CRISPR therapy takes on rare swelling disorder

NCT ID NCT06634420

Ongoing Disease control Sponsor: Intellia Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jul 30, 2026 · Updated 3 times

Summary

This phase 3 trial tests a one-time gene editing treatment called NTLA-2002 for people with hereditary angioedema (HAE), a rare genetic condition causing sudden, painful swelling attacks. About 80 participants will receive either the treatment or a placebo. The goal is to see if NTLA-2002 can reduce the number of attacks by targeting the root genetic cause.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: NTLA-2002 (a gene editing therapy using CRISPR/Cas9 delivered via lipid nanoparticles)
What this could lead to: If it works, this could provide a long-lasting reduction in HAE attacks, potentially freeing patients from regular preventive medications.
What could go wrong: This is still an early-stage trial with only 80 participants. The treatment may not work for everyone, and long-term safety is unknown. Gene editing carries risks like unintended genetic changes.

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Conditions

The condition(s) this trial relates to.

hereditary angioedema hereditary angioedema type 1 hereditary angioedema type 2 hereditary angioedema with C1Inh deficiency

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HEREDITARY ANGIOEDEMA

Contacts and locations

Locations

AARA Research Center

Dallas, Texas, 75231, United States
AP-HM - Hopital de la Timone

Marseille, 13385, France
Allergy & Asthma Clinical Research

Walnut Creek, California, 94598, United States
Amsterdam UMC - Locatie AMC

Amsterdam, 1105 AZ, Netherlands
Asthma & Allergy Associates

Colorado Springs, Colorado, 80907, United States
Bernstein Clinical Research Center, LLC

Cincinnati, Ohio, 45236, United States
CHU Grenoble-Alpes - Hopital Michallon

Grenoble, 38043, France
CHU de Lille - Hopital Claude Huriez

Lille, 59037, France
Cambridge University Hospitals NHS Foundation Trust

Cambridge, CB2 0QQ, United Kingdom
Campbelltown Hospital

Campbelltown, New South Wales, 2560, Australia
Charite Universitaetsmedizin Berlin

Berlin, 12203, Germany
IMMUNOe International Research Centers

Centennial, Colorado, 80112, United States
Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States
Massachusetts General Hospital

Boston, Massachusetts, 02114, United States
Medical Research of Arizona

Scottsdale, Arizona, 85251, United States
Medizinische Hochschule Hannover (MHH)

Hanover, 30625, Germany
NYU Langone Health - Long Island

Mineola, New York, 11501, United States
New Zealand Clinical Research (NZCR)

Auckland, 1010, New Zealand
Ottawa Allergy Research Corporation

Ottawa, Ontario, K1H 1E4, Canada
Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States
Raffi Tachdjian MD, Inc

Santa Monica, California, 90404, United States
Royal Melbourne Hospital

Parkville, Victoria, 3050, Australia
The Alfred Hospital

Melbourne, Victoria, 3004, Australia
Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet

Frankfurt, 60590, Germany
University of Alberta

Edmonton, Alberta, T6G 2G3, Canada
University of California, San Diego (UCSD)

San Diego, California, 92122, United States
University of Cape Town - Lung Institute - Lung Clinical Research Unit

Cape Town, 7700, South Africa
University of South Florida

Tampa, Florida, 33613, United States
Washington University in St. Louis

St Louis, Missouri, 63141, United States

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