lonvoguran

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Ligands
lonvoguran

GtoPdb Ligand ID: 13712

Synonyms: NTLA-2002 | NTLA2002

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.

References
1. Cohn DM, Gurugama P, Magerl M, Katelaris CH, Launay D, Bouillet L, Petersen RS, Lindsay K, Aygören-Pürsün E, Maag D et al.. (2025) CRISPR-Based Therapy for Hereditary Angioedema. N Engl J Med, 392 (5): 458-467. [PMID:39445704]
2. Longhurst HJ, Lindsay K, Petersen RS, Fijen LM, Gurugama P, Maag D, Butler JS, Shah MY, Golden A, Xu Y et al.. (2024) CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema. N Engl J Med, 390 (5): 432-441. [PMID:38294975]
3. (2022) Corrections. J Allergy Clin Immunol Pract, 10 (6): 1673. [PMID:35688510]

References

1. Cohn DM, Gurugama P, Magerl M, Katelaris CH, Launay D, Bouillet L, Petersen RS, Lindsay K, Aygören-Pürsün E, Maag D et al.. (2025)
CRISPR-Based Therapy for Hereditary Angioedema.
N Engl J Med, 392 (5): 458-467. [PMID:39445704]

2. Longhurst HJ, Lindsay K, Petersen RS, Fijen LM, Gurugama P, Maag D, Butler JS, Shah MY, Golden A, Xu Y et al.. (2024)
CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema.
N Engl J Med, 390 (5): 432-441. [PMID:38294975]

3. (2022)
Corrections.
J Allergy Clin Immunol Pract, 10 (6): 1673. [PMID:35688510]