Also known as ATTR. A rare genetic disease caused by accumulation of misfolded transthyretin (TTR) protein, which affects the nerves, heart, kidneys and eyes. Patients can develop amyloidosis by inheriting the faulty TTR gene from a parent (hereditary or ATTRv) or due to a natural form of this protein, without genetic mutation (wild-type or ATTRwt).

NTLA-2001, Intellia’s first investigational therapy, is being evaluated in a Phase 1 clinical trial as a treatment for people who have hereditary ATTR with polyneuropathy.