CRISPR therapy shows promise against debilitating amyloid disease
Disease control
Completed
This early-stage study tested a single dose of NTLA-2001, a gene-editing treatment, in 72 adults with hereditary transthyretin amyloidosis, a condition that causes nerve damage and heart problems. The goal was to see if the treatment is safe and can lower a harmful protein called…
Phase: PHASE1 • Sponsor: Intellia Therapeutics • Aim: Disease control
Last updated May 12, 2026 13:42 UTC