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访谈2025年诺贝尔生理学或医学奖三位获奖者
生物世界· 2025-10-10 08:00
我们的 免疫系统 是一个错综复杂的制衡机制——能够提供强大的抗感染防御,同时又能够在大多数情况下 避免对自身组织产生破坏性反应。 这种平衡是如何维持的? 这个问题困扰了免疫学家一个多世纪。 编辑丨王多鱼 排版丨水成文 整理丨王聪 在公布获奖名单后 , 诺贝尔奖外联办公室首席科学官 Adam Smith 通过电话分别采访了三位获奖者。 2025 年诺贝尔生理学或医学奖 授予了 Mary Brunkow 、 Fred Ramsdell 、 坂口志文 ( Shimon Sakaguchi ) , 通过一系列富有洞察力的观察和精心设计的实验,做出了关键发现,为上述问题提供了重 要答案—— 他们发现并定义了 CD4 + CD25 + FOXP3 + 调节性 T 细胞 (Treg 细胞) 及其在控制自身反应性响应中的 重要性,开创了 Treg 细胞介导的 外周免疫耐受 这一新领域。这一历程体现了科学的好奇心、坚持不懈的 探索以及关键发现,彻底改变了我们对免疫调控的理解,对自身耐受、自身免疫和肿瘤逃逸具有重要意 义。 Mary Brunkow :我还没完全反应过来 Mary Brunkow 凌晨四点半坐在餐桌旁接受采访,这 ...
浙江大学最新Nature论文:发现系统性红斑狼疮新型致病基因突变——PLD4
生物世界· 2025-09-11 04:06
Core Viewpoint - The research identifies loss-of-function mutations in the PLD4 gene as a cause of systemic lupus erythematosus (SLE), providing a theoretical basis for precise diagnosis and treatment of the disease [3][10]. Group 1: Research Findings - The study published in Nature confirms that mutations in the PLD4 gene can lead to SLE and elucidates the pathogenic mechanism involved [3]. - The research team reported five SLE patients with renal lesions exhibiting PLD4 biallelic mutations [8]. - PLD4 is highly expressed in dendritic cells, monocytes, and B cells, and functions as a 5' nucleic acid exonuclease that limits the overactivation of TLR7 and TLR9 [7]. Group 2: Mechanism of Action - TLR7 and TLR9 play critical roles in sensing RNA and DNA, initiating downstream inflammatory signaling pathways that contribute to SLE development [6]. - The absence of PLD4 leads to excessive activation of TLR7 and TLR9, resulting in heightened inflammatory responses and the production of autoantibodies [10]. Group 3: Potential Therapeutic Implications - PLD4-deficient mice exhibited autoimmune phenotypes and responded to the JAK inhibitor baricitinib, suggesting that targeting type I interferon may be a potential therapy for SLE patients with PLD4 deficiency [10].
协和专家:什么是好的免疫力?
Hu Xiu· 2025-08-29 08:31
Core Viewpoint - The discussion emphasizes the importance of understanding autoimmune diseases, particularly lupus and antiphospholipid syndrome, and highlights advancements in treatment options, including cell therapy, which can significantly improve patients' quality of life and reproductive outcomes [1][30][36]. Group 1: Autoimmune Diseases Overview - There are over a hundred types of autoimmune diseases, affecting a significant population, yet awareness remains low [3]. - Common autoimmune diseases include lupus, antiphospholipid syndrome, and arthritis, with a notable prevalence among women [4][14]. - Patients often form support groups to share experiences and encourage each other, especially regarding expensive treatments [4][18]. Group 2: Patient Experience and Treatment - The role of physicians extends beyond treatment; they provide emotional support and guidance to help patients navigate their conditions [7][10]. - Many patients initially experience anxiety and uncertainty about their conditions, but with ongoing care, they often become more at ease [7][12]. - The importance of accurate diagnosis and treatment plans is emphasized, particularly in managing conditions like antiphospholipid syndrome, which can significantly impact pregnancy outcomes [16][23]. Group 3: Advances in Treatment - Cell therapy shows promise in treating autoimmune diseases by specifically targeting B cells, potentially leading to long-term remission [30][33]. - New biological agents, particularly monoclonal antibodies, are emerging as effective treatments for lupus and other autoimmune conditions [35]. - The success rate for lupus patients achieving healthy pregnancies has improved significantly, with over 80% of children born to lupus patients being healthy [28][36]. Group 4: Misconceptions and Patient Education - There is a tendency for patients to self-diagnose with conditions like antiphospholipid syndrome due to its treatable nature, which can lead to over-treatment and misinformation [21][22]. - The need for standardized antibody testing across different regions is highlighted to ensure accurate diagnosis and treatment [24]. - Patients are encouraged to maintain a balanced lifestyle and not to overly focus on their autoimmune conditions, as this can lead to unnecessary anxiety [50][53].
自免领域迈入双抗时代,康诺亚成下一代自免药王的领路人?
