Intellia Therapeutics, Inc. is a clinical-stage biotechnology company pioneering the development of transformative gene editing therapies using CRISPR/Cas9 technology. The company’s core mission is to create single-course treatments that address the underlying causes of severe genetic diseases. Its proprietary platform enables both in vivo and ex vivo genome editing applications, with an emphasis on precision, safety and durable therapeutic benefit.
The company’s pipeline includes NTLA-2001, a first-in-human in vivo CRISPR therapy designed to treat transthyretin amyloidosis (ATTR) through lipid nanoparticle delivery of Cas9 mRNA and guide RNA directly to hepatocytes. Intellia is also advancing ex vivo programs targeting hematologic disorders by engineering patient-derived cells before reinfusion. Strategic collaborations, notably with Regeneron Pharmaceuticals, support the discovery and development of additional in vivo gene editing candidates for liver and cardiovascular diseases.
Founded in 2014 and headquartered in Cambridge, Massachusetts, Intellia emerged from foundational work led by CRISPR pioneers, including co-founder Jennifer Doudna. The company completed its initial public offering on the NASDAQ under the ticker NTLA, establishing a research presence in both the United States and the United Kingdom. Over the years, Intellia has built a robust intellectual property portfolio covering core CRISPR/Cas9 gene editing technology and delivery modalities.
Intellia’s leadership team is headed by President and CEO John Leonard, whose prior experience spans both biotechnology research and commercial operations. The organization’s scientific advisory board features leading figures in genomics and molecular biology, underscoring its commitment to advancing genome editing to address unmet medical needs. With multiple clinical milestones ahead, Intellia continues to position itself at the forefront of genetic medicine innovation.
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