KR20210126004A - Hmgb1 발현을 억제하기 위한 조성물 및 방법 - Google Patents

Hmgb1 발현을 억제하기 위한 조성물 및 방법 Download PDF

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KR20210126004A
KR20210126004A KR1020217023455A KR20217023455A KR20210126004A KR 20210126004 A KR20210126004 A KR 20210126004A KR 1020217023455 A KR1020217023455 A KR 1020217023455A KR 20217023455 A KR20217023455 A KR 20217023455A KR 20210126004 A KR20210126004 A KR 20210126004A
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seq
set forth
oligonucleotide
sequence set
artificial sequence
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Korean (ko)
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마크 애브람스
기리시 촙다
지혜 박
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다이서나 파마수이티컬, 인크.
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    • C12N15/09Recombinant DNA-technology
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    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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KR1020217023455A 2018-12-28 2019-12-20 Hmgb1 발현을 억제하기 위한 조성물 및 방법 Withdrawn KR20210126004A (ko)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201862786287P 2018-12-28 2018-12-28
US62/786,287 2018-12-28
US201862787038P 2018-12-31 2018-12-31
US62/787,038 2018-12-31
US201962788111P 2019-01-03 2019-01-03
US62/788,111 2019-01-03
PCT/US2019/067883 WO2020139764A1 (en) 2018-12-28 2019-12-20 Compositions and methods for inhibiting hmgb1 expression

Publications (1)

Publication Number Publication Date
KR20210126004A true KR20210126004A (ko) 2021-10-19

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KR1020217023455A Withdrawn KR20210126004A (ko) 2018-12-28 2019-12-20 Hmgb1 발현을 억제하기 위한 조성물 및 방법

Country Status (13)

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US (1) US20220072024A1 (https=)
EP (1) EP3883581A4 (https=)
JP (2) JP2022517742A (https=)
KR (1) KR20210126004A (https=)
CN (1) CN113874025A (https=)
AU (1) AU2019417585A1 (https=)
BR (1) BR112021012516A2 (https=)
CA (1) CA3124664A1 (https=)
CL (2) CL2021001718A1 (https=)
IL (2) IL284327B2 (https=)
MX (1) MX2021007855A (https=)
SG (1) SG11202106857VA (https=)
WO (1) WO2020139764A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20200023427A (ko) 2017-06-29 2020-03-04 다이서나 파마수이티컬, 인크. Hmgb1 발현을 억제하기 위한 조성물 및 방법
WO2023208023A1 (zh) * 2022-04-26 2023-11-02 上海拓界生物医药科技有限公司 氘代化学修饰和包含其的寡核苷酸
TWI868755B (zh) 2022-06-24 2025-01-01 丹麥商諾佛 儂迪克股份有限公司 抑制跨膜絲胺酸蛋白酶6(tmprss6)表現的組成物及方法
CN117264954A (zh) * 2022-09-20 2023-12-22 北京福元医药股份有限公司 用于抑制hmgb1基因表达的双链核糖核酸及其修饰物、缀合物和用途
WO2024233864A2 (en) * 2023-05-10 2024-11-14 Dicerna Pharmaceuticals, Inc. Galnac-conjugated rnai oligonucleotides

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007076200A2 (en) * 2005-11-28 2007-07-05 Medimmune, Inc. Antagonists of hmgb1 and/or rage and methods of use thereof
WO2007150071A1 (en) * 2006-06-23 2007-12-27 Myriad Genetics, Inc. Gene amplifications and deletions
US9278108B2 (en) * 2009-07-16 2016-03-08 Nec Solution Innovators, Ltd. HMGB1 binding nucleic acid molecule and applications thereof
WO2012036215A1 (ja) * 2010-09-17 2012-03-22 Taniguchi Tadatsugu Hmgbタンパクによって仲介される免疫応答の活性化の抑制剤及びスクリーニング方法
WO2012177639A2 (en) * 2011-06-22 2012-12-27 Alnylam Pharmaceuticals, Inc. Bioprocessing and bioproduction using avian cell lines
AU2015330670B2 (en) * 2014-10-10 2022-01-06 Novo Nordisk Health Care Ag Therapeutic inhibition of lactate dehydrogenase and agents therefor
JP7105065B2 (ja) * 2014-12-15 2022-07-22 ダイセルナ ファーマシューティカルズ, インコーポレイテッド リガンド修飾二本鎖核酸
WO2017079227A1 (en) * 2015-11-05 2017-05-11 University Of Connecticut Compositions and methods for the treatment of liver fibrosis
KR102350647B1 (ko) * 2016-09-02 2022-01-14 다이서나 파마수이티컬, 인크. 4'-포스페이트 유사체 및 이를 포함하는 올리고뉴클레오타이드
KR20200023427A (ko) * 2017-06-29 2020-03-04 다이서나 파마수이티컬, 인크. Hmgb1 발현을 억제하기 위한 조성물 및 방법
JP2021508491A (ja) * 2017-12-18 2021-03-11 アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. 高移動度グループボックス−1(HMGB1)iRNA組成物及びその使用方法

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Publication number Publication date
MX2021007855A (es) 2021-10-26
CA3124664A1 (en) 2020-07-02
CN113874025A (zh) 2021-12-31
SG11202106857VA (en) 2021-07-29
EP3883581A4 (en) 2023-03-29
WO2020139764A1 (en) 2020-07-02
CL2023002984A1 (es) 2024-03-08
CL2021001718A1 (es) 2022-02-18
IL284327A (en) 2021-08-31
IL313425A (en) 2024-08-01
US20220072024A1 (en) 2022-03-10
IL284327B2 (en) 2024-11-01
EP3883581A1 (en) 2021-09-29
BR112021012516A2 (pt) 2021-09-14
IL284327B1 (en) 2024-07-01
AU2019417585A1 (en) 2021-07-08
JP2024105308A (ja) 2024-08-06
JP2022517742A (ja) 2022-03-10

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