Trending Now - Intellia's CRISPR Therapy Shows Promise in Rare Disease Trial
WHO:
STAT News - Intellia Therapeutics, a biotechnology company.
CNBC - John Leonard, CEO of Intellia Therapeutics.
WHAT:
STAT News - Intellia Therapeutics reported that a single dose of its gene editing treatment, lonvo-z, reduced swelling attacks in patients with hereditary angioedema (HAE) in a Phase 3 trial.
CNBC - The treatment reduced attack rates by 87% compared to a placebo in an 80-patient study.
CNBC - Six months after treatment, 62% of patients were free from attacks and not using other therapies.
CNBC - The most common side effects reported were infusion-related reactions, headaches, and fatigue.
WHERE:
CNBC - The treatment is administered through an infusion that makes edits directly in the liver.
WHEN:
CNBC - Intellia plans to complete its rolling application with the FDA in the second half of this year.
CNBC - The company expects to launch the treatment in the U.S. in the first half of next year, if approved.
WHY:
CNBC - The treatment aims to turn off the gene that controls the production of a peptide that is overactive in people with hereditary angioedema, which causes potentially life-threatening swelling attacks.
HOW:
STAT News - The therapy, lonvo-z, is the first in vivo treatment that edits patients’ DNA directly in the body.
CNBC - The treatment uses CRISPR technology to edit DNA.
(UPDATED April 27, 2026 23:19 UTC)
Intellia Therapeutics has announced promising results from a Phase 3 trial of its gene editing treatment, lonvo-z, which significantly reduced swelling attacks in patients with hereditary angioedema. In an 80-patient study, the treatment cut attack rates by 87%, with 62% of patients remaining attack-free six months after a single infusion, although some experienced common side effects like headaches and fatigue. Intellia plans to submit its application to the FDA in the second half of this year, aiming for a potential U.S. launch of the treatment in the first half of next year, if approved.