当司美格鲁肽、替尔泊肽靠着 "减肥神药" 的光环席卷全球市场，靶点、剂型、疗效升级不断，另一群 糖尿病患者的困境似乎被掩盖在这场"技术狂欢"之下—— 1型糖尿病（T1D）患者，全球约915万人，每天必须与胰岛素注射笔、血糖监测仪为伴，稍有不慎就可 能因严重低血糖或高血糖并发症危及生命。 直到几个月前，Vertex在ADA年会上抛出的一组数据，将这一领域推到聚光灯下：其干细胞疗法 Zimislecel治疗1型糖尿病的1/2期临床中，83%（10/12）患者在注射一年后完全摆脱胰岛素依赖，且未 再发生致命性低血糖。 更关键的是，这款疗法无需依赖稀缺的捐献胰岛，而是通过干细胞体外分化生成胰岛，一旦 2026年顺 利申报上市，有可能彻底改写1型糖尿病的治疗格局。 尽管还有待最终临床数据的确认，以及Zimislecel与免疫抑制剂长期共同使用带来的安全性问题，但其 仍是对一个百年未破的治疗困局的正面冲击，让患者看到"功能性治愈"的曙光。 那么，这款被寄予厚望的疗法，真能掀翻1型糖尿病治疗的旧桌子吗？ 01 915万患者的临床困境 当我们谈及糖尿病，近几年的焦点几乎全被2型糖尿病的 "减肥神药" 占据。司美格鲁肽上市后迅 ...

Core Insights - CRISPR Therapeutics is at a pivotal moment, having developed revolutionary gene editing technology that has the potential to deliver life-changing therapies, with the focus now on replicating success across its pipeline [1][2] - The company has experienced significant stock decline, over 80% from its peak in 2021, but is now positioned for potential growth with the commercialization of its first therapy [2][12] Company Developments - The first therapy, Casgevy, has been approved for treating sickle cell disease and transfusion-dependent beta thalassemia, marking a significant milestone as the first CRISPR-based cell therapy approved by the FDA [4] - CRISPR Therapeutics has begun selling Casgevy, with revenue projections of $45 million for the current year and $214 million by 2026 [5] Pipeline and Future Potential - The company has five therapies in clinical trials and an additional ten in preclinical stages, targeting major health issues such as cancers, cardiovascular diseases, and type 1 diabetes [7] - The potential for high returns exists if the company successfully commercializes additional therapies, as the gene editing market offers vast opportunities [10][13] Financial Position - CRISPR Therapeutics maintains a strong cash position with $1.85 billion available, allowing it to sustain operations for years without needing additional funding [9] - The current enterprise value is approximately $1.2 billion, equating to about six times next year's revenue estimates, indicating a potentially attractive entry point for investors [12] Investment Considerations - The company wholly owns its five clinical therapies, meaning it stands to gain fully from any successful commercialization, which could lead to significant investment returns [13] - While there are risks associated with drug development, the potential for blockbuster therapies could position CRISPR Therapeutics favorably in the market [14]