search results:

ATC
Classification
Product Name Synonymous Phase Description ID
    AEG35156   Country Canada Type Antisense DrugBank  
Adaptation Diseases Hepatocellular carcinoma
Company Aegera Therapeutics Inc. Target Molecule X-Linked Inhibitor of Apoptosis (XIAP) Target Protein
UniProt
P98170
Remarks A multicenter randomized open label phase II trial with sorafenib was conducted for patients with advanced hepatocellular carcinoma (HCC).
The combination group with sorafenib showed good tolerability with respect to the objective response rate.
Clinical trials have already been refused.
Phase R
(II)
Nucleic Acid Sequence d(P-thio)[(2'-O-Me)(rU-rG-rC-rA)-C-C-C-T-G-G-A-T-A-C-C-(2'-O-Me)(rA-rU-rU-rU)] PDB 4KJU
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2009-013669-25/DE
https://www.ncbi.nlm.nih.gov/pubmed/24977690
    aganirsen GS101 Country France Type Antisense DrugBank  
Adaptation Diseases inhibition of corneal neovascularisation, a major risk factor of corneal graft rejection
Company Les Laboratoires CTRS Target Molecule insulin receptor substrate-1 (IRS1) Target Protein
UniProt
P35568
Remarks Aganirsen is an antisense oligonucleotide ophthalmic solution that inhibits the function of insulin receptor IRS1.
In Phase II study, inhibition of corneal neovascularization was confirmed.
Double mask, randomization, placebo-controlled Phase III trials at multiple locations examined clinical benefits for vision, QoL, and the need for transplantation.
The trial showed significant inhibition of corneal angiogenesis in patients with keratitis, a significant reduction in transplantation need in viral keratitis and central angiogenic patients. The safety of topical application was also confirmed.
Phase II/III Nucleic Acid Sequence   PDB 1IRS, 1QQG
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2008-005388-33/DE
https://www.clinicaltrialsregister.eu/ctr-search/trial/2004-005015-29/DE
https://www.clinicaltrialsregister.eu/ctr-search/trial/2004-005015-29/results
http://www.ncbi.nlm.nih.gov/pubmed/19643487
https://www.ncbi.nlm.nih.gov/pubmed/24811963
http://www.genesignal.com/GS-101_Aganirsen_Clinical_Tr.32.0.html
    AP 12009   Country Germany Type Antisense DrugBank  
Adaptation Diseases Recurrent or Refractory Anaplastic Astrocytoma (WHO grade III) or Secondary Glioblastoma (WHO grade IV)
Company Antisense Pharma GmbH Target Molecule TGF硫2 Target Protein
UniProt
P61812
Remarks It is an antisense drug that was once developed for the purpose of treating liver cancer.
Selectively inhibits mRNA of TGF硫2 overexpressed in liver malignant tumors.
Clinical trials have already been refused.
Phase R
(III)
Nucleic Acid Sequence   PDB 1M9Z
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2007-005802-38/AT
https://www.ncbi.nlm.nih.gov/pubmed/21366804
    Apatorsen OGX-427 Country UK Type Antisense DrugBank  
Adaptation Diseases advanced squamous cell lung cancers
Company Queen Mary, University of London Target Molecule Hsp27 Target Protein
UniProt
P04792
Remarks OGX-427 is a drug under development for the treatment of advanced non-small cell lung cancer (NSCLC).
Combined use of gemcitabine and carboplatin chemotherapy has been studied.
Phase II Nucleic Acid Sequence   PDB 4PCW, 4MJH
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2014-000199-25/GB
http://urotoday.com/recent-abstracts/urologic-oncology/mcrpc-treatment/87811-a-phase-i-dose-escalation-study-of-apatorsen-ogx-427-an-antisense-inhibitor-targeting-heat-shock-protein-27-hsp27-in-patients-with-castration-resistant-prostate-cancer-and-other-advanced-cancers.html
    ATL1103   Country Australia Type Antisense DrugBank  
Adaptation Diseases Acromegaly
Company Antisense Therapeutics Ltd Target Molecule growth hormone receptor (GHr) Target Protein
UniProt
P10912
Remarks ATL 1103 is a drug under development for the treatment of growth hormone overdiagnosis, acromegaly.
Significant results were obtained in the intermediate analysis stage in the open label phase II study of safety, tolerability, pharmacokinetics and efficacy.
Phase II Nucleic Acid Sequence   PDB 2AEW
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2012-003147-30/GB
http://www.antisense.com.au/atl1103
http://www.cancerresearchuk.org/about-cancer/find-a-clinical-trial/a-trial-apatorsen-chemotherapy-non-small-cell-lung-cancer-spread-cedar#undefined
http://www.evaluategroup.com/Universal/View.aspx?type=Story&id=657335
    AVT-02 UE   Country Germany Type decoy DrugBank  
Adaptation Diseases psoriasis vulgaris
Company Avontec GmbH Target Molecule STAT-1 Target Protein
UniProt
P42224
Remarks A multicenter, randomized, double-blind, placebo-controlled, intraindividual-comparison phase IIa trial, to evaluate the efficacy and safety of 2% AVT-02 UE ointment in the treatment of mild to moderate psoriasis vulgaris, has been carried out.
