这些细胞不仅在数月内持续分泌胰岛素，还能逃避免疫检测，使植入接受者无需服用免疫抑制剂。 此研究于已发表在《新英格兰医学杂志》上，并作为新闻报道登上了Nature官网。 那么，这项突破究竟是如何实现的呢？ CRISPR基因编辑为治疗糖尿病带来希望 糖尿病治疗迎来新突破！ 最新研究显示，科学家首次将CRISPR编辑的胰岛细胞移植到一名1型糖尿病患者体内。 1型糖尿病是一种自身免疫性疾病，患者的免疫系统会错误地攻击胰腺中分泌胰岛素的细胞。 一旦胰岛素"停产"，身体里的血糖就会失控。 如果不通过注射等方式补充胰岛素，此疾病可能会损害神经和器官，特别是心脏、肾脏和眼睛。 针对这一问题，华盛顿西雅图Sana Biotechnology公司提出了一项新研究，为全球约950万1型糖尿病病人带来了治愈的希望。 首先，在乌普萨拉大学医院，研究人员从一名没有糖尿病的60岁遗体捐赠者体内提取出胰岛细胞。 随后，这些细胞被运送至奥斯陆大学医院，利用CRISPR-Cas12b基因编辑技术进行改造。 主要做法为敲除了两个关键基因——B2M（I类HLA）和CIITA（II类HLA），这两个基因通常会向T细胞（免疫系统的前线防御者）标记外来入 ...

时令 发自 凹非寺 量子位 | 公众号 QbitAI 糖尿病治疗迎来新突破！ 最新研究显示，科学家首次将 CRISPR编辑的胰岛细胞 移植到一名1型糖尿病患者体内。 这些细胞不仅在数月内持续分泌胰岛素，还能 逃避免疫检测 ，使植入接受者无需服用免疫抑制剂。 此研究于已发表在《新英格兰医学杂志》上，并作为新闻报道登上了Nature官网。 那么，这项突破究竟是如何实现的呢？ 针对这一问题，华盛顿西雅图 Sana Biotechnology公司 提出了一项新研究，为全球约950万1型糖尿病病人带来了治愈的希望。 首先，在乌普萨拉大学医院，研究人员从一名没有糖尿病的60岁遗体捐赠者体内提取出胰岛细胞。 随后，这些细胞被运送至奥斯陆大学医院，利用CRISPR-Cas12b基因编辑技术进行改造。 主要做法为 敲除了两个关键基因 ——B2M（I类HLA）和CIITA（II类HLA），这两个基因通常会向T细胞（免疫系统的前线防御者）标记外来 入侵者。 CRISPR基因编辑为治疗糖尿病带来希望 1型糖尿病是一种 自身免疫性疾病 ，患者的免疫系统会错误地攻击胰腺中分泌胰岛素的细胞。 一旦胰岛素"停产"，身体里的血糖就会失控。 如 ...

Sana has also significantly expanded its financial runway by raising funds through an at-the-market sale and a public equity offering. It ended the second quarter with $72.7 million in cash, which increased to a pro forma $177.2 million after the capital raises. The company expects this to fund operations until the second half of 2026. Sana's breakthrough for diabetes is one of the most promising initiatives to date in developing a functional treatment that does not require lifelong immunosuppression. This ...

Summary of Sana Biotechnology FY Conference Call Company Overview - **Company**: Sana Biotechnology (NasdaqGS:SANA) - **Focus**: Developing engineered cells as medicines, specifically targeting type 1 diabetes and allogeneic CAR-T therapies [2][19] Key Platforms - **HypoImmune Platform**: Aims to overcome allogeneic rejection of cells by modifying immune response through gene editing [2][3] - **Fusogen Platform**: A cell-specific in vivo delivery system designed to deliver genetic material directly to target cells without lymphodepletion [16][17] Type 1 Diabetes Focus - **Unmet Need**: Approximately 9 million people suffer from type 1 diabetes, with significant health risks and no meaningful change in standard care for over 100 years [5][6] - **Clinical Data**: Recent proof of concept study published in The New England Journal of Medicine shows promising results for HypoImmune-modified islet cell therapy, with patients producing insulin for the first time in over 30 years [10][19] - **Study Design**: Cadaveric islets were genetically modified and transplanted without immunosuppression, achieving all primary and secondary endpoints [9][10] Clinical Results - **C-peptide Levels**: Patients showed stable C-peptide levels, indicating insulin production, with significant improvement in response to meals over six months [10][11] - **Immune Evasion**: HypoImmune-modified islets demonstrated survival against pre-existing immune responses, with no T cell or antibody recognition [12][14] Allogeneic CAR-T Programs - **Programs in Development**: - SC291: HypoImmune-modified CD19-directed CAR-T for autoimmune diseases [15] - SC262: CD22-directed allogeneic CAR-T for patients relapsing post-CD19 CAR-T therapy [16] - **Clinical Trials**: Ongoing studies with expected data release in 2025 [16] Fusogen Platform Development - **Lead Product Candidate**: SG299, a CD8 targeted fusosome for delivering CD19-directed CAR-T cells, with IND filing expected as early as 2026 [18][19] Conclusion - **Progress**: Sana Biotechnology is making significant strides in overcoming allogeneic rejection and advancing its CAR-T and Fusogen platforms, with broad applicability across various diseases anticipated [19]

PresentationSo with that, I'd like to introduce Steve Harr. And for those in the audience who aren't familiar, maybe Steve, if you can give us an introduction on Sana and key takeaways.Before we get started, I just want to read a brief disclosure. For important disclosures, please see the Morgan Stanley research disclosure website at www.morganstanley.com/researchdisclosures. If you have any questions, please reach out to your Morgan Stanley sales representative.Steven HarrPresident, CEO & Director Sure. Fi ...

