Intellia reported Phase 3 results for lonvo-z, its in vivo CRISPR medicine for hereditary angioedema (HAE), showing attack rates fell 87% versus placebo in an 80-patient pivotal study. More than 60% of treated patients were attack-free over the evaluation period compared with 11% on placebo, with no serious safety signals reported in the company’s disclosure. Intellia said it has initiated a rolling submission with the FDA, positioning the therapy as the second CRISPR-based medicine in the market after Vertex’s Casgevy. An in vivo approach edits genes directly in the body, differing from ex vivo systems that rely on collecting and modifying cells outside the patient. The company also framed the outcome as a functional cure-type improvement, while investors continue to debate commercial potential given the broader category’s pricing and competition dynamics.
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