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Tag: Intellia

Intellia has released clinical data for NTLA-2001, a gene therapy for hereditary transthyretin amyloidosis. What is transthyretin amyloidosis? Transthyretin (ATTR) amyloidosis is the buildup of misfolded transthyretin, a protein generated by the liver. Normally, this protein is partially responsible for the transport of thyroid hormones, hence its name; however, transthyretin is vulnerable to mutations and...