Randomized Trialhigh evidence

Lonvoguran Ziclumeran - In Vivo CRISPR Gene Editing in Hereditary Angioedema.

The New England journal of medicine·June 13, 2026·PMID 42294842

Why it matters

This trial provides high-level evidence for a novel, single-dose, disease-modifying approach to hereditary angioedema that directly targets the underlying genetic defect, potentially reducing long-term attack burden and the need for chronic prophylaxis.

Infographic summary of Lonvoguran Ziclumeran - In Vivo CRISPR Gene Editing in Hereditary Angioedema.
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