Phase II Nucleic Acid Sequence   PDB 1BF5
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2007-000462-21/DE
https://www.clinicaltrialsregister.eu/ctr-search/trial/2008-002765-31/DE
https://www.clinicaltrialsregister.eu/ctr-search/trial/2007-001969-15/DE
    Bevasiranib Sodium   Country US Type siRNA DrugBank  
Adaptation Diseases Exudative Age-Related Macular Degeneration
Company Opko Health, Incorporated Target Molecule VEGF Target Protein
UniProt
P15692
Remarks A Phase III sutdy, to patients with exudative age-related macular degeneration (AMD) every 8 weeks or 12 weeks as maintenance therapy after 3 injections of Lucentis compared to Lucentis? monotherapy every 4 weeks a randomized double-blind parallel assessment trial had been continued.
The Phase III trial had been discontinued.
Phase R
(III)
Nucleic Acid Sequence   PDB 3BDY
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2007-003042-15/PT
    BMN 044 PRO44 Country US Type Antisense DrugBank  
Adaptation Diseases Duchenne muscular dystrophy
Company BioMarin Pharmaceutical Inc. Target Molecule dystrophin RNA Target Protein
UniProt
 
Remarks BMN 044 had been developed as a medicine to treat genetic mutation that causes duchenne muscular dystrophy.
In a phase I / II dose escalation study conducted in Europe, the tolerability was good and no serious adverse events were reported.
On May 31, 2016, BioMarin International Limited notified the Pharmaceuticals Committee (CHMP) of withdrawal of marketing approval application.
Phase R
(II)
Nucleic Acid Sequence   PDB  
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2015-003681-87/BE
https://www.clinicaltrialsregister.eu/ctr-search/trial/2013-003605-26/SE
http://www.biomarin.com/products/clinical-trials/duchenne-muscular-dystrophy-bmn-044/
    BMN 053 PRO53 Country US Type Antisense DrugBank  
Adaptation Diseases Duchenne muscular dystrophy
Company BioMarin Pharmaceutical Inc. Target Molecule dystrophin RNA Target Protein
UniProt
 
Remarks BMN 053 had been developed as a medicine to treat genetic mutation that causes duchenne muscular dystrophy.
A phase I / II dose escalation study had been conducted in Europe, but on May 31, 2016, BioMarin International Limited notified the Pharmaceuticals Committee (CHMP) of withdrawal of marketing approval application.
Phase R
(I/II)
Nucleic Acid Sequence   PDB  
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2011-005042-35/GB
http://www.remudy.jp/dystrophinopathy/news/2016/06/000544.html
http://www.biomarin.com/products/clinical-trials/duchenne-muscular-dystrophy-bmn-053/
    CpG 7909 AMA1-C1
PF-3512676
Country US
UK
Germany
Type CpG oligo DrugBank  
Adaptation Diseases matastatic or recurrent malignancies
advanced non-small cell lung cancer
Company Pfizer Inc
Ludwig Institute For Cancer Research
University of Oxford
Pfizer Pharma GmbH
Target Molecule TLR9 Target Protein
UniProt
Q9NR96
Remarks A phase I/II study of immunization with multiple peptides mixed with the immunological adjuvant CpG 7909 in HLA-A2 patients with metastatic melanoma has been initiated.
On the other hand, a phase I/IIa trial of the Safety, Immunogenicity and Parasite Growth Inhibitory Activity of AMA1-C1/AlhydrogelR + CPG 7909, an Asexual Blood Stage Vaccine for Plasmodium falciparum Malaria has been initiated.
Phase III Nucleic Acid Sequence   PDB 4QDH
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2004-003813-18/GB
https://www.clinicaltrialsregister.eu/ctr-search/trial/2004-004686-15/DE
https://www.clinicaltrialsregister.eu/ctr-search/trial/2004-004684-30/DE
https://www.clinicaltrialsregister.eu/ctr-search/trial/2007-005389-11/GB
https://www.clinicaltrialsregister.eu/ctr-search/search?query=CpG
https://www.clinicaltrialsregister.eu/ctr-search/trial/2005-004557-10/results
https://www.clinicaltrialsregister.eu/ctr-search/trial/2006-001588-52/DE
https://www.clinicaltrialsregister.eu/ctr-search/trial/2006-001588-52/results
    Custirsen OGX-011 Country US
Canada
Type Antisense DrugBank  
Adaptation Diseases Metastatic Castrate Resistant Prostate Cancer
Company Teva Pharmaceutical Industries, Ltd
enex Technologies Inc
Target Molecule Clusterin Target Protein
UniProt
P10909
Remarks Custirsen is a drug under development for the purpose of treating metastatic castration-resistant prostate cancer by inhibiting the production of clusterin.
A phase II study with custirsen and chemotherapy suggested the possibility of improved clinical outcome due to clusterin inhibition.
As a result of conducting a phase III study with Cabazitaxel / Prednisone, custirsen did not show a significant prolongation effect compared with Cabazitaxel / Prednisone alone.
