Details of the Drug

General Information of Drug (ID: DMWUN1S)

Drug Name

Drisapersen

Synonyms

2402968

Indication

Disease Entry	ICD 11	Status	REF
Duchenne dystrophy	8C70	Phase 3	[1]
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Drug Type

Antisense drug

Structure

3D MOL is unavailable

2D MOL

#Ro5 Violations (Lipinski): 5

Molecular Weight (mw)

6978

Logarithm of the Partition Coefficient (xlogp)

-17

Rotatable Bond Count (rotbonds)

136

Hydrogen Bond Donor Count (hbonddonor)

46

Hydrogen Bond Acceptor Count (hbondacc)

172

Chemical Identifiers

Formula: C211H275N76O119P19S19
IUPAC Name: 1-[(2R,3R,4R,5R)-5-[[[(2R,3R,4R,5R)-2-[[[(2R,3R,4R,5R)-2-[[[(2R,3R,4R,5R)-2-[[[(2R,3R,4R,5R)-2-[[[(2R,3R,4R,5R)-2-[[[(2R,3R,4R,5R)-2-[[[(2R,3R,4R,5R)-5-(2-amino-6-oxo-1H-purin-9-yl)-2-[[[(2R,3R,4R,5R)-5-(2-amino-6-oxo-1H-purin-9-yl)-2-[[[(2R,3R,4R,5R)-2-[[[(2R,3R,4R,5R)-2-[[[(2R,3R,4R,5R)-5-(2-amino-6-oxo-1H-purin-9-yl)-2-[[[(2R,3R,4R,5R)-2-[[[(2R,3R,4R,5R)-2-[[[(2R,3R,4R,5R)-5-(2-amino-6-oxo-1H-purin-9-yl)-2-[[[(2R,3R,4R,5R)-5-(2-amino-6-oxo-1H-purin-9-yl)-2-[[[(2R,3R,4R,5R)-2-[[[(2R,3R,4R,5R)-2-[[[(2R,3R,4R,5R)-5-(4-amino-2-oxopyrimidin-1-yl)-2-[[[(2R,3R,4R,5R)-5-(2,4-dioxopyrimidin-1-yl)-2-(hydroxymethyl)-4-methoxyoxolan-3-yl]oxy-hydroxyphosphinothioyl]oxymethyl]-4-methoxyoxolan-3-yl]oxy-sulfanylphosphoryl]oxymethyl]-5-(6-aminopurin-9-yl)-4-methoxyoxolan-3-yl]oxy-hydroxyphosphinothioyl]oxymethyl]-5-(6-aminopurin-9-yl)-4-methoxyoxolan-3-yl]oxy-hydroxyphosphinothioyl]oxymethyl]-4-methoxyoxolan-3-yl]oxy-hydroxyphosphinothioyl]oxymethyl]-4-methoxyoxolan-3-yl]oxy-hydroxyphosphinothioyl]oxymethyl]-5-(6-aminopurin-9-yl)-4-methoxyoxolan-3-yl]oxy-hydroxyphosphinothioyl]oxymethyl]-5-(6-aminopurin-9-yl)-4-methoxyoxolan-3-yl]oxy-hydroxyphosphinothioyl]oxymethyl]-4-methoxyoxolan-3-yl]oxy-hydroxyphosphinothioyl]oxymethyl]-5-(6-aminopurin-9-yl)-4-methoxyoxolan-3-yl]oxy-hydroxyphosphinothioyl]oxymethyl]-5-(2,4-dioxopyrimidin-1-yl)-4-methoxyoxolan-3-yl]oxy-hydroxyphosphinothioyl]oxymethyl]-4-methoxyoxolan-3-yl]oxy-hydroxyphosphinothioyl]oxymethyl]-4-methoxyoxolan-3-yl]oxy-hydroxyphosphinothioyl]oxymethyl]-5-(4-amino-2-oxopyrimidin-1-yl)-4-methoxyoxolan-3-yl]oxy-hydroxyphosphinothioyl]oxymethyl]-5-(6-aminopurin-9-yl)-4-methoxyoxolan-3-yl]oxy-hydroxyphosphinothioyl]oxymethyl]-5-(2,4-dioxopyrimidin-1-yl)-4-methoxyoxolan-3-yl]oxy-hydroxyphosphinothioyl]oxymethyl]-5-(2,4-dioxopyrimidin-1-yl)-4-methoxyoxolan-3-yl]oxy-hydroxyphosphinothioyl]oxymethyl]-5-(2,4-dioxopyrimidin-1-yl)-4-methoxyoxolan-3-yl]oxy-hydroxyphosphinothioyl]oxymethyl]-5-(4-amino-2-oxopyrimidin-1-yl)-4-methoxyoxolan-3-yl]oxy-hydroxyphosphinothioyl]oxymethyl]-4-hydroxy-3-methoxyoxolan-2-yl]pyrimidine-2,4-dione
