The U.S. Food and Drug Administration (FDA) on Tuesday approved dosing in Sarepta Therapeutics Inc.’s (NASDAQ:SRPT) Cohort 8 of ENDEAVOR (Study 9001-103). • SRPT shares are advancing steadily. Get the market research here.The purpose of Cohort 8 is to evaluate the use of an enhanced immunosuppressive regimen as part of treatment with Elevidys (delandistrogene moxeparvovec-rokl) for non-ambulant (patients who cannot walk) individuals with Duchenne muscular dystrophy.Data from Cohort 8 will be used to determi ...

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 ...

Accessibility StatementSkip Navigation Of the 12 patients treated with escalating doses of REQORSA and standard doses of osimertinib, all of whom had progressed on osimertinib containing regimens, three patients had experienced prolonged time to progression, including one with continuing partial response. Specifically, one patient at the 0.06 mg/kg dose level, previously treated with carboplatin, pemetrexed and osimertinib, had a partial remission by investigator evaluation, and as of the data from April 20 ...

记者丨季媛媛 编辑丨张星 一家制药公司市值突破万亿美元大关，礼来刷新了行业纪录。然而，在专利到期、市场竞争加 剧以及价格压力之下，这家行业巨擘能否再创辉煌，寻找下一个媲美替尔泊肽的药品，成为了 无法忽视的潜在隐忧。 11月21日，美国制药巨头礼来股价攀升1.59%，收盘报1059.70美元/股， 市值成功突破1万亿 美元大关，成为全球首个市值跨越万亿美元里程碑的医药企业。 礼来市值跃升至万亿美元级别，得益于其旗舰产品替尔泊肽（tirzepatide）的强劲表现。据礼 来公布的第三季度财务报告显示，当季营收达到176亿美元，同比增长54%。在此之中，替尔 泊肽的降糖版本Mounjaro在第三季度实现了65.15亿美元的销售额，而前三季度的累计销售额 高达155.56亿美元。同时，替尔泊肽的减重版本Zepbound在第三季度取得了35.88亿美元的销 售额，前三季度累计销售额达到92.81亿美元。 从宏观市场结构审视，现阶段， GLP-1领域显现出鲜明的"双寡头"竞争态势。 面对潜力无限 的市场蓝海，众多国内创新制药公司正从多个维度着手，积极部署下一代GLP-1类药物的研 发。譬如，信达生物近期宣布，其独立研发的 ...

礼来市值的急剧膨胀，主要得益于其产品替尔泊肽的卓越销售业绩。这款GIP/GLP-1双重受体激动剂在 2023年获得减重适应症的批准后，便踏上了业绩的飞速增长之路。目前，在美国GLP-1类药物的处方量 中，替尔泊肽的占比已经超越了其老对手司美格鲁肽。据公开数据显示，截至2025年第三季度，替尔泊 肽在美国市场的处方量占比达到了57.9%，而司美格鲁肽则为41.7%。 然而，诺和诺德在减重药物领域的霸主地位依然坚如磐石，其凭借明星产品司美格鲁肽，继续稳居"药 界王者"之位。在2025年前三季度，诺和诺德的糖尿病及肥胖护理业务收入总计2156.61亿丹麦克朗（约 332.4亿美元），同比增长15%。在此之中，GLP-1类药物的营收为1745.77亿丹麦克朗（约269.04亿美 元），胰岛素产品的营收为397.36亿丹麦克朗（约61.24亿美元）。旗下Ozempic（司美格鲁肽注射液的 降糖版本）、Rybelsus（口服司美格鲁肽片）及Wegovy（司美格鲁肽注射液的减肥版本）三个品牌分别 礼来市值跃升至万亿美元级别，得益于其旗舰产品替尔泊肽（tirzepatide）的强劲表现。据礼来公布的 第三季度财务报告显示，当 ...

2025 年 11 月 23 日 生物医药Ⅱ 新药周观点：小核酸药物治疗乙肝取 得突破性数据 本周新药行情回顾： 2025 年 11 月 17 日-2025 年 11 月 23 日，新药板块涨幅前 5：来凯医 药（+13.34%）、北海康成（+2.67%）、嘉和生物（+2.19%）、永泰生 物 （ +1.94% ）、 友 芝 友 （ +1.14% ）； 跌 幅 前 5 ： 药 明 巨 诺 （ - 15.75%）、艾迪药业（-14.56%）、盟科药业（-14.34%）、宜明昂科 （-13.40%）、凯因科技（-13.04%）。 本周建议关注标的： 考虑板块后续仍有多个催化值得期待，包括学术会议、数据读出、 多个 BD 兑现等，仍建议关注： 1）已获 MNC 认证未来海外放量确定性高的品种：三生制药、联邦制 药、科伦博泰等； 2）存在海外数据催化的品种：贝达药业、和黄医药、映恩生物等； 3）下一个可能海外授权 MNC 的重磅品种：复宏汉霖、石药集团、益 方生物等； 4）新的创新药技术突破领域：小核酸、体内 CAR-T、减脂增肌、自 免 CAR-T/双抗、基因疗法等。 本周新药行业重点分析： 小核酸药物治疗乙肝取得 ...

