Intellia said its lonvo-z CRISPR therapy achieved the primary endpoint in a Phase 3 trial for hereditary angioedema and has now moved toward an FDA submission. The biotech reported an 87% reduction in attack rates versus placebo, with more than 60% of patients remaining attack-free over the assessment period. The program is designed as a one-time in vivo gene-editing approach that targets disease biology in patients’ bodies, rather than manufacturing edited cell products ex vivo. Intellia also highlighted that it began rolling submission activities, aiming to accelerate regulatory review if safety data continue to support the efficacy signal. With Vertex’s Casgevy as the only recently approved CRISPR-based medicine, lonvo-z is positioned as the next major regulatory benchmark for permanent editing therapies across rare disease indications.