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BioWorld - Thursday, December 25, 2025
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Home » Transthyretin amyloidosis space heats up with a potential cure
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Transthyretin amyloidosis space heats up with a potential cure

Oct. 26, 2020
By Brian Orelli
Intellia Therapeutics Inc. is looking to disrupt the transthyretin (TTR) amyloidosis (ATTR) market with NTLA-2001, its CRISPR-based treatment designed to be a potential cure for the disease. The drug, which is delivered via a lipid nanoparticle, edits the patient's DNA in vivo to create a stop codon and eliminate the expression of TTR, the protein that aggregates in ATTR patients' nervous systems and hearts, disrupting their functions.
BioWorld Analysis and data insight Endocrine/metabolic

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