Volanesorsen

(natri salt)
| ATC_prefix = None
| ATC_suffix =
| ATC_supplemental =
| PubChem = 122409770
| DrugBank =
| ChemSpiderID = 34982934

| UNII = 2O4BE0K238
| ChEMBL =
| synonyms = ISIS 304801, ISIS-APOCIIIRx
| chemical_formula =
| C = 230
| H = 320
| N = 63
| O = 125
| P = 19
| S = 19
| molecular_weight =
| SMILES = Cc1cn(c(=O)[nH]c1=O)[C@H]2C[C@@H]([C@H](O2)COP(=O)(O[C@H]3C[C@@H](O[C@@H]3COP(=O)(O[C@H]4C[C@@H](O[C@@H]4COP(=O)(O[C@@H]5[C@H](O[C@H]([C@@H]5OCCOC)n6cc(c(=O)[nH]c6=O)C)COP(=O)(O[C@@H]7[C@H](O[C@H]([C@@H]7OCCOC)n8cc(c(=O)[nH]c8=O)C)COP(=O)(O[C@@H]9[C@H](O[C@H]([C@@H]9OCCOC)n1cc(c(nc1=O)N)C)COP(=O)(O[C@@H]1[C@H](O[C@H]([C@@H]1OCCOC)n1cnc2c1nc([nH]c2=O)N)COP(=O)(O[C@@H]1[C@H](O[C@H]([C@@H]1OCCOC)n1cnc2c1ncnc2N)CO)S)S)S)S)S)n1cc(c(nc1=O)N)C)S)n1cc(c(=O)[nH]c1=O)C)S)OP(=O)(OC[C@@H]1[C@H](C[C@@H](O1)n1cnc2c1nc([nH]c2=O)N)OP(=O)(OC[C@@H]1[C@H](C[C@@H](O1)n1cc(c(=O)[nH]c1=O)C)OP(=O)(OC[C@@H]1[C@H](C[C@@H](O1)n1cc(c(nc1=O)N)C)OP(=O)(OC[C@@H]1[C@H](C[C@@H](O1)n1cc(c(nc1=O)N)C)OP(=O)(OC[C@@H]1[C@H](C[C@@H](O1)n1cnc2c1ncnc2N)OP(=O)(OC[C@@H]1[C@H](C[C@@H](O1)n1cnc2c1nc([nH]c2=O)N)OP(=O)(OC[C@@H]1[C@H](C[C@@H](O1)n1cc(c(nc1=O)N)C)OP(=O)(OC[C@@H]1[C@H]([C@H]([C@@H](O1)n1cc(c(=O)[nH]c1=O)C)OCCOC)OP(=O)(OC[C@@H]1[C@H]([C@H]([C@@H](O1)n1cc(c(=O)[nH]c1=O)C)OCCOC)OP(=O)(OC[C@@H]1[C@H]([C@H]([C@@H](O1)n1cc(c(=O)[nH]c1=O)C)OCCOC)OP(=O)(OC[C@@H]1[C@H]([C@H]([C@@H](O1)n1cnc2c1ncnc2N)OCCOC)OP(=O)(OC[C@@H]1[C@H]([C@H]([C@@H](O1)n1cc(c(=O)[nH]c1=O)C)OCCOC)O)S)S)S)S)S)S)S)S)S)S)S)S
| StdInChI = 1S/C230H320N63O125P19S19/c1-98-55-274(217(308)254-178(98)231)142-45-112(400-419(322,438)361-70-124-113(46-143(381-124)275-56-99(2)179(232)255-218(275)309)401-423(326,442)367-76-130-119(52-149(387-130)288-92-248-152-183(236)242-89-245-186(152)288)407-427(330,446)369-78-132-121(54-151(389-132)290-94-252-156-190(290)260-215(240)263-202(156)306)408-425(328,444)365-72-125-114(47-144(382-125)276-57-100(3)180(233)256-219(276)310)406-428(331,447)372-81-134-160(171(354-38-28-344-18)206(393-134)283-64-107(10)196(300)269-226(283)317)412-432(335,451)375-84-137-163(173(356-40-30-346-20)208(395-137)285-66-109(12)198(302)271-228(285)319)415-435(338,454)377-86-139-165(175(358-42-32-348-22)210(397-139)287-68-111(14)200(304)273-230(287)321)416-436(339,455)379-87-140-166(176(359-43-33-349-23)212(398-140)292-96-250-154-185(238)244-91-247-188(154)292)417-430(333,449)371-80-133-158(295)168(351-35-25-341-15)204(391-133)282-63-106(9)195(299)268-225(282)316)123(380-142)71-362-421(324,440)403-117-50-147(279-61-104(7)193(297)266-223(279)314)386-128(117