Drug General Information
Drug ID
D0X4DB
Former ID
DNCL003619
Drug Name
Drisapersen
Synonyms
2402968
Drug Type
Antisense drug
Indication Duchenne muscular dystrophy [ICD10:G71.0] Phase 3 [524346]
Company
GlaxoSmithKline
Structure
Download
2D MOL

3D MOL

Formula
C211H275N76O119P19S19
Canonical SMILES
COC1C(C(OC1N2C=CC(=O)NC2=O)COP(=S)(O)OC3C(OC(C3OC)N4C=C<br />C(=NC4=O)N)COP(=S)(O)OC5C(OC(C5OC)N6C=CC(=O)NC6=O)COP(=<br />S)(O)OC7C(OC(C7OC)N8C=CC(=O)NC8=O)COP(=S)(O)OC9C(OC(C9O<br />C)N1C=CC(=O)NC1=O)COP(=S)(O)OC1C(OC(C1OC)N1C=NC2=C1N=CN<br />=C2N)COP(=S)(O)OC1C(OC(C1OC)N1C=CC(=NC1=O)N)COP(=S)(O)O<br />C1C(OC(C1OC)N1C=NC2=C1N=C(NC2=O)N)COP(=S)(O)OC1C(OC(C1O<br />C)N1C=NC2=C1N=C(NC2=O)N)COP(=S)(O)OC1C(OC(C1OC)N1C=CC(=<br />O)NC1=O)COP(=S)(O)OC1C(OC(C1OC)N1C=NC2=C1N=CN=C2N)COP(=<br />S)(O)OC1C(OC(C1OC)N1C=NC2=C1N=C(NC2=O)N)COP(=S)(O)OC1C(<br />OC(C1OC)N1C=NC2=C1N=CN=C2N)COP(=S)(O)OC1C(OC(C1OC)N1C=N<br />C2=C1N=CN=C2N)COP(=S)(O)OC1C(OC(C1OC)N1C=NC2=C1N=C(NC2=<br />O)N)COP(=O)(OC1C(OC(C1OC)N1C=NC2=C1N=C(NC2=O)N)COP(=S)(<br />O)OC1C(OC(C1OC)N1C=NC2=C1N=CN=C2N)COP(=S)(O)OC1C(OC(C1O<br />C)N1C=NC2=C1N=CN=C2N)COP(=S)(O)OC1C(OC(C1OC)N1C=CC(=NC1<br />=O)N)COP(=S)(O)OC1C(OC(C1OC)N1C=CC(=O)NC1=O)CO)S)O
InChI
1S/C211H275N76O119P19S19/c1-329-136-116(295)77(369-178(136)271-33-24-99(289)252-206(271)304)40-349-407(310,426)389-118-79(371-180(138(118)331-3)268-30-21-96(212)249-203(268)301)42-351-409(312,428)392-121-81(373-183(141(121)334-6)273-35-26-101(291)254-208(273)306)44-352-410(313,429)393-122-82(374-184(142(122)335-7)274-36-27-102(292)255-209(274)307)45-353-411(314,430)394-123-83(375-185(143(123)336-8)275-37-28-103(293)256-210(275)308)46-355-415(318,434)396-125-85(377-187(145(125)338-10)277-65-238-105-156(215)226-59-232-162(105)277)48-358-413(316,432)391-120-80(372-182(140(120)333-5)270-32-23-98(214)251-205(270)303)43-354-417(320,436)402-131-94(386-193(151(131)344-16)283-71-244-111-168(283)258-198(221)263-173(111)296)57-366-424(327,443)405-132-91(383-194(152(132)345-17)284-72-245-112-169(284)259-199(222)264-174(112)297)54-363-414(317,433)395-124-84(376-186(144(124)337-9)276-38-29-104(294)257-211(276)309)47-356-416(319,435)397-126-89(381-188(146(126)339-11)278-66-239-106-157(216)227-60-233-163(106)278)52-361-422(325,441)403-133-92(384-195(153(133)346-18)285-73-246-113-170(285)260-200(223)265-175(113)298)55-364-420(323,439)400-130-88(380-192(150(130)343-15)282-70-243-110-161(220)231-64-237-167(110)282)51-360-419(322,438)399-128-90(382-190(148(128)341-13)280-68-241-108-159(218)229-62-235-165(108)280)53-362-423(326,442)404-134-95(387-196(154(134)347-19)286-74-247-114-171(286)261-201(224)266-176(114)299)58-367-425(328,444)406-135-93(385-197(155(135)348-20)287-75-248-115-172(287)262-202(225)267-177(115)300)56-365-421(324,440)401-129-87(379-191(149(129)342-14)281-69-242-109-160(219)230-63-236-166(109)281)50-359-418(321,437)398-127-86(378-189(147(127)340-12)279-67-240-107-158(217)228-61-234-164(107)279)49-357-412(315,431)390-119-78(370-181(139(119)332-4)269-31-22-97(213)250-204(269)302)41-350-408(311,427)388-117-76(39-288)368-179(137(117)330-2)272-34-25-100(290)253-207(272)305/h21-38,59-95,116-155,178-197,288,295H,39-58H2,1-20H3,(H,310,426)(H,311,427)(H,312,428)(H,313,429)(H,314,430)(H,315,431)(H,316,432)(H,317,433)(H,318,434)(H,319,435)(H,320,436)(H,321,437)(H,322,438)(H,323,439)(H,324,440)(H,325,441)(H,326,442)(H,327,443)(H,328,444)(H2,212,249,301)(H2,213,250,302)(H2,214,251,303)(H2,215,226,232)(H2,216,227,233)(H2,217,228,234)(H2,218,229,235)(H2,219,230,236)(H2,220,231,237)(H,252,289,304)(H,253,290,305)(H,254,291,306)(H,255,292,307)(H,256,293,308)(H,257,294,309)(H3,221,258,263,296)(H3,222,259,264,297)(H3,223,260,265,298)(H3,224,261,266,299)(H3,225,262,267,300)
InChIKey
HUGILZFVSVLCAO-UHFFFAOYSA-N
PubChem Compound ID
Target and Pathway
Target(s) mRNA of Dystrophin Target Info [531075]
KEGG Pathway Hypertrophic cardiomyopathy (HCM)
Arrhythmogenic right ventricular cardiomyopathy (ARVC)
Dilated cardiomyopathy
Viral myocarditis
NetPath Pathway IL4 Signaling Pathway
IL5 Signaling Pathway
Reactome Non-integrin membrane-ECM interactions
Striated Muscle Contraction
WikiPathways Striated Muscle Contraction
Ectoderm Differentiation
Extracellular matrix organization
Arrhythmogenic Right Ventricular Cardiomyopathy
Muscle contraction
References
Ref 524346ClinicalTrials.gov (NCT01890798) Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment Protocol. U.S. National Institutes of Health.
Ref 531075PRO-051, an antisense oligonucleotide for the potential treatment of Duchenne muscular dystrophy. Curr Opin Mol Ther. 2010 Aug;12(4):478-86.

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