FDA announcements or Intellia Therapeutics press releases

Intellia's NTLA-2002 Shows 81% Attack Reduction in 8 of 11 Patients in Phase 2 HAE Study; FDA Filing Expected in 2026

Oct 24, 2024, 12:18 PM

Intellia Therapeutics has reported promising results from its Phase 2 study of NTLA-2002, an investigational CRISPR gene editing treatment for hereditary angioedema (HAE). In the study, 8 out of 11 patients receiving the mid-level dose showed no attacks during a median follow-up of 9 months post-infusion. The treatment demonstrated an 81% reduction in swelling attacks, indicating a potential for long-term efficacy. The company is planning a larger Phase 3 study with 60 participants and aims to file for FDA approval in 2026. The results position NTLA-2002 as a strong contender in the gene editing landscape, with expectations for it to be one of the first in vivo CRISPR therapies to receive regulatory approval. Other companies, such as Editas Medicine and Beam Therapeutics, are also exploring similar approaches in gene editing for conditions like sickle cell disease and HAE, reflecting a broader shift in the field towards targeted therapies.