Intellia Therapeutics, Inc.

Intellia Therapeutics, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on developing novel, potentially curative therapeutics leveraging CRISPR-based gene editing technologies. The company's mission is to transform the lives of people with severe diseases by developing and commercializing potentially curative treatments.

Intellia utilizes the CRISPR/Cas9 system to develop both in vivo and ex vivo therapies. Its in vivo programs involve intravenously administered CRISPR for precise editing of disease-causing genes directly within specific target tissues, with lead candidates including lonvoguran ziclumeran (lonvo-z, formerly NTLA-2002) for hereditary angioedema (HAE) and nexiguran ziclumeran (nex-z, formerly NTLA-2001) for transthyretin (ATTR) amyloidosis. The company also pursues ex vivo programs, engineering human cells to treat cancer and autoimmune diseases. Intellia has established research and development collaborations with various partners, including Regeneron, Novartis, AvenCell, Kyverna, and ONK Therapeutics.

In April 2026, Intellia announced positive Phase 3 results for lonvo-z in hereditary angioedema, demonstrating an 87% reduction in attack rates compared to placebo, positioning it as potentially the world's first in-vivo CRISPR gene editing therapy to seek regulatory approval. The company has initiated a rolling Biologics License Application (BLA) submission with the U.S. FDA, with completion expected in the second half of 2026 and a potential U.S. launch in the first half of 2027, pending approval. John Leonard serves as the President and CEO, having been appointed in January 2018. As a clinical-stage company, Intellia's market positioning is characterized by significant scientific advancements, alongside investor focus on its cash position and path to profitability.