CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema

This phase 1 study evaluates NTLA 2002, a CRISPR Cas9 based in vivo gene-editing therapy targeting KLKB1 for hereditary angioedema. Ten patients received single intravenous doses of 25 mg, 50 mg, or 75 mg. The treatment showed dose dependent reductions in plasma kallikrein levels (67% to 95%) and angioedema attacks (91% to 97%) with no serious adverse events. The therapy uses lipid nanoparticles to deliver Cas9 mRNA and sgRNA to hepatocytes, permanently disrupting KLKB1 expression. NTLA 2002 demonstrated promising safety and efficacy, supporting further investigation as a one-time treatment for hereditary angioedema.

This phase 1 study evaluates NTLA 2002, a CRISPR Cas9 based in vivo gene-editing therapy targeting KLKB1 for hereditary angioedema. Ten patients received single intravenous doses of 25 mg, 50 mg, or 75 mg. The treatment showed dose dependent reductions in plasma kallikrein levels (67% to 95%) and angioedema attacks (91% to 97%) with no serious adverse events. The therapy uses lipid nanoparticles to deliver Cas9 mRNA and sgRNA to hepatocytes, permanently disrupting KLKB1 expression. NTLA 2002 demonstrated promising safety and efficacy, supporting further investigation as a one-time treatment for hereditary angioedema.