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Cell子刊:CAR-T细胞疗法又攻克一种自身免疫疾病
生物世界· 2025-05-06 00:00
Core Viewpoint - The article discusses the promising application of CD19 CAR-T cell therapy in treating MDA5+ dermatomyositis with rapidly progressive interstitial lung disease (RPILD), highlighting its effectiveness and safety in a pediatric patient [2][3][7]. Group 1: Disease Overview - Dermatomyositis (DM) is an autoimmune disease primarily affecting the skin and muscles, with MDA5+ DM being a severe subtype associated with high mortality due to rapidly progressive interstitial lung disease [2]. - MDA5+ DM-RPILD is considered one of the most challenging conditions in the medical field, with no curative therapies currently available [2]. Group 2: Research Findings - The study reports the first use of second-generation CD19 CAR-T cell therapy in a child with severe MDA5+ DM-RPILD, demonstrating both efficacy and safety [5][7]. - Prior to CAR-T cell injection, conventional treatment stabilized the patient's condition, and CAR-T cell expansion indicated the presence of CD19+ B lymphocytes in deep tissues [6]. - Over 11 months, the patient showed gradual improvement in lung function, skin, motor, respiratory, and neurological functions without the use of immunosuppressants [6][7][9]. Group 3: Implications - The results suggest that CD19 CAR-T cell therapy may provide a new treatment avenue for complex autoimmune diseases that are resistant to traditional immunosuppressive therapies [2][9].
三次“左手倒右手”后,三生国健靠减负重回业绩巅峰
Hua Xia Shi Bao· 2025-03-28 06:31
Core Viewpoint - Sanofi's strategic shift towards focusing on the autoimmune sector has led to significant revenue and profit growth, despite challenges in the competitive landscape and pricing pressures from centralized procurement policies [1][4][7]. Financial Performance - In 2024, Sanofi achieved revenue of 1.194 billion yuan, a year-on-year increase of 17.70%, and a net profit attributable to shareholders of 705 million yuan, up 139.15% [1]. - The company reported a non-recurring net profit of 246 million yuan, reflecting an 18.99% increase year-on-year [1]. - Cash flow from operating activities showed a net inflow of 281 million yuan, a decrease of 32.48% compared to the previous year [2]. Product Portfolio and Market Position - Sanofi currently has three therapeutic antibody drugs on the market, with Yisaipu being the core product, alongside Jianipai and Saiputing [3]. - The market share of Yisaipu has declined from 40.14% in 2016 to 27.00% in 2022 due to increased competition [3]. - The company has faced pricing pressures due to the expansion of centralized procurement policies, with Yisaipu's average price decreasing as it entered over 20 provincial procurement programs in 2024 [3][4]. Strategic Focus and Business Model - Sanofi has divested its oncology pipeline to concentrate on the autoimmune sector, which is seen as a strategic move to enhance its core competencies and improve financial performance [5][6][7]. - The company has signed multiple agreements to transfer oncology and ophthalmology projects to its affiliate, Shenyang Sanofi, to reduce R&D costs and focus on autoimmune drug development [6][7]. - The autoimmune disease market is projected to reach $6.1 billion by 2025, with a compound annual growth rate (CAGR) of over 20% [9]. Industry Trends and Opportunities - The autoimmune disease market is characterized by a growing patient population and a significant unmet clinical need, with only about 30 treatment options currently available globally [8][9]. - The market for biological agents in autoimmune diseases is expected to grow from 12 billion dollars in 2022 to 138 billion dollars by 2030, indicating a robust opportunity for innovation and development in this sector [9].