作者｜ 张婉莹 编辑｜ 黄茗婷 新媒体编辑｜ 江江 视觉 | 顾芗 "2024年底，我们的股价跌到谷底。直到2025年，18A股市回暖，我们1月到9月股价也在上涨，这次的触 底反弹不仅给了我一种信心，也给了我们一次涅槃重生的机会。" 戈谢病临床表现 2025年，薛群终于等来转机。 5月，北海康成首款自研戈谢病药物注射用维拉苷酶β注射液（商品名：戈芮宁）获批上市， 承诺将患者年治疗费用降低至少50%； 7月，戈芮宁在上海交通大学医学院附属新华医院开出首方正式进入临床应用； 作为北海康成的创始人兼董事长，薛群从公司上市时身家过亿美元，到股价跌至谷底时市值近乎"归 零"，这段过山车般的经历，在薛群口中却显得云淡风轻。 北海康成的创始人兼董事长薛群 公司股价从2021年12月上市当天12.18港元的高光，到2025年1月最低时每股仅0.095港元，市值一度蒸发 超99%。他带领北海康成"向死而生"，又在2025年前9个月杀回港股涨幅榜第一。 "二级市场曾给我泼了 一盆冰水，但2025年股价的回升又让我重拾信心。" 对薛群而言，财富的起伏远不如另一组数字重要： 中国戈谢病患者约有3000人。 作为一种常染色体隐性遗传的 ...

Core Insights - The company Beihai Kangcheng-B (01228) announced that its domestically developed enzyme replacement therapy CAN103 has been included in China's first version of the "Commercial Insurance Innovative Drug Directory," which will be implemented starting January 1, 2026 [1] Group 1: Product Development - CAN103 was approved for market launch on May 13, 2025, making it the first domestically developed enzyme replacement therapy for adolescents aged 12 and older, as well as adults with Type I and Type III Gaucher disease in China [1] - On July 2, 2025, CAN103 was officially prescribed at Xinhua Hospital affiliated with Shanghai Jiao Tong University School of Medicine, marking its entry into clinical application [1] Group 2: Market Impact - The inclusion of CAN103 in the insurance directory is considered a milestone achievement for the company, as it ensures that Gaucher disease patients will have access to safe and effective domestic enzyme replacement therapy, providing broader and more comprehensive treatment coverage [1]

Core Viewpoint - The recent research on "AAV9-mediated gene therapy for infantile Pompe disease" led by Professor Feng Zhichun represents a significant breakthrough in treating this rare genetic disorder, potentially offering a "one-shot cure" solution for patients [1][5]. Group 1: Disease Overview - Pompe disease, also known as Glycogen Storage Disease Type II, is a rare autosomal recessive disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA), leading to glycogen accumulation in lysosomes, particularly affecting skeletal, cardiac, and smooth muscles [2][3]. - The infantile form of Pompe disease progresses rapidly, often resulting in heart failure and respiratory failure by the age of one if untreated [2]. Group 2: Current Treatment Limitations - Enzyme replacement therapy (ERT) has been the primary treatment since its introduction in 2006, significantly reducing mortality rates and improving quality of life for some patients; however, it does not penetrate the blood-brain barrier and has limitations in addressing central nervous system involvement [3][4]. - ERT requires bi-weekly administration and can be costly, with some patients developing antibodies that reduce treatment efficacy or cause allergic reactions [3]. Group 3: Gene Therapy Development - The newly developed GC301 injection, utilizing AAV9 as a vector to deliver a codon-optimized human GAA gene, aims to provide a long-lasting solution by correcting the genetic defect and restoring GAA enzyme synthesis [4][5]. - Initial trials with four infants showed promising results, with three achieving significant developmental milestones and improvements in cardiac function within a 52-week observation period [5]. Group 4: Safety and Future Directions - Preliminary findings indicate that GC301 is safe, with no severe immune-related adverse reactions reported; however, ongoing assessments are necessary to evaluate long-term safety and efficacy [6]. - Future research may explore the application of GC301 in late-onset Pompe disease and the development of personalized treatment plans based on different genotypes [7].

Core Viewpoint - The approval of domestic enzyme replacement therapy, Gorenin, for Gaucher disease patients in China is expected to significantly reduce treatment costs compared to imported alternatives [1][3][5]. Group 1: Product Overview - Gorenin is the first domestically developed enzyme replacement therapy for type I and III Gaucher disease patients aged 12 and above, fully replacing similar imported products [1][2]. - The drug has been approved for a comprehensive range of indications, covering both type I and III patients, including those who are intolerant or unresponsive to other treatments [2][5]. Group 2: Market Context - The annual treatment costs for imported therapies like imiglucerase can range from $100,000 to $300,000, with domestic adult patients potentially facing costs exceeding 1 million yuan [3][5]. - Approximately 3,000 Gaucher disease patients are reported in China, making it challenging for pharmaceutical companies to justify the investment in research and development for such a small patient population [5][6]. Group 3: Cost Management Strategies - WuXi Biologics, the CRDMO service provider, aims to control the development and production costs of rare disease drugs through innovative technology, allowing for competitive pricing [5][6]. - Gorenin is expected to reduce annual treatment costs for Gaucher disease patients by over 50% compared to imported drugs [3][5]. Group 4: Future Directions - The company plans to push for Gorenin's inclusion in medical insurance systems to ensure broader access for patients [6][7]. - There is a strategic focus on developing international markets for rare disease drugs, leveraging local clinical resources and competitive pricing strategies [7].