lonvoguran   Click here for help

GtoPdb Ligand ID: 13712

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.
Classification Click here for help
Compound class Nucleic acid
Compound subclass
Target kallikrein B1 (KLKB1) gene
International Nonproprietary Names Click here for help
INN number INN
13064 lonvoguran
Synonyms Click here for help
NTLA-2002 | NTLA2002
Database Links Click here for help
CAS Registry No. 2844372-34-3 (source: WHO INN record)
GtoPdb PubChem SID 507750346
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