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Intellia Therapeutics Says Drug Shows Potential in Phase 1 Transthyretin Amyloidosis Trial
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Intellia Therapeutics Says Drug Shows Potential in Phase 1 Transthyretin Amyloidosis Trial
May 26, 2025 10:02 AM

03:58 AM EDT, 05/19/2025 (MT Newswires) -- Intellia Therapeutics ( NTLA ) said Sunday it received "positive" two-year follow-up data from an ongoing phase 1 trial of its investigational drug nexiguran ziclumeran for the potential treatment of hereditary transthyretin amyloidosis with polyneuropathy, a genetic disease associated with the liver.

The company said a one-time dose of the drug resulted in a reduction of the levels of the protein that cause the illness by a mean level of 90%.

It also said the study provided evidence that the drug could result in disease stability or "clinically meaningful improvement" based on the evaluation of certain clinical measures.

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