Intellia Therapeutics reported Phase 3 success for lonvo-z, an in vivo CRISPR-based therapy for hereditary angioedema, hitting the study’s primary endpoint and cutting attack rates by 87% versus placebo. The company said it has begun preparing a rolling submission to the FDA for potential approval. In the 80-patient HAELO study, Intellia reported that just over 60% of participants in the lonvo-z arm became attack-free during the assessment period, compared with 11% on placebo. The company said no serious safety signals emerged, positioning the treatment as a potential one-time, genome-editing approach. The Phase 3 milestone makes Intellia’s therapy a key market test case for in vivo CRISPR, where commercialization hinges on durability, safety management, and how regulators weigh permanent genome editing in humans.
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