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

Intellia Therapeutics, building exterior and company sign, Cambridge, Massachusetts, USA. Spencer Grant | Universal Images Group | 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. The company described the safety and tolerability of the treatment as "favorable," reporting the most common side effects were infusion-related reactions, headaches and fatigue. Analysts were closely watching safety in the trial since a patient in a separate trial of a different treatment from Intellia died from liver toxicity. …