mongersen   Click here for help

GtoPdb Ligand ID: 8277

Synonyms: DNA, d(P-thio)(G-T-m5C-G-C-C-C-C-T-T-C-T-C-C-C-m5C-G-C-A-G-C) | GED-0301 | GED0301
Immunopharmacology Ligand
Compound class: Synthetic organic
Comment: Mongersen (GED-0301) is a phosphorothioate single-stranded oligonucleotide matching the region 107-128 (5'-GCTGCGGGGAGAAGGGGCGAC-3') of the human Smad7 complementary DNA sequence. The antisense oligonucleotide sequence is (3'-5')d(P-thio)(G-T-m5C-G-C-C-C-C-T-T-C-T-C-C-C-m5C-G-C-A-G-C), where m5C represents 2'-deoxy-5-methyl-P-thiocytidylyl bases.
Mongersen was investigated as a treatment for Smad7-induced colitis associated with Crohn's disease and irritable bowel disease (IBD), but following failure to demonstrate efficacy in Phase 3 evaluation, development was terminated in late 2017.
2D Structure
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2D Structure

An image of the ligand's 2D structure. For small molecules with SMILES these are drawn using the NCI/CADD Chemical Identifier Resolver. Click on the image to access the chemical structure search tool with the ligand pre-loaded in the structure editor. For other types of ligands, e.g. longer nucleotides and peptides, a manually drawn representation of the molecule may be provided.

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Physico-chemical Properties
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Physico-chemical Properties

Calculated molecular properties are available for small molecules and natural products (not peptides). Properties were generated using the CDK toolkit. All properties were selected to enable the prediction of the Lipinski Rule-of-Five profile or ‘druglikeness’ for each ligand. For more info on each category see the help pages.

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Hydrogen bond acceptors 176
Hydrogen bond donors 47
Rotatable bonds 122
Topological polar surface area 3146.67
Molecular weight 6604.42
XLogP -6.74
No. Lipinski's rules broken 4
SMILES / InChI / InChIKey
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SMILES / InChI / InChIKey

SMILES (Simplified Molecular Input Line Entry Specification) A specification for unambiguously describing the structure of chemical molecules using short ASCII strings. Canonical SMILES specify a unique representation of the 2D structure without chiral or isotopic specifications. Isomeric SMILES include chiral specification and isotopes.

Standard InChI (IUPAC International Chemical Identifier) and InChIKey InChI is a non-proprietary, standard, textual identifier for chemical substances designed to facilitate linking of information and database searching. An InChIKey is a simplified version of a full InChI, designed for easier web searching.

