Intellia cleared to start Phase 3 trial for gene editing drug
PhonlamaiPhoto/iStock via Getty Images

Intellia Therapeutics (NASDAQ:NTLA) said Wednesday that the U.S. FDA cleared the companyâs Investigational New Drug (IND) application for NTLA-2001, allowing it to start a pivotal Phase 3 trial for the gene editing candidate in transthyretin (ATTR) amyloidosis with cardiomyopathy.
Accordingly, the biotech and its partner, Regeneron Pharmaceuticals (NASDAQ:REGN), expect to start a global Phase 3 study for NTLA-2001 later this year for patients with cardiomyopathy linked to ATTR amyloidosis.

âThe FDA clearance of the NTLA-2001 IND application allows us to initiate a pivotal Phase 3 trial in the United States, marking the first in vivo CRISPR-based candidate to begin late-stage clinical development,â Intellia (NTLA) CEO John Leonard remarked.
Additional details about the study are expected on November 9, when the company announces its Q3 2023 results.



More on Intellia, Regeneron, etc.

Intellia Therapeutics: Massive OpEx Begin To Weigh On HAE Prospects
Regeneron Pharmaceuticals: Game-Changing Discoveries In Healthcare
Regeneron Pharmaceuticals, Inc. (REGN) Morgan Stanley 21st Annual Global Healthcare Conference (Transcript)
Intellia's gene editing therapy for hereditary angioedema gets PRIME status in EU
Regeneron says Q3 results to be impacted by $100M IPR&D expense