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.