Phase III Nucleic Acid Sequence   PDB  
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2010-021011-16/HU
http://oncogenex.com/physicians/custirsen-ogx-011
https://www.clinicaltrialsregister.eu/ctr-search/trial/2010-020802-13/ES
https://www.clinicaltrialsregister.eu/ctr-search/trial/2012-002447-14/HU
http://www.esmo.org/Conferences/Past-Conferences/ESMO-2016-Congress/Press-Media/Custirsen-Shows-no-Survival-Benefits-in-Metastatic-Prostate-Cancer
https://www.cancerit.jp/52338.html
    Drisapersen Kyndrisa Country Netherlands Type Antisense DrugBank  
Adaptation Diseases Duchenne Muscular Dystrophy
Company BioMarin Nederland BV Target Molecule dystrophin RNA Target Protein
UniProt
P11532
Remarks Drisapersen had been developed as a medicine to treat genetic mutation that causes duchenne muscular dystrophy.
Phase III trials were conducted on patients with Duchenne muscular dystrophy, but did not meet the criteria for significance.
On May 31, 2016, BioMarin International Limited notified the Pharmaceuticals Committee (CHMP) of withdraw of marketing approval application.
Phase R
(III)
Nucleic Acid Sequence   PDB 1DXX
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2015-001955-54/NL
http://www.remudy.jp/dystrophinopathy/news/2016/06/000544.html
http://www.medscape.com/viewarticle/857406
    Eteplirsen Exondys 51 Country US Type Antisense DrugBank DB06014
Adaptation Diseases Duchenne muscular dystrophy
Company Sarepta Therapeutics, Inc. Target Molecule DMD-001 Target Protein
UniProt
P11532
Remarks Eteplirsen is a phosphoramidite morpholino sequence complementary to a portion of exon 51. It exerts it's mechanism of action by forcing the exclusion of exon 51 from the mature DMD mRNA.
Phase AD Nucleic Acid Sequence 5'-CTCCAACATCAAGGAAGATGGCATTTCTAG-3' PDB 1EG3
Source https://www.sarepta.com/our-product
https://www.drugbank.ca/drugs/DB06014
http://www.rxlist.com/exondys-51-drug.htm
https://www.drugs.com/ingredient/eteplirsen.html
S01AD08 S01A Fomivirsen Fomivirsen Sodium
Vitravene
Vitravene Preservative
Country US
EU
Type Antisense DrugBank  
Adaptation Diseases Cytomegalovirus Retinitis, HIV Infections
Company Ionis Pharmaceuticals Target Molecule Cytomegalovirus mRNA Target Protein
UniProt
 
Remarks Fomivirsen is used for the topical treatment of cytomegalovirus (CMV) retinitis in AIDS patients.
Phase AD Nucleic Acid Sequence 5'-GCG TTT GCT CTT CTT CTT GCG-3', 20 Na PDB  
Source http://www.rxlist.com/vitravene-drug.htm
http://www.kegg.jp/dbget-bin/www_bget?D07989
    Fovista   Country US Type Aptamer DrugBank  
Adaptation Diseases subfoveal neovascular age-related macular degeneration
Company OPHTHOTECH CORPORATION Target Molecule PDGF-B Target Protein
UniProt
P01127
Remarks Fovista is a new drug expected as a remedy for neovascular age-related macular degeneration.
Currently, 3 kinds of tests shown below has been initiated.
・a phase IIb randomized, double-masked, controlled trial to establish the safety and efficacy of intravitreous administration of Fovista administered in combination with Avastin compared to Avastin monotherapy in subjects with subfoveal neovascular age-related macular degeneration.
・a phase III randomized, double-masked, controlled trial to establish the safety and efficacy of intravitreous administration of Fovista (Anti PDGF-B pegylated aptamer) administered in combination with either Avastin or Eylea compared to Avastin or Eylea monotherapy in subjects with subfoveal neovascular age-related macular degeneration
・a phase III randomized, double-masked, controlled trial in order to establish the safety and effectiveness of intravitreal administration with combination of Fovista and Lucentis compared with administration of only Lucentis
Phase III Nucleic Acid Sequence   PDB 4QCI
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2015-000518-23/NL
https://www.clinicaltrialsregister.eu/ctr-search/trial/2013-003018-42/PT
https://www.clinicaltrialsregister.eu/ctr-search/trial/2013-003017-18/HU
https://www.clinicaltrialsregister.eu/ctr-search/trial/2013-002997-33/GB
https://www.clinicaltrialsregister.eu/ctr-search/trial/2010-018741-65/LV
    HBVAXPRO   Country France Type decoy DrugBank  
Adaptation Diseases Hepatitis B
Company Sanofi Pasteur MSD S.N.C. Target Molecule HBV Target Protein
UniProt
 
Remarks This is an open-label, controlled, multi-center phase III trial of the immunogenicity and safety of a challenge dose of HBVAXPRO?.
