Intellia Therapeutics, Inc. announced that the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) has approved a protocol amendment for the Company?s ongoing Phase 1 study of NTLA-2001 to include patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM). The study of NTLA-2001 now includes patients with ATTR-CM to be enrolled in new dose-escalation and expansion cohorts. The inclusion of the ATTR-CM patient population is in addition to the original Phase 1 study population, which is currently evaluating NTLA-2001 in patients with ATTR amyloidosis with polyneuropathy (ATTRv-PN). The first investigational therapy of its kind, NTLA-2001 is an in vivo CRISPR/Cas9-based genome editing candidate being developed as a single-dose treatment for transthyretin (ATTR) amyloidosis. It is designed to inactivate the TTR gene in liver cells to prevent the production of transthyretin (TTR) protein and is being developed to potentially be the first single-dose ATTR amyloidosis treatment to not only halt but also reverse disease progression in both ATTRv-PN and ATTR-CM patients. NTLA-2001 has received orphan drug designation for the treatment of ATTR amyloidosis by both the European Commission and the U.S. FDA. The protocol amendment to the Phase 1 study allows for enrollment of up to 36 adults in the United Kingdom with either hereditary ATTR-CM (ATTRv-CM) or wild-type cardiomyopathy (ATTRwt-CM), and New York Heart Association Class I ? III heart failure, across the two-part study. The trial?s primary objectives are to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2001, which will include the measurement of serum TTR levels following a single intravenous infusion. The secondary objectives are to evaluate the efficacy of NTLA-2001 on clinical measures of cardiac disease in ATTR-CM patients.