Intellia: Interim Data Show NTLA-2001 Can Profoundly Reduce Serum TTR Levels

(RTTNews) - Intellia Therapeutics, Inc. (NTLA) and Regeneron Pharmaceuticals, Inc. (REGN) reported positive interim results from an ongoing phase 1 clinical trial of NTLA-2001, an investigational in vivo CRISPR/Cas9 genome editing therapy in development as a single-dose treatment for transthyretin amyloidosis. The company said the interim data from the cardiomyopathy arm of the phase 1 study showed deep and sustained mean serum transthyretin reductions of 93% and 92% at 0.7 mg/kg and 1.0 mg/kg doses, respectively, at day 28. NTLA-2001 was generally well-tolerated at both dose levels, the company noted.

Intellia CEO John Leonard, said: "Together with the previously reported data from the polyneuropathy arm of this landmark study, these results strongly suggest that NTLA-2001 could serve as a single-dose treatment regardless of disease manifestation."

Shares of Intellia Therapeutics are up 10% in pre-market trade on Friday.

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