Core Insights - The prevalence of thalassemia carriers in China exceeds 30 million, with an estimated number of severe patients in the tens of thousands [1][3][6] - Thalassemia is a hereditary hemolytic anemia caused by defects in globin genes, leading to ineffective erythropoiesis and varying degrees of chronic anemia [2][3] - The disease is primarily divided into α-thalassemia and β-thalassemia, with severe cases requiring lifelong blood transfusions and iron chelation therapy [2][3] Disease Characteristics - Thalassemia is often confused with iron deficiency anemia, but they have fundamentally different causes; thalassemia is genetic while iron deficiency anemia is acquired [2][3] - Severe thalassemia can lead to life-threatening complications, including organ damage and severe deformities in fetuses [3][4] Treatment Developments - Current treatment for severe thalassemia mainly relies on allogeneic hematopoietic stem cell transplantation, which faces challenges such as donor matching and high costs [4][5] - Emerging gene editing technologies, particularly base editing, show promise for potentially curing β-thalassemia in a single treatment [5][6] - Clinical trials have demonstrated that patients treated with gene editing have stable hemoglobin levels and no longer require regular blood transfusions [6][7] Future Directions - The research team plans to conduct long-term follow-up studies on treated patients to monitor the safety and efficacy of gene editing [7] - There are intentions to expand the application of gene editing technology to other rare blood disorders, such as sickle cell anemia [7] - The cost of gene editing treatments is expected to decrease with technological advancements and policy support, making it accessible to more patients [7][8] Public Awareness and Prevention - There is a significant need for increased public awareness regarding thalassemia, as many individuals lack basic knowledge about the disease and its screening [8] - Efforts are being made by health authorities to promote pre-pregnancy and prenatal screening for thalassemia, but misconceptions hinder effective prevention [8]

新华社上海7月26日电（记者杨有宗）中国生物医药产业高地上海市浦东新区近期发布《浦东新区生物 医药产业园区功能提升方案（2025—2027年）》，首次提出打造全球创新药械首发地、科学家创新创业 首选地、制度改革首创地。方案还提出，到2027年，浦东新区生物医药产业规模将突破5000亿元。 正序生物是一家在浦东新区创业、孵化自上海科技大学的生物医药企业。其最新科研成果——碱基编辑 疗法，以类似修正笔的方法对患者突变碱基精准修正，力求损伤小、疗效好。正序生物副总裁武冰冰介 绍，刚刚为这一疗法提交了关键临床申请。 近年来，浦东新区加快建设生物医药世界级产业集群。浦东新区生物医药产业规模由2020年的2408亿元 增至2024年的4100亿元、年均复合增长14%；创新药在研管线约占中国全国的1/5、全球6%。像正序生 物这种基于新靶点、新机制的创新药械产品并不少见。 空中远望位于上海浦东新区张江科学城的形如鹦鹉螺的大科学装置上海光源（中）（2023年11月25日 摄，无人机照片）。新华社记者 方喆 摄 这是2023年9月10日拍摄的中国（上海）自由贸易试验区张江片区（无人机照片）。新华社记者 方喆 摄 为打造全球创新 ...