Intellia Starts Phase 3 on Single-Dose CRISPR Treatment for Transthyretin Amyloidosis with Cardiomyopathy

 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced the first patient dosed in the global pivotal, Phase 3 MAGNITUDE trial of NTLA-2001. NTLA-2001 is an investigational in vivo CRISPR-based therapy designed as a single-dose treatment to inactivate the TTR gene and thereby prevent the production of TTR protein for the treatment of transthyretin (ATTR) amyloidosis. The MAGNITUDE trial is evaluating the efficacy and safety of NTLA-2001 in patients with ATTR amyloidosis with cardiomyopathy.

https://www.biospace.com/article/releases/intellia-therapeutics-announces-first-patient-dosed-in-the-phase-3-magnitude-study-of-ntla-2001-as-a-single-dose-crispr-based-treatment-for-transthyretin-amyloidosis-with-cardiomyopathy/