RNA, Small Untranslated

RNA, Small Untranslated
Accession Number

Short RNA, about 200 base pairs in length or shorter, that does not code for protein.

DrugDrug Description
InclisiranA PCSK9-targeting short interfering RNA (siRNA) that lowers plasma LDL-cholesterol levels.
BevasiranibInvestigated for use/treatment in macular degeneration and diabetic retinopathy.
PatisiranA transthyretin-directed small interfering RNA used to treat polyneuropathy of hereditary transthyretin-mediated amyloidosis.
FitusiranFitusiran is under investigation in clinical trial NCT02554773 (An Open-label Extension Study of an Investigational Drug, ALN-AT3SC, in Patients With Moderate or Severe Hemophilia A or B).
LumasiranLumasiran is an interfering RNA that silences hydroxyacid oxidase 1 for the treatment of primary hypoxaluria type 1.
RevusiranRevusiran is under investigation in clinical trial NCT02319005 (ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)).
NTLA-2001NTLA-2001 is a clustered regularly interspaced short palindromic repeats CRISPR/ Cas9-based gene therapy consisting of a single guide RNA targeting the human TTR gene and a messenger RNA encoding Cas9.
OlpasiranOlpasiran is under investigation in clinical trial NCT05581303 (Olpasiran Trials of Cardiovascular Events and Lipoprotein(a) Reduction (Ocean(a)) - Outcomes Trial).
Drugs & Drug Targets
InclisiranProprotein convertase subtilisin/kexin type 9target
BevasiranibVascular endothelial growth factor Atarget
PatisiranTransthyretin mRNAtarget
PatisiranSerum albumincarrier
Patisiranalpha1-acid glycoproteincarrier
LumasiranHydroxyacid oxidase 1target
LumasiranSerum albumincarrier
LumasiranCytochrome P450 2C8enzyme