Study summary
A one-time CRISPR gene-editing treatment resumed its phase 3 trial
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
- The FDA paused the trials in October 2025 after a patient had severe liver injury and later died.
- The hold was lifted in March 2026 with closer liver monitoring and new exclusion criteria.
What it means
Could become a one-time treatment if efficacy and safety hold up.
Last reviewed 2026-11-15 by the Find a Trial editorial team.
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