Lipid Nanoparticle Encapsulating mRNA Encoding Cas9 Protein and TTR-targeting Single Guide RNA NTLA-2001

**Semantic type:** Pharmacologic Substance

**Definition:** A lipid nanoparticle (LNP) formulation encapsulating messenger RNA (mRNA) encoding the clustered regularly interspaced short palindromic repeat (CRISPR)-associated protein 9 (Cas9) protein and a single guide RNA targeting transthyretin (TTR), that may decrease serum TTR concentration. Upon administration of LNP encapsulating mRNA encoding Cas9 protein and TTR-targeting single guide RNA NTLA-2001, the LNP moiety binds to the plasma membrane of nearby cells and releases the Cas9 mRNA and the TTR-targeting single guide RNA into the cells. The mRNA is then translated by the cellular protein translation machinery to produce Cas9, which gene-edits the TTR gene in vivo, thereby lowering the levels of misfolded TTR in the blood. This may reduce the accumulation of misfolded TTR protein in the tissues.

**Synonyms:** - LNP Encapsulating mRNA Encoding Cas9 Protein and TTR-targeting Single Guide RNA NTLA-2001 - NTLA 2001 - NTLA-2001 - NTLA2001