核心观点 - AAV基因治疗在脑科和眼科领域进展迅速，重点关注康弘药业、特宝生物和海特生物 [1][2] - rAAV具备小尺寸、非致病性、可工程化等优势，适用于多种适应症 [10][11] - 全球AAV基因治疗商业化进展显著，Zolgensma销售额突破10亿美金 [22][27] - 国内AAV基因治疗管线丰富，涉及眼科、中枢神经系统和罕见病等领域 [42][43] AAV基因治疗技术优势 - rAAV具有小尺寸、多种组织嗜性、稳定转基因表达等特点 [10][11] - 不同AAV血清型具有独特组织趋向性，可针对不同适应症 [14][17] - AAV在神经元中可持续表达治疗蛋白数年，实现长效治疗 [2][37] - 眼部具有免疫豁免特性，是AAV基因治疗重要应用领域 [2][41] 商业化进展 - 诺华Zolgensma用于治疗SMA，2024年销售额突破10亿美金 [22][27] - Sarepta的Elevidys用于治疗DMD，2024年销售额8.21亿美元 [27] - Regenxbio/艾伯维ABBV-RGX-314预计2025年完成关键性临床 [2][27] - 信念医药信玖凝获批成为中国首个血友病B基因治疗药物 [20] 适应症拓展 - 中枢神经领域：SMA、帕金森病、阿尔茨海默病等 [2][35] - 眼科领域：湿性AMD、视网膜色素变性等 [38][41] - 从罕见病向常见病拓展，如AD和PD等 [37] - 康弘药业AAVv128展现更高转导效率，潜力显著 [41] 国内企业布局 - 康弘药业：KH631和KH658进度领先，布局湿性AMD等 [2][47] - 特宝生物：脊髓型肌萎缩症、扩张型心肌病等 [48] - 海特生物：控股中眸医疗，布局视网膜色素变性等 [49][50] - 国内临床阶段管线丰富，涉及眼科、CNS和罕见病 [43][44] 行业动态 - 恒瑞医药与GSK达成协议，潜在总金额120亿美元 [3] - 石药集团与Madrigal达成协议，总额20.75亿美金 [3] - 赛诺菲引进维亚臻APOC siRNA新药，合作金额3.95亿美元 [3] - 2025年世界肺癌大会将展示43项中国研究 [3]

NEW YORK, Aug. 03, 2025 (GLOBE NEWSWIRE) -- Leading securities law firm Bleichmar Fonti & Auld LLP announces that a lawsuit has been filed against Sarepta Therapeutics, Inc. (NASDAQ: SRPT) and certain of the Company’s senior executives for potential violations of the federal securities laws. If you invested in Sarepta, you are encouraged to obtain additional information by visiting: https://www.bfalaw.com/cases-investigations/sarepta-therapeutics-inc-class-action. Investors have until August 25, 2025, to as ...

NEW YORK, Aug. 01, 2025 (GLOBE NEWSWIRE) -- Leading securities law firm Bleichmar Fonti & Auld LLP announces that a lawsuit has been filed against Sarepta Therapeutics, Inc. (NASDAQ: SRPT) and certain of the Company’s senior executives for potential violations of the federal securities laws. If you invested in Sarepta, you are encouraged to obtain additional information by visiting: https://www.bfalaw.com/cases-investigations/sarepta-therapeutics-inc-class-action. Investors have until August 25, 2025, to as ...

NEW YORK, July 30, 2025 (GLOBE NEWSWIRE) -- Leading securities law firm Bleichmar Fonti & Auld LLP announces that a lawsuit has been filed against Sarepta Therapeutics, Inc. (NASDAQ: SRPT) and certain of the Company’s senior executives for potential violations of the federal securities laws. If you invested in Sarepta, you are encouraged to obtain additional information by visiting: https://www.bfalaw.com/cases-investigations/sarepta-therapeutics-inc-class-action. Investors have until August 25, 2025, to as ...

公司动态 - 美国食品药品管理局（FDA）调查结果显示，八岁男孩的死亡与Sarepta Therapeutics的基因疗法Elevidys无关 [1]

NEW YORK, July 28, 2025 (GLOBE NEWSWIRE) -- Leading securities law firm Bleichmar Fonti & Auld LLP announces that a lawsuit has been filed against Sarepta Therapeutics, Inc. (NASDAQ: SRPT) and certain of the Company’s senior executives for potential violations of the federal securities laws. If you invested in Sarepta, you are encouraged to obtain additional information by visiting: https://www.bfalaw.com/cases-investigations/sarepta-therapeutics-inc-class-action. Investors have until August 25, 2025, to as ...

美联储政策与市场预期 - 美联储预计维持利率不变 联邦基金期货显示98%概率维持现有利率 [3] - 特朗普持续施压美联储降息 甚至考虑解雇鲍威尔 但市场反应有限 [4] - 财政部长贝森特多次安抚市场 强调美联储主席不会被解雇 以稳定长期国债收益率波动 [5] - 德意志银行指出 市场已定价2026年8月前累计降息200基点 历史显示此类幅度通常伴随经济衰退 [7] 科技巨头财报前瞻 - **苹果(AAPL)**：预计EPS 1.43美元 营收888.9亿美元 投资者关注服务业务增长及iPhone销售放缓影响 [8] - **微软(MSFT)**：预计EPS 3.38美元 营收738亿美元 增长动力来自AI产品生态、云服务及数据中心投资 [9] - **Meta(META)**与**亚马逊(AMZN)**将于周三、周四公布财报 同期还有高通、Robinhood等公司业绩 [7][10] 欧美贸易协议 - 美欧达成协议 对多数欧盟出口商品征收15%关税 避免原定周五生效的更高关税 [11] - 特朗普最初提议50%关税 后降至30% 最终谈判达成15%的折中方案 [12] 财政政策与民意 - 特朗普新税法被52%民众反对 被认为偏向富人和大企业 预计未来十年增加3.4万亿美元赤字 [13][14] 生物医药事件 - Sarepta Therapeutics声明巴西患者死亡与基因疗法Elevidys无关 合作伙伴罗氏亦确认该结论 [15] 股息动态 - 蒙特利尔银行(BMO)周三除息 8月26日派息 达美航空(DAL)与摩根士丹利(MS)周四除息 [15][16] - 好市多(COST)周五除息 派息日为8月15日 [16]

NEW YORK, July 26, 2025 (GLOBE NEWSWIRE) -- Leading securities law firm Bleichmar Fonti & Auld LLP announces that a lawsuit has been filed against Sarepta Therapeutics, Inc. (NASDAQ: SRPT) and certain of the Company’s senior executives for potential violations of the federal securities laws. If you invested in Sarepta, you are encouraged to obtain additional information by visiting: https://www.bfalaw.com/cases-investigations/sarepta-therapeutics-inc-class-action. Investors have until August 25, 2025, to as ...

Sarepta Therapeutics发布澄清公告：8岁儿童的死亡与本公司基因疗法Elevidys无关。 6月18日已经向美国食品药品管理局（FDA）通报了这起事故。 ...

On Friday, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion on the conditional marketing authorisation (CMA) for Elevidys (delandistrogene moxeparvovec) for ambulatory individuals aged three to seven years with Duchenne muscular dystrophy (DMD).Sarepta Therapeutics, Inc. SRPT and Roche Holdings AG RHHBY are collaborating to develop and commercialize gene therapy.Also Read: Elevidys Halt Sparks Analyst Downgrades—Sarepta Races To Revis ...