Intellia Therapeutics announces FDA granted RMAT to NTLA-2002

Intellia Therapeutics announced that the U.S. Food and Drug Administration, FDA, granted Regenerative Medicine Advanced Therapy, RMAT, designation to NTLA-2002 for the treatment of hereditary angioedema, HAE. NTLA-2002 is an in vivo CRISPR-based investigational therapy designed to inactivate the target gene, kallikrein B1, to potentially prevent life-threatening swelling attacks in people with HAE. "The RMAT designation is important recognition for our early clinical data. It indicates that a single dose of NTLA-2002 has the potential to address serious unmet medical need for people living with hereditary angioedema," said Intellia President and Chief Executive Officer John Leonard, M.D. "We look forward to continuing our productive dialogue with the FDA to accelerate the development of NTLA-2002, an investigational in vivo CRISPR-based therapy, with the goal of bringing forth a potentially transformative treatment to patients more quickly."