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ACAAI: Gene-Editing Therapy Reduces Angioedema Attacks in Hereditary Angioedema

By Elana Gotkine HealthDay Reporter

Medically reviewed by Carmen Pope, BPharm. Last updated on Oct 24, 2024.

via HealthDay

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.

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