MedKoo Biosciences, Inc.
Tel:   +1-919-636-5577    Fax:   +1-919-980-4831    Email:   sales@medkoo.com
Search
Login Register
Search
MedKoo Cat#: 413924 | Name: Drisapersen Na

Description:

WARNING: This product is for research use only, not for human or veterinary use.

Drisapersen Na is a synthetic, modified RNA molecule; Intramuscular injection of antisense oligonucleotide PRO051 induced dystrophin synthesis in four patients with Duchenne's muscular dystrophy who had suitable mutations

Chemical Structure

Drisapersen Na
Drisapersen Na
CAS#1181666-20-5 (Na)

Theoretical Analysis

MedKoo Cat#: 413924

Name: Drisapersen Na

CAS#: 1181666-20-5 (Na)

Chemical Formula: C211H256N76Na19O119P19S19

Exact Mass:

Molecular Weight: 7395.23

Elemental Analysis: C, 34.27; H, 3.49; N, 14.39; Na, 5.91; O, 25.74; P, 7.96; S, 8.24

Price and Availability

This product is currently not in stock but may be available through custom synthesis. To ensure cost efficiency, the minimum order quantity is 1 gram. The estimated lead time is 2 to 4 months, with pricing dependent on the complexity of the synthesis (typically high for intricate chemistries). Quotes for quantities below 1 gram will not be provided. To request a quote, please click the button below. Note: If this product becomes available in stock in the future, pricing will be listed accordingly.
Bulk Inquiry
Related CAS #
1181666-20-5 (Na) 1251830-50-8 (free base)
Synonym
Drisapersen Na; GSK2402968B; Drisapersen sodium; h51AON23 sodium salt
IUPAC/Chemical Name
All-P-ambo-2'-O-methyl-P-thiouridylyl-(3'->5')-2'-O-methyl-P-thiocytidylyl-(3'->5')-2'-O- methyl-P-thioadenylyl-(3'->5')-2'-O-methyl-P-thioadenylyl-(3'->5')-2'-O-methyl-P- thioguanylyl-(3'->5')-2'-O-methyl-P-thioguanylyl-(3'->5')-2'-O-methyl-P-thioadenylyl- (3'->5')-2'-O-methyl-P-thioadenylyl-(3'->5')-2'-O-methyl-P-thioguanylyl-(3'->5')-2'-O- methyl-P-thioadenylyl-(3'->5')-2'-O-methyl-P-thiouridylyl-(3'->5')-2'-O-methyl-P- thioguanylyl-(3'->5')-2'-O-methyl-P-thioguanylyl-(3'->5')-2'-O-methyl-P-thiocytidylyl- (3'->5')-2'-O-methyl-P-thioadenylyl-(3'->5')-2'-O-methyl-P-thiouridylyl-(3'->5')-2'-O- methyl-P-thiouridylyl-(3'->5')-2'-O-methyl-P-thiouridylyl-(3'->5')-2'-O-methyl-P- thiocytidylyl-(3'->5')-2'-O-methyluridine, nonadeca sodium
InChi Key
FZJHBHYSGPESDI-FWLJYILUSA-A
InChi Code
InChI=1S/C211H275N76O119P19S19.19Na/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);;;;;;;;;;;;;;;;;;;/q;19*+1/p-19/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
SMILES Code
CO[C@@H]1[C@@H]([C@H](O[C@H]1N2C=CC(NC2=O)=O)COP([S-])(O[C@@H]3[C@H](O[C@@H](N4C=CC(N)=NC4=O)[C@@H]3OC)COP([S-])(O[C@@H]5[C@H](O[C@@H](N6C=CC(NC6=O)=O)[C@@H]5OC)COP([S-])(O[C@@H]7[C@H](O[C@@H](N8C=CC(NC8=O)=O)[C@@H]7OC)COP([S-])(O[C@@H]9[C@H](O[C@@H](N%10C=CC(NC%10=O)=O)[C@@H]9OC)COP([S-])(O[C@@H]%11[C@H](O[C@@H](n%12c%13ncnc(N)c%13nc%12)[C@@H]%11OC)COP([S-])(O[C@@H]%14[C@H](O[C@@H](N%15C=CC(N)=NC%15=O)[C@@H]%14OC)COP([S-])(O[C@@H]%16[C@H](O[C@@H](n%17c%18c(C(NC(N)=N%18)=O)nc%17)[C@@H]%16OC)COP([S-])(O[C@@H]%19[C@H](O[C@@H](n%20c%21c(C(NC(N)=N%21)=O)nc%20)[C@@H]%19OC)COP([S-])(O[C@@H]%22[C@H](O[C@@H](N%23C=CC(NC%23=O)=O)[C@@H]%22OC)COP([S-])(O[C@@H]%24[C@H](O[C@@H](n%25c%26ncnc(N)c%26nc%25)[C@@H]%24OC)COP([S-])(O[C@@H]%27[C@H](O[C@@H](n%28c%29c(C(NC(N)=N%29)=O)nc%28)[C@@H]%27OC)COP([S-])(O[C@@H]%30[C@H](O[C@@H](n%31c%32ncnc(N)c%32nc%31)[C@@H]%30OC)COP([S-])(O[C@@H]%33[C@H](O[C@@H](n%34c%35ncnc(N)c%35nc%34)[C@@H]%33OC)COP([S-])(O[C@@H]%36[C@H](O[C@@H](n%37c%38c(C(NC(N)=N%38)=O)nc%37)[C@@H]%36OC)COP([S-])(O[C@@H]%39[C@H](O[C@@H](n%40c%41c(C(NC(N)=N%41)=O)nc%40)[C@@H]%39OC)COP([S-])(O[C@@H]%42[C@H](O[C@@H](n%43c%44ncnc(N)c%44nc%43)[C@@H]%42OC)COP([S-])(O[C@@H]%45[C@H](O[C@@H](n%46c%47ncnc(N)c%47nc%46)[C@@H]%45OC)COP([S-])(O[C@@H]%48[C@H](O[C@@H](N%49C=CC(N)=NC%49=O)[C@@H]%48OC)COP([S-])(O[C@@H]%50[C@H](O[C@@H](N%51C=CC(NC%51=O)=O)[C@@H]%50OC)CO)=O)=O)=O)=O)=O)=O)=O)=O)=O)=O)=O)=O)=O)=O)=O)=O)=O)=O)=O)O.[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+].[Na+]
Appearance
Solid powder
Purity
>98% (or refer to the Certificate of Analysis)
Shipping Condition
Shipped under ambient temperature as non-hazardous chemical. This product is stable enough for a few weeks during ordinary shipping and time spent in Customs.
Storage Condition
Dry, dark and at 0 - 4 C for short term (days to weeks) or -20 C for long term (months to years).
Solubility
To be determined
Shelf Life
>2 years if stored properly
Drug Formulation
To be determined
Stock Solution Storage
0 - 4 C for short term (days to weeks), or -20 C for long term (months).
HS Tariff Code
2934.99.9001
More Info

