JP2017500277A5 - - Google Patents

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JP2017500277A5
JP2017500277A5 JP2016519876A JP2016519876A JP2017500277A5 JP 2017500277 A5 JP2017500277 A5 JP 2017500277A5 JP 2016519876 A JP2016519876 A JP 2016519876A JP 2016519876 A JP2016519876 A JP 2016519876A JP 2017500277 A5 JP2017500277 A5 JP 2017500277A5
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JP
Japan
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strand
optionally
composition
nucleotides
nucleic acid
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JP2016519876A
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Japanese (ja)
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JP2017500277A (ja
JP6694811B2 (ja
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Priority claimed from PCT/US2014/058705 external-priority patent/WO2015051045A2/en
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JP2016519876A 2013-10-04 2014-10-01 RNA干渉に使用するためのRNAi剤用の3’末端キャップ Active JP6694811B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361886739P 2013-10-04 2013-10-04
US61/886,739 2013-10-04
PCT/US2014/058705 WO2015051045A2 (en) 2013-10-04 2014-10-01 3'END CAPS FOR RNAi AGENTS FOR USE IN RNA INTERFERENCE

Related Child Applications (1)

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JP2019053123A Division JP6841859B2 (ja) 2013-10-04 2019-03-20 RNA干渉に使用するためのRNAi剤用の3’末端キャップ

Publications (3)

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JP2017500277A JP2017500277A (ja) 2017-01-05
JP2017500277A5 true JP2017500277A5 (enExample) 2017-11-09
JP6694811B2 JP6694811B2 (ja) 2020-05-20

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JP2016519876A Active JP6694811B2 (ja) 2013-10-04 2014-10-01 RNA干渉に使用するためのRNAi剤用の3’末端キャップ
JP2019053123A Active JP6841859B2 (ja) 2013-10-04 2019-03-20 RNA干渉に使用するためのRNAi剤用の3’末端キャップ

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Country Status (8)

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US (2) US10227588B2 (enExample)
EP (2) EP3052464B1 (enExample)
JP (2) JP6694811B2 (enExample)
KR (1) KR102298475B1 (enExample)
CN (1) CN105683163B (enExample)
AU (2) AU2014329560B2 (enExample)
CA (2) CA2925129C (enExample)
WO (1) WO2015051045A2 (enExample)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3052627B1 (en) 2013-10-04 2018-08-22 Novartis AG Novel formats for organic compounds for use in rna interference
KR102298475B1 (ko) 2013-10-04 2021-09-06 노파르티스 아게 RNA 간섭에 사용하기 위한 RNAi 작용제를 위한 3' 말단 캡
JP6546161B2 (ja) 2013-10-04 2019-07-17 ノバルティス アーゲー B型肝炎ウイルスを治療するための有機化合物
SMT202000004T1 (it) * 2015-07-21 2020-03-13 Immunogen Inc Metodi di preparazione dei derivati citotossici di benzodiazepina
WO2017047807A1 (ja) * 2015-09-17 2017-03-23 国立大学法人大阪大学 トラン化合物
EP3564375A4 (en) * 2016-12-27 2020-12-16 The University Of Tokyo ARNM FUNCTIONALIZATION PROCESS

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