)75-366-426(329,445)409-120-53-150(289-93-251-155-189(289)259-214(239)262-201(155)305)388-131(120)77-368-424(327,443)405-118-51-148(280-62-105(8)194(298)267-224(280)315)385-127(118)74-364-422(325,441)404-116-49-146(278-60-103(6)192(296)265-222(278)313)384-126(116)73-363-420(323,439)402-115-48-145(277-58-101(4)181(234)257-220(277)311)383-129(115)79-370-429(332,448)411-162-136(394-207(172(162)355-39-29-345-19)284-65-108(11)197(301)270-227(284)318)83-374-434(337,453)414-164-138(396-209(174(164)357-41-31-347-21)286-67-110(13)199(303)272-229(286)320)85-376-433(336,452)413-161-135(392-205(170(161)353-37-27-343-17)281-59-102(5)182(235)258-221(281)312)82-378-437(340,456)418-167-141(399-213(177(167)360-44-34-350-24)293-97-253-157-191(293)261-216(241)264-203(157)307)88-373-431(334,450)410-159-122(69-294)390-211(169(159)352-36-26-342-16)291-95-249-153-184(237)243-90-246-187(153)291/h55-68,89-97,112-151,158-177,204-213,294-295H,25-54,69-88H2,1-24H3,(H,322,438)(H,323,439)(H,324,440)(H,325,441)(H,326,442)(H,327,443)(H,328,444)(H,329,445)(H,330,446)(H,331,447)(H,332,448)(H,333,449)(H,334,450)(H,335,451)(H,336,452)(H,337,453)(H,338,454)(H,339,455)(H,340,456)(H2,231,254,308)(H2,232,255,309)(H2,233,256,310)(H2,234,257,311)(H2,235,258,312)(H2,236,242,245)(H2,237,243,246)(H2,238,244,247)(H,265,296,313)(H,266,297,314)(H,267,298,315)(H,268,299,316)(H,269,300,317)(H,270,301,318)(H,271,302,319)(H,272,303,320)(H,273,304,321)(H3,239,259,262,305)(H3,240,260,263,306)(H3,241,261,264,307)/t112-,113-,114-,115-,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+,158+,159+,160+,161+,162+,163+,164+,165+,166+,167+,168+,169+,170+,171+,172+,173+,174+,175+,176+,177+,204+,205+,206+,207+,208+,209+,210+,211+,212+,213+,419?,420?,421?,422?,423?,424?,425?,426?,427?,428?,429?,430?,431?,432?,433?,434?,435?,436?,437?/m0/s1
| StdInChIKey = IJUQCWMZCMFFJP-GQSLRNSLSA-N
}}
Volanesorsen là thuốc giảm triglyceride. Nó là một oligonucleotide điều trị bằng tinh bột trị liệu thế hệ thứ hai 2'- O -methoxyethyl (2'-MOE) (ASO) nhắm vào RNA thông tin cho apolipoprotein C 3 (apo-CIII).

Đó là trong các thử nghiệm lâm sàng giai đoạn III để điều trị tăng triglycid máu, hội chứng chylomicronemia gia đình và loạn dưỡng mỡ một phần gia đình.

Thuốc được phát hiện và phát triển bởi Ionis Pharmaceuticals.

Hóa học
Trình tự hoàn chỉnh của volanesorsen là:

: 3'—A—G—mC—T—T—dmC —dT —dT —dG —dT —dmC —dmC —dA —dG —dmC —T—T—T—A—T—5'
: = 2'- O - (2-metoxyethyl)
: m = 5-metyl
: d = 2'-deoxy

Tên gọi
Volanesorsen là một INN.

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