The purpose of this trial is to explore the anamnestic immune response in healthy children vaccinated 10 years ago with a primary series (3 doses) of either HEXAVAC? or INFANRIX?-HEXA.
Phase AD Nucleic Acid Sequence   PDB 4K7F
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2013-001602-28/IT
    IONIS-APO(a)-LRx   Country US Type Antisense DrugBank  
Adaptation Diseases High Lipoprotein
Company Ionis Pharmaceuticals Target Molecule Apolipoprotein A Target Protein
UniProt
P02647
Remarks IONIS-APO (a) -LRx is an antisense oligonucleotide designed to lower the Lp (a) concentration, which is a genetic risk factor for cardiovascular disease and calcified aortic valve stenosis.
As a result of randomized, double-blind, placebo-controlled phase I / IIa study, it reduced Lp (a) -mediated cardiovascular risk.
Phase III Nucleic Acid Sequence   PDB 1KIV
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2014-000701-13/DE
http://www.sciencedirect.com/science/article/pii/S0140673616310091
https://www.ncbi.nlm.nih.gov/pubmed?term=ionis-apo (a) lrx&cmd=correctspelling
    IONIS-APO(a)-Rx   Country US Type Antisense DrugBank  
Adaptation Diseases High Lipoprotein
Company Ionis Pharmaceuticals Target Molecule Apolipoprotein A Target Protein
UniProt
P02647
Remarks IONIS-APO (a) -Rx is an antisense oligonucleotide designed to lower the Lp (a) concentration, which is a genetic risk factor for cardiovascular disease and calcified aortic valve stenosis.
As a result of a randomized, double-blind, placebo-controlled Phase II study, this decreased Lp (a) by 80%.
Phase II Nucleic Acid Sequence   PDB 1KIV
Source https://www.ncbi.nlm.nih.gov/pubmed?term=ionis-apo (a) lrx&cmd=correctspelling
https://www.ncbi.nlm.nih.gov/pubmed/28128058
    IONIS-FXIRx Factor XI Antisense Oligonucleotide
BAY 2306001
Country US Type Antisense DrugBank  
Adaptation Diseases Total Knee Arthroplasty
Company Ionis Pharmaceuticals Target Molecule Factor XI gene Target Protein
UniProt
P03951
Remarks IONIS-FXIRx is an antisense drug designed to reduce the production of blood coagulation factor XI, which is responsible for thrombosis.
As a result of a phase II study on patients undergoing total knee replacement surgery, it showed good tolerability.
Safety was equivalent to existing drug enoxaparin.
Severe drug-related adverse events were not reported.
Phase II Nucleic Acid Sequence   PDB 2J8J
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2012-001836-72/LV
http://www.ionispharma.com/spotlight-3/
    IONIS-GCGRRx   Country US Type Antisense DrugBank  
Adaptation Diseases Type 2 Diabetes
Company Ionis Pharmaceuticals Target Molecule Glucagon Receptor Target Protein
UniProt
P47871
Remarks IONIS-GCGRRx is an antisense oligonucleotide inhibitor of the glucagon receptor (GCGR), which is under development for the treatment of type 2 diabetes.
A phase II studies showed that hemoglobin A1c (HbA1c) was significantly reduced compared to the placebo group.
Phase II Nucleic Acid Sequence   PDB 4ERS
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2015-003337-10/HU
https://link.springer.com/article/10.1007/s10928-017-9505-5
http://www.biospace.com/news_story.aspx?StoryID=443134
    IONIS-TTRRx Human Transthyretin Antisense Oligonucleotide Country US Type Antisense DrugBank  
Adaptation Diseases Familial Amyloid Polyneuropathy (FAP)
Company Ionis Pharmaceuticals Target Molecule Human Transthyretin Target Protein
UniProt
P02766
Remarks IONIS-TTRRx is a drug under development for the treatment of TTR familial amyloid polyneuropathy and TTR-related cardiomyopathy.
Following the results of an Open Label phase II study, a phase III sutdy has been carried out.
Phase AD Nucleic Acid Sequence   PDB 3NES
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2013-004561-13/PT
https://www.clinicaltrialsregister.eu/ctr-search/trial/2012-001831-30/GB
https://www.zacks.com/stock/news/218774/ionis-down-glaxo-dumps-ionisttrrx-phase-iii-study-plans
https://www.ncbi.nlm.nih.gov/pubmed/27355239
    ISIS EIF4E Rx eIF-4E Antisense Oligonucleotide Country US Type Antisense DrugBank  
Adaptation Diseases Castrate-Resistant Prostate Cancer
Company Ionis Pharmaceuticals Target Molecule eIF-4E Target Protein
UniProt
P06730
Remarks ISIS EIF4E Rx is an antisense drug designed to bind eukaryotic translation initiation factor 4E (eIF4E) , which is a powerful cancer gene including colorectal carcinogenesis (CRC), and inhibit the production of eIF4E protein.
In a phase I / II study conducted on patients with irinotecan refractory colorectal cancer, the peripheral blood concentration of eIF4E mRNA decreased in 13 of 19 cases.
