Gene-Editing breakthrough tested for rare, fatal heart and nerve disease
Disease control
Completed
This early-stage study tested a new, one-time gene-editing treatment called NTLA-2001 for people with hereditary forms of amyloidosis, a disease where a harmful protein builds up and damages the heart or nerves. The main goal was to check the safety of the treatment and see how i…
Phase: PHASE1 • Sponsor: Intellia Therapeutics • Aim: Disease control
Last updated Mar 17, 2026 12:55 UTC