Intellia's CRISPR Therapy Shows 90% Reduction in ATTR-CM Over 12 Months

Nov 18, 2024, 04:25 AM

Intellia Therapeutics has announced promising results from its ongoing Phase 1 study of its CRISPR therapy, nexiguran ziclumeran (nex-z), for treating transthyretin amyloidosis (ATTR-CM). The therapy demonstrated a 90% reduction in transthyretin levels over 12 months, indicating potential disease stabilization. This result is significant compared to Alnylam's siRNA therapy, which showed an 81% reduction. Additionally, Intellia's therapy showed gains in cardiomyopathy, further highlighting its efficacy.