Canonical SMILES: CO[C@@H]1[C@@H]([C@H](O[C@H]1N2C=CC(=O)NC2=O)COP(=S)(O)O[C@@H]3[C@H](O[C@H]([C@@H]3OC)N4C=CC(=NC4=O)N)COP(=S)(O)O[C@@H]5[C@H](O[C@H]([C@@H]5OC)N6C=CC(=O)NC6=O)COP(=S)(O)O[C@@H]7[C@H](O[C@H]([C@@H]7OC)N8C=CC(=O)NC8=O)COP(=S)(O)O[C@@H]9[C@H](O[C@H]([C@@H]9OC)N1C=CC(=O)NC1=O)COP(=S)(O)O[C@@H]1[C@H](O[C@H]([C@@H]1OC)N1C=NC2=C(N=CN=C21)N)COP(=S)(O)O[C@@H]1[C@H](O[C@H]([C@@H]1OC)N1C=CC(=NC1=O)N)COP(=S)(O)O[C@@H]1[C@H](O[C@H]([C@@H]1OC)N1C=NC2=C1N=C(NC2=O)N)COP(=S)(O)O[C@@H]1[C@H](O[C@H]([C@@H]1OC)N1C=NC2=C1N=C(NC2=O)N)COP(=S)(O)O[C@@H]1[C@H](O[C@H]([C@@H]1OC)N1C=CC(=O)NC1=O)COP(=S)(O)O[C@@H]1[C@H](O[C@H]([C@@H]1OC)N1C=NC2=C(N=CN=C21)N)COP(=S)(O)O[C@@H]1[C@H](O[C@H]([C@@H]1OC)N1C=NC2=C1N=C(NC2=O)N)COP(=S)(O)O[C@@H]1[C@H](O[C@H]([C@@H]1OC)N1C=NC2=C(N=CN=C21)N)COP(=S)(O)O[C@@H]1[C@H](O[C@H]([C@@H]1OC)N1C=NC2=C(N=CN=C21)N)COP(=S)(O)O[C@@H]1[C@H](O[C@H]([C@@H]1OC)N1C=NC2=C1N=C(NC2=O)N)COP(=S)(O)O[C@@H]1[C@H](O[C@H]([C@@H]1OC)N1C=NC2=C1N=C(NC2=O)N)COP(=S)(O)O[C@@H]1[C@H](O[C@H]([C@@H]1OC)N1C=NC2=C(N=CN=C21)N)COP(=S)(O)O[C@@H]1[C@H](O[C@H]([C@@H]1OC)N1C=NC2=C(N=CN=C21)N)COP(=O)(O[C@@H]1[C@H](O[C@H]([C@@H]1OC)N1C=CC(=NC1=O)N)COP(=S)(O)O[C@@H]1[C@H](O[C@H]([C@@H]1OC)N1C=CC(=O)NC1=O)CO)S)O
InChI: 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)/t76-,77-,78-,79-,80-,81-,82-,83-,84-,85-,86-,87-,88-,89-,90-,91-,92-,93-,94-,95-,116-,117-,118-,119-,120-,121-,122-,123-,124-,125-,126-,127-,128-,129-,130-,131-,132-,133-,134-,135-,136-,137-,138-,139-,140-,141-,142-,143-,144-,145-,146-,147-,148-,149-,150-,151-,152-,153-,154-,155-,178-,179-,180-,181-,182-,183-,184-,185-,186-,187-,188-,189-,190-,191-,192-,193-,194-,195-,196-,197-,407?,408?,409?,410?,411?,412?,413?,414?,415?,416?,417?,418?,419?,420?,421?,422?,423?,424?,425?/m1/s1
InChIKey: HUGILZFVSVLCAO-XVKRXUDYSA-N

Cross-matching ID

PubChem CID: 121494119
CAS Number: 1251830-50-8
DrugBank ID: DB13688
TTD ID: D0X4DB

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Dystrophin messenger RNA (DMD mRNA)	TTWLFXU	DMD_HUMAN	Not Available	[2]

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Molecular Interaction Atlas (MIA)

MIA Detail

Jump to Detail Molecular Interaction Atlas of This Drug

Molecular Expression Atlas of This Drug

The Studied Disease

Duchenne dystrophy

ICD Disease Classification

8C70

Molecule Name	Molecule Type	Gene Name	p-value	Fold-Change	Z-score
Dystrophin messenger RNA (DMD mRNA)	DTT	DMD	1.15E-07	-1.07	-2.94

Molecular Expression Atlas (MEA)

MEA Detail

Jump to Detail Molecular Expression Atlas of This Drug

References

1	ClinicalTrials.gov (NCT01890798) Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment Protocol. U.S. National Institutes of Health.
2	PRO-051, an antisense oligonucleotide for the potential treatment of Duchenne muscular dystrophy. Curr Opin Mol Ther. 2010 Aug;12(4):478-86.