FDA Clears Way for Intellia to Move In Vivo CRISPR Therapy for HAE into Clinical Testing

Rare Daily Staff

The U.S. Food and Drug Administration has cleared Intellia Therapeutics’ New Drug application for NTLA-2002, an experimental gene-editing therapy for the treatment of hereditary angioedema.

The clearance will allow the company to include the United States in the global phase 2 portion of its ongoing phase 1/2 study.

Hereditary angioedema (HAE) is a rare, genetic disorder characterized by severe, recurring, and unpredictable inflammatory attacks in various organs and tissues of the body, which can be painful, debilitating and life-threatening. It is estimated that one in 50,000 people are affected by HAE, and current treatment options often include life-long therapies, which may require chronic intravenous or subcutaneous administration as often as twice per week, or daily oral administration to ensure constant pathway suppression for disease control. Despite chronic administration, breakthrough attacks still occur.

NTLA-2002 is the first single-dose investigational treatment being explored in clinical trials for the potential to continuously reduce kallikrein activity and prevent attacks in people living with HAE. NTLA-2002 is a wholly owned investigational CRISPR therapeutic candidate designed to inactivate the kallikrein B1 (KLKB1) gene, which encodes for prekallikrein, the kallikrein precursor protein.

NTLA-2002 is Intellia’s second investigational CRISPR therapeutic candidate to be administered systemically, by intravenous infusion, to edit disease-causing genes inside the human body with a single dose of treatment. Intellia’s proprietary non-viral platform deploys lipid nanoparticles to deliver to the liver a two-part genome editing system: guide RNA specific to the disease-causing gene and messenger RNA that encodes the Cas9 enzyme, which together carry out the precision editing.

“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,” said John Leonard, president and CEO of Intellia. “The NTLA-2002 IND clearance marks an important milestone for Intellia.”

Photo: John Leonard, president and CEO of Intellia