FDA取消Intellia的CRISPR在安全停顿后对罕见神经疾病的试验。 FDA clears Intellia's CRISPR trial for rare nerve disease after safety pause.

FDA解除了对Intellia Therapeutics针对nexiguran ziclumeran遗传性转甲状腺淀粉样变性多发性神经病变（ATTRv-PN）第三期MAGNITUDE-2试验的临床暂停，允许该试验于2025年10月发生涉及肝脏问题的安全事件后恢复。 The FDA lifted a clinical hold on Intellia Therapeutics' Phase 3 MAGNITUDE-2 trial for nexiguran ziclumeran in hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), allowing the trial to resume after a safety event in October 2025 involving liver issues. 该公司加强了肝脏监测,将患者人数从50人增至60人。 The company implemented enhanced liver monitoring and increased enrollment from 50 to 60 patients. 治疗是一种基于CRISPR的基因编辑治疗,在早先的试验中,这种治疗显示出显著的TTR减少。 The therapy, a CRISPR-based gene-editing treatment, showed strong TTR reduction in earlier trials. 由于Intellia继续与监管者进行讨论,对ATTRv-心血管病的审理仍被搁置。 The trial for ATTRv-cardiomyopathy remains on hold as Intellia continues discussions with regulators.