RNA, Untranslated

RNA, Untranslated
Accession Number

RNA which does not code for protein but has some enzymatic, structural or regulatory function. Although ribosomal RNA (RNA, RIBOSOMAL) and transfer RNA (RNA, TRANSFER) are also untranslated RNAs they are not included in this scope.

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