Intellia Therapeutics

Intellia Therapeutics is a leading genomics company, focused on the development of CRISPR gene-editing therapies. The company was founded in 2014 and is headquartered in Cambridge, Massachusetts.

Overview

Intellia Therapeutics is dedicated to improving patient care using a broad range of genetic engineering techniques, with a primary focus on the CRISPR/Cas9 system. The company's mission is to develop curative gene therapy treatments that can directly edit the DNA in the body.

Technology

Intellia's technology is based on the revolutionary CRISPR/Cas9 system, a tool for genome editing that allows scientists to add, delete, or alter sections of the DNA sequence. The company is developing in vivo treatments to edit genes inside the body and ex vivo treatments that modify cells outside the body before they are reintroduced to the patient.

Pipeline

Intellia's pipeline includes therapies for a variety of diseases, including transthyretin amyloidosis (ATTR), acute myeloid leukemia (AML), and hepatitis B. The company's lead program, NTLA-2001, is an in vivo treatment for ATTR that is currently in Phase 1 clinical trials.

Partnerships

Intellia has established several strategic partnerships to advance its research and development efforts. These include collaborations with Regeneron, Novartis, and the Caribou.

See Also

• CRISPR
• Gene therapy
• Genome editing
• Transthyretin amyloidosis
• Acute Myeloid Leukemia
• Hepatitis B

References

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