结果显示，在人源化小鼠模型和非人灵长类动物中，经过 AERA-109 治疗后， 血液和组织中的 B 细胞被 深度耗竭。Aera 公司计划在 2026 年年中推进 AERA-109 进入临床开发阶段。 AERA-109 利用专有的 靶向脂质纳米颗粒 （tLNP） 递送平台和 CAR-T 技术，直接在体内靶向重编程 CD8 + T 细胞，为严重自身免疫疾病提供了一种潜在疗法，具有更高的精准度和更好的安全性。 撰文丨王聪 编辑丨王多鱼 排版丨水成文 2025 年 9 月 24 日，在 第十届 CAR-TCR 峰会上， Aera Therapeutics 展示了其候选疗法 AERA-109 的临床前数据，这是一款 in vivo CAR-T 细胞疗法 ，旨在治疗多种 B 细胞介导的自身免疫疾病 （例如系 统性红斑狼疮） 。 Aera Therapeutics 由 CRISPR 基因编辑先驱 张锋 教授等人创立，该公司于 2023 年宣布完成 1.93 亿美 元融资并正式亮相 。 创始团队还包括 RNAi 疗法巨 头 Alnylam 公司前高管 Akin Akinc 和前创始 CEO John Maraganore 。 ...

Core Viewpoint - Avidity Biosciences is experiencing significant market interest due to potential acquisition talks with Novartis, leading to a notable increase in its stock price by 26% [3][4]. Group 1: Company Overview - Avidity Biosciences focuses on developing Antibody Oligonucleotide Conjugates (AOCs) for rare diseases, with a current market capitalization of $5.8 billion [4]. - The company has a strong cash position of approximately $1.4 billion, providing funding into mid-2027 [7]. - Avidity is advancing three AOCs in clinical development, targeting Duchenne Muscular Dystrophy (DMD), Myotonic Dystrophy Type 1 (DM1), and Facioscapulohumeral Muscular Dystrophy (FSHD) [26][39]. Group 2: Clinical Development and Pipeline - Avidity's lead therapy, del-zota, for DMD has received breakthrough therapy designation from the FDA and is expected to submit a Biologics License Application (BLA) by the end of 2025 [5][7]. - The company is preparing for three planned BLA submissions within 12 months, with ongoing registrational trials [7]. - Avidity's AOC platform has successfully delivered RNA to muscle tissue, marking a significant advancement in RNA therapy [7][39]. Group 3: Recent Developments and Collaborations - Avidity recently entered a collaboration with Bristol Myers Squibb (BMS) for the development of up to five cardiovascular targets, with potential payments totaling $2.3 billion [18][19]. - The collaboration includes an upfront payment of $100 million and milestone payments based on development progress [19]. - Avidity's stock surged by 40% following the announcement of this partnership, reflecting strong market confidence in its pipeline and strategic direction [18][19].