Sana Biotechnology (NasdaqGS:SANA) FY Conference September 08, 2025 11:30 AM ET Company ParticipantsMaxwell Skor - VP - Biotech Equity ResearchSteve Harr - President, CEO & DirectorMaxwell SkorHello everyone. I'm Maxwell Score, biotech analyst with Morgan Stanley. I'm happy to host Sana Biotechnology and Steve Harr, CEO. Before we get started, I just want to read a brief disclosure. For important disclosures, please see the Morgan Stanley Research Disclosure website at www.morganstanley.com/researchdisclosu ...

撰文丨王聪 编辑丨王多鱼 排版丨水成文 2025 年 8 月，国际顶尖医学期刊《 新英格兰医学杂志 》 （NEJM） 发表了一篇重磅论文 ： Survival of Transplanted Allogeneic Beta Cells with No Immunosuppression （无需免疫抑制的同种异体 胰岛β细胞移植存活 ） 【1】 。 在异体细胞移植后，需要抑制患者的免疫系统，而这会带来广泛的副作用。在这项研究中， 研究人员 使用 CRISPR-Cas12b 基因编辑了来自捐赠者的 胰岛 β 细 胞以避免排斥反应，并将这些细胞注射移植到一名患有 1 型糖尿病 的男性患者 前臂肌肉中 ，且 患者未接受任何 免疫抑制药物，移植后 12 周内，未出现对这些 基因编辑细胞的免疫反应，这些细胞在体内稳定且持续分泌着调控 血糖的胰岛素。 其中， Vertex 公司进展最快。今年 6 月，该公司在 《 新英格兰医学杂志 》 （NEJM） 发表了一篇论文： Stem Cell–Derived, Fully Differentiated Islets for Type 1 Diabetes （ 干细胞来源的完全分化 ...

Core Insights - The company believes that cell and gene therapy will be a significant transformation in medicine over the coming decades [1] - The industry has faced challenges, but the company remains optimistic about its progress and future developments [1] Company Perspective - The CEO emphasizes the importance of understanding stock catalysts while focusing on building the business [1] - The company is committed to navigating the complexities of the cell and gene therapy field [1]

Financial Data and Key Metrics Changes - The company is focused on cell and gene therapy, which is expected to transform medicine over the coming decades [6] - The diabetes program is highlighted as potentially one of the most valuable therapies in development, with a large and unsatisfied market [19] Business Line Data and Key Metrics Changes - The company is advancing three categories of therapies: type one diabetes, in vivo CAR T cells, and allogeneic CAR T cells [10][17] - The type one diabetes program aims to provide a single treatment that allows patients to maintain normal blood sugar levels without insulin or immunosuppression [14][19] Market Data and Key Metrics Changes - Type one diabetes affects over nine million people, with estimates suggesting it will grow to fifteen million within fifteen years [12] - The company is optimistic about the scalability of its therapies, particularly in the context of the growing diabetes market [19] Company Strategy and Development Direction - The company aims to overcome allogeneic rejection in cell therapies, which has been a significant challenge in the field [7][17] - The focus is on developing scalable and effective therapies that can be broadly accessible to patients worldwide [62] Management's Comments on Operating Environment and Future Outlook - Management acknowledges the challenges in the cell and gene therapy space but remains optimistic about the potential of their diabetes program [75] - The company is preparing for an IND filing as early as 2026, contingent on successful completion of necessary studies [58][60] Other Important Information - The company has made significant progress in gene editing and manufacturing processes, which are critical for the success of their therapies [46][48] - The management emphasizes the importance of human data to unlock value in their programs, particularly for CAR T therapies [73] Q&A Session Summary Question: Can you provide an overview of the type one diabetes program and recent data? - The type one diabetes program has shown promising results, with the first patient treated demonstrating functioning cells for over six months [24][28] Question: What are the next steps for the iPSC product? - The company is focused on finalizing the master cell bank and completing the necessary toxicology studies before filing for IND [50][55] Question: How does the company plan to address the challenges in manufacturing stem cell-derived products? - The management highlighted the importance of purity, potency, and yield in manufacturing, which are critical for safety and efficacy [52][53] Question: What is the company's strategy for partnerships in the CAR T space? - The company is exploring partnerships to advance its CAR T programs, recognizing the need for capital to move forward [75][78]

Core Insights - The company has been operational for approximately 6.5 years, indicating a relatively young age in the industry while having gained valuable experience through successes and mistakes [1] Company Vision - The company was founded with the vision that cell modification will be a significant advancement in medicine over the coming decades, suggesting a long-term commitment to this innovative approach [1] - Despite current skepticism in the industry regarding cell modification as a viable treatment, the company remains confident in its potential and the likelihood of its success [1]