FDA lifts hold on clinical trial testing gene-editing therapy for ATTR-CM

The U.S. Food and Drug Administration (FDA) has lifted its hold on a large-scale clinical trial evaluating nexiguran ziclumeran, Intellia Therapeutics’ gene-editing therapy candidate, known as nex-z for short, in adults with transthyretin amyloid cardiomyopathy (ATTR-CM).

The regulatory agency had placed the clinical hold on the Phase 3 MAGNITUDE trial (NCT06128629) — which had then already enrolled more than 600 patients — late last year due to safety concerns. Specifically, an older man with ATTR-CM developed life-threatening liver damage and eventually died after receiving the therapy in the trial.