Intellia Therapeutics Announces Initiation of HAELO Phase 3 Study of NTLA-2002, an Investigational In Vivo CRISPR Gene Editing Treatment for Hereditary Angioedema (HAE)

NTLA-2002 Development and Clinical Trial - NTLA-2002 is a single-dose CRISPR-based gene editing therapy designed to treat hereditary angioedema (HAE) by inactivating the kallikrein B1 (KLKB1) gene, which encodes for prekallikrein, the kallikrein precursor protein [1][4] - The HAELO Phase 3 trial is a global, randomized, double-blind, placebo-controlled study involving 60 adults with Type I or Type II HAE, with patients randomized 2:1 to receive a single 50 mg infusion of NTLA-2002 or placebo [2] - The primary endpoint of the Phase 3 trial is the change in the number of HAE attacks from week 5 through week 28 [2] - Interim Phase 1 clinical data showed dramatic reductions in attack rates and consistent, deep, and durable reductions in kallikrein levels [3] Regulatory Designations and Potential Impact - NTLA-2002 has received five notable regulatory designations, including Orphan Drug and RMAT Designation by the U.S. FDA, Innovation Passport by the U.K. MHRA, PRIME Designation by the European Medicines Agency, and Orphan Drug Designation by the European Commission [4] - NTLA-2002 has the potential to become the first one-time treatment for HAE, addressing the real-world needs of patients, physicians, and payors [1][4] Hereditary Angioedema (HAE) Overview - HAE is a rare, genetic disease affecting approximately one in 50,000 people, characterized by severe, recurring, and unpredictable inflammatory attacks that can be life-threatening [5] - Current treatment options for HAE include lifelong therapies requiring chronic intravenous or subcutaneous administration, often with breakthrough attacks despite treatment [5] Intellia Therapeutics Overview - Intellia Therapeutics is a leading clinical-stage gene editing company focused on CRISPR-based therapies, with in vivo programs targeting disease-causing genes directly inside the human body and ex vivo programs for cancer and autoimmune diseases [6][7] - The company is advancing its modular gene editing platform, with NTLA-2002 being its second in vivo candidate to enter late-stage clinical development [1]