People with hereditary angioedema live with constant fear and One gene editing dose may change that.

Ajouté : 2026-06-05

[00:00:00]People with hereditary angodma live under the constant threat of a swelling attack that can seal their airway. And every treatment requires ongoing medication without fixing the gene that causes it. One dose may have just changed that. Hereditary angodma is a genetic condition where a faulty gene triggers sudden life-threatening swelling in the face airways and abdomen without warning. Every approved treatment manages the attacks. None address the gene responsible. And here's where gene editing crossed from experimental into latestage clinical validation for the first time anywhere in the world. Meet John Leonard, CEO of Intellia Therapeutics, who announced April 27th that the phase 3 halo trial of Longhorn met its primary endpoint and all key secondary endpoints. He explained how Longo Z uses crisper to inactivate the faulty gene directly inside the liver through a single outpatient injection. A single dose freed most patients from both attacks and ongoing therapy throughout the six-month evaluation period with no treatment related serious adverse events. Intellia has begun a rolling FDA application with a planned US launch in the first half of 2027 if approved. And here's what made this a milestone beyond just one disease. Every crisper therapy before this either edited cells outside the body or only reached early stage trials. Lone Z edits DNA inside a living person and just became the first invivo gene editing therapy to report positive phase 3 results anywhere in the world. For patients who have spent years managing a condition that could kill them at any moment, this may be the result they have been waiting for. Follow me for the latest clinical trials and medical updates.