Phase Ib/II Nucleic Acid Sequence   PDB 4TPW
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2010-022239-12/HU
https://www.clinicaltrialsregister.eu/ctr-search/trial/2010-022240-21/HU
https://www.ncbi.nlm.nih.gov/pubmed/27194579
    Lucanix? Belagenpumatucel-L Country US Type Antisense DrugBank  
Adaptation Diseases Advanced Non-small Cell Lung Cancer
Company NovaRx Corporation Target Molecule TGFβ2 Target Protein
UniProt
P61812
Remarks Lucanix is a drug under development for the treatment of NSCLC.
As a result of an international multicenter, randomized, double-blind, placebo-controlled phase III trial for patients with stage III / IV NSCLC, the trials did not meet the survival endpoint.
Patients who were randomized within 12 weeks of completion of chemotherapy and had received previous radiation suggested an improvement in the survival rate of belagenpumatucel-L.
Phase III Nucleic Acid Sequence   PDB 1M9Z
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2007-005234-36/GB
http://www.cancernetwork.com/articles/lucanix-anti-tgf-%CE%B22-vaccine-may-prolong-nsclc-survival
https://clinicaltrials.gov/ct2/show/NCT00676507
https://www.ncbi.nlm.nih.gov/pubmed/26283035
S01LA03 S01L Macugen Pegaptanib Country Germany
US
Type Aptamer DrugBank DB04895
Adaptation Diseases Wet Macular Degeneration
neovascular age-related macular degeneration
Company Society (Institute) for clinical research
(OSI) Eyetech, Inc
Target Molecule VEGF Target Protein
UniProt
P15692
Remarks Macugen is used for the treatment of angiogenic age-related macular degeneration (AMD).
Phase AD Nucleic Acid Sequence ((2'-deoxy-2'-fluoro)C-Gm-Gm-A-A-(2'-deoxy-2'-fluoro)U-(2'-deoxy-2'-fluoro)C-Am-Gm-(2'-deoxy-2'-fluoro)U-Gm-Am-Am-(2'-deoxy-2'-fluoro)U-Gm-(2'-deoxy-2'-fluoro)C-(2'-deoxy-2'-fluoro)U-(2'-deoxy-2'fluoro)U-Am-(2'-deoxy-2'-fluoro)U-Am-(2'-deoxy-2'-fluoro)C-Am-(2'-deoxy-2'-fluoro)U-(2'deoxy-2'-fluoro)C-(2'-deoxy-2'-fluoro)C-Gm-(3'→3')-dT), 5'-ester with α,α'-[4,12-dioxo-6[[[5-(phosphoonoxy)pentyl]amino] carbonyl]-3,13-dioxa-5,11-diaza-1,15pentadecanediyl]bis[ω-methoxypoly(oxy-1,2-ethanediyl)] PDB 3BDY
Source http://www.rxlist.com/macugen-drug.htm
http://www.kegg.jp/dbget-bin/www_bget?D05386
https://www.clinicaltrialsregister.eu/ctr-search/trial/2007-006290-90/DE
https://www.clinicaltrialsregister.eu/ctr-search/trial/2006-000722-31/DE
C10AX11 C10A Mipomersen Mipomersen Sodium
Kynamro
Country US
EU
JPN
Type Antisense DrugBank DB05528
Adaptation Diseases Hypercholesterolemia
Company Ionis Pharmaceuticals
Genzyme Corporation and its Affiliates
Genzyme Europe B.V.
Hospital of the University of Munich
Target Molecule apolipoprotein B mRNA Target Protein
UniProt
P04114
Remarks Mipomersen is used to treat homozygotes of patients with hypercholesterolemia and familial hypercholesterolemia.
This can be administered systemically.
Phase AD Nucleic Acid Sequence 5'-mG-mC*-mC*-mU*-mC*-dA-dG-dT-dC*-dT-dG-dC*-dT-dT-dC*-mG-mC*-mA-mC*-mC*-3' PDB 5K81, 5K82, 5K83
Source http://www.rxlist.com/kynamro-drug.htm
http://www.kegg.jp/dbget-bin/www_bget?D08946
https://www.clinicaltrialsregister.eu/ctr-search/trial/2004-003934-32/DE
https://www.clinicaltrialsregister.eu/ctr-search/trial/2005-003449-15/GB
http://www.ncbi.nlm.nih.gov/pubmed/21210756
https://www.clinicaltrialsregister.eu/ctr-search/trial/2005-003450-10/GB
https://www.clinicaltrialsregister.eu/ctr-search/trial/2007-005140-24/NL
https://www.clinicaltrialsregister.eu/ctr-search/trial/2011-001480-42/ES
https://www.clinicaltrialsregister.eu/ctr-search/trial/2008-006020-53/DE
https://www.clinicaltrialsregister.eu/ctr-search/trial/2011-002539-24/DE
    Miravirsen SPC3649 Country Denmark Type Antisense DrugBank  
Adaptation Diseases Chronic Hepatitis C (CHC) Infection
Company Santaris Pharma A/S Target Molecule miRNA-122 Target Protein
UniProt
 
Remarks As a result of randomized, double-blind, placebo-controlled, elevated multiple-dose phase IIa trial of untreated patients with chronic HCV genotype 1 infection, high dose-dependent antiviral activity was demonstrated Indicated.
