Intellia's CRISPR Therapy: Phase II Success Cuts HAE Attacks Amid Stock Drop

Intellia Therapeutics has achieved promising results in its Phase II trial for NTLA-2002, an in vivo CRISPR-based therapy targeting hereditary angioedema (HAE). The treatment demonstrated a significant reduction in monthly HAE attacks, achieving an 81% reduction at higher doses. In particular, eight out of eleven patients receiving the 50 mg dose experienced no attacks over a median follow-up of eight months[1][2]. Despite these promising outcomes, Intellia's stock fell, possibly due to unmet high expectations and competition from other treatments[1][2][3]. Nonetheless, the therapy is viewed as a potential game-changer, preparing to enter late-stage development[1][3].