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Substance Name: Patisiran [USAN:INN]
RN: 1420706-45-1
UNII: 50FKX8CB2Y
InChIKey: UBJHFPNBJDGZHL-UHFFFAOYSA-N

Note

  • Small interfering RNA encapsulated within lipid nanoparticles for treatment of familial amyloidotic polyneuropathy.

Molecular Formula

  • C412-H520-N148-O290-P40

Molecular Weight

  • 13424.346
 

Classification Code

  • Treatment of Amyloidosis
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Names and Synonyms

Name of Substance

  • Patisiran [USAN:INN]

Synonyms

  • ALN-18328
  • ALN-TTR02
  • Patisiran
  • Patisiran [INN]
  • 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)
  • UNII-50FKX8CB2Y

Registry Numbers

CAS Registry Number

  • 1420706-45-1

FDA UNII

  • 50FKX8CB2Y

System Generated Number

  • 1420706451

Structure Descriptors

InChI

1S/C210H268N76O143P20.C202H252N72O147P20/c1-63-14-268(200(318)249-147(63)211)178-110(290)129(413-433(335,336)372-29-80-131(112(292)180(393-80)270-16-65(3)149(213)251-202(270)320)415-437(343,344)380-37-88-138(119(299)187(401-88)277-53-239-99-151(215)225-46-232-158(99)277)422-442(353,354)377-33-84-133(114(294)182(397-84)272-20-69(7)170(310)261-206(272)324)412-431(331,332)370-27-78-75(13-98(390-78)267-19-68(6)169(309)260-205(267)323)410-430(329,330)369-26-77-74(288)12-97(389-77)266-18-67(5)168(308)259-204(266)322)79(392-178)31-374-435(339,340)417-135-85(398-184(116(135)296)274-22-71(9)172(312)263-208(274)326)34-375-436(341,342)418-136-86(399-185(117(136)297)275-23-72(10)173(313)264-209(275)327)35-378-443(355,356)423-139-89(402-188(120(139)300)278-54-240-100-152(216)226-47-233-159(100)278)39-383-440(349,350)420-137-87(400-186(118(137)298)276-24-73(11)174(314)265-210(276)328)36-379-444(357,358)428-145-95(408-195(126(145)306)285-61-247-107-166(285)254-198(223)257-176(107)316)44-387-447(363,364)427-144-94(407-193(125(144)305)283-59-245-105-157(221)231-52-238-164(105)283)43-386-449(367,368)429-146-96(409-196(127(146)307)286-62-248-108-167(286)255-199(224)258-177(108)317)45-388-448(365,366)426-143-93(406-192(124(143)304)282-58-244-104-156(220)230-51-237-163(104)282)42-385-446(361,362)424-141-90(403-190(122(141)302)280-56-242-102-154(218)228-49-235-161(102)280)38-381-438(345,346)416-132-81(394-181(113(132)293)271-17-66(4)150(214)252-203(271)321)30-373-434(337,338)414-130-82(395-179(111(130)291)269-15-64(2)148(212)250-201(269)319)32-376-441(351,352)421-140-92(405-189(121(140)301)279-55-241-101-153(217)227-48-234-160(101)279)41-384-445(359,360)425-142-91(404-191(123(142)303)281-57-243-103-155(219)229-50-236-162(103)281)40-382-439(347,348)419-134-83(396-183(115(134)295)273-21-70(8)171(311)262-207(273)325)28-371-432(333,334)411-128-76(25-287)391-194(109(128)289)284-60-246-106-165(284)253-197(222)256-175(106)315;1-62-23-262(199(317)250-160(62)301)97-21-66(276)69(381-97)28-361-422(321,322)402-67-22-98(263-24-63(2)161(302)251-200(263)318)382-70(67)29-362-423(323,324)404-128-71(384-168(109