Intellia Phase 1 in vivo CRISPR therapy nex-z shows disease stabilization in ATTR amyloidosis

Intellia Therapeutics, Inc.

Single dose of nex-z achieved mean 90% serum TTR reduction at month 12 in ATTR-CM (n=36); sustained >24 months.
81% of ATTR-CM patients stable/improved in NT-proBNP, 94% in hs-Troponin T at month 12; hospitalization rate 0.16/yr.
In ATTRv-PN, mean TTR reduction 91% at month 12 (n=33); neuropathy scores stable or improved through month 24.
Favorable safety: infusion-related reactions mild/moderate, no discontinuations; Phase 3 MAGNITUDE trials ongoing.
Data published in NEJM and presented at AHA 2024; supports hypothesis greater TTR reduction yields greater clinical benefit.

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