex healthcare challenges. We continue to build upon our 50-year legacy in Japan while expanding our global footprint into the U.S., Europe, and Latin America. We improve patients' lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies. Our approved products in Japan include therapies for the treatment of growth disorder, MPS II (Hunter syndrome), Fabry disease, acute graft-versus host disease, and renal anemia. Our investigational products in development worldwide are aimed at treating rare diseases including MPS I (Hurler, Hurler-Scheie and Scheie syndrome), MPS II, MPS IIIA and B (Sanfilippo syndrome type A and B), and more. Our core values – Putting people first, Forging our own path, Always advancing, and Committed to excellence – mean that the work we do benefits all our stakeholders, including employees, partners, and patients. We strive to expand the possibilities for patients while accelerating medical advancement at a global level. For more information, please visit the global website:
Yahoo
a day ago
- Business
- Yahoo
JCR Pharmaceuticals Announces Launch of Mucopolysaccharidosis (MPS) Type II Disease Awareness Film Featured in The Next Frontier Series
- Story Highlights Severity and Impact of MPS II, a Rare, Life-Threatening Lysosomal Storage Disorder - HYOGO, Japan, June 18, 2025--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE 4552; "JCR"), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, announced the launch of a film featured in The Next Frontier, a global branded film series presented by the Biotechnology Innovation Organization (BIO) and produced by BBC StoryWorks Commercial Productions. The Next Frontier series explores how biotechnology can be used effectively to address urgent challenges the world faces. As part of the series, the JCR-sponsored film tells the story of a family affected by mucopolysaccharidosis type II (MPS II, or Hunter syndrome), a rare, progressive, life-threatening lysosomal storage disorder. "This film demonstrates the hardships and challenges that people living with MPS II face on a daily basis from the perspective of a family affected by this devastating and life-limiting disease," said Shin Ashida, Chairman, President and CEO of JCR Pharmaceuticals. "We are honored to participate in this important video series to shine a spotlight on MPS II and increase awareness worldwide about this debilitating rare disease for which patients desperately need new treatment options. This impactful story is a great illustration of how collaborating with patient communities drives progress. We are committed to supporting the MPS II patient community, and we look forward to advancing our investigational therapy for people living with MPS II." The Next Frontier series highlights biotechnology's potential to help create a healthier and more sustainable future for all. The series delves into how powerful storytelling can turn complexity into clarity and breakthrough science into emotional connection. The series debuted at the BIO International Convention, taking place from June 16-19, 2025. Watch the JCR film here, and view the full The Next Frontier series here. About The Next Frontier SeriesThe Next Frontier is a global branded film series presented by Biotechnology Innovation Organization (BIO) and produced by BBC StoryWorks Commercial Productions, and it explores how biotechnology can be used effectively to address urgent challenges the world faces. The series highlights biotechnology's potential to help create a healthier and more sustainable future for all. The series delves into how powerful storytelling can turn complexity into clarity and breakthrough science into emotional connection and features the tools that help the public not just understand biotech but believe in its potential. If the future depends on science, it also depends on the stories we tell. About Biotechnology Innovation Organization (BIO)BIO is the world's largest biotechnology organization, providing advocacy, business development and communications services, worldwide. BIO members are at the forefront of researching and developing innovative biotechnologies across healthcare, agriculture, industry, and the environment. Corporate members range from entrepreneurial companies developing a first product to Fortune 500 multinationals. BIO is helping to drive a revolution that aims to cure patients, protect our climate, and nourish humanity. About Mucopolysaccharidosis Type II (Hunter Syndrome)Mucopolysaccharidosis type II (MPS II, or Hunter syndrome) is an X-linked recessive lysosomal storage disorder caused by a deficiency of iduronate-2-sulfatase, an enzyme that breaks down complex carbohydrates called glycosaminoglycans (GAGs, also known as mucopolysaccharides) in the body. Hunter syndrome, which affects an estimated 2,000-3,000 individuals worldwide (according