Core Viewpoint - The article discusses the successful transplantation of genetically edited pig lungs into a brain-dead human, marking a significant advancement in xenotransplantation, which aims to address the global organ shortage crisis [1][5][18]. Group 1: Background and Importance of Xenotransplantation - Approximately 2 million people worldwide require organ transplants each year, but only about 10% receive suitable organs, highlighting the critical shortage of transplantable organs [5]. - Xenotransplantation, particularly using pig organs, is considered a promising solution to the organ shortage problem [5][9]. Group 2: Research and Development - The research team led by Professor He Jianxing from Guangzhou Medical University successfully transplanted a genetically edited Bama pig's left lung into a brain-dead patient, maintaining ventilation and gas exchange for 9 days [5][18]. - Since 2021, several cases of pig-to-human organ transplants have been reported globally, involving various organs such as hearts, kidneys, and livers [7]. Group 3: Advantages of Using Pigs - Pigs are preferred over primates for organ donation due to their abundant resources, smaller size, high survival rates, and genetic stability, along with significant anatomical and physiological similarities to humans [9][10]. - The evolutionary distance between pigs and humans reduces the risk of zoonotic diseases and ethical concerns associated with using primates [9]. Group 4: Challenges in Xenotransplantation - Despite the advantages, xenotransplantation faces challenges such as cross-species rejection and the risk of infections, which have historically hindered research progress [10][18]. - Recent advancements in gene editing technologies, such as CRISPR/Cas9, have enabled the development of genetically modified pigs that minimize immune rejection and viral infection risks [10][12]. Group 5: Clinical Trials and Future Prospects - Currently, xenotransplantation trials are primarily conducted in China and the United States, with significant milestones achieved in heart and kidney transplants [14][16]. - The recent lung transplant case represents a critical step towards clinical applications, although experts emphasize the need for cautious progression through further trials and research [18][19].

Core Viewpoint - Tibet Pharmaceutical plans to establish a wholly-owned subsidiary, TopRidge Pharma Limited, in Hong Kong to enhance sustainable development and overcome research and development bottlenecks [1] Group 1: Company Overview - Accuredit Therapeutics Limited focuses on developing in vivo gene editing technologies and products based on LNP and other non-viral vectors, aiming to provide innovative treatment solutions that require only a single administration for patients globally, with cost advantages [1] - Ruizheng Gene (Suzhou), established in 2021, specializes in the development, industrialization, and commercialization of in vivo gene editing drugs based on non-viral vectors, supported by a core team with successful experience across the entire biopharmaceutical cycle [1] Group 2: Product Pipeline - Key products under development by Ruizheng Gene include ART001, targeting transthyretin amyloidosis (ATTR), and ART002, aimed at familial hypercholesterolemia, with additional products focusing on metabolic diseases, particularly in the liver disease sector [1]

Core Insights - The successful transplantation of genetically edited pig lungs into a human patient marks a significant milestone in the field of xenotransplantation, addressing the critical shortage of human lung donors [2][4][10] - The research indicates that the genetically modified pig lung can survive and function for 9 days in a human body, demonstrating the potential for clinical applications in the future [5][8] Group 1: Research Breakthrough - The study published in "Nature Medicine" details the process of transplanting a genetically edited pig lung into a 39-year-old male patient who was brain dead, with a total cold ischemia time of 206 minutes [4][5] - This is the first successful case of pig lung transplantation into a human, which is considered a major advancement in the scientific community [4][7] Group 2: Challenges and Future Directions - Despite the success, significant challenges remain, including immune rejection and the need for optimized immunosuppressive protocols [5][6][15] - The research team aims to enhance gene editing strategies and anti-rejection treatments to prolong the survival and functionality of transplanted organs [8][19] Group 3: Industry Context - The demand for transplantable organs far exceeds supply, with a projected donor-to-recipient ratio of 1:8 in China by 2024, highlighting the urgency for alternative sources like xenotransplantation [10][15] - The use of genetically modified pigs is seen as a promising solution to the organ shortage, as their organs are similar in size to human organs and can be produced without the same limitations as human donors [10][17] Group 4: Technological Advancements - The genetic editing technology used to create the donor pigs is at the forefront of scientific innovation, allowing for the removal of genes that trigger strong immune responses in humans [6][17] - Companies like Zhongke Aoge and Dashuo Group are leading efforts in developing these genetically modified pigs, contributing to China's position in the global xenotransplantation landscape [18][19]

