The US Food and Drug Administration (FDA) has accepted Intellia Therapeutics’ Investigational New Drug (IND) application for NTLA-2002, a potential gene editing therapy for hereditary angioedema (HAE), the company announced. The acceptance, announced via news release, allows the company to include the United States in its global phase 2 clinical study.
“The FDA’s acceptance of our IND application to initiate clinical evaluation of NTLA-2002 brings us one step closer to introducing a potentially paradigm-shifting treatment for people living with hereditary angioedema,” Intellia’s president and chief executive officer, John Leonard, MD, said.
Current treatments for HAE involve lifelong, chronic (up to twice weekly) intravenous or subcutaneous injections or daily oral administration of kallikrein and other pathway inhibitors. Breakthrough attacks are known to occur despite treatment, and the continuous need for treatment is a burden on patients and healthcare systems.
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NTLA-2002 is the first single-dose, in vivo gene editing therapy designed to inactivate KLKB1 (kallikrein B1), the causative gene in HAE, thereby permanently reducing its plasma protein activity and preventing HAE attacks. It is based on Nobel Prize-winning clustered regularly interspaced short palindromic repeats (CRISPR)/Cas 9 technology, which employs an enzyme and a sequence of guide RNA to cut DNA at a specific, damaged location and trigger the cell to “edit” or repair it.
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The current phase 1/2 open-label study on adults with type 1 or type 2 HAE employs a single-ascending dose design to determine 2 dose levels of NTLA-2002 to be assessed in the upcoming randomized, placebo-controlled phase 2 study.
Results from the phase 1 study demonstrated significant and sustained dose-dependent reductions in plasma kallikrein levels and HAE attacks.
HAE is a rare genetic condition characterized by severe and unpredictable attacks of swelling in various tissues and organ systems such as the face, airway, and intestinal tract. The potentially life-threatening attacks can be triggered by stress or trauma, but they often occur without a specific trigger.
Reference
Intellia Therapeutics announces FDA clearance of Investigational New Drug (IND) application for NTLA-2002, an in vivo CRISPR-based investigational therapy for the treatment of hereditary angioedema (HAE). News release. Intellia Therapeutics, Inc; March 2, 2023.
