In un paragrafo
Intellia's bet is in vivo editing: instead of taking cells out to edit them, deliver the editing machinery into the body directly. It was the first company to do that in a person, in 2021. Its two lead programmes — for ATTR amyloidosis and hereditary angioedema — are both in late-stage trials, and one of them produced the first positive Phase 3 result for in vivo gene editing anywhere.
Intellia Therapeutics was founded in 2014 with Jennifer Doudna among its scientific founders. Its lipid-nanoparticle CRISPR platform delivers editors to hepatocytes systemically. Nexiguran ziclumeran (TTR knockout for ATTR amyloidosis) and lonvoguran ziclumeran (KLKB1 knockout for hereditary angioedema) are its lead programmes, both in Phase 3; the HAELO study in hereditary angioedema reported positive results described as the first Phase 3 success for in vivo gene editing.
Both ends of what in vivo editing means
Intellia's two programmes illustrate the promise and the risk of the approach in the same company and the same year. Hereditary angioedema produced the field's first positive Phase 3 result. The ATTR programme saw a participant die of liver dysfunction after dosing, leading to a voluntary pause and an FDA clinical hold that was subsequently lifted for the MAGNITUDE trial. Neither event tells you what to conclude about the other, and the Atlas records both.
Sources
- Intellia Therapeutics · 2026
Intellia Therapeutics corporate overview ↗