(128)283)254-13-5-89(203)234-191(254)309)31-368-434(345,346)414-138-81(394-179(119(138)293)267-54-226-100-147(207)216-49-221-152(100)267)40-373-432(341,342)412-136-79(392-176(117(136)291)264-25-64(3)162(303)252-201(264)319)38-371-429(335,336)409-134-78(391-174(115(134)289)260-19-11-95(280)240-197(260)315)37-370-436(349,350)418-142-87(400-183(123(142)297)271-58-230-104-156(271)242-187(211)246-164(104)305)46-379-440(357,358)420-144-86(399-185(125(144)299)273-60-232-106-158(273)244-189(213)248-166(106)307)45-378-431(339,340)411-135-77(390-175(116(135)290)261-20-12-96(281)241-198(261)316)36-367-428(333,334)408-133-76(389-173(114(133)288)259-18-10-94(279)239-196(259)314)35-365-425(327,328)405-129-72(385-169(110(129)284)255-14-6-90(204)235-192(255)310)32-364-427(331,332)407-132-75(388-172(113(132)287)258-17-9-93(278)238-195(258)313)34-366-426(329,330)406-130-73(386-170(111(130)285)256-15-7-91(205)236-193(256)311)33-369-435(347,348)415-139-82(395-180(120(139)294)268-55-227-101-148(208)217-50-222-153(101)268)41-374-433(343,344)413-137-80(393-177(118(137)292)265-26-65(4)163(304)253-202(265)320)39-372-437(351,352)416-140-83(396-181(121(140)295)269-56-228-102-149(209)218-51-223-154(102)269)42-375-438(353,354)417-141-84(397-182(122(141)296)270-57-229-103-150(210)219-52-224-155(103)270)43-376-439(355,356)419-145-88(401-186(126(145)300)274-61-233-107-159(274)245-190(214)249-167(107)308)47-380-441(359,360)421-143-85(398-184(124(143)298)272-59-231-105-157(272)243-188(212)247-165(105)306)44-377-430(337,338)410-131-74(387-171(112(131)286)257-16-8-92(277)237-194(257)312)30-363-424(325,326)403-127-68(27-275)383-178(108(127)282)266-53-225-99-146(206)215-48-220-151(99)266/h14-24,46-62,74-98,109-146,178-196,287-307H,12-13,25-45H2,1-11H3,(H,329,330)(H,331,332)(H,333,334)(H,335,336)(H,337,338)(H,339,340)(H,341,342)(H,343,344)(H,345,346)(H,347,348)(H,349,350)(H,351,352)(H,353,354)(H,355,356)(H,357,358)(H,359,360)(H,361,362)(H,363,364)(H,365,366)(H,367,368)(H2,211,249,318)(H2,212,250,319)(H2,213,251,320)(H2,214,252,321)(H2,215,225,232)(H2,216,226,233)(H2,217,227,234)(H2,218,228,235)(H2,219,229,236)(H2,220,230,237)(H2,221,231,238)(H,259,308,322)(H,260,309,323)(H,261,310,324)(H,262,311,325)(H,263,312,326)(H,264,313,327)(H,265,314,328)(H3,222,253,256,315)(H3,223,254,257,316)(H3,224,255,258,317);5-20,23-26,48-61,66-88,97-98,108-145,168-186,275-276,282-300H,21-22,27-47H2,1-4H3,(H,321,322)(H,323,324)(H,325,326)(H,327,328)(H,329,330)(H,331,332)(H,333,334)(H,335,336)(H,337,338)(H,339,340)(H,341,342)(H,343,344)(H,345,346)(H,347,348)(H,349,350)(H,351,352)(H,353,354)(H,355,356)(H,357,358)(H,359,360)(H2,203,234,309)(H2,204,235,310)(H2,205,236,311)(H2,206,215,220)(H2,207,216,221)(H2,208,217,222)(H2,209,218,223)(H2,210,219,224)(H,237,277,312)(H,238,278,313)(H,239,279,314)(H,240,280,315)(H,241,281,316)(H,250,301,317)(H,251,302,318)(H,252,303,319)(H,253,304,320)(H3,211,242,246,305)(H3,212,243,247,306)(H3,213,244,248,307)(H3,214,245,249,308)