Moreover, sufficient safety and durability were confirmed, and no side effects leading to cessation of trial were confirmed.
Phase IIa Nucleic Acid Sequence   PDB  
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2010-019057-17/NL
http://nar.oxfordjournals.org/content/42/1/609.full
http://www.news-medical.net/news/20111004/14772/Japanese.aspx
http://mirnablog.com/antiviral-drug-miravirsen-final-phase-2a-results-show-dose-dependent-prolonged-antiviral-activity-in-hepatitis-c-patients/
    Mongersen GED-0301 Country US
Italy
Type Antisense DrugBank  
Adaptation Diseases Crohn’s disease
Company Celgene Corporation
GIULIANI
Target Molecule SMAD7 Target Protein
UniProt
O15105
Remarks Mongersen is a drug under development treating inflammation associated with Crohn's disease.
As a result of double-blind, placebo-controlled phase II study on patients with Crohn's disease, the remission rate and clinical response were significantly higher compared to the placebo control group.
A phase III study has been carried out.
Phase III Nucleic Acid Sequence   PDB 2LTW
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2015-001963-37/LV
http://www.nejm.org/doi/full/10.1056/NEJMoa1407250
https://www.clinicaltrialsregister.eu/ctr-search/trial/2011-002640-27/IT
https://www.clinicaltrialsregister.eu/ctr-search/trial/2015-001925-18/GB
http://www.nejm.org/doi/full/10.1056/NEJMoa1407250
    ND-L02-s0201   Country JPN Type siRNA DrugBank  
Adaptation Diseases Extensive Hepatic Fibrosis
Company Nitto Denko Corporation Target Molecule HSP47 gene Target Protein
UniProt
P50454
Remarks ND L02 s0201 Injection is a Vitamin A-coupled Lipid Nanoparticle Containing siRNA.
A Phase Ib/II trial, Open Label, Randomized, Repeat Dose, Dose Escalation Study to Evaluate the Safety, Tolerability, Biological Activity and Pharmacokinetics of ND L02 s0201 Injection, in Subjects with Moderate to Extensive Hepatic Fibrosis (METAVIR F3-4), has been initiated.
Phase II Nucleic Acid Sequence   PDB 4AU4
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2014-004882-26/BG
    Nusinersen Spinraza, Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Country US
UK
Netherlands
Type Antisense DrugBank  
Adaptation Diseases Infantile-onset Spinal Muscular Atrophy
Company Ionis Pharmaceuticals
BioMarin Nederland BV
Target Molecule Survival of Motor Neuron 2 (SMN2) Splicing Modulator Target Protein
UniProt
Q16637
Remarks Nusinersen is a drug under development for the treatment of spinal muscular atrophy.
In an open-label phase II clinical dose clinical trial, safety, tolerability, pharmacokinetics and clinical efficacy of multiple intrathecal administration to infantile onset spinal muscular atrophy patients were evaluated.
Since both items were satisfied, a phase III trial has been carried out.
Phase AD Nucleic Acid Sequence 5’-mU-mC-A-mC-mU-mU-mU-mC-A-mU-A-A-mU-G-mC-mU-G-G-3’ PDB 2LEH
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2013-004422-29/IT
https://www.clinicaltrialsregister.eu/ctr-search/trial/2014-001947-18/DE
https://www.clinicaltrialsregister.eu/ctr-search/trial/2015-001870-16/DE
https://www.clinicaltrialsregister.eu/ctr-search/trial/2014-002098-12/IT
http://www.thelancet.com/journals/lancet/article/PIIS0140-6736(16)31408-8/abstract
L01XX36 L01X Oblimersen Genasense Country EU Type Antisense DrugBank  
Adaptation Diseases Melanoma
Company   Target Molecule Bcl-2 gene Target Protein
UniProt
P10415
Remarks Oblimersen is an anti-Bcl-2 drug once used to treat cancers such as follicular lymphoma, breast cancer, colon cancer and prostate cancer, and middle and high malignant lymphoma.
By the Human Use Pharmaceuticals Committee (CHMP), marketing approval was refused on July 19, 2007.
Phase III Nucleic Acid Sequence   PDB 1G5M
Source http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/000711/human_med_000806.jsp&mid=WC0b01ac058001d124
http://www.kegg.jp/dbget-bin/www_bget?D08290
http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Public_assessment_report/human/000711/WC500070766.pdf
http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/000711/human_med_000806.jsp&mid=WC0b01ac058001d124
http://www.kegg.jp/dbget-bin/www_bget?D05216
    Resten-MP   Country US Type Antisense DrugBank  
Adaptation Diseases de novo Native Coronary Artery Lesions
Company AVI BioPharma Inc. Target Molecule c-myc Target Protein
UniProt
 
Remarks The active ingredient AVI-4126 of RESTEN-MP is an antisense drug designed to interfere with the ability of the human c-myc gene to translate mRNA into MYC protein.
