Core Insights - Intellia Therapeutics announced positive follow-up data from the Phase 1 clinical trial of its investigational product nexiguran ziclumeran (nex-z) for patients with transthyretin (ATTR) amyloidosis with cardiomyopathy, showcasing significant clinical benefits and disease stabilization [1][2][10] Group 1: Clinical Trial Results - The Phase 1 trial demonstrated a consistent and durable reduction in serum TTR levels, with a mean reduction of 87% observed in patients followed for 36 months [3] - At 24 months, 70% of patients showed stability or improvement in NT-proBNP levels, and 85% showed stability or improvement in hs-Troponin T levels, indicating positive outcomes in disease progression [5] - Functional status preservation was noted, with 69% of patients showing stability or improvement in the 6-minute walk test, and 81% of patients stable or improved in their NYHA classification [5] Group 2: Mortality Assessment - A post-hoc mortality assessment indicated an all-cause mortality rate of 3.9 per 100 patient-years for patients treated with nex-z, compared to 12.7 per 100 patient-years in a matched cohort, suggesting a significant reduction in mortality risk [6][7] Group 3: Safety Profile - Nex-z was generally well tolerated, with infusion-related reactions and transaminase elevations being the most common treatment-related adverse events, and liver enzyme elevations did not exceed Grade 2 [8] Group 4: Product Overview - Nex-z is based on CRISPR/Cas9 gene editing technology and aims to be the first one-time treatment for ATTR amyloidosis with cardiomyopathy and/or polyneuropathy, currently under investigation in ongoing Phase 3 trials [10][11]