to JCR research), gives rise to a wide range of somatic and neurological symptoms. The current standard of care for Hunter syndrome is enzyme replacement therapy. Central nervous system symptoms related to MPS II have been unmet medical needs so far. About JCR Pharmaceuticals Co., Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceutical company that develops treatments that go beyond rare diseases to solve the world's most complex healthcare challenges. We continue to build upon our 50-year legacy in Japan while expanding our global footprint into the U.S., Europe, and Latin America. We improve patients' lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies. Our approved products in Japan include therapies for the treatment of growth disorder, MPS II (Hunter syndrome), Fabry disease, acute graft-versus host disease, and renal anemia. Our investigational products in development worldwide are aimed at treating rare diseases including MPS I (Hurler, Hurler-Scheie and Scheie syndrome), MPS II, MPS IIIA and B (Sanfilippo syndrome type A and B), and more. Our core values – Putting people first, Forging our own path, Always advancing, and Committed to excellence – mean that the work we do benefits all our stakeholders, including employees, partners, and patients. We strive to expand the possibilities for patients while accelerating medical advancement at a global level. For more information, please visit the global website: View source version on Contacts Investors & Media:JCR Pharmaceuticals Co., Communicationsir-info@
Associated Press
14-05-2025
- Business
- Associated Press
FDA Accepts Biologics License Application for Mucopolysaccharidosis II Treatment
PARAMUS, N.J., May 14, 2025 /PRNewswire/ -- NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku) announced today that the U.S. Food and Drug Administration has accepted for review the Biologics License Application (BLA) submission by REGENXBIO Inc. (REGENXBIO; Headquarters: Rockville, Maryland, USA; CEO: Curran M. Simpson, NASDAQ: RGNX) for RGX-121 (clemidsogene lanparvovec), a potential first-in-class, investigational gene therapy for the treatment of Mucopolysaccharidosis II (MPS II). The FDA granted REGENXBIO's BLA Priority Review with a Prescription Drug User Fee Act ('PDUFA') target action date of November 9, 2025. In January 2025, Nippon Shinyaku and REGENXBIO entered into a strategic partnership for the development and commercialization of RGX-121, as well as RGX-111, which is for the treatment of MPS I. Upon potential approval of RGX-121, NS Pharma will be exclusively responsible for commercializing RGX-121 in the U.S. 'This FDA decision represents a significant milestone in bringing a new, potentially life-changing treatment option to patients in the MPS community,' said NS Pharma President, Yukiteru Sugiyama, Ph.D. 'We are excited about our partnership with REGENXBIO and the value of our combined expertise in generating renewed hope for MPS families.' For more details, please see the press release from REGENXBIO: About RGX-121 (clemidsogene lanparvovec) RGX-121 is a potential one-time AAV therapeutic for the treatment of boys with MPS II, designed to deliver the iduronate-2-sulfatase (IDS) gene to the central nervous system (CNS). Delivery of the IDS gene within cells in the CNS could provide a permanent source of secreted iduronate-2-sulfatase (I2S) protein beyond the blood-brain barrier, allowing for long-term cross correction of cells throughout the CNS. RGX-121 expressed protein is structurally identical to normal I2S. RGX-121 has received Orphan Drug Product, Rare Pediatric Disease, Fast Track and Regenerative Medicine Advanced Therapy designations from the FDA. About MPS II MPS II, or Hunter Syndrome, is a rare, X-linked recessive disease caused by a deficiency in the lysosomal enzyme I2S leading to an accumulation of glycosaminoglycans (GAGs), including heparan sulfate (HS) in tissues which ultimately results in cell, tissue, and organ dysfunction, including in the CNS. In severe forms of the disease, early developmental milestones may be met, but developmental delay is readily apparent by 18 to 24 months. Specific treatment to address the neurological manifestations of MPS II remains a significant unmet medical need. Key biomarkers of I2S enzymatic activity in MPS II patients include its substrate heparan sulfate (HS) D2S6, which has been shown to correlate with neurocognitive manifestations of the disorder. About REGENXBIO Inc. 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. For more information, please visit About NS Pharma, Inc. NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. NS Pharma is a registered trademark of the Nippon Shinyaku Co., Ltd. For more information, please visit US Media Contact: [email protected] View original content to download multimedia: SOURCE NS Pharma, Inc.
