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Synonyms: NTLA-2002 | NTLA2002
Compound class:
Nucleic acid
Comment: Lonvoguran (NTLA-2002) is a synthetic single guide RNA CRISPR/Cas drug that targets the human KLKB1 gene and knocks out plasma kallikrein expression [1-3]. The guide RNA (G012267) is encapsulated within lipid nanoparticles for effective delivery. A single dose of lonvoguran is proposed to provide lifelong control of angioedema attacks, for patients with uncontrolled plasma kallikrein production due to mutations in the SERPING1 gene.
We have been unable to locate a full chemical SMILES for the drug, or its HELM notation. We provide the basic nucleic acid structure for the molecule here. Full specifications for all modifications are available from its INN record. |
| No information available. |
Summary of Clinical Use  |
| Lonvoguran (NTLA-2002) is being evaluated for safety and efficacy as a potential treatment for hereditary angioedema (HAE). The agent was designated as an orphan drug for HAE by the FDA in September 2022. |
| Clinical Trials | |||||
| Clinical Trial ID | Title | Type | Source | Comment | References |
| NCT05120830 | NTLA-2002 in Adults With Hereditary Angioedema (HAE) | Phase 1/Phase 2 Interventional | Intellia Therapeutics | ||
| NCT06634420 | HAELO: a Phase 3 Study to Evaluate NTLA-2002 in Participants with Hereditary Angioedema (HAE) | Phase 3 Interventional | Intellia Therapeutics | ||
