Clinical research
Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.
本试验正在测试什么
A late-stage trial of a one-time infusion designed to stop hereditary angioedema attacks by switching off a liver gene. It reported positive results, and its sponsor described it as the first successful Phase 3 trial of gene editing performed inside the body.
A global Phase 3 study of lonvoguran ziclumeran (NTLA-2002), a lipid-nanoparticle CRISPR-Cas9 therapy knocking out hepatic KLKB1, in adults with hereditary angioedema. Intellia reported positive results, described as marking a global first for in vivo gene editing, with a biologics licence application signalled for the second half of 2026.
Sources
- Intellia Therapeutics · 2026
Intellia reports positive Phase 3 results in hereditary angioedema ↗
试验结果意味着什么，又不意味着什么
早期试验结果来自少量参与者和较短的随访周期，随着人数增加和时间延长，结论可能发生变化。正在招募、进行中甚至已完成的试验，并不代表某种疗法已被证实有效。入组标准十分严格，是否符合条件由试验机构决定，而非由网站判断。