Core Viewpoint - Gene editing technology is revolutionizing the understanding of life by allowing precise modifications of genetic sequences, akin to using a "molecular scissors" to correct genetic errors [2][3][4] Group 1: Technology Development - The evolution of gene editing technology has progressed rapidly, particularly with the advent of CRISPR technology in 2012, which significantly lowered the technical barriers and costs associated with gene editing [4][5] - Newer techniques such as base editing and guided editing have emerged, providing more precise tools for genetic modifications, enhancing both basic scientific research and translational medicine [5][6] Group 2: Applications in Medicine - Gene editing technology offers innovative treatment methods for genetic diseases, such as using CRISPR to edit hematopoietic stem cells for conditions like thalassemia, leading to significant symptom relief in patients [6] - In cancer treatment, gene editing is utilized in CAR-T therapy, which modifies patients' immune cells to better target and combat cancer cells [6] Group 3: Applications in Agriculture and Bio-manufacturing - In agriculture, gene editing has been used to develop new rice varieties that are resistant to diseases and environmental stressors, contributing to global food security [6] - The technology also plays a crucial role in bio-manufacturing, enhancing the efficiency of biofuel production and reducing costs in the synthesis of scarce drugs [6] Group 4: Ethical Considerations - The advancement of gene editing technology raises significant ethical concerns, particularly regarding the editing of human germline cells, which could permanently alter the human gene pool and pose risks to future generations [7][8] - There is a need for strict ethical guidelines and international collaboration to ensure responsible use of gene editing technologies, prioritizing non-heritable somatic cell editing for therapeutic purposes [7][8] Group 5: Regulatory Framework - In July 2024, the Ministry of Science and Technology released ethical guidelines for human genome editing research, addressing the ethical challenges and promoting healthy development in this field [8]

Core Viewpoint - Prime Editing technology, a prominent member of the CRISPR gene editing family, has made its debut in the medical field with the treatment of Chronic Granulomatous Disease (CGD) using the PM359 therapy, showing promising early clinical results [2][5][8]. Group 1: Prime Editing Technology - Prime Editing technology, developed by Professor David Liu, allows for precise gene editing without relying on DNA templates, enabling the correction of 89% of known pathogenic human genetic variations [5][7]. - The technology has been upgraded to improve editing efficiency, and it is particularly suitable for diseases like CGD, where common mutations can be corrected by inserting two missing bases in the DNA sequence [9][10]. Group 2: Clinical Trials and Results - Initial clinical data from Prime Medicine indicates that after one month of treatment with PM359, a teenage patient showed significant recovery in NADPH oxidase activity, with 66% of neutrophils fully restored, exceeding the expected clinical benefit threshold of 20% [2][8]. - The company has reported a 92% editing efficiency in correcting the most common mutation type associated with CGD in preclinical studies [7]. Group 3: Market and Economic Considerations - Prime Medicine announced a strategic restructuring, exploring external partnerships for the clinical development of PM359, highlighting the economic challenges in developing gene editing therapies for rare diseases [8][11]. - The only currently marketed gene editing therapy, Casgevy, has faced slow commercialization despite its approval, with projected sales of less than $10 million in 2024 [8]. Group 4: Future Directions - Prime Medicine plans to focus on developing gene editing therapies for hereditary liver diseases and continue supporting in vivo gene editing for cystic fibrosis, while also collaborating with Bristol-Myers Squibb on CAR-T cell therapies [11][13]. - The company aims to enhance its financial resources and accelerate innovation to ensure the widespread application of Prime Editing technology [11].