Accession Number

Complex compounds of high molecular weight occurring in living cells. These are basically of two types, ribonucleic (RNA) and deoxyribonucleic (DNA) acids, both of which consist of nucleotides (nucleoside phosphates linked together by phosphate bridges).

DrugDrug Description
PegaptanibA selective vascular endothelial growth factor (VEGF) antagonist used for the treatment of neovascular (wet) age-related macular degeneration.
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].
VolanesorsenAn antisense oligonucleotide indicated to treat familial chylomicronemia.
ViltolarsenAn antisense phosphorodiamidate morpholino oligonucleotide specific for exon 53 of the human DMD gene that is capable of inducing exon 53 skipping to produce a functional truncated dystrophin protein in Duchenne muscular dystrophy patients with specific underlying mutations.
CasimersenAn antisense phosphorodiamidate morpholino oligonucleotide used to treat Duchenne muscular dystrophy patients with mutations amenable to exon 45 skipping.
Adenylyl-(3'-5')-uridine 3'-monophosphateNot Available
DefibrotideA mixture of single-stranded oligonucleotides used in the treatment of severe hepatic veno-occlusive disease with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation.
LErafAONIntended for the treatment of various forms of cancer.
AGRO100Investigated for use/treatment in cancer/tumors (unspecified), kidney cancer, leukemia (myeloid), and pancreatic cancer.
LY2181308Investigated for use/treatment in cancer/tumors (unspecified) and solid tumors.
LY2275796Investigated for use/treatment in cancer/tumors (unspecified) and solid tumors.
Egaptivon pegolPlatelet aggregation, thrombosis and acute coronary syndromes
GTI-2501Investigated for use/treatment in prostate cancer and solid tumors.
ISS-1018Investigated for use/treatment in colorectal cancer, hepatitis (viral, B), and lymphoma (non-hodgkin's). ISS 1018 is currently being investigated in combination with HBsAg vaccine as a prophylactic treatment for to prevent...
MipomersenAn oligonucleotide drug used for the treatment of homozygous familial hypercholesterolemia.
CPG 10101Investigated for use/treatment in hepatitis (viral, C) and viral infection.
NF-kappaB DecoyInvestigated for use/treatment in atopic dermatitis, inflammatory disorders (unspecified), and skin infections/disorders.
TrabedersenInvestigated for use/treatment in brain cancer, colorectal cancer, melanoma, and pancreatic cancer.
GEM-231Investigated for use/treatment in solid tumors.
ArchexinInvestigated for use/treatment in solid tumors.
EteplirsenAn antisense oligonucleotide used to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene that is amenable to exon 51 skipping.
ApatorsenInvestigated for use/treatment in cancer/tumors (unspecified).
IMO-2055Investigated for use/treatment in renal cell carcinoma, solid tumors, and lung cancer.
AEG35156Investigated for use/treatment in cancer/tumors (unspecified) and leukemia (myeloid).
AlicaforsenInvestigated for use/treatment in crohn's disease and ulcerative colitis.
PegnivacoginInvestigated for use/treatment in thrombosis, coronary artery disease, and vascular diseases.
G4460Investigated for use/treatment in cancer/tumors (unspecified) and leukemia (myeloid).
AprinocarsenInvestigated for use/treatment in lung cancer.
EdifoligideInvestigated for use/treatment in cardiac surgery, peripheral vascular disease, and kidney disease.
AbetimusInvestigated for use/treatment in kidney disease and systemic lupus erythematosus.
Lexaptepid pegolLexaptepid pegol has been used in trials studying the treatment of Anemia, Inflammation, Chronic Diseases, End Stage Renal Disease, and Anemia of Chronic Disease.
MiravirsenMiravirsen has been investigated for the treatment of Hepatitis C and Hepatitis C, Chronic.
NusinersenA medication used to treat spinal muscular atrophy.
DrisapersenNot Annotated
OblimersenNot Annotated
InotersenAn antisense oligonucleotide used to treat polyneuropathy of hereditary transthyretin-mediated amyloidosis.
TofersenAn antisense oligonucleotide indicated for the treatment of adult amyotrophic lateral sclerosis caused by SOD1 mutation.
CobitolimodCobitolimod is under investigation in clinical trial NCT03178669 (The Efficacy of Cobitolimod in Patients With Moderate to Severe Active Ulcerative Colitis).
DanvatirsenDanvatirsen is under investigation in clinical trial NCT03334617 (Phase II Umbrella Study of Novel Anti-cancer Agents in Patients With NSCLC Who Progressed on an Anti-PD-1/PD-L1 Containing Therapy.).
ImetelstatImetelstat is under investigation in clinical trial NCT01916187 (Imetelstat Given Intravenously Alone and With Standard 13-Cis-Retinoic Acid in Children With Neuroblastoma).
BazlitoranBazlitoran is under investigation in clinical trial NCT02092909 (Phase 1/2 Dose Escalation Study in Patients With Relapsed or Refractory Waldenstrom's Macroglobulinemia).
MongersenMongersen is under investigation in clinical trial NCT02957474 (A Study to Evaluate the Effect of Food, Formulation Strength, and a Proton Pump Inhibitor on GED 0301 Pharmacokinetics in Healthy Adult...
GTI-2040GTI-2040 is under investigation in clinical trial NCT00084643 (GTI-2040, Oxaliplatin, and Capecitabine in Treating Patients With Locally Advanced or Metastatic Colorectal Cancer or Other Solid Tumors).
