FDA clearance of investigational new drug (IND) application to initiate a pivotal phase III trial of NTLA 2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.- Intellia + Regeneron

The global Phase III study of NTLA 2001, an in vivo CRISPR-based gene editing candidate, is expected to initiate by year-end 2023.

“The FDA clearance of the NTLA 2001 IND application allows us to initiate a pivotal Phase III trial in the United States, marking the first in vivo CRISPR-based candidate to begin late-stage clinical development. This is another important step forward for Intellia and our collaborator, Regeneron, as we aim to establish a new standard of care for the treatment of ATTR amyloidosis,” said Intellia President and Chief Executive Officer John Leonard, M.D. “We are thrilled to further advance NTLA 2001 and our pipeline of investigational gene editing therapies as we embark on a new era in medicine. We look forward to sharing additional information about the Phase III study at our upcoming quarterly earnings webcast, being held on Thursday, November 9.”