Gene editing breakthrough? CRISPR therapy targets rare amyloidosis
Disease control
Completed
This early-stage trial tests NTLA-2001, a CRISPR-based gene editing therapy, in 72 adults with hereditary ATTR amyloidosis—a condition where abnormal protein builds up in nerves or the heart. The treatment aims to turn off the faulty gene to reduce protein levels. The study focus…
Phase: PHASE1 • Sponsor: Intellia Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC