克利夫兰诊所完成的一项第一期人体临床试验显示，单次注射CRISPR基因编辑疗法CTX310能够将难治性血脂异常患者的LDL胆固醇水平降低约50%1。该研究涉及15名患者，在最高剂量组（0.8 mg/kg）中，LDL胆固醇降低幅度达到52.5%，甘油三酯降低47.8%，且这些改善在12个月的随访期间持续保持1。在整个一年的观察期内，研究未报告与疗法相关的严重不良事件1。
CTX310通过靶向肝脏中的ANGPTL3基因进行治疗1。研究结果已发表在2026年《新英格兰医学杂志》，并在欧洲心脏病学会2026年年会上呈报1。研究者计划继续进行15年的长期安全随访，以符合美国食品药品监督管理局对基因编辑疗法的建议1。该项研究由瑞士楚格的CRISPR Therapeutics AG公司资助1。
A phase one clinical trial conducted at Cleveland Clinic has demonstrated that a single injection of the CRISPR gene-editing therapy CTX310 reduced LDL cholesterol and triglycerides by approximately 50% in patients with treatment-resistant dyslipidemia, with effects persisting at the 12-month follow-up mark 1. The highest-dose group showed a reduction of 52.5% in LDL cholesterol and 47.8% in triglycerides 1. No serious adverse events related to the therapy were reported during the one-year follow-up period 1.
The trial enrolled 15 patients and tested CTX310 at doses ranging from 0.1 to 0.8 mg/kg, with the therapy targeting the ANGPTL3 gene in the liver 1. The findings were published in the New England Journal of Medicine in 2026 and presented at the European Society of Cardiology Congress 2026 1. Researchers plan to continue a 15-year long-term safety follow-up study, aligning with FDA recommendations for gene-editing therapies 1. The trial was funded by CRISPR Therapeutics AG, based in Zug, Switzerland 1.
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