Drug intelligence / Profile preview

lonvoguran ziclumeran

Development stage
Unknown
Lead developer
Intellia Therapeutics
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

**Lonvoguran ziclumeran** is an investigational, wholly owned **Intellia Therapeutics** in vivo CRISPR/Cas9 gene-editing therapy for hereditary angioedema. It is administered as a single outpatient intravenous lipid-nanoparticle infusion that delivers Cas9 messenger RNA and a guide RNA to hepatocytes, permanently disrupting the **KLKB1** gene. KLKB1 encodes prekallikrein; its inactivation lowers plasma kallikrein and downstream bradykinin production, aiming to prevent the recurrent edema attacks characteristic of hereditary angioedema. The global randomized Phase 3 HAELO study met its primary and key secondary efficacy endpoints, and Intellia initiated a rolling US biologics license application submission in April 2026. ([intelliatx.com](https://www.intelliatx.com/wp-content/uploads/ACAAI-2025_Lonvo-z-Ph1-2-50mg-and-LTFU_Oral_FINAL.pdf))

Other names
lonvo-z
02

Targets

KLKB1 (Plasma kallikrein)

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