Intellia, Regeneron report positive initial data from phase 1 study of NTLA?2001 for transthyretin amyloidosis treatment

Intellia Therapeutics, Inc. and Regeneron Pharmaceuticals, Inc. announced positive interim results from an ongoing phase 1 clinical trial of NTLA─2001, an investigational, in vivo CRISPR/Cas9 genome editing therapy in development as a single─dose treatment for transthyretin