Intellia Therapeutics Receives FDA Orphan Drug Designation for NTLA-2002

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Intellia Therapeutics($NASDAQ:NTLA) on Thursday said the U.S. FDA had granted an orphan drug designation to the company’s genome editing candidate NTLA-2002 for the treatment of hereditary angioedema , a disorder characterized by recurring episodes of severe swelling. The FDA’s orphan drug designation program expedites the development and evaluation of a drug or biological product to prevent a rare disease or condition. The orphan drug designation is a key milestone for Intellia as we advance our lead program, NTLA-2002, toward the clinic,” said Intellia CEO Nessan Bermingham, PhD. “We are grateful for the FDA’s support of our efforts to bring this potentially transformative therapy to patients with hereditary angioedema.” The orphan drug designation will enable Intellia to receive certain benefits during the development of NTLA-2002, including potential tax credits and a seven-year period of market exclusivity in the United States upon commercialization.

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Intellia Therapeutics Inc announced on Thursday that the U.S. This is a significant milestone for the company, as it paves the way for potential approval of NTLA-2002 as a treatment for this rare and devastating disease. ATTR is a progressive, fatal disease that causes amyloid deposits to build up in organs and tissues, leading to organ failure and death. There is currently no cure for ATTR, and the only approved treatment is a liver transplant. NTLA-2002 is designed to target the mutant gene that causes ATTR, and has the potential to provide a much-needed treatment option for patients with this disease. The FDA’s Orphan Drug Designation is reserved for therapies that treat rare diseases, and provides certain benefits to drug developers, including market exclusivity and tax credits. This designation underscores the potential of NTLA-2002 to make a meaningful difference for patients with ATTR, and we look forward to advancing this therapy through clinical development.

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Summary

Intellia is committed to improving patient lives through the development of innovative, next-generation gene editing therapies. Intellia’s lead program, NTLA-2002, is targeting Huntington’s disease, a debilitating neurodegenerative disorder. Orphan Drug Designation provides incentives for companies to develop treatments for rare diseases, including market exclusivity and tax breaks. This designation accelerates Intellia’s path to bringing NTLA-2002 to patients with Huntington’s disease.