InChIKey

UBJHFPNBJDGZHL-UHFFFAOYSA-N

Smiles

CC1=CN(C2OC(COP(=O)(O)OC3C(O)C(OC3COP(=O)(O)OC4C(O)C(OC4COP(=O)(O)OC5C(O)C(OC5COP(=O)(O)OC6C(O)C(OC6COP(=O)(O)OC7C(O)C(OC7COP(=O)(O)OC8C(O)C(OC8COP(=O)(O)OC9C(O)C(OC9COP(=O)(O)OC%10C(O)C(OC%10COP(=O)(O)OC%11C(O)C(OC%11COP(=O)(O)OC%12C(O)C(OC%12COP(=O)(O)OC%13C(O)C(OC%13COP(=O)(O)OC%14C(O)C(OC%14COP(=O)(O)OC%15C(O)C(OC%15COP(=O)(O)OC%16C(O)C(OC%16COP(=O)(O)OC%17C(O)C(OC%17CO)n%18cnc%19C(=O)NC(=Nc%18%19)N)N%20C=C(C)C(=O)NC%20=O)n%21cnc%22c(N)ncnc%21%22)n%23cnc%24c(N)ncnc%23%24)N%25C=C(C)C(=NC%25=O)N)N%26C=C(C)C(=NC%26=O)N)n%27cnc%28c(N)ncnc%27%28)n%29cnc%30c(N)ncnc%29%30)n%31cnc%32C(=O)NC(=Nc%31%32)N)n%33cnc%34c(N)ncnc%33%34)n%35cnc%36C(=O)NC(=Nc%35%36)N)N%37C=C(C)C(=O)NC%37=O)n%38cnc%39c(N)ncnc%38%39)N%40C=C(C)C(=O)NC%40=O)N%41C=C(C)C(=O)NC%41=O)C(OP(=O)(O)OCC%42OC(C(O)C%42OP(=O)(O)OCC%43OC(C(O)C%43OP(=O)(O)OCC%44OC(C(O)C%44OP(=O)(O)OCC%45OC(CC%45OP(=O)(O)OCC%46OC(CC%46O)N%47C=C(C)C(=O)NC%47=O)N%48C=C(C)C(=O)NC%48=O)N%49C=C(C)C(=O)NC%49=O)n%50cnc%51c(N)ncnc%50%51)N%52C=C(C)C(=NC%52=O)N)C2O)C(=O)N=C1N.CC%53=CN(C%54CC(O)C(COP(=O)(O)OC%55CC(OC%55COP(=O)(O)OC%56C(O)C(OC%56COP(=O)(O)OC%57C(O)C(OC%57COP(=O)(O)OC%58C(O)C(OC%58COP(=O)(O)OC%59C(O)C(OC%59COP(=O)(O)OC%60C(O)C(OC%60COP(=O)(O)OC%61C(O)C(OC%61COP(=O)(O)OC%62C(O)C(OC%62COP(=O)(O)OC%63C(O)C(OC%63COP(=O)(O)OC%64C(O)C(OC%64COP(=O)(O)OC%65C(O)C(OC%65COP(=O)(O)OC%66C(O)C(OC%66COP(=O)(O)OC%67C(O)C(OC%67COP(=O)(O)OC%68C(O)C(OC%68COP(=O)(O)OC%69C(O)C(OC%69COP(=O)(O)OC%70C(O)C(OC%70COP(=O)(O)OC%71C(O)C(OC%71COP(=O)(O)OC%72C(O)C(OC%72COP(=O)(O)OC%73C(O)C(OC%73COP(=O)(O)OC%74C(O)C(OC%74CO)n%75cnc%76c(N)ncnc%75%76)N%77C=CC(=O)NC%77=O)n%78cnc%79C(=O)NC(=Nc%78%79)N)n%80cnc%81C(=O)NC(=Nc%80%81)N)n%82cnc%83c(N)ncnc%82%83)n%84cnc%85c(N)ncnc%84%85)N%86C=C(C)C(=O)NC%86=O)n%87cnc%88c(N)ncnc%87%88)N%89C=CC(=NC%89=O)N)N%90C=CC(=O)NC%90=O)N%91C=CC(=NC%91=O)N)N%92C=CC(=O)NC%92=O)N%93C=CC(=O)NC%93=O)n%94cnc%95C(=O)NC(=Nc%94%95)N)n%96cnc%97C(=O)NC(=Nc%96%97)N)N%98C=CC(=O)NC%98=O)N%99C=C(C)C(=O)NC%99=O)n1cnc2c(N)ncnc12)N3C=CC(=NC3=O)N)N4C=C(C)C(=O)NC4=O)O%54)C(=O)NC%53=O