CRISPR therapy takes on rare swelling disorder
NCT ID NCT06634420
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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AARA Research Center
Dallas, Texas, 75231, United States
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AP-HM - Hopital de la Timone
Marseille, 13385, France
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Allergy & Asthma Clinical Research
Walnut Creek, California, 94598, United States
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Amsterdam UMC - Locatie AMC
Amsterdam, 1105 AZ, Netherlands
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Asthma & Allergy Associates
Colorado Springs, Colorado, 80907, United States
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Bernstein Clinical Research Center, LLC
Cincinnati, Ohio, 45236, United States
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CHU Grenoble-Alpes - Hopital Michallon
Grenoble, 38043, France
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CHU de Lille - Hopital Claude Huriez
Lille, 59037, France
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Cambridge University Hospitals NHS Foundation Trust
Cambridge, CB2 0QQ, United Kingdom
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Campbelltown Hospital
Campbelltown, New South Wales, 2560, Australia
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Charite Universitaetsmedizin Berlin
Berlin, 12203, Germany
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IMMUNOe International Research Centers
Centennial, Colorado, 80112, United States
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Icahn School of Medicine at Mount Sinai
New York, New York, 10029, United States
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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Medical Research of Arizona
Scottsdale, Arizona, 85251, United States
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Medizinische Hochschule Hannover (MHH)
Hanover, 30625, Germany
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NYU Langone Health - Long Island
Mineola, New York, 11501, United States
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New Zealand Clinical Research (NZCR)
Auckland, 1010, New Zealand
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Ottawa Allergy Research Corporation
Ottawa, Ontario, K1H 1E4, Canada
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Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033, United States
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Raffi Tachdjian MD, Inc
Santa Monica, California, 90404, United States
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Royal Melbourne Hospital
Parkville, Victoria, 3050, Australia
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The Alfred Hospital
Melbourne, Victoria, 3004, Australia
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Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet
Frankfurt, 60590, Germany
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University of Alberta
Edmonton, Alberta, T6G 2G3, Canada
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University of California, San Diego (UCSD)
San Diego, California, 92122, United States
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University of Cape Town - Lung Institute - Lung Clinical Research Unit
Cape Town, 7700, South Africa
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University of South Florida
Tampa, Florida, 33613, United States
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Washington University in St. Louis
St Louis, Missouri, 63141, United States