RNA, Small Untranslated

Name
RNA, Small Untranslated
Accession Number
DBCAT002970
Description

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

Drugs
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.
Drugs & Drug Targets
DrugTargetType
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
RevusiranTransthyretintarget