Pioneering In Vivo Therapies for Transthyretin Amyloidosis

NTLA-2001, an in vivo CRISPR therapy by Intellia Therapeutics and Regeneron, was the first to be administered directly into the bloodstream to treat transthyretin amyloidosis (ATTR), demonstrating rapid and sustained reductions of the disease-causing protein by up to 96%.

Sources

• Clinical Update: CRISPR Therapy for Transthyretin Amyloidosis Results in Rapid and Prolonged Responses
• CRISPR/Cas9 in the treatment of sickle cell disease (SCD) and its comparison with traditional treatment approaches: a review - PMC
• CRISPR Cas9 in-vivo gene editing for transthyretin amyloidosis | IDT
• CRISPR injected into the blood treats genetic disease - Front Line Genomics
• Intellia Announces First Clinical Evidence from Ongoing Phase 1 Study that Nexiguran Ziclumeran (nex-z), an In Vivo CRISPR/Cas9-Based Gene Editing Therapy, May Favorably Impact Disease Progression in Transthyretin (ATTR) Amyloidosis

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