Intellia finds genetic suspect for liver safety signals with ATTR gene therapy
Intellia finds genetic suspect for liver safety signals with ATTR gene therapy
Intellia Therapeutics
Intellia Therapeutics is a clinical-stage biotechnology company developing in vivo and ex vivo CRISPR/Cas9-based gene-editing therapies. Its lead program NTLA-2001 is being studied for transthyretin (ATTR) amyloidosis, with additional programs in hereditary angioedema, hemophilia A/B, and other genetic diseases. Intellia has also invested heavily in commercial-launch infrastructure, including a dedicated team working on cell-and-gene therapy readiness in advance of its first potential approvals.
Company Info
Sector:Biotechnology / Gene Editing (CRISPR/Cas9)
Headquarters:Cambridge, Massachusetts, USA
Founded:2014
Website:www.intelliatx.com/
Upcoming Milestones & Key Dates
H2 2026
Clinical Trial
MAGNITUDE Phase 3 topline data in ATTR amyloidosis with cardiomyopathy
Q4 2026
Regulatory
BLA submission readiness update for NTLA-2002 in HAE
Clinical Development Pipeline
NTLA-2001 (nexiguran ziclumeran)
Target: In vivo CRISPR/Cas9 knockout of TTR geneNTLA-2002
Target: In vivo CRISPR/Cas9 knockout of KLKB1NTLA-6001
Target: Ex vivo CRISPR-edited allogeneic CAR-TRecent News
The Commercial Launch Playbook: Team Structure, Readiness and Execution
Intellia Therapeutics' SVP Maria Natale outlines the commercial launch playbook for gene-editing therapies, arguing the most successful launches are shaped long before approval with strategy, structure, and patient insight at the core.
Leadership Team
John Leonard
President and Chief Executive Officer
Maria Natale
Senior Vice President, Commercial
Dr. David Lebwohl
Chief Medical Officer