Intellia Therapeutics said its in vivo CRISPR therapy lonvoguran ziclumeran (lonvo-z) hit a Phase 3 primary endpoint in hereditary angioedema, prompting the company to begin a rolling submission to the FDA. In the 80-patient study, attack rates dropped 87% versus placebo, with more than 60% of patients becoming entirely attack-free during the study period. The result positions lonvo-z for potential approval as a first-in-class in vivo gene editing medicine. Intellia is framing the outcome as a major upgrade over existing hereditary angioedema options, given the magnitude of the reduction in swelling attacks and the absence of new major safety concerns highlighted in the company’s disclosure. Investors will now focus on the regulatory path and commercial readiness for a one-time in-body treatment, as well as durability of response as the program advances toward filing and post-trial follow-up.
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