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

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Intellia Therapeutics

said its Crispr-based treatment for a rare swelling condition met its goals in a late-stage trial, marking a milestone for the field of gene editing and putting the company on track to seek approval from the U.S. Food and Drug Administration.

The company’s treatment uses Nobel Prize-winning technology Crispr to edit DNA and turn off the gene that controls production of a peptide that’s overactive in people with hereditary angioedema, causing them to experience potentially life-threatening swelling attacks. Intellia’s treatment is administered once through an hourslong infusion, making the edits directly in the liver.

Intellia said the one-time treatment reduced attacks by 87% compared with a placebo, meeting the study’s main goal. Six months after treatment, 62% of patients were free from attacks and weren’t using other therapies, Intellia said.