Expert opinion on investigational drugsJul 27, 2026

Gene therapy using CRISPR-Cas9 to treat hereditary angioedema

CRISPR Gene Editing Weekly Brief ↗PubMed ↗DOI ↗

Updated Aug 3, 2026

Abstract

Lonvoguran ziclumeran (Lonvo-z; NTLA-2002) is the first gene-editing therapy designed to permanently disrupt the KLKB1 gene associated with hereditary angioedema (HAE).

Hereditary angioedema (HAE) is caused by a genetic disorder that leads to recurrent swelling.
Current therapies can reduce the frequency of HAE attacks but require lifelong treatment.
Lonvoguran ziclumeran aims to provide a long-lasting solution by editing genes involved in the disorder.
The therapy targets the kallikrein-kinin pathway, which is dysregulated in HAE.

Simplified

Hereditary angioedema (HAE) is a rare genetic disorder characterized by recurrent swelling caused by dysregulation of the kallikrein-kinin pathway. Although current therapies effectively reduce attack frequency, treatment remains lifelong. Lonvoguran ziclumeran (Lonvo-z; NTLA-2002) is the first systemically administered in vivo CRISPR/Cas9 gene-editing therapy designed to provide durable suppression of plasma kallikrein through permanent disruption of the KLKB1 gene.

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