Intellia Therapeutics says its Crispr-based treatment succeeds in pivotal trial

intellia therapeutics said its crispr-based treatment for hereditary angioedema hit its goals in a late-stage trial, putting it on track for fda approval. the one-time infusion reduced swelling attacks by 87% compared to placebo. six months after treatment, 62% of patients were attack-free and off other therapies. the treatment uses crispr to edit dna in the liver, turning off the gene that controls a peptide overactive in people with hae. the most common side effects were infusion-related reactions, headaches, and fatigue. analysts were watching safety closely after a patient died from liver toxicity in a separate intellia trial. intellia has started a rolling fda application and expects to complete it in the second half of this year. if approved, the treatment — lonvoguran ziclumeran — would launch in the us in the first half of next year. it would compete with about a dozen chronic drugs for hae. intellia ceo john leonard said the company hasn't seen a single case in almost six years where the effects diminished, but he stopped short of calling it a functional cure.