Phase IIa Nucleic Acid Sequence   PDB  
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2005-000713-35/DE
https://clinicaltrials.gov/ct2/show/NCT00244647
    SB010   Country Germany Type Antisense DrugBank  
Adaptation Diseases mild allergic asthma
Company sterna biologicals GmbH & Co. KG Target Molecule GATA-3 Target Protein
UniProt
P23771
Remarks A multicenter, randomized, double-blind, placebo-controlled clinical trial of SB010 was conducted for patients who had allergic asthma accompanied by eosinophilia in sputum and showed immediate / delayed biphasic asthmatic response after inhalation of allergen in the laboratory.
In allergic asthma patients, both delayed asthmatic response and immediate asthmatic response after allergen inhalation induction were significantly attenuated in allergic asthma patients.
Biomarker analysis showed that inflammatory responses regulated by Th2 are attenuated.
Phase IIa Nucleic Acid Sequence   PDB 4HC7
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2012-003570-77/DE
http://www.sterna-biologicals.com/images/stories/PDF/pr17may15.pdf
http://nejm.jp/abstract/vol372.p1987
http://www.nejm.org/doi/full/10.1056/NEJMoa1411776
    SPC2996   Country Denmark Type Antisense DrugBank  
Adaptation Diseases Chronic Lymphocytic Leukaemia
Company Santaris Pharma Target Molecule Bcl-2 gene Target Protein
UniProt
P10415
Remarks SPC2996 is an antisense drug that was developed for the purpose of treating chronic lymphocytic leukemia, which selectively inhibits Bcl-2 gene causes cancer.
Clinical trials have already been discontinued.
Phase R
(I/II)
Nucleic Acid Sequence   PDB 1G5M
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2004-004741-17/DK
http://www.nature.com/leu/journal/v25/n4/full/leu2010322a.html
    SRP-4053   Country US Type Antisense DrugBank  
Adaptation Diseases Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
Company Sarepta Therapeutics, Inc. Target Molecule AcpP Target Protein
UniProt
P15309
Remarks SRP-4053 is a drug under development of duchenne muscular dystrophy.
Phase III Nucleic Acid Sequence   PDB 4KEH
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2014-002008-25/GB
https://clinicaltrials.gov/ct2/show/NCT02310906
    Volanesorsen ApoC-III Antisense Oligonucleotide Country US Type Antisense DrugBank  
Adaptation Diseases Hypertriglyceridemia
Company Ionis Pharmaceuticals Target Molecule ApoC-III Target Protein
UniProt
P02656
Remarks Volanesorsen reduces the amount of blood triglycerides, a serious risk factor for pancreatitis by binding to ApoC-III in the blood.
A phase III study of the efficacy and safety of Varanesen for familial partial lipodystrophy (FPL) patients has been carried out.
Phase III Nucleic Acid Sequence   PDB 2JQ3
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2014-003434-93/DE
https://www.clinicaltrialsregister.eu/ctr-search/trial/2015-000493-35/DE
https://www.clinicaltrialsregister.eu/ctr-search/trial/2014-002421-35/GB
https://www.clinicaltrialsregister.eu/ctr-search/trial/2015-003755-21/GB
http://adisinsight.springer.com/drugs/800031453
http://apociii.com/about-isis-apociiix/
http://www.raredr.com/news/phase-3-volanesorsen-fpl
    Zimura   Country US Type Aptamer DrugBank  
Adaptation Diseases Age-Related Macular Degeneration
Company OPHTHOTECH CORPORATION Target Molecule C5 Target Protein
UniProt
P01031
Remarks A Phase II / III randomized, double-masked, controlled trial has been carried out to assess the safety and efficacy of intravitreous administration of Zimura (anti-C5 aptamer), in subjects with geographic atrophy secondary to dry age-related macular degeneration (AMD).
Phase II/III Nucleic Acid Sequence   PDB 3KM9
Source https://www.clinicaltrialsregister.eu/ctr-search/trial/2015-003991-56/HU
    Patisiran Onpattro, ALN-18328 Country US
EU
Type siRNA DrugBank DB14582
Adaptation Diseases Hereditary transthyretin-mediated amyloidosis
Company Alnylam Pharmaceuticals Inc Target Molecule Transthyretin mRNA Target Protein
UniProt
 
Remarks Parisiran is a first in class short interfering RNA for the treatment of patients with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis [4]. It is marketed as Onpattro which is formulated as patisiran within a liposome envelope for better delivery to the liver, where transthyretin is produced. The approval for Onpattro was granted to Alnylam Pharmaceuticals, Inc. in August of 2018. Onpattro has been granted Fast Track, Priority Review and Breakthrough Therapy, and Orphan Drug designations.
