Problem: The intended effect of treatment with NTLA-2002 is to permanently rebalance the disease pathway by decreasing the production of bradykinin, which results in the prevention of angioedema attacks. Here, we report data from the phase 1 portion of a phase 1-2 trial to evaluate the safety, pharmacodynamics, pharmacokinetics, and preliminary efficacy of NTLA-2002 for the treatment of patients with hereditary angioedema. Methods Study Design and Oversight The phase 1 portion of this phase 1-2 trial is an ongoing multicenter, open-label, dose-escalation study with up to 30 patients that is assessing the safety, side-effect profile, pharmacodynamics, pharmacokinetics, and preliminary efficacy of NTLA-2002 therapy in patients with hereditary angioedema. The study does not have a placebo control. Phase 1 was designed to include up to three dose-escalation cohorts and two optional dose-reduction cohorts, with three to six evaluable patients per cohort for the assessment of dose-limiting toxic effects. Patients were enrolled at three sites in New Zealand, the Netherlands, and the United Kingdom. Need Assignment Help?