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lonvoguran ziclumeran (Intellia Therapeutics)
- Development stage
- Unknown
- Lead developer
- Intellia Therapeutics
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
**Lonvoguran ziclumeran** is an investigational, one-time, intravenously administered in vivo CRISPR-based gene-editing therapy developed by Intellia Therapeutics for hereditary angioedema. The lipid nanoparticle-delivered CRISPR system is designed to permanently inactivate the hepatic **KLKB1** gene, reducing production of plasma prekallikrein and thereby lowering plasma kallikrein and bradykinin activity that drive swelling attacks in hereditary angioedema. The global Phase 3 HAELO study met its primary and key secondary endpoints, and Intellia has initiated a rolling U.S. biologics license application submission. ([ir.intelliatx.com](https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-reports-positive-phase-3-results))
- Brand names
- Lonvo-z
02
Targets
KLKB1 (Plasma kallikrein)
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