明星基因疗法公司股价一夜暴跌40%，相关药物临床被暂停

Core Insights - Intellia Therapeutics' stock plummeted over 40% following the suspension of two late-stage clinical trials due to a serious liver injury in a trial participant [1] - The suspended trials involve the company's lead product, a gene therapy for ATTR-CM, a rare and fatal heart disease [1][2] - ATTR-CM is projected to be a multi-billion dollar market, with an estimated 200,000 to 300,000 patients globally, many of whom remain undiagnosed [2] Company Overview - Intellia Therapeutics focuses on drug development using CRISPR gene editing technology to treat genetic diseases [1] - The halted clinical trial involved the drug nexiguran ziclumeran (nex-z), which aims to "turn off" the gene responsible for producing transthyretin protein [1][3] - The company is facing significant risks as safety issues in clinical trials are not uncommon, and repeated incidents could jeopardize the drug pipeline [2] Competitive Landscape - Following Intellia's announcement, competitor BridgeBio Pharma's stock rose over 17%, indicating market reaction to the news [2] - Other companies, including Alnylam Pharmaceuticals and Pfizer, have already received approval for treatments targeting ATTR-CM, utilizing different technological approaches to inhibit abnormal transthyretin production [2] Market Potential - The ATTR-CM market is valued in the billions, with significant unmet medical needs due to the number of undiagnosed patients [2] - In China, genetic therapies for ATTR-CM are also in clinical trials, with the first domestic gene editing drug YOLT-201 having completed its initial patient dosing [2]