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Intellia Therapeutics Receives Authorization to Initiate Phase 1/2 Clinical Trial of NTLA-3001 for the Treatment of Alpha-1 Antitrypsin Deficiency
NTLA-3001 is a potential one-time gene editing treatment that may normalize AAT protein levels and halt the progression of lung disease associated with
About this update from Intellia Therapeutics, Inc.
[{"type":"text","content":"NTLA-3001 is a potential one-time gene editing treatment that may normalize AAT protein levels and halt the progression of lung disease associated with alpha-1 antitrypsin deficiency (AATD)NTLA-3001 is Intellia’s first wholly owned CRISPR-based in vivo targeted gene insertion candidate to advance into the clinicOn track to dose the first patient in 2H 2024 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. “NTLA-3001 is a groundbreaking in vivo CRISPR-based gene insertion candidate designed to durably produce functional AAT protein at normal levels after a one-time treatment. We are excited to receive regulatory authorization to begin this important first-in-human study of NTLA-3001 for people living with AATD,” said Intellia President and Chief Executive Officer John Leonard, M.D. “In addition, this study serves to validate our modular gene insertion platform, which we plan to leverage to address numerous diseases caused by a missing or defective protein.” The Phase 1/2 study will be an international, multicenter, single-arm, open-label study of NTLA-3001 in adults with AATD-associated lung disease. The study will enroll up to 30 patients and consist of a dose-escalation phase, followed by a dose-expansion phase to confirm the recommended dose. The study ...