NCI Drug Dictionary

The NCI Drug Dictionary contains technical definitions and synonyms for drugs/agents used to treat patients with cancer or conditions related to cancer. Each drug entry includes links to check for clinical trials listed in NCI's List of Cancer Clinical Trials.

anti-transthyretin siRNA ALN-TTR02
A lipid nanoparticle (LNP)-based formulation consisting of small-interfering RNAs (siRNAs) directed against transthyretin (TTR)-encapsulated in lipids, which has potential use in the treatment of TTR-mediated amyloidosis (ATTR). Upon intravenous administration of ALN-TTR02, the lipid formulation promotes the uptake by cells. The siRNAs bind to TTR mRNAs, which may result in the inhibition of both the translation and expression of the TTR protein. ATTR is caused by mutations in the TTR gene and results in the formation of abnormal amyloid proteins that accumulate in and cause damage to various body organs and tissues. Check for active clinical trials using this agent. (NCI Thesaurus)

Synonym:RNAi therapeutic-targeting transthyretin ALN-TTR02
US brand name:Patisiran
Code name:ALN-TTR02