ARC-19499ARC-19499 is under investigation in clinical trial NCT01191372 (First-in-Human and Proof-of-Mechanism Study of ARC19499 Administered to Hemophilia Patients).
RadavirsenRadavirsen is under investigation in clinical trial NCT01375985 (Safety Study of Single Administration Intravenous Treatment for Influenza).
AganirsenAganirsen is under investigation in clinical trial NCT02947867 (Trial of Aganirsen in iCRVO Patients at Risk of Developing NVG).
CenersenCenersen is under investigation in clinical trial NCT02243124 (A Study of Aezea® (Cenersen) in Transfusion Dependent Anemia Associated With Myelodysplastic Syndrome (MDS)).
GolodirsenA drug used to treat certain mutations that cause Duchenne muscular dystrophy (DMD).
AnivamersenAnivamersen is under investigation in clinical trial NCT01848106 (A Study to Determine the Efficacy and Safety of REG1 Compared to Bivalirudin in Patients Undergoing PCI).
EluforsenEluforsen is under investigation in clinical trial NCT02532764 (Dose Escalation Study of QR-010 in Homozygous ΔF508 Cystic Fibrosis Patients).
ISIS 2503ISIS 2503 is under investigation in clinical trial NCT00004193 (ISIS 2503 in Treating Patients With Metastatic And/or Locally Recurrent Colorectal Cancer).
ISIS 104838ISIS 104838 is under investigation in clinical trial NCT00048321 (ISIS 104838, an Inhibitor of Tumor Necrosis Factor, for Active Rheumatoid Arthritis).
PelacarsenPelacarsen is under investigation in clinical trial NCT03070782 (Phase 2 Study of ISIS 681257 (Akcea-apo(a)-lrx) in Participants With Hyperlipoproteinemia(a) and Cardiovascular Disease).
RintatolimodNot Annotated
MonarsenNot Annotated
RosomidnarNot Available
HiltonolNot Available
AgatolimodAgatolimod is under investigation in clinical trial NCT00471471 (Vaccine Therapy in Treating Patients With Recurrent Stage III or Stage IV Melanoma That Cannot Be Removed by Surgery).
AVR118AVR118 represents a new type of biopolymer chemistry that also possesses novel immunomodulator activity. This non-toxic peptide-nucleic acid, which to date has shown no indication of human toxicity, appears to...
CobomarsenCobomarsen is under investigation in clinical trial NCT02580552 (Safety, Tolerability and Pharmacokinetics of MRG-106 in Patients With Mycosis Fungoides (MF), CLL, DLBCL or ATLL).
TilsotolimodTilsotolimod is under investigation in clinical trial NCT03865082 (Study of Tilsotolimod in Combination With Nivolumab and Ipilimumab for the Treatment of Solid Tumors (ILLUMINATE-206)).
IMT504IMT504, a non-CpG 24-mer oligodeoxynucleotide, is an immunomodulatory oligonucleotide currently being investigated as a rabies vaccine.
AVI-7288Not Annotated
TominersenNot Annotated
AtesidorsenNot Annotated
ISTH0036ISTH0036 is a synthetic 14-mer phosphorothioate antisense oligonucleotide directed against TGF-beta2 mRNA
DonidalorsenNot Annotated
Drugs & Drug Targets
PegaptanibVascular endothelial growth factor Atarget
Fomivirsen30 kDa immediate-early protein 2target
Fomivirsen45 kDa immediate-early protein 2target
VolanesorsenApolipoprotein C-IIItarget
ViltolarsenDMD gene (exon 53 viltolarsen target site)target
CasimersenDMD gene (exon 45 casimersen target site)target
CasimersenCytochrome P450 3A4enzyme
CasimersenCytochrome P450 3A5enzyme
CasimersenCytochrome P450 2C9enzyme
CasimersenCytochrome P450 2C19enzyme
Adenylyl-(3'-5')-uridine 3'-monophosphateDiphtheria toxintarget
DefibrotideAdenosine receptor A1target
DefibrotideAdenosine receptor A2atarget
DefibrotideAdenosine receptor A2btarget
LErafAONRAF proto-oncogene serine/threonine-protein kinasetarget
AGRO100NF-kappa-B essential modulatortarget
LY2181308Baculoviral IAP repeat-containing protein 5target
LY2275796Eukaryotic translation initiation factor 4Etarget
Egaptivon pegolvon Willebrand factortarget
Egaptivon pegolPlatelet glycoprotein Ib alpha chaintarget
ISS-1018Toll-like receptor 9target
MipomersenmRNA of ApoB-100target
CPG 10101Toll-like receptor 9target
NF-kappaB DecoyNuclear factor NF-kappa-Btarget
TrabedersenTransforming growth factor beta-2target
GEM-231cAMP-dependent protein kinase type II-alpha regulatory subunittarget
GEM-231cAMP-dependent protein kinase type I-alpha regulatory subunittarget
ArchexinRAC-alpha serine/threonine-protein kinasetarget
EteplirsenDMD-001 gene (exon 51 target site)target
ApatorsenHeat shock protein beta-1target
AEG35156E3 ubiquitin-protein ligase XIAPtarget
G4460Transcriptional activator Mybtarget
AprinocarsenProtein kinase C alpha typetarget
NusinersenSurvival motor neuron proteintarget
InotersenTransthyretin mRNAtarget
TofersenSuperoxide dismutase 1 (SOD1)target
TilsotolimodToll-like receptor 9target