Nucleic Acids

Nucleic Acids
Accession Number

High molecular weight polymers containing a mixture of purine and pyrimidine nucleotides chained together by ribose or deoxyribose linkages.

DrugDrug Description
AmolimogeneIntended for the treatment of cervical dysplasia associated with the human papillomavirus (HPV).
MipomersenAn oligonucleotide drug used for the treatment of homozygous familial hypercholesterolemia.
BevasiranibInvestigated for use/treatment in macular degeneration and diabetic retinopathy.
FomivirsenIndicated for the local treatment of cytomegalovirus (CMV) retinitis in patients with acquired immunodeficiency syndrome (AIDS), when other therapy has been ineffective or is considered unsuitable [FDA Label, L1428].
PatisiranA transthyretin-directed small interfering RNA used to treat polyneuropathy of hereditary transthyretin-mediated amyloidosis.
OblimersenNot Annotated
InotersenAn antisense oligonucleotide used to treat polyneuropathy of hereditary transthyretin-mediated amyloidosis.
InclisiranA PCSK9-targeting short interfering RNA (siRNA) that lowers plasma LDL-cholesterol levels.
7-methyl-5'-guanylic acidNot Available
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)).
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).
GolodirsenA drug used to treat certain mutations that cause Duchenne muscular dystrophy (DMD).
LumasiranLumasiran is an interfering RNA that silences hydroxyacid oxidase 1 for the treatment of primary hypoxaluria type 1.
Golnerminogene pradenovecNot Annotated
Allovectin-7Allovectin-7 or allovectin-7(r) is a lipid complex that contains the DNA sequences encoding HLA-B7 and beta-2 microglobulin. It was discontinued after it failed to improve the objective response rate or...
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
MipomersenmRNA of ApoB-100target
BevasiranibVascular endothelial growth factor Atarget
Fomivirsen30 kDa immediate-early protein 2target
Fomivirsen45 kDa immediate-early protein 2target
PatisiranTransthyretin mRNAtarget
PatisiranSerum albumincarrier
Patisiranalpha1-acid glycoproteincarrier
InotersenTransthyretin mRNAtarget
InclisiranProprotein convertase subtilisin/kexin type 9target
7-methyl-5'-guanylic acidm7GpppX diphosphatasetarget
LumasiranHydroxyacid oxidase 1target
LumasiranSerum albumincarrier
LumasiranCytochrome P450 2C8enzyme