世界首例！新一代基因编辑技术——“先导编辑”完成首次人体试验，修复患者免疫细胞功能

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].