Intellia Therapeutics ( (NTLA) ) just unveiled an announcement.
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Intellia Therapeutics has unveiled promising Phase 1 clinical trial results for Nexiguran Ziclumeran, a CRISPR-based gene-editing therapy aimed at treating transthyretin amyloidosis. The therapy demonstrated significant reduction in serum TTR levels and showed stabilization or improvement in disease markers over 12 months. The trial highlights Nexiguran’s potential as a one-time treatment option, with favorable safety and tolerability, providing hope for patients with advanced heart failure and neuropathy conditions.
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