Preparing Stock Solutions

The following data is based on the product molecular weight 7,395.23 Batch specific molecular weights may vary from batch to batch due to the degree of hydration, which will affect the solvent volumes required to prepare stock solutions.

Recalculate based on batch purity %
Concentration / Solvent Volume / Mass 1 mg 5 mg 10 mg
1 mM 1.15 mL 5.76 mL 11.51 mL
5 mM 0.23 mL 1.15 mL 2.3 mL
10 mM 0.12 mL 0.58 mL 1.15 mL
50 mM 0.02 mL 0.12 mL 0.23 mL
1: Goemans N, Mercuri E, Belousova E, Komaki H, Dubrovsky A, McDonald CM, Kraus JE, Lourbakos A, Lin Z, Campion G, Wang SX, Campbell C; DEMAND III study group. A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy. Neuromuscul Disord. 2018 Jan;28(1):4-15. doi: 10.1016/j.nmd.2017.10.004. Epub 2017 Dec 6. PMID: 29203355. 2: McDonald CM, Wong B, Flanigan KM, Wilson R, de Kimpe S, Lourbakos A, Lin Z, Campion G; DEMAND V study group. Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy. Ann Clin Transl Neurol. 2018 Jul 11;5(8):913-926. doi: 10.1002/acn3.579. PMID: 30128316; PMCID: PMC6093847. 3: Shimizu-Motohashi Y, Murakami T, Kimura E, Komaki H, Watanabe N. Exon skipping for Duchenne muscular dystrophy: a systematic review and meta-analysis. Orphanet J Rare Dis. 2018 Jun 15;13(1):93. doi: 10.1186/s13023-018-0834-2. PMID: 29907124; PMCID: PMC6003096. 4: McMillan HJ, Amid A, Gonorazky H, Almobarak S, Campbell C. Drisapersen associated with elevated serum factor VIII levels in Duchenne muscular dystrophy. Neurology. 2020 Mar 24;94(12):538-540. doi: 10.1212/WNL.0000000000009139. Epub 2020 Feb 19. PMID: 32075891. 5: Kole R, Krieg AM. Exon skipping therapy for Duchenne muscular dystrophy. Adv Drug Deliv Rev. 2015 Jun 29;87:104-7. doi: 10.1016/j.addr.2015.05.008. Epub 2015 May 14. PMID: 25980936. 6: Hilhorst N, Spanoudi-Kitrimi I, Goemans N, Morren MA. Injection site reactions after long-term subcutaneous delivery of drisapersen: a retrospective study. Eur J Pediatr. 2019 Feb;178(2):253-258. doi: 10.1007/s00431-018-3272-1. Epub 2018 Nov 17. PMID: 30448867. 7: Reinig AM, Mirzaei S, Berlau DJ. Advances in the Treatment of Duchenne Muscular Dystrophy: New and Emerging Pharmacotherapies. Pharmacotherapy. 2017 Apr;37(4):492-499. doi: 10.1002/phar.1909. Epub 2017 Mar 10. PMID: 28152217. 8: Hoffman E. A rebirth for drisapersen in Duchenne muscular dystrophy? Lancet Neurol. 2014 Oct;13(10):963-5. doi: 10.1016/S1474-4422(14)70158-9. Epub 2014 Sep 7. PMID: 25209737. 9: Pascual-Morena C, Cavero-Redondo I, Álvarez-Bueno C, Mesas AE, Pozuelo- Carrascosa D, Martínez-Vizcaíno V. Restorative treatments of dystrophin expression in Duchenne muscular dystrophy: A systematic review. Ann Clin Transl Neurol. 2020 Sep;7(9):1738-1752. doi: 10.1002/acn3.51149. Epub 2020 Aug 10. PMID: 33325654; PMCID: PMC7480922. 10: Willcocks RJ, Forbes SC, Walter GA, Vandenborne K. Magnetic resonance imaging characteristics of injection site reactions after long-term subcutaneous delivery of drisapersen. Eur J Pediatr. 2019 May;178(5):777-778. doi: 10.1007/s00431-019-03349-0. Epub 2019 Feb 21. PMID: 30790036; PMCID: PMC6530557.