基因编辑疗法连破纪录，国产器械引领全球创新 | Healthcare View

Group 1 - The first successful clinical treatment of hyperlipidemia using APOC3 gene editing was announced by Zhengxu Biotech, marking a significant breakthrough in gene therapy [2][3] - The CS-121 injection utilizes transformer Base Editor technology to achieve precise single-base correction without damaging the DNA double helix, simulating beneficial mutations found in nature [2] - Clinical data showed a significant decrease in fasting triglyceride levels within three days post-treatment, with no adverse events reported, indicating a promising safety profile [2] Group 2 - New芽基因 reported positive results from a 52-week follow-up of GEN6050X, the first FDA-approved gene editing therapy for Duchenne Muscular Dystrophy (DMD), showing significant improvements in cardiac and pulmonary function [6] - The therapy employs a dual mechanism to restore dystrophin expression and enhance cell stability, providing new hope for DMD patients [6] Group 3 - 精锋医疗's 精锋云® remote surgery system received NMPA approval, representing a leap in domestic surgical robotics technology [8] - The system has successfully assisted over 500 remote surgeries globally with a 100% success rate, showcasing its potential in enhancing healthcare accessibility [8] Group 4 - 凌意生物's LY-M003 injection for Wilson's disease received FDA approval to directly enter Phase II clinical trials, utilizing a novel copper ion dynamic regulation technology [11][12] - Initial clinical observations indicated good safety and positive treatment effects, with no serious adverse events reported [11] Group 5 - 瑞吉生物's freeze-dried mRNA vaccine for tuberculosis received CDE approval for clinical trials, marking a significant advancement in mRNA vaccine technology [13] - This vaccine aims to address a major public health challenge and represents a new technological pathway for tuberculosis prevention [13] Group 6 - 启函生物's QT-019B, a universal dual-target CAR-T cell product for refractory systemic lupus erythematosus, received CDE approval, highlighting its innovative design and potential applications in various autoimmune diseases [15] - The product aims to reduce the risk of graft-versus-host disease and improve clinical outcomes for patients [15] Group 7 - 心泰医疗's interventional guidewire received market approval, enhancing precision and safety in structural heart disease interventions [18] - The guidewire's innovative design allows for multiple functions, improving surgical efficiency and reducing risks [18] Group 8 - 同心医疗's BrioVAD system successfully completed its first clinical enrollment in the US, aiming to provide long-term support for patients with advanced heart failure [19][20] - The system's design minimizes surgical trauma and infection risk, enhancing patient experience and quality of life [19] Group 9 - 唯柯医疗's transcatheter mitral valve implantation project was approved for the 2025 National Key R&D Program, focusing on developing a durable and low-complication valve replacement system [23] - This project represents a significant step in addressing clinical challenges in heart valve diseases and enhancing domestic medical device capabilities [23] Group 10 - 微光医疗's project on coronary atherosclerosis diagnosis and treatment technology won the Beijing Science and Technology Progress Award, showcasing its innovative contributions to medical imaging [26] - The project has established a comprehensive evaluation system and achieved significant technological breakthroughs, enhancing the quality of care in cardiovascular diseases [26] Group 11 - 迪必尔生物工程 was recognized as a "Little Giant" enterprise, reflecting its innovation in high-performance bioreactor technology [30] - The recognition underscores the company's role in advancing the biomanufacturing industry and addressing global challenges in process scaling [30] Group 12 - 重庆壹永科技 was also recognized as a "Little Giant" enterprise, highlighting its capabilities in tumor big data and artificial intelligence [32] - This acknowledgment supports the company's ongoing innovation and market competitiveness in the healthcare sector [32] Group 13 - 红杉中国 has invested over 200 healthcare companies with distinctive technological features and high growth potential, covering various segments including innovative drugs and medical devices [33] - The investment strategy reflects a commitment to fostering innovation and supporting the growth of the healthcare industry [33]