NEJM publishes first clinical data from systemic in vivo CRISPR editing: NTLA-2001 lowers serum TTR

Intellia Therapeutics/Nex-z (NTLA-2001)

NEJM published Phase 1 results of NTLA-2001, the first systemic CRISPR therapy delivered via IV infusion, in patients with hereditary transthyretin amyloidosis. A single 0.3 mg/kg dose achieved an 87% mean reduction in serum TTR, with maximum reductions reaching 96% by day 28, and no serious adverse events in the first six patients.

• NTLA-2001 uses Cas9 delivered in lipid nanoparticles to inactivate the TTR gene in liver cells and prevent production of misfolded transthyretin protein.
• Response was dose-dependent; the mechanism operates with precision editing of a target gene inside the body.
• No serious adverse events were noted in the first six patients by day 28 post-infusion.