Phase 3 trial results published in scientific journals or press releases

Intellia Therapeutics Initiates HAELO Phase 3 Trial of NTLA-2002, a CRISPR Treatment for HAE

Oct 7, 2024, 11:33 AM

Intellia Therapeutics announced the initiation of HAELO, a global, 60-patient Phase 3 study of NTLA-2002, an investigational in vivo CRISPR gene editing treatment for hereditary angioedema (HAE). NTLA-2002 aims to disrupt the HAE market by potentially offering a one-time treatment solution. Compared to current prophylactic drugs such as lanadelumab and berotralstat, which cost approximately $500,000 annually, NTLA-2002 is expected to cost a fraction over a lifetime, even at a projected price of $3 million. The treatment boasts best overall efficacy and eliminates the need for repeated administrations, potentially improving patient quality of life. The initiation comes ahead of the upcoming Phase 2 data presentation at the American College of Allergy, Asthma & Immunology (ACAAI), which could significantly catalyze enrollment in the study.