Breakthrough in CRISPR Gene Therapy for ATTR Amyloidosis

Cambridge, Saturday, 16 November 2024.
Intellia Therapeutics reports promising Phase 1 results for nexiguran ziclumeran (nex-z), a one-time CRISPR-based gene editing therapy for transthyretin (ATTR) amyloidosis. The treatment showed a 90% reduction in serum TTR levels at 12 months, with evidence of disease stabilization or improvement in advanced heart failure patients.