Intellia Therapeutics

This early-stage study is testing a single injection of an experimental gene therapy called NTLA-2002 in adults with hereditary angioedema (HAE). The main goals are to see if the treatment is safe and if it can reduce the number of painful, unpredictable swelling attacks people e…

This Phase 3 trial is testing whether a single dose of an experimental gene therapy called NTLA-2001 can safely slow or stop the progression of hereditary amyloidosis with polyneuropathy (ATTRv-PN), a rare genetic disease that damages nerves. About 50 participants worldwide will …

This study is testing whether a single infusion of an experimental gene therapy called NTLA-2002 can safely and effectively reduce the number of sudden, severe swelling attacks in people with hereditary angioedema (HAE). About 60 participants will be randomly assigned to receive …

This study is monitoring people who previously received an investigational gene therapy called NTLA-2002 for hereditary angioedema (HAE). The main goal is to track their long-term health, safety, and how well the treatment continues to control their HAE attacks over time. It is a…

This large, late-stage study is testing whether a single dose of an experimental gene therapy, called NTLA-2001, can help people with a serious inherited heart condition called ATTR-CM. The main goal is to see if the treatment reduces heart-related deaths and hospital visits comp…