Nexiguran ziclumeran is an investigational in vivo CRISPR/Cas9 gene-editing therapy for transthyretin amyloidosis, developed as a potential one-time treatment. It is being studied in hereditary ATTR polyneuropathy and ATTR cardiomyopathy programs, with a pivotal Phase 3 study underway in ATTRv-PN. The program has received FDA RMAT and orphan designations but is not approved.
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 |
