Study summary

A one-time CRISPR gene-editing treatment resumed its phase 3 trial

Nex-z (MAGNITUDE) clinical hold lifted · Phase 3 (1,200 people) · FDA hold placed October 2025; lifted March 2026

What was tested

Nexiguran ziclumeran (nex-z), a single infusion that uses CRISPR to permanently switch off the TTR gene in the liver.

What they found

What it means

Could become a one-time treatment if efficacy and safety hold up.

Global Genes (RARE Daily) ↗ ← Back to the outlook
Last reviewed 2026-11-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.