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Canonical SMILES CC1=CN([C@H]2C[C@@H]([C@@H](COP(=S)(O)O[C@H]3C[C@H](N4C=CC(=NC4=O)N)O[C@@H]3COP(=S)(O)O[C@H]5C[C@H](N6C=CC(=NC6=O)N)O[C@@H]5COP(=S)(O)O[C@H]7C[C@H](N8C=CC(=NC8=O)N)O[C@@H]7COP(=S)(O)O[C@H]9C[C@H](N%10C=C(C)C(=O)NC%10=O)O[C@@H]9COP(=S)(O)O[C@H]%11C[C@H](N%12C=CC(=NC%12=O)N)O[C@@H]%11COP(=S)(O)O[C@H]%13C[C@H](N%14C=C(C)C(=O)NC%14=O)O[C@@H]%13COP(=S)(O)O[C@H]%15C[C@H](N%16C=C(C)C(=O)NC%16=O)O[C@@H]%15COP(=S)(O)O[C@H]%17C[C@H](N%18C=CC(=NC%18=O)N)O[C@@H]%17COP(=S)(O)O[C@H]%19C[C@H](N%20C=CC(=NC%20=O)N)O[C@@H]%19COP(=S)(O)O[C@H]%21C[C@H](N%22C=CC(=NC%22=O)N)O[C@@H]%21COP(=S)(O)O[C@H]%23C[C@H](N%24C=CC(=NC%24=O)N)O[C@@H]%23COP(=S)(O)O[C@H]%25C[C@H](N%26C=NC%27=C%26N=C(N)NC%27=O)O[C@@H]%25COP(=S)(O)O[C@H]%28C[C@H](N%29C=C(C)C(=NC%29=O)N)O[C@@H]%28COP(=S)(O)O[C@H]%30C[C@H](N%31C=C(C)C(=O)NC%31=O)O[C@@H]%30COP(=S)(O)O[C@H]%32C[C@H](N%33C=NC%34=C%33N=C(N)NC%34=O)O[C@@H]%32CO)O2)OP(=S)(O)OC[C@@H]%35[C@H](C[C@H](N%36C=NC%37=C%36N=C(N)NC%37=O)O%35)OP(=S)(O)OC[C@@H]%38[C@H](C[C@H](N%39C=CC(=NC%39=O)N)O%38)OP(=S)(O)OC[C@@H]%40[C@H](C[C@H](N%41C=NC%42=C%41N=CN=C%42N)O%40)OP(=S)(O)OC[C@@H]%43[C@H](C[C@H](N%44C=NC%45=C%44N=C(N)NC%45=O)O%43)OP(=S)(O)OC[C@@H]%46[C@H](C[C@H](N%47C=CC(=NC%47=O)N)O%46)O)C(=O)N=C1N
Isomeric SMILES CC1=CN(C(=O)NC1=O)[C@H]2C[C@@H]([C@H](O2)COP(=S)(O)O[C@H]3C[C@@H](O[C@@H]3CO)N4C=NC5=C4N=C(NC5=O)N)OP(=S)(O)OC[C@@H]6[C@H](C[C@@H](O6)N7C=C(C(=NC7=O)N)C)OP(=S)(O)OC[C@@H]8[C@H](C[C@@H](O8)N9C=NC%10=C9N=C(NC%10=O)N)OP(=S)(O)OC[C@@H]%11[C@H](C[C@@H](O%11)N%12C=CC(=NC%12=O)N)OP(=S)(O)OC[C@@H]%13[C@H](C[C@@H](O%13)N%14C=CC(=NC%14=O)N)OP(=S)(O)OC[C@@H]%15[C@H](C[C@@H](O%15)N%16C=CC(=NC%16=O)N)OP(=S)(O)OC[C@@H]%17[C@H](C[C@@H](O%17)N%18C=CC(=NC%18=O)N)OP(=S)(O)OC[C@@H]%19[C@H](C[C@@H](O%19)N%20C=C(C(=O)NC%20=O)C)OP(=S)(O)OC[C@@H]%21[C@H](C[C@@H](O%21)N%22C=C(C(=O)NC%22=O)C)OP(=S)(O)OC[C@@H]%23[C@H](C[C@@H](O%23)N%24C=CC(=NC%24=O)N)OP(=S)(O)OC[C@@H]%25[C@H](C[C@@H](O%25)N%26C=C(C(=O)NC%26=O)C)OP(=S)(O)OC[C@@H]%27[C@H](C[C@@H](O%27)N%28C=CC(=NC%28=O)N)OP(=S)(O)OC[C@@H]%29[C@H](C[C@@H](O%29)N%30C=CC(=NC%30=O)N)OP(=S)(O)OC[C@@H]%31[C@H](C[C@@H](O%31)N%32C=CC(=NC%32=O)N)OP(=S)(O)OC[C@@H]%33[C@H](C[C@@H](O%33)N%34C=C(C(=NC%34=O)N)C)OP(=S)(O)OC[C@@H]%35[C@H](C[C@@H](O%35)N%36C=NC%37=C%36N=C(NC%37=O)N)OP(=S)(O)OC[C@@H]%38[C@H](C[C@@H](O%38)N%39C=CC(=NC%39=O)N)OP(=S)(O)OC[C@@H]%40[C@H](C[C@@H](O%40)N%41C=NC%42=C(N=CN=C%42%41)N)OP(=S)(O)OC[C@@H]%43[C@H](C[C@@H](O%43)N%44C=NC%45=C%44N=C(NC%45=O)N)OP(=S)(O)OC[C@@H]%46[C@H](C[C@@H](O%46)N%47C=CC(=NC%47=O)N)O
