Clinical research
Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.
この試験で検証していること
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 ↗
試験結果が意味すること・意味しないこと
初期段階の結果は少人数の参加者と短い追跡期間から得られたものです。参加者が増え時間が経つにつれて変わる可能性があり、募集中・実施中・完了済みの試験であっても、治療効果が確立されたわけではありません。登録基準は厳格で、適格性の判断は試験実施施設が行うものであり、ウェブサイトが行うものではありません。