Key Takeaways AbbVie is acquiring Capstan Therapeutics for up to $2.1B to expand its immunology portfolio. The acquisition adds CPTX2309, a phase I in vivo CAR-T therapy for autoimmune diseases, to AbbVie's pipeline. Capstan's CellSeeker tLNP platform enables RNA delivery to engineer specific cell types inside the body.AbbVie (ABBV) announced that it has entered into a definitive agreement to acquire privately held biotech, Capstan Therapeutics, for up to $2.1 billion in cash.The impending acquisition wil ...

日前， 艾伯维 （AbbVie） 宣布 全力进军 in vivo CAR-T 市场，将斥资 21 亿美元现金 收购 Capstan Therapeutics ，以期将一款处于 1 期临床实验阶段的自 身免疫疾病候选 invivo CAR-T 细胞疗法 （ CPTX2309 ） 纳入其研发管线。 撰文丨王聪 编辑丨王多鱼 排版丨水成文 创立于 2022 年的 Capstan 公司，致力于通过在体内 （ in vivo ） 而非体外重新编程 T 细胞，从而清除阻碍 CAR-T 细胞疗法普及面临的制造和可扩展性方面 的挑战，且患者在治疗前无需接受淋巴细胞清除化疗。 Capstan 公司创立始于 2022 年初的一篇论文。 体内生成 CAR-T 细胞，治疗心脏损伤 2022 年 1 月 6 日，宾夕法尼亚大学的研究人员在 Science 期刊发表了题为： CAR T cells produced in vivo to treat cardiac injury 研究论文 【1】 ，开发了一 种 在体内原位生成 CAR-T 细胞的新技术 （ in vivo CAR-T） ，通过 注射脂质纳米颗粒 （LNP） 递送 mRN ...

Preclinical studies achieved therapeutically relevant gene editing levels of the HBG1/2 promoter & favorable biodistribution profile in non-human primates using a clinically validated editing strategy Data reinforces continued development as a potentially transformative, in vivo approach to treating sickle cell disease and beta thalassemia CAMBRIDGE, Mass., May 14, 2025 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company, today announced that new data from a study in ...

Summary of Editas' Conference Call Company Overview - **Company**: Editas Medicine - **Industry**: Biotechnology, specifically focusing on gene editing and therapy Key Points and Arguments Focus on In Vivo Development - Editas aims to be a premier in vivo gene editing company, refining its focus since the CEO's arrival three years ago [5] - The company has generated promising data in in vivo gene editing, particularly for sickle cell disease, which is seen as a significant opportunity [6][7] Market Potential and Treatment Efficacy - In vivo gene editing is expected to expand the market and reduce patient burden compared to ex vivo therapies [6] - The company believes that the efficacy bar for in vivo treatments is on par or better than ex vivo options, allowing for a larger patient population [9] Preclinical Data and Optimization - Editas is applying learnings from its ex vivo program to optimize its in vivo gene editing approach, focusing on delivery mechanisms [10][11] - Proprietary TLNP technology is showing great potential in preclinical settings [11] Business Development and Pipeline Expansion - The company is exploring additional investments and partnerships to expand its pipeline, particularly in in vivo applications [13][17] - Editas is focused on being best-in-class in vivo gene therapy, with a strategic approach to target functional proteins [15][16] Financial Position and Capital Efficiency - Editas has a cash runway extending into Q2 2027, providing breathing room to advance its in vivo programs [19][21] - The company is being capital efficient, particularly in managing wind-down costs from previous programs [26] Licensing Agreements and Intellectual Property - Current licensing agreements remain unaffected by recent legal decisions, and Editas is open to new licensing opportunities [28][29] Upcoming Data and Milestones - Editas presented data at ASGCT, showing over 80% reduction in a disease biomarker in its liver program [32] - The company plans to declare two drug candidates by mid-2025 and file an IND for one candidate in 2026, with potential human proof of mechanism expected by late 2026 to early 2027 [36] Conclusion - Editas is focused on advancing its in vivo gene editing capabilities while maintaining a strong financial position and exploring new partnerships to enhance its pipeline [41]