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Intellia Therapeutics' Gene-Editing Drug Gets FDA Designation
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Intellia Therapeutics' Gene-Editing Drug Gets FDA Designation
Mar 26, 2025 6:13 AM

08:36 AM EDT, 03/26/2025 (MT Newswires) -- Intellia Therapeutics ( NTLA ) said Wednesday that the US Food and Drug Administration granted the Regenerative Medicine Advanced Therapy designation to its nex-z treatment for transthyretin amyloidosis with cardiomyopathy or ATTR-CM.

The company said the CRISPR-based therapy has shown it can sharply reduce serum TTR levels with a single dose, potentially slowing disease progression in patients with cardiomyopathy and polyneuropathy.

Intellia added that nex-z has also received RMAT designation for polyneuropathy and Orphan Drug Designation from US and European regulators.

The company's shares were up over 1.9% in recent Wednesday premarket activity.

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