FIX-Padua 是天然 FIX 的 高活性突变体，使用靶向肝脏的 腺相关病毒 （AAV） 载体递送 FIX-Padua，能够有效治疗 B 型血友病。 信念医药/华东理工大学 肖啸 教授团队开发了 亚洲首个 B 型血友病基因疗法—— BBM-H901 ，在临床 试验中显示出了良好的安全性和治疗效果，该疗法已于 2025 年 4 月 10 日在国内获批上市，商品名"信玖凝"。 2025 年 11 月 20 日 ， 中国医学科学院血液病医院 （中国医学科学院血液学研究所） 张磊 / 杨仁池 团队在国际顶尖 学术期刊 Nature Medicine 上发表了题为： Factor IX Padua gene therapy in hemophilia B: phase 1/2 and 3 trials 的临床研究论文。 撰文丨王聪 编辑丨王多鱼 排版丨水成文 B 型血友病 是一种 X 连锁隐性遗传疾病，其特征为 凝血因子 IX （FIX，由 F9 基因编码） 缺乏，临床上表现为自发 性/创伤性出血 （关节积血、软组织血肿和颅内出血） 。严重病例 （FIX:C < 1 IU/dl） 会出现反复关节积血，且常发 展为致 ...

Group 1: Healthcare Innovation and Technology - The construction of the heavy ion radiation therapy system in Jinan is accelerating, showcasing China's advancements in high-end medical equipment for cancer treatment [1] - The approval of the first gene therapy drug for hemophilia B, developed by a Shanghai company, marks a significant milestone in China's biopharmaceutical industry [2] - The emergence of AI-driven drug discovery platforms is revolutionizing the pharmaceutical industry, with over 200 innovative drugs approved in China since the start of the 14th Five-Year Plan [3] Group 2: Medical Equipment Development - A range of high-end medical equipment has been developed, including intelligent minimally invasive surgical systems and advanced imaging devices, enhancing surgical precision and capabilities [5][6] - The introduction of AI-assisted surgical navigation has improved surgical accuracy by 8% and reduced error rates by 43%, demonstrating the effectiveness of integrating AI in medical procedures [6] - The approval of 265 innovative medical devices, including the carbon ion therapy system, highlights China's progress in developing autonomous high-end medical equipment [6] Group 3: Smart Healthcare Initiatives - The launch of the "Yizhi Ark" medical model cluster in Shenzhen aims to improve healthcare resource distribution and enhance public health services through data integration [7] - The development of national-level specialized medical model clusters is facilitating cross-institutional collaboration and improving healthcare delivery [8] - AI technologies are being utilized to enhance diagnostic and treatment processes, significantly improving patient outcomes and healthcare accessibility [8]

Investment Rating - The investment rating for the pharmaceutical and biotechnology industry is "Positive" and the rating has been maintained [2] Core Insights - The pharmaceutical and biotechnology industry index increased by 4.90%, ranking third among 31 primary industries, outperforming the CSI 300 index, which decreased by 0.09% [4][15] - The sub-industries of medical research outsourcing and vaccines saw significant gains of 12.70% and 6.59%, respectively, while blood products and offline pharmacies lagged with increases of only 0.35% and 2.41% [4][15] - As of August 1, 2025, the overall PE (TTM) for the pharmaceutical and biotechnology industry was 30.97x, up from 30.09x in the previous period, indicating an upward valuation trend [4][21] - The top three sub-industries by PE (TTM) are vaccines (62.06x), hospitals (38.94x), and other biological products (38.44x), while the lowest valuation is in pharmaceutical circulation (16.12x) [4][21] Industry Review - The National Medical Products Administration (NMPA) is seeking public opinion on the "Regulations on the Filing Management of Internet Drug and Medical Device Information Services" [6] - The State Council's news office held a press conference discussing multiple measures for drug price management [6] - Bristol-Myers Squibb's "Nivolumab Injection" combined with "Ipilimumab Injection" received NMPA approval, marking it as China's first approved dual immune therapy for lung cancer [6] - The total transaction amount for innovative drug BD in China exceeded 60 billion USD in the first half of 2025, indicating a significant market shift towards platform-based pipeline output [7] Investment Recommendations - The National Healthcare Security Administration (NHSA) has clarified a "reverse inward competition" direction in the 11th batch of centralized procurement, promoting a shift from price competition to a quality-first and reasonably priced ecosystem [7] - The report suggests focusing on companies with "true innovation" capabilities, strong barriers (complex formulations/high-end device domestic replacements), platform-based operations, and internationalization [7]