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