Core Viewpoint - The article highlights the significant achievements of Shanghai Bangyao Biotechnology Co., Ltd. in the field of gene editing and cell therapy, particularly their recognition through the Shanghai Science and Technology Progress Award for their innovative projects in these areas [2][5]. Group 1: Award Recognition - The project "Development and Translational Application of Key Technologies in Gene Editing and Cell Therapy" won the first prize at the Shanghai Science and Technology Progress Award, marking a significant milestone for Bangyao Biotechnology [2][5]. - The award signifies the project's achievement at an internationally advanced or domestically leading level, emphasizing its substantial social and economic benefits [5][6]. Group 2: Technological Innovations - Bangyao Biotechnology is recognized as one of the earliest teams in China to apply CRISPR-Cas9 gene editing technology, having published the first successful application in mammals in 2013 [8]. - The company has developed a gene therapy for β-thalassemia, achieving a "one-time treatment, lifelong cure" for 15 patients, which has been acknowledged as a benchmark in the international field of thalassemia treatment [10]. - The non-viral PD1-CAR-T technology, developed by the team, has shown a 100% objective response rate and an 85.7% complete response rate in clinical studies, significantly improving safety and cost-effectiveness compared to traditional CAR-T therapies [12][15]. - The allogeneic universal CAR-T cell therapy, targeting CD19, has been developed to address the challenges of personalized treatment cycles and costs, allowing for immediate use and significantly reducing production costs [16][17]. Group 3: Clinical Research and Future Directions - Bangyao Biotechnology is conducting two registration clinical studies focusing on relapsed/refractory B-cell acute lymphoblastic leukemia and B-cell non-Hodgkin lymphoma, aiming to expand the application of their therapies to solid tumors and autoimmune diseases [22]. - The company has established a robust innovation platform with over 100 patents and multiple projects in clinical trials, demonstrating its commitment to advancing gene and cell therapy [25].

Core Viewpoint - The article highlights the significant breakthrough in gene editing therapy for β-thalassemia, particularly through the case of a patient named "Xixi," who was successfully treated using CRISPR technology, marking a milestone in the treatment of hereditary blood disorders [1][4]. Group 1: Patient Case Study - Xixi, diagnosed with the most severe form of β0/β0 thalassemia at 9 months old, required lifelong blood transfusions and iron removal therapy, placing a heavy burden on his family [3]. - In 2020, at the age of 7, Xixi became the first patient globally to receive CRISPR gene editing therapy (BRL-101) and successfully became independent from blood transfusions just 56 days post-treatment [6]. - Five years later, Xixi has maintained his health without transfusions, with hemoglobin levels around 140g/L, showcasing the long-term efficacy and safety of the treatment [6][10]. Group 2: Scientific Breakthrough - β-thalassemia is a hereditary blood disorder caused by defects in the globin gene, traditionally treated through costly and complex allogeneic stem cell transplants, which carry high risks [10]. - The BRL-101 gene therapy utilizes CRISPR technology to modify the BCL11A locus in the patient's hematopoietic stem cells, allowing for a one-time treatment that can potentially cure the disease [10]. - The therapy's delivery method avoids safety issues associated with viral vectors, enhancing its safety profile [10]. Group 3: Company Development and Achievements - Shanghai Bangyao Biotechnology has been deeply involved in gene therapy, with BRL-101 receiving IND approval in August 2022 and achieving significant clinical milestones since its inception [14]. - The company has published influential research in top journals and has been recognized at major international conferences, receiving awards for its contributions to rare disease treatment [15]. - Future plans include exploring treatments for sickle cell disease (BRL-102) and expanding global clinical collaborations to benefit patients with various hereditary blood disorders [14][17].

