First In-Body CRISPR Gene Therapy Cures Hereditary Angioedema in Clinical Trial

Intellia Therapeutics announced groundbreaking Phase 3 results for an in vivo CRISPR gene-editing therapy, Lonvo-Z, which achieved a functional cure for hereditary angioedema with high efficacy and safety.

The top 3

  1. First Ex Vivo Cures for Sickle Cell and Beta-Thalassemia: Casgevy (exagamglogene autotemcel), developed by Vertex Pharmaceuticals and CRISPR Therapeutics, became the first FDA-approved CRISPR therapy, effectively treating severe sickle cell disease and transfusion-dependent beta-thalassemia by editing patients' stem cells outside the body to increase fetal hemoglobin.
  2. Pioneering In Vivo Therapies for Transthyretin Amyloidosis: NTLA-2001, an in vivo CRISPR therapy by Intellia Therapeutics and Regeneron, was the first to be administered directly into the bloodstream to treat transthyretin amyloidosis (ATTR), demonstrating rapid and sustained reductions of the disease-causing protein by up to 96%.
  3. CRISPR's Role in Enhancing Agricultural Resilience: CRISPR technology is transforming agriculture by enabling precise genetic modifications in crops to enhance disease resistance (e.g., in rice, wheat, tomatoes), improve drought tolerance, and boost yields, contributing to global food security.

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