Intellia Therapeutics Receives Authorization to Initiate Phase 1/2 Clinical Trial of NTLA-3001 for the Treatment of Alpha-1 Antitrypsin Deficiency
CAMBRIDGE, Mass., July 30, 2024 (GLOBE NEWSWIRE) — Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced the authorization of its Clinical Trial Application (CTA) by the United Kingdom’s Medicine and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1/2 study evaluating NTLA-3001 for the treatment of alpha-1 antitrypsin deficiency (AATD)-associated lung disease. AATD is a rare, genetic disease that most commonly manifests in lung dysfunction due to insufficient levels of alpha-1 antitrypsin (AAT) protein. NTLA-3001 is a systemically administered in vivo CRISPR/Cas9-based targeted gene insertion candidate. It is designed to precisely insert a healthy copy of the SERPINA1 gene, which encodes the AAT protein, with the potential to restore permanent expression of functional AAT protein to therapeutic levels after a single dose. This approach seeks to improve patient outcomes, including eliminating the need for weekly intravenous infusions of AAT augmentation therapy or lung transplant in severe cases.