Core Viewpoint - Recent advancements in gene editing technology, particularly through pioneering editing techniques, show promise in treating severe neurological diseases, although significant technical and funding challenges remain to be addressed [1][4]. Group 1: Breakthroughs in Gene Editing - Harvard University and Jackson Laboratory successfully utilized pioneering editing technology to correct pathogenic gene mutations in a mouse model of Alternating Hemiplegia of Childhood (AHC), achieving an 85% mutation correction rate [2]. - The treatment led to significant improvements in the mice's brain function, reducing seizure frequency and doubling their lifespan, alongside enhancements in motor and cognitive abilities [2]. - A separate team, led by Professor Qiu Zilong, demonstrated the ability to reverse behavioral abnormalities in MEF2C mutation mice using base editing technology, which is crucial for addressing epilepsy and developmental disorders in children [2][3]. Group 2: Safety and Feasibility - The precision of gene editing technology allows for targeted correction of pathogenic mutations, making it an ideal treatment for neurodevelopmental disorders and autism in children [3]. - The pioneering editing technique requires only a single brain injection for treatment, with minimal off-target effects, confirming its safety and feasibility [3]. - The technology has shown the capability to simultaneously correct five mutations, indicating its broad applicability [3]. Group 3: Challenges Ahead - Despite promising results in mouse models, significant hurdles remain before gene editing can benefit human patients, including the need for advanced delivery systems to target brain cells effectively [4]. - The use of adeno-associated virus 9 (AAV9) as a delivery vehicle poses risks of severe immune reactions at high doses, necessitating the development of improved viral vectors and exploration of non-viral delivery methods [4]. - The biotechnology sector is currently facing a funding crisis, which complicates the lengthy and complex development processes for gene therapies, potentially deterring investors [5].

泓博医药（301230.SZ）8月12日在投资者互动平台表示，公司暂不涉及相关领域。 （文章来源：每日经济新闻） 每经AI快讯，有投资者在投资者互动平台提问：请问贵公司有无布局基因编辑技术，和哪些制药公司 有深度合作？ ...

Group 1: Industry Overview - The new gene editing technology developed by the Institute of Genetics and Developmental Biology of the Chinese Academy of Sciences allows precise manipulation of large DNA segments ranging from thousands to millions of bases, significantly enhancing the scale and capability of gene editing [1] - The gene editing market is projected to reach an annual revenue of $30 billion by 2030, indicating a strong growth trajectory [2] - Gene editing technology is transitioning from theoretical research to practical applications, with significant implications in medicine for treating genetic diseases and in agriculture for developing new crop varieties [2] Group 2: Company Developments - Approximately 20 listed companies in the A-share market are involved in gene editing technology [3] - A company has established a cotton research institute in collaboration with Huazhong Agricultural University, utilizing gene editing and hybridization techniques for cotton seed development [3] - Another company is conducting research on the water buffalo genome and has developed a breeding model based on whole genome data analysis to enhance breeding efficiency [3] - A biotechnology company focuses on providing gene-modified animal models and related technical services for drug development and disease research, having developed over 21,000 models by the end of 2024 [3] - Beijing Baipu Sais Biotechnology Co., Ltd. has launched CAS series proteins for targeted gene editing applications [4]

Core Viewpoint - The research team from Uppsala University in Sweden has successfully demonstrated the survival and functionality of donor pancreatic β cells obtained through CRISPR-Cas gene editing technology in a type 1 diabetes patient for 12 weeks without the use of immunosuppressants, indicating a potential breakthrough in curing type 1 diabetes [1] Industry Summary - Over 20 listed companies in the A-share market are involved in gene editing technology [1] - According to data from Zhitongcaijing, a total of 21 gene editing concept stocks have received "positive" ratings from institutions, including buy, increase, and strong recommendation ratings [1] - Among these, Shenguan Medical (300888) is the most favored by institutions, with 27 institutional research reports mentioning this stock [1]

Core Viewpoint - The research team from Uppsala University has successfully demonstrated the world's first instance of donor pancreatic β cells edited by CRISPR-Cas technology surviving and functioning for 12 weeks in a type 1 diabetes patient without the use of immunosuppressants, offering hope for a potential cure for the disease [1][2]. Group 1: Research Breakthrough - The study utilized CRISPR-Cas12b technology to modify donor pancreatic cells by knocking out B2M and CIITA genes to reduce immunogenicity and overexpressing CD47 through lentiviral transduction [1]. - The modified low-immunogenicity cells were implanted into a 42-year-old subject's left brachioradialis muscle, with the entire procedure conducted without glucocorticoids, anti-inflammatory drugs, or immunosuppressants [1]. Group 2: Clinical Outcomes - Monitoring over 84 days showed that the transplanted cells successfully evaded the immune system, with no T cell activation or antibody response observed [2]. - Imaging confirmed the good survival of the graft, and the patient's hemoglobin A1c levels significantly decreased by 42% [2]. - Although the number of transplanted cells was only 7% of what is needed for treatment, this experiment marks a critical breakthrough in demonstrating survival and functionality [2]. Group 3: Future Implications - The low-immunogenic gene editing technology used in this study may lead to the development of more curative β cells, potentially allowing for a diabetes cure without the need for immunosuppression [2]. - This advancement could relieve millions of patients from the daily pain of injections and the long-term complications associated with diabetes [2].