ACAAI: Gene-Editing Therapy Reduces Angioedema Attacks in Hereditary Angioedema
By Elana Gotkine HealthDay Reporter
THURSDAY, Oct. 24, 2024 -- For adults with hereditary angioedema, the in vivo gene-editing therapy NTLA-2002, which is based on clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9, administered as a single dose reduces angioedema attacks, according to a study published online Oct. 24 in the New England Journal of Medicine to coincide with the annual meeting of the American College of Allergy, Asthma & Immunology, held from Oct. 24 to 28 in Boston.
Danny M. Cohn, M.D., Ph.D., from the Amsterdam University Medical Center, and colleagues randomly assigned adults with hereditary angioedema to receive NTLA-2002 in a single dose of 25 mg or 50 mg or placebo in a 2:2:1 ratio (10, 11, and six patients, respectively). The number of angioedema attacks per month from week 1 to 16 was the primary end point.
The researchers found that the estimated mean monthly attack rate was 0.70, 0.65, and 2.82 with 25-mg NTLA-2002, 50-mg NTLA-2002, and placebo, respectively (differences, −75 and −77 percent, respectively, compared with placebo). Overall, 40 and 73 percent of the patients who received 25-mg and 50-mg NTLA-2002, respectively, were attack-free with no additional treatment during the period from weeks 1 to 16. Headache, fatigue, and nasopharyngitis were the most common adverse events among patients receiving NTLA-2002.
"These results show the potential of a single dose of the new CRISPR-Cas9-based in vivo gene editing therapy NTLA-2002 to be a functional cure for patients with hereditary angioedema and support continued investigation in a larger phase 3 trial," the authors write.
The study was funded by Intellia Therapeutics, which is developing NTLA-2002.
Abstract/Full Text (subscription or payment may be required)
Editorial (subscription or payment may be required)
Disclaimer: Statistical data in medical articles provide general trends and do not pertain to individuals. Individual factors can vary greatly. Always seek personalized medical advice for individual healthcare decisions.
© 2025 HealthDay. All rights reserved.
Posted October 2024
Read this next
Urinary Metal Levels Tied to Increased Risk for Later Heart Failure
THURSDAY, July 3, 2025 -- There are consistent associations between elevated urinary metal levels and increased heart failure risk over time across geographically diverse cohorts...
Higher Temperatures Tied to Increased Probability of Obstructive Sleep Apnea
THURSDAY, July 3, 2025 -- Higher temperatures across the globe are associated with an increased probability of having obstructive sleep apnea (OSA), according to a study published...
Tons of Turkey Bacon Products Recalled Due to Possible Listeria Contamination
THURSDAY, July 3, 2025 — Kraft Heinz Foods is recalling some 370,000 pounds of fully cooked turkey bacon products that may be contaminated with listeria, the U.S...
More news resources
- FDA Medwatch Drug Alerts
- Daily MedNews
- News for Health Professionals
- New Drug Approvals
- New Drug Applications
- Drug Shortages
- Clinical Trial Results
- Generic Drug Approvals
Subscribe to our newsletter
Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.