STAT+: Intellia says CRISPR-based treatment for rare disease reduced swelling attacks in pivotal trial

STAT PlusBiotech Intellia says CRISPR-based treatment for rare disease reduced swelling attacks in pivotal trial Data set up approval of what would be second gene editing medicine Manage alerts for this article Email this article Share this article By Jason MastApril 27, 2026 General Assignment Reporter Jason Mast[email protected]Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason on Signal at JasonMast.05. Intellia Therapeutics said Monday that a single dose of its gene editing treatment dramatically reduced swelling attacks in patients with a rare genetic disorder in a Phase 3 trial, setting up a potential approval.

The therapy, known as lonvo-z, would be the second approved CRISPR-based medicine, after Vertex Pharmaceutical’s sickle cell treatment Casgevy. Intellia has already initiated a rolling submission with the agency. It would be the first in vivo treatment, meaning it edits patients’ DNA directly in the body.

Advertisement In the 80-patient study, volunteers with hereditary angioedema (HAE) who received lonvo-z saw their attack rates drop 87%, relative to placebo. Just over 60% were entirely attack-free over the period, compared to 11% for the placebo patients. STAT+ Exclusive Story Already have an account?

Log in This article is exclusive to STAT+ subscribers Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+. Already have an account? Log in Monthly $39 Totals $468 per year $39/month Get Started Totals $468 per year Starter $30 for 3 months, then $399/year $30 for 3 months Get Started Then $399/year Annual $399 Save 15% $399/year Get Started Save 15% 11+ Users Custom Savings start at 25%!

Request A Quote Request A Quote Savings start at 25%! 2-10 Users $300 Annually per user $300/year Get Started $300 Annually per user View All Plans To read the rest of this story subscribe to STAT+. Subscribe Log In biotechnology, CRISPR, drug development, FDA, gene editing, rare diseases, STAT+ Submit a correction requestReprints Jason Mast General Assignment Reporter Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients.

You can reach Jason on Signal at JasonMast.05. Newsletter Understand how science, health policy, and medicine shape the world every day Recommended Biotech April 27, 2026 STAT Plus: Veradermics’ hair loss drug succeeds in late-stage trial Biotech April 27, 2026 STAT Plus: Oruka’s long-acting psoriasis therapy posts strong results in mid-stage study Advertisement Biotech April 24, 2026 STAT Plus: FDA to speed up review of three psychedelics as mental health treatments The Readout April 24, 2026 STAT Plus: Sanofi MS drug gets positive review in Europe following FDA Biotech April 23, 2026 STAT Plus: Trump celebrates closing first round of drug pricing deals, promises more ahead Subscriber Picks