Intellia Therapeutics Authorized for Phase 1/2 Trial of NTLA-3001 for Alpha-1 Antitrypsin Deficiency

Intellia Therapeutics, Inc., a renowned clinical-stage gene editing company, has received authorization from the United Kingdom’s Medicine and Healthcare products Regulatory Agency (MHRA) to start a Phase 1/2 study of NTLA-3001, a potential treatment for alpha-1 antitrypsin deficiency (AATD)-related lung disease. AATD is a genetic disorder that typically results in lung dysfunction due to low levels of the alpha-1 antitrypsin (AAT) protein.

NTLA-3001 represents a pioneering approach in gene therapy, being a systemically administered in vivo CRISPR/Cas9-based gene insertion treatment. The therapy aims to insert a functional copy of the SERPINA1 gene, which encodes the AAT protein, directly into patients' genomes. This mechanism is designed to restore the production of functional AAT protein to therapeutic levels after just one dose, potentially eliminating the need for regular AAT augmentation therapy or even lung transplants in severe cases.

John Leonard, M.D., President and CEO of Intellia, emphasized the significance of this development, stating, “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.”

The Phase 1/2 study will be a multicenter, international, single-arm, open-label study. It will involve up to 30 adult patients suffering from AATD-associated lung disease. The trial will commence with a dose-escalation phase to determine the optimal dose, followed by a dose-expansion phase to confirm the recommended dosage. The primary goals of the study are to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of NTLA-3001. Details about the study will be available on clinicaltrials.gov as the trial progresses.

In addition to the trial in the UK, Intellia is also preparing to submit regulatory applications in other countries as part of its strategy to develop NTLA-3001 on a global scale.

AATD is a hereditary condition that increases the risk of liver and lung diseases. The disorder is caused by mutations in the SERPINA1 gene, which is responsible for producing the AAT protein in the liver. This protein is then secreted to protect the lungs. Due to these genetic mutations, the AAT protein is produced abnormally and accumulates in the liver, leading to significantly low levels of AAT in the blood and lungs. The deficiency in AAT puts the lungs at risk for emphysema, a severe type of chronic obstructive pulmonary disease (COPD). In the United States, AATD affects more than 60,000 people, with approximately 250,000 cases worldwide.

Intellia Therapeutics, Inc., listed on NASDAQ under NTLA, is dedicated to transforming medicine through CRISPR-based therapies. The company’s in vivo programs leverage CRISPR technology to enable precise editing of genes directly within the human body. Moreover, Intellia’s ex vivo programs focus on engineering human cells outside the body for treating conditions such as cancer and autoimmune diseases. Intellia combines extensive scientific and clinical experience to set new standards in gene editing, continually expanding its platform with innovative editing and delivery technologies.

In summary, Intellia’s authorization to proceed with the NTLA-3001 study marks a significant milestone in the development of treatments for AATD-associated lung disease, with the potential to significantly improve patient outcomes through a one-time gene therapy solution.

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