Intellia Therapeutics ( (NTLA) ) has provided an announcement.
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Intellia Therapeutics has announced promising Phase 2 results for NTLA-2002, a CRISPR-based gene editing therapy aimed at treating hereditary angioedema (HAE). The study showed that a single 50 mg dose significantly reduced attack rates and could potentially offer a functional cure, with most patients remaining attack-free for months. The treatment was well-tolerated with no serious adverse events, paving the way for the pivotal Phase 3 HAELO study. These findings suggest NTLA-2002 might transform HAE treatment, offering hope for eliminating the need for chronic therapies.
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