Phase AD Nucleic Acid Sequence RNA, (A-U-G-G-A-A-Um-A-C-U-C-U-U-G-G-U-Um-A-C-dT-dT), complex with RNA (G-Um-A-A-Cm-Cm-A-A-G-A-G-Um-A-Um-Um-Cm-Cm-A-Um-dT-dT) (1:1) PDB
Source https://www.drugbank.ca/drugs/DB14582
https://clinicaltrials.gov/ct2/show/NCT01960348?term=patisiran&phase=2&rank=3
https://www.kegg.jp/entry/D11116
    Emapticap Pegol NOX-E36 Country Germany Type Aptamer DrugBank  
Adaptation Diseases Systemic Lupus Erythematosus
Type 2 Diabetes Mellitus
Chronic Inflammatory Diseases
Albuminuria
Renal Impairment
Company NOXXON Pharma Target Molecule CCL2 Target Protein
UniProt
P13500
Remarks A study for patients with type 2 diabetes mellitus and albuminuria has been carried out.
Phase II Nucleic Acid Sequence   PDB 1DOK
Source https://clinicaltrials.gov/ct2/show/NCT01547897
https://clinicaltrials.gov/ct2/show/NCT01372124
https://clinicaltrials.gov/ct2/show/NCT01085292
https://clinicaltrials.gov/ct2/show/NCT00976729
    Olaptesed Pegol NOX-A12 Country Germany Type Aptamer DrugBank DB11707
Adaptation Diseases Chronic Lymphocytic Leukemia
Multiple Myeloma
Hematopoietic Stem Cell Transplantation
Autologous Stem Cell Transplantatio
Glioblastoma
Metastatic Colorectal Cancer
Metastatic Pancreatic Cancer
Company NOXXON Pharma Target Molecule CXCL12 Target Protein
UniProt
P48061
Remarks NOX-A12 has been used in trials studying the treatment of hematopoietic stem cell transplantation.
Phase II Nucleic Acid Sequence   PDB 1A15
Source https://clinicaltrials.gov/ct2/show/NCT01486797
https://clinicaltrials.gov/ct2/show/NCT01521533
https://clinicaltrials.gov/ct2/show/NCT01194934
https://clinicaltrials.gov/ct2/show/NCT00976378
https://clinicaltrials.gov/ct2/show/NCT04121455
https://clinicaltrials.gov/ct2/show/NCT03168139
    Lexaptepid Pegol NOX-H94 Country Germany Type Aptamer DrugBank DB12578
Adaptation Diseases Anemia
End Stage Renal Disease
Anemia of Chronic Disease
Chronic Diseases
Inflammation
Company NOXXON Pharma Target Molecule Hepcidin Target Protein
UniProt
P81172
Remarks NOX-H94 has been investigated for the treatment of Anemia, Inflammation, Chronic Diseases, End Stage Renal Disease, and Anemia of Chronic Disease.
Phase II Nucleic Acid Sequence   PDB 1M4E
Source https://clinicaltrials.gov/ct2/show/NCT02079896
https://clinicaltrials.gov/ct2/show/NCT01522794
https://clinicaltrials.gov/ct2/show/NCT01372137
https://clinicaltrials.gov/ct2/show/NCT01691040
    Egaptivon pegol ARC1779 Country US Type Aptamer DrugBank DB05202
Adaptation Diseases Intracranial Embolism
Cerebral Thromboembolism
Carotid Stenosis
Von Willebrand Disease
Purpura, Thrombotic Thrombocytopenic
Thrombotic Microangiopathy
Thrombosis
Acute Myocardial Infarction
Company Archemix Target Molecule VWF
GP1BA
Target Protein
UniProt
P04275
P07359
Remarks A study of ARC1779 in patients with Von Willebrand factor-related platelet function disorders has been carried out.
Phase II Nucleic Acid Sequence   PDB 1AO3
1GWB
Source https://clinicaltrials.gov/ct2/show/NCT00742612
https://clinicaltrials.gov/ct2/show/NCT00694785
https://clinicaltrials.gov/ct2/show/NCT00632242
https://clinicaltrials.gov/ct2/show/NCT00726544
https://clinicaltrials.gov/ct2/show/NCT00432770
https://clinicaltrials.gov/ct2/show/NCT00507338
    ARC19499   Country US Type Aptamer DrugBank DB15285
Adaptation Diseases Hemophilia
Company Archemix Target Molecule TFPI Target Protein
UniProt
P10646
Remarks A study for hemophilia patients has been carried out.
Phase I Nucleic Acid Sequence   PDB 1ADZ
Source https://clinicaltrials.gov/ct2/show/NCT01191372
https://www.ncbi.nlm.nih.gov/pubmed/21389323
    AGRO100 AS1411 Country UK Type Aptamer DrugBank DB04998
Adaptation Diseases Acute Myeloid Leukemia
Advanced Solid Tumors
Metastatic Renal Cell Carcinoma
Leukemia, Myeloid
Company Antisoma Target Molecule IKBKG Target Protein
UniProt
Q9Y6K9
Remarks AS1411 has been investigated for the treatment of advanced solid tumors and myeloid leukemia.
Phase II Nucleic Acid Sequence   PDB 2JVX
Source https://clinicaltrials.gov/ct2/show/NCT01034410
https://clinicaltrials.gov/ct2/show/NCT00881244
https://clinicaltrials.gov/ct2/show/NCT00740441
https://clinicaltrials.gov/ct2/show/NCT00512083