Idursulfase beta

【Suzhou, China, July 30, 2026】 – CANbridge Pharmaceuticals Inc. (“CANbridge” or the “Company”, Stock Code: 1228.HK), a global biopharmaceutical company focused on rare diseases, is committed to the research, development and commercialization of innovative therapies. The Company announced that it has fully initiated registration procedures and commercial readiness work for Gaurunning (velaglucerase-beta for injection) (“Gaurunning”) covering a key North African market. Launch preparations for Gaurunning in the region have commenced. Gaucher disease in North Africa presents a substantial and growing market with significant unmet clinical needs. Based on publicly available data from health authorities across North African countries, the Gaucher disease treatment market in the region is projected to reach nearly USD 100 million by 2030. Gaurunning was CANbridge’s first in-house developed enzyme replacement therapy (“ERT”) approved in China for adolescents aged 12 and above and adults with Type I and Type III Gaucher disease. As an innovative biologic drug, it is positioned as an alternative to comparable enzyme replacement products. The differentiated label, state of art manufacturing and unique value proposition will position Gaurunning as a strong contender to substantially enhance treatment accessibility and sustainability in China, and to global Gaucher disease patients. An NMPA-approved registration trial is ongoing, aiming to expand Gaurunning’s label to patients aged 2 to under 12. The commercial entry into the North African market marks the first step for Gaurunning to expand into a growing global Gaucher disease market. According to data from IMARC Group, the global Gaucher disease market size reached USD 1.9 billion in 2025 and is expected to expand to USD 3.1 billion by 2034[ https://www.imarcgroup.com/gaucher-disease-market-report]. “CANbridge is actively developing and pursuing its global registration and market access strategy to provide a meaningful and cost-effective treatment alternative to the Gaucher community. We will continue to innovate and develop new solutions toward the goal of leaving no Gaucher patient behind”, said Dr. James Xue, Founder, Chairman and Chief Executive Officer of CANbridge. About Gaucher disease Gaucher disease, one of the most common lysosomal storage disorders, is a rare inherited genetic metabolic disease caused by autosomal recessive mutations in the GBA gene located on chromosome 1q22. It affects both males and females equally and was included in China’s First List of Rare Diseases on 11 May 2018. Gaucher disease is a clinical spectrum that comprises Type 0 (perinatal-lethal), Type I (non-neuronopathic), Type II (acute neuronopathic), and Type III (chronic neuronopathic) forms, with Types I and III surviving into adulthood. Gaucher disease is caused by a deficiency of glucocerebrosidase (acid β-glucosidase), an enzyme that helps break down a cellular membrane glycosphingolipid called glucocerebroside (glucosylceramide) inside lysosomes. As a result, glucocerebroside accumulates primarily in cells of the monocyte-macrophage lineage (Gaucher cells) within certain organs, leading to splenomegaly, hepatomegaly, anemia, thrombocytopenia, bone pain and fractures, and in the most severe forms (perinatal-lethal, Types II and III), early neurological symptoms. About Gaurunning Gaurunning (velaglucerase-beta for injection) has been granted marketing approval by the National Medical Products Administration (the “NMPA”) of the People’s Republic of China (the “PRC”) on 15 May 2025. It is the first domestically independently developed long-term ERT for adolescents aged 12 and above and adults with Type I and Type III Gaucher disease. The Company holds global proprietary rights to develop and commercialize the product. As an innovative biologic drug, Gaurunning can serve as an alternative to comparable imported products. Treatment costs are expected to be markedly lower, which will substantially reduce the long-term financial burden on patients and enhance treatment accessibility and sustainability for Gaucher disease patients globally. About CANbridge CANbridge Pharmaceuticals Inc. (Stock Code: 1228.HK) was founded in 2012 and listed on the Main Board of HKEX in 2021. The Company is a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies to treat rare diseases. As of December 31, 2025, the Company has a comprehensive pipeline of 7 drug assets targeting prevalent rare diseases that have high unmet needs and significant market potential. The robust pipeline includes 3 marketed products (Hunterase®, Livmarli®, Gaurunning®) and 1 drug candidate at the late clinical stage, covering biologics, small molecules and gene therapies. In the rare disease area, the Company currently has seven biologic and small molecule product candidates. These include MPS II (Hunter syndrome) and other lysosomal storage disorders (LSDs), complement-mediated disorders, hemophilia A, metabolic disorders and rare cholestatic liver diseases including Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC). Furthermore, CANbridge is investing in the R&D of next-generation gene therapy technologies, which will provide novel, potentially curative therapies for rare genetic diseases with limited treatment options. To support the research, development and commercialization of its pipeline, the Company has built a global partner network including, but not limited to, WuXi AppTec, WuXi Biologics, Baheal Medical, GC Pharma, Mirum, Privus and Scriptr Global.