Core Insights - Bayer has received European Commission approval for elinzanetant, branded as Lynkuet, to treat moderate to severe vasomotor symptoms (VMS) associated with menopause or adjuvant endocrine therapy related to breast cancer [1][7] - The approval is based on positive results from the OASIS program, which demonstrated a favorable safety profile and met all primary and key secondary endpoints [2][7] - Bayer's shares have increased by 62.1% year to date, significantly outperforming the industry average gain of 14.3% [2] Product Details - Elinzanetant is a dual neurokinin-targeted therapy, specifically an NK-1 and NK-3 receptor antagonist, and is the only hormone-free treatment for moderate to severe VMS [4] - VMS affects up to 80% of women during menopause, with approximately 40% of women in Europe reporting moderate to severe symptoms [4][5] - Lynkuet is already approved in the UK and the US for treating VMS associated with menopause [5] Portfolio Expansion - The approval of Lynkuet enhances Bayer's pharmaceutical portfolio, adding to the momentum of existing drugs like Nubeqa and Kerendia [2][9] - Bayer's Pharmaceuticals division is experiencing growth, compensating for declines in sales of Xarelto, which is co-developed with Johnson & Johnson [8] - Bayer is also working on label expansions for Nubeqa and Kerendia, which could further drive sales growth [9] Pipeline Development - Bayer is making progress in its pipeline, with a new drug application for gadoquatrane accepted for review in the US and China [10] - The company is expanding its capabilities in cell and gene therapy through acquisitions of BlueRock and AskBio, targeting various diseases [11]

Pacira BioSciences (NasdaqGS:PCRX) 2025 Conference November 18, 2025 08:30 AM ET Company ParticipantsFrank Lee - CEOConference Call ParticipantsNone - AnalystFrank LeeIf you know a little bit about Pacira, you know a little bit about Pacira. You know that we're focused on musculoskeletal pain and adjacencies. We have three products: Exparel, our flagship product, which is a nerve block; Zilretta, a long-acting steroid for osteoarthritis; and Iovera, which is a medical device that uses cryoneurolysis to alle ...

Core Insights - The 7th Guangdong-Hong Kong-Macao Greater Bay Area Biopharmaceutical Innovation Conference emphasizes the theme "Bay Area Innovation Navigates New Journey," focusing on discussions around innovative drug development, technology transfer, global compliance, and AI empowerment in new drug development [1][5] Group 1: Industry Trends - China's pharmaceutical industry is transitioning from a focus on generic drugs to early-stage innovation represented by Me too drugs and biosimilars, facing challenges such as insufficient payment systems, difficulties in international expansion, and limited capital patience [2] - The Guangdong province has reduced drug approval times from 200 working days to 60, supporting innovative drug projects with comprehensive policy assistance, resulting in 27 approved national Class 1 innovative drugs, ranking third in the country [2] - The Greater Bay Area has seen 14 overseas authorizations by Guangdong biopharmaceutical companies in 2024, accounting for nearly 16% of the national total, indicating initial success in internationalization [2] Group 2: Company Innovations - Dongyang Sunshine Pharmaceutical has developed multiple technology platforms and is transitioning from "following" to "leading" in the market, exemplified by a $1 billion licensing deal with UK-based Apollo for HEC88473 [3] - Kangfang Biopharma has achieved breakthroughs in tumor immunotherapy and set records for international licensing, including a $5 billion deal for its drug with Summit in the U.S., marking a significant milestone for Chinese innovative drugs [3] - Micron Biotech highlights that over 50% of IND approvals from the FDA in 2024 are from China, with 30% of global license-outs originating from the country, showcasing a shift from "following" to "reshaping the global landscape" [4] Group 3: Technological Advancements - AI is evolving from a supportive tool to a core engine in drug development, with companies like Baidu Biotech achieving significant improvements in research efficiency through AI collaborations [4] - The FDA's approval process for gene therapies reflects the balance between unmet clinical needs and safety concerns, emphasizing the importance of maintaining scientific rigor and patient safety in innovation [4] - The conference illustrates the confidence and capability of China's pharmaceutical innovation to transition from local to global markets, driven by policy support, technological breakthroughs, and corporate practices [5]

Core Insights - The ophthalmic CGT (cell and gene therapy) sector is emerging as a new focus for pharmaceutical companies, with significant investments from major players like Eli Lilly, who recently made two strategic acquisitions in this field [1][2][3] Group 1: Eli Lilly's Strategic Moves - Eli Lilly has made two notable transactions: acquiring Adverum for up to $262 million for the wet age-related macular degeneration (wAMD) gene therapy Ixo-vec, and partnering with MeiraGTx for a $75 million upfront payment and up to $475 million in milestone payments for a gene therapy targeting Leber congenital amaurosis type 4 (LCA4) [1][2] - The collaboration with MeiraGTx focuses on the AAV-AIPL1 gene therapy, which has shown significant vision improvement in all 11 treated LCA4 children, highlighting its potential in treating severe inherited retinal diseases [2][3] - The acquisition of Adverum reflects Eli Lilly's ambition in the common eye disease market, as wAMD is a leading cause of blindness among the elderly, with 190 million patients globally in 2020 [3] Group 2: The Unique Advantages of Ophthalmic CGT - The eye is considered an "immune-privileged organ," making it a suitable target for gene therapy, as many blinding eye diseases are caused by single-gene mutations, providing clear therapeutic targets [4][5] - The success of Spark Therapeutics' Luxturna, the first FDA-approved ophthalmic gene therapy, has validated the effectiveness of AAV vectors in this field, leading to increased interest from various pharmaceutical companies [5][6] - Over 10 ophthalmic AAV gene therapies are currently in phase III clinical trials, with a balanced focus on both rare and common diseases [6] Group 3: Market Dynamics and Future Outlook - The commercial viability of high-priced CGT therapies is supported by the robust payment systems for rare diseases in the U.S., making it feasible for companies like Eli Lilly to invest heavily in this market [8][9] - The potential for gene therapies to address unmet clinical needs in retinal diseases, despite existing treatments, positions them favorably for commercialization [8][9] - The upcoming years are expected to be a critical window for the ophthalmic CGT industry, with multiple therapies poised to enter the market, potentially reshaping the landscape [11]