Zhi Tong Cai Jing· 2025-08-02 02:40
Core Insights - The innovative drug market in China is experiencing significant growth, with the total amount of License out transactions for innovative drugs expected to reach nearly $66 billion by the first half of 2025, surpassing the total BD transaction amount for 2024 [1] - The immune system drug market is the second largest prescription drug market globally, with an increasing share of BD transactions, particularly in the autoimmune disease sector, which is gaining attention due to unmet medical needs [2][23] - The emergence of bispecific antibodies (dual antibodies) in the autoimmune disease field is anticipated to create new blockbuster drugs, as they can target multiple antigens simultaneously, enhancing treatment efficacy [7][8][23] Group 1: Market Dynamics - The market for autoimmune disease treatments is projected to reach $119.35 billion by 2027, driven by high demand and long treatment cycles for conditions like systemic lupus erythematosus (SLE) and rheumatoid arthritis (RA) [2] - The TNFα inhibitors, particularly AbbVie's Humira, have generated over $200 billion in revenue over its lifecycle, showcasing the potential for successful drug development in the immune market [2][4] - The competition in the immune drug market is intensifying, with a growing number of patented drugs and a shift towards precision medicine in clinical and commercial strategies [5] Group 2: Bispecific Antibodies - Bispecific antibodies are gaining traction in autoimmune disease treatment, with the ability to simultaneously target different antigens, potentially leading to more effective disease management [7][8] - Clinical data from Roche's bispecific antibody shows significant efficacy in treating refractory SLE patients, indicating the potential of this class of drugs in the autoimmune sector [8] Group 3: Company Insights - Kangnuo Pharmaceutical is positioned as a leading player in the autoimmune field, with its core product CM310 being the first domestically approved IL-4Rα antibody, currently in the commercialization stage [13][23] - The success of CM310 in the market is contingent on its inclusion in the medical insurance negotiation, which is crucial for its sales growth [16][23] - Kangnuo's innovative approach through the NewCo model for licensing out its products has proven beneficial, providing cash flow support and reducing risks for the company [22][23] Group 4: Future Outlook - The potential for Kangnuo's CM310 to achieve significant sales in the Chinese market is optimistic, with projections suggesting it could reach nearly $5 billion by 2030 if it successfully navigates the insurance landscape [23] - The company's advancements in the ADC drug CMG901 for cancer treatment are also noteworthy, with clinical progress ahead of competitors [19][23]
Cell:王俊团队等开发新型LAG-3/TCR双抗,治疗T细胞驱动的自身免疫病
生物世界· 2025-07-01 04:04
Core Viewpoint - The article discusses a novel therapeutic approach targeting pathogenic T cells in autoimmune diseases, focusing on the mechanism of LAG-3 and T Cell Receptor (TCR) interaction, which offers a new strategy for treatment [4][10]. Group 1: Mechanism of Action - Autoimmune diseases are caused by overactive immune responses leading to tissue damage, with T cells playing a crucial role in diseases like type 1 diabetes and rheumatoid arthritis [2][3]. - LAG-3, an inhibitory immune checkpoint receptor, is regulated by its classical ligand MHC-II, and its activation is dependent on the spatial proximity to TCR, which is essential for effective suppression of CD4+ T cells [4][5][7]. - The study reveals that LAG-3's optimal function requires not just interaction with MHC-II but also the formation of a spatial proximity with TCR, which is a key molecular mechanism for T cell inhibition [7][8]. Group 2: Therapeutic Development - The research team developed a bispecific T cell silencer (BiTS) that targets the interaction between LAG-3 and TCR, allowing for selective modulation of pathogenic T cells while preserving beneficial T cell functions [5][10]. - This innovative approach has shown significant therapeutic effects in various animal models of autoimmune diseases, indicating its potential for treating conditions like refractory multiple sclerosis and rheumatoid arthritis [5][10]. - The study provides a unique opportunity for precise intervention in T cell-driven autoimmune diseases, addressing the current lack of safe and effective therapies [10].
周杰伦病情让自身免疫类药物受关注 和美药业能否借势闯关港股?核心产品还未上市,竞品集采已降价超93%
Mei Ri Jing Ji Xin Wen· 2025-06-25 12:43
Core Viewpoint - The rising public awareness of autoimmune diseases, particularly ankylosing spondylitis, has led to increased interest in the pharmaceutical company Ganzhou Hemei Pharmaceutical Co., Ltd. as it seeks to go public with its core drug Mufemilast targeting psoriasis and ankylosing spondylitis [1][6]. Company Overview - Ganzhou Hemei Pharmaceutical is preparing for an IPO under the Hong Kong Stock Exchange's 18A biotechnology rules, focusing on high investment and potential returns typical of biopharmaceutical companies [2]. - The company has not yet generated revenue and relies heavily on government subsidies and continuous financing to sustain operations [2][11]. Product Pipeline - The company has seven clinical-stage small molecule candidates aimed at addressing unmet medical needs in autoimmune diseases and oncology [2]. - Mufemilast is a dual-action PDE4 inhibitor that has entered the NDA priority review process, targeting multiple indications including psoriasis and ankylosing spondylitis [2][3]. Market Potential - The market for psoriasis drugs in China has seen rapid growth, with a compound annual growth rate (CAGR) of 30.4% from 2019 to 2023, reaching a market size of 13.9 billion yuan in 2023, projected to soar to 89.4 billion yuan by 2032 [3]. - The autoimmune disease drug market in China has expanded from $2.5 billion in 2019 to $4 billion in 2023, with expectations to reach $26.3 billion by 2032, reflecting a CAGR of 23.3% [6]. Competitive Landscape - The competitive environment for Mufemilast is intense, with existing competitors having undergone significant price reductions due to national procurement policies, with price drops exceeding 93% for some products [5]. - There are currently five approved small molecule targeted therapies for psoriasis in China, with 37 more in clinical development, indicating a crowded market for Mufemilast [5][8]. Financial Status - The company reported significant losses of 156 million yuan in 2023 and 123 million yuan in 2024, with minimal other income [11]. - The company has undergone multiple rounds of financing since 2021, raising over 500 million yuan to support its operations, with a post-investment valuation of 3.9 billion yuan [11]. Leadership - The founder and chairman, Zhang Hesheng, has over 20 years of experience in biopharmaceuticals and holds a significant voting power in the company [12].
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].