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Shane Stone

Pennsylvania State University

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JUL 27, 2026 · JOURNAL ARTICLE

Lonvoguran ziclumeran: a CRISPR-CAS9-based gene therapy for the treatment of hereditary angioedema

Expert Opinion on Investigational Drugs

Phase 3 RCT with sustained reductions in disease biomarkers and clinical attack frequency from a single administration; rigorous design supports potential practice impact, though long-term durability data remain incomplete.

Strong EvidenceVan Bui Bhattarai Bk Timothy Craig

Study details

DesignPhase 3 randomized controlled trial (HAELO)

InterventionLonvoguran ziclumeran (Lonvo-z; NTLA-2002):…

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