Intellia Therapeutics Completes Enrollment in the Global Phase 3 HAELO Study of Lonvoguran Ziclumeran (lonvo-z) for Hereditary Angioedema

Core Insights - Intellia Therapeutics has completed enrollment in the global Phase 3 HAELO study for lonvoguran ziclumeran (lonvo-z) targeting hereditary angioedema (HAE) with topline data expected in the first half of 2026 [1][2] - The company plans to submit a biologics license application (BLA) in the second half of 2026, aiming for a U.S. launch in the first half of 2027 [1][6] Company Overview - Intellia Therapeutics is a clinical-stage gene editing company focused on CRISPR-based therapies [1][7] - The company aims to address unmet medical needs through innovative gene editing technologies [7] Study Details - The Phase 3 HAELO study is a randomized, double-blind, placebo-controlled trial involving at least 60 adults and adolescents aged 16 years and older with Type I or Type II HAE [2][4] - Key endpoints include the number of HAE attacks and the number of patients achieving attack-free status from week 5 through week 28 [2][4] Treatment Potential - Lonvo-z is designed to be a one-time treatment for HAE, utilizing CRISPR technology to inactivate the KLKB1 gene, which is crucial for preventing HAE attacks [4][5] - Interim Phase 1/2 clinical data indicated significant reductions in attack rates and consistent decreases in kallikrein levels [4] Regulatory Designations - Lonvo-z has received multiple regulatory designations, including Orphan Drug and RMAT Designation from the FDA, Innovation Passport from the U.K. MHRA, and PRIME Designation from the European Medicines Agency [4]