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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