FDA halts Intellia gene-editing trial over liver issues

The FDA clinical hold covers two Phase 3 studies for nexiguran ziclumeran, or nex-z, an experimental gene-editing therapy for transthyretin amyloidosis. Nex-z uses the CRISPR gene-editing technology to inactivate the gene that codes for the protein driving this rare disease.

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Top Story Liver Complications Lead FDA to Stop Tests of Intellia Gene-Editing Therapy for a Rare Disease The FDA clinical hold covers two Phase 3 studies for nexiguran ziclumeran, or nex-z, an experimental gene-editing therapy for transthyretin amyloidosis. Nex-z uses the CRISPR gene-editing technology to inactivate the gene that codes for the protein driving this rare disease. Sponsored Enhanced Direct Enrollment: Georgia Access and the Power of Partnership Presented by GetInsured Across the nation, state-based health insurance exchanges are searching for ways to better serve their communities while making enrollment easier, more personal, and more accessible for everyone. Learn More.

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