NTLA-3001
Alpha-1 Antitrypsin Deficiency (AATD)-associated Lung Disease
Key Facts
Indication
Alpha-1 Antitrypsin Deficiency (AATD)-associated Lung Disease
Phase
Phase 1/2
Status
Active
Company
About Intellia Therapeutics
Intellia Therapeutics is a pioneer in the development of CRISPR/Cas9-based therapeutics, aiming to cure severe genetic and other diseases through precise genome editing. The company has achieved significant milestones, including the first-ever clinical data demonstrating in vivo CRISPR genome editing in humans. Its strategy leverages a dual-platform approach, developing both in vivo (systemically administered) and ex vivo (cell-based) therapies, with key partnerships including Regeneron and Novartis to advance its pipeline.
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