Intellia Therapeutics is an American clinical-stage biotechnology company developing potentially curative therapeutics based on CRISPR technologies. Its programs include both in vivo and ex vivo gene-editing approaches for genetic and oncologic diseases.
The FDA removed the clinical hold on Intellia Therapeutics' Phase 3 trial of nexiguran ziclumeran for transthyretin amyloidosis with cardiomyopathy. The hold was imposed in October following liver safety concerns in one patient.
The FDA removed the clinical hold on Intellia Therapeutics' MAGNITUDE-2 Phase 3 trial of nexiguran ziclumeran for ATTRv-PN, allowing patient enrollment and dosing to resume with enhanced safety monitoring.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT06672237 |
A Phase 3 Study of NTLA-2001 in ATTRv-PN |
RECRUITING | PHASE3 |
| NCT06634420 |
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE) |
ACTIVE_NOT_RECRUITING | PHASE3 |
| NCT06622668 |
NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease |
WITHDRAWN | PHASE1/PHASE2 |
| NCT06379789 |
A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B |
RECRUITING | PHASE1/PHASE2 |
| NCT06262399 |
Long-Term Follow-Up (LTFU) of Subjects Treated With NTLA 2002 |
ENROLLING_BY_INVITATION | |
| NCT06128629 |
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM) |
RECRUITING | PHASE3 |
| NCT05697861 |
Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001 |
RECRUITING | |
| NCT05120830 |
NTLA-2002 in Adults With Hereditary Angioedema (HAE) |
ACTIVE_NOT_RECRUITING | PHASE1/PHASE2 |
| NCT05066165 |
Study Investigating NTLA-5001 in Subjects With Acute Myeloid Leukemia |
TERMINATED | PHASE1/PHASE2 |
| NCT04601051 |
Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM) |
COMPLETED | PHASE1 |