YONGIN, South Korea, Aug. 21, 2025 /PRNewswire/ -- GC Biopharma, a leading global pharmaceutical company based in South Korea, announced today that it has revealed the delivery mechanism of Hunterase (idursulfase beta), a recombinant enzyme replacement therapy for Hunter syndrome (MPS II). The research findings, detailing the role of N-glycosylation in lysosomal targeting, have been published in the International Journal of Biological Macromolecules, a prestigious SCIE-indexed journal. Hunter syndrome (MPS II) is a rare genetic disorder caused by mutations in the iduronate-2-sulfatase (IDS) gene, resulting in a deficiency of the IDS enzyme, which is essential for the degradation of glycosaminoglycans (GAGs). This deficiency leads to the progressive accumulation of GAG within lysosomes, causing a range of symptoms. Thus, effective treatment relies on delivering the therapeutic enzyme to lysosomes to facilitate GAG breakdown. For effective lysosomal targeting, the N-glycan structures of the therapeutic enzyme must contain mannose-6-phosphate (M6P). M6P acts as a targeting signal, guiding the enzyme to bind to cells, facilitating its uptake and delivery into lysosomes to degrade GAGs. The research utilized high-resolution Orbitrap mass spectrometry to analyze the site-specific N-glycan structures of idursulfase beta (Hunterase). A total of 42 N-glycan structures were identified, with two sites, Asn221 and Asn255, found to be predominantly modified with bis-mannose-6-phosphate (bis-M6P), a structure containing two phosphate groups. The research team explains that this bis-M6P structure ensures the efficient and stable delivery of idursulfase beta (Hunterase) to lysosomes. The research team also demonstrated idursulfase beta's high-affinity binding to the M6P receptors using surface plasmon resonance. Additionally, fluorescence-labeled cellular studies confirmed efficient uptake and lysosomal delivery of idursulfase beta (Hunterase). Moreover, several N-glycan structures of idursulfase beta (Hunterase) were found to be modified with sialic acid, which is known to prolong the enzyme's circulation in the blood, extending its half-life. Hunterase (idursulfase beta), according to GC Biopharma, is highly effective for treating Hunter syndrome due to its targeted lysosomal delivery, driven by N-glycan structures modified with M6P and prolonged circulation enabled by sialic acid modifications. "This study analyzed the lysosomal delivery mechanism of Hunterase (idursulfase beta), supported by robust scientific evidence," said Jae Uk Jeong, Head of R&D at GC Biopharma. "With clear evidence of its therapeutic effectiveness, patients with Hunter syndrome can have greater confidence in using Hunterase throughout their treatment journey." About GC Biopharma GC Biopharma (formerly known as Green Cross Corporation) is a biopharmaceutical company headquartered in Yong-in, South Korea. The company has over half a century of experience in the development and manufacturing of plasma derivatives and vaccines, and is expanding its global presence with successful US market entry of Alyglo®(intravenous immunoglobulin G) in 2024. In line with its mission to meet the demands of future healthcare, GC Biopharma continues to drive innovation by leveraging its core R&D capabilities in engineering of proteins, mRNAs, and lipid nanoparticle (LNP) drug delivery platform to develop therapeutics for the field of rare disease as well as I&I (Immunology & Inflammation). To learn more about the company, visit This press release may contain biopharmaceuticals in forward-looking statements, which express the current beliefs and expectations of GC Biopharma's management. Such statements do not represent any guarantee by GC Biopharma or its management of future performance and involve known and unknown risks, uncertainties, and other factors. GC Biopharma undertakes no obligation to update or revise any forward-looking statement contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule. GC Biopharma Contacts (Media) Sohee Kim [email protected] Yelin Jun [email protected] Yoonjae Na [email protected] SOURCE GC Biopharma WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED