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糖尿病治愈新希望:CRISPR基因编辑细胞在人体内分泌胰岛素,且无需使用免疫抑制药物
生物世界· 2025-09-06 04:05
Core Viewpoint - The research led by Sana Biotechnology offers a potential breakthrough in treating Type 1 Diabetes (T1D) by enabling the survival of transplanted allogeneic beta cells without the need for immunosuppression, which could significantly improve patient outcomes and reduce the risks associated with long-term immunosuppressive therapy [2][4][7]. Group 1: Research Findings - A study published in NEJM demonstrated that CRISPR-Cas12b gene editing was used to modify donor beta cells to avoid immune rejection, allowing these cells to be transplanted into a Type 1 Diabetes patient without immunosuppressive drugs [2][4]. - The patient showed no immune response to the transplanted cells within 12 weeks, and the cells continued to produce insulin, effectively regulating blood sugar levels [2][9]. - This approach represents a significant advancement towards achieving a long-term cure for T1D, with the potential for a one-time treatment that eliminates the need for insulin injections and immunosuppressive medications [9][10]. Group 2: Comparison with Other Companies - Other companies, such as Vertex and Reprogenix, are also exploring stem cell-derived therapies for T1D, but these methods still require immunosuppressive drugs to prevent immune attacks on either donor or patient-derived cells [6][7]. - Vertex's research involved transplanting stem cell-derived islets into 12 patients, with 10 no longer needing insulin after one year, while Reprogenix successfully reprogrammed patients' own fat cells into insulin-producing cells [6][7]. - Unlike these approaches, Sana's method aims to eliminate the need for immunosuppression entirely, which could revolutionize treatment for T1D [7][9]. Group 3: Future Directions and Challenges - The ultimate goal of Sana's research is to apply the "immune stealth" gene editing technique to stem cells, guiding their development into insulin-secreting beta cells, with clinical trials expected to begin next year [9][10]. - However, there are concerns regarding the reproducibility of the protective effects of CD47 and the limited scope of the initial study, which involved only one patient and a small number of transplanted cells [10].
首发|宁美浩维获数千万天使轮融资,细胞+基因双引擎驱动精准医疗新纪元
Sou Hu Cai Jing· 2025-05-12 00:21
Core Viewpoint - Beijing Ningmei Haowei Biotechnology Co., Ltd. has completed tens of millions in angel round financing to support research and industrialization in gene-edited cell therapy, focusing on clinical applications in precision anti-aging and chronic disease treatment such as diabetes [1][4] Group 1: Company Overview - Ningmei Haowei was founded in 2015 and aims to provide comprehensive precision medical and health management solutions from disease prevention and diagnosis to treatment, leveraging cutting-edge life science technologies [1] - The founding team consists of top domestic and international experts in life sciences and clinical medicine, with over a decade of experience in cell research and gene therapy, achieving multiple breakthrough research results [1] Group 2: Precision Medical Ecosystem - The company has established a precision medical ecosystem driven by "cell + gene" technology, covering the entire lifecycle [3] - Precision Anti-aging: Offers personalized anti-aging solutions through cell preparation and immune cell storage services based on leading global cell technology research and industrialization platforms [3] - Chronic Disease Treatment: Actively promotes stem cell therapy for major chronic diseases such as diabetes, cardiovascular diseases, and neurological disorders, collaborating with clinical institutions for efficacy evaluation to ensure treatment safety and durability [3] - Tumor Treatment: Focuses on gene-reprogrammed cell technology to create a comprehensive tumor cell treatment system, including personalized cell bank establishment, immune cell and stem cell differentiation preparation, and engineered cell line technology [3] Group 3: Quality Control and Future Plans - Ningmei Haowei has a strict quality control system ensuring safe cell sources with low immunogenicity and no potential infectious diseases or cancer risks, with cells stored in national authoritative institutions using top global storage equipment [4] - The company is equipped with GMP-grade independent laboratories and top-tier R&D, production, and quality inspection equipment, ensuring a sterile operating environment and automated, closed cell culture systems [4] - The recent financing will inject strong momentum into the company's R&D in cell therapy and gene editing technology, accelerating the transition of innovative therapies from laboratory research to clinical applications [4] - Future development plans include expanding the application scenarios of stem cell therapy in precision anti-aging and chronic disease treatment, continuously deepening the precision medical sector, and promoting the industrialization of cell therapy technology [4]