In vivo CRISPR therapy nears approval after striking Phase 3 results in HAE
Intellia Therapeutics’ Phase 3 study of lonvo-z, its in vivo CRISPR-based treatment for hereditary angioedema, showed an 87% reduction in attack rates versus placebo after a single dose, with over 60% of patients attack-free, prompting an FDA rolling submission and positioning lonvo-z as the second approved CRISPR medicine and the first in vivo gene-editing therapy.