InChI InChI=1S/C200H261N69O107P20S20/c1-81-48-259(195(290)235-165(81)211)149-38-98(367-392(311,412)333-73-127-107(47-159(355-127)269-80-224-164-172(269)240-184(217)244-180(164)279)376-395(314,415)326-64-118-97(37-148(346-118)258-26-16-138(210)234-194(258)289)366-387(306,407)332-71-125-104(44-155(353-125)265-76-220-160-167(213)218-75-219-168(160)265)374-396(315,416)335-72-126-105(45-157(354-126)267-78-222-162-170(267)238-182(215)242-178(162)277)373-377(296,397)316-55-109-87(271)27-139(337-109)249-17-7-129(201)225-185(249)280)119(347-149)66-327-384(303,404)363-94-34-145(255-23-13-135(207)231-191(255)286)341-113(94)59-321-380(299,400)359-91-31-142(252-20-10-132(204)228-188(252)283)339-111(91)57-319-382(301,402)361-92-32-143(253-21-11-133(205)229-189(253)284)343-115(92)61-323-388(307,408)369-101-41-152(262-51-84(4)174(273)246-198(262)293)350-122(101)68-328-386(305,406)365-96-36-147(257-25-15-137(209)233-193(257)288)344-116(96)62-324-389(308,409)370-102-42-153(263-52-85(5)175(274)247-199(263)294)352-124(102)70-331-391(310,411)372-103-43-154(264-53-86(6)176(275)248-200(264)295)351-123(103)69-329-385(304,405)364-95-35-146(256-24-14-136(208)232-192(256)287)342-114(95)60-322-381(300,401)360-90-30-141(251-19-9-131(203)227-187(251)282)338-110(90)56-318-379(298,399)358-89-29-140(250-18-8-130(202)226-186(250)281)340-112(89)58-320-383(302,403)362-93-33-144(254-22-12-134(206)230-190(254)285)345-117(93)63-325-394(313,414)375-106-46-158(268-79-223-163-171(268)239-183(216)243-179(163)278)356-128(106)74-334-393(312,413)368-99-39-150(260-49-82(2)166(212)236-196(260)291)348-120(99)67-330-390(309,410)371-100-40-151(261-50-83(3)173(272)245-197(261)292)349-121(100)65-317-378(297,398)357-88-28-156(336-108(88)54-270)266-77-221-161-169(266)237-181(214)241-177(161)276/h7-26,48-53,75-80,87-128,139-159,270-271H,27-47,54-74H2,1-6H3,(H,296,397)(H,297,398)(H,298,399)(H,299,400)(H,300,401)(H,301,402)(H,302,403)(H,303,404)(H,304,405)(H,305,406)(H,306,407)(H,307,408)(H,308,409)(H,309,410)(H,310,411)(H,311,412)(H,312,413)(H,313,414)(H,314,415)(H,315,416)(H2,201,225,280)(H2,202,226,281)(H2,203,227,282)(H2,204,228,283)(H2,205,229,284)(H2,206,230,285)(H2,207,231,286)(H2,208,232,287)(H2,209,233,288)(H2,210,234,289)(H2,211,235,290)(H2,212,236,291)(H2,213,218,219)(H,245,272,292)(H,246,273,293)(H,247,274,294)(H,248,275,295)(H3,214,237,241,276)(H3,215,238,242,277)(H3,216,239,243,278)(H3,217,240,244,279)/t87-,88-,89-,90-,91-,92-,93-,94-,95-,96-,97-,98-,99-,100-,101-,102-,103-,104-,105-,106-,107-,108+,109+,110+,111+,112+,113+,114+,115+,116+,117+,118+,119+,120+,121+,122+,123+,124+,125+,126+,127+,128+,139+,140+,141+,142+,143+,144+,145+,146+,147+,148+,149+,150+,151+,152+,153+,154+,155+,156+,157+,158+,159+,377?,378?,379?,380?,381?,382?,383?,384?,385?,386?,387?,388?,389?,390?,391?,392?,393?,394?,395?,396?/m0/s1
InChI Key ABASMUXUCSQFKC-PKEKLUKKSA-N