JP2021514190A - コード化リボ核酸の器官保護発現および調節のための組成物および方法 - Google Patents

コード化リボ核酸の器官保護発現および調節のための組成物および方法 Download PDF

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JP2021514190A
JP2021514190A JP2020543932A JP2020543932A JP2021514190A JP 2021514190 A JP2021514190 A JP 2021514190A JP 2020543932 A JP2020543932 A JP 2020543932A JP 2020543932 A JP2020543932 A JP 2020543932A JP 2021514190 A JP2021514190 A JP 2021514190A
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cells
mrna
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sequence
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ミコル,ロマン
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Combined Therapeutics Inc
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Combined Therapeutics Inc
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JP2020543932A 2017-09-07 2019-02-19 コード化リボ核酸の器官保護発現および調節のための組成物および方法 Pending JP2021514190A (ja)

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JP2024015220A JP2024059656A (ja) 2017-09-07 2024-02-02 コード化リボ核酸の器官保護発現および調節のための組成物および方法

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862632056P 2018-02-19 2018-02-19
US62/632,056 2018-02-19
USPCT/US2018/049772 2018-09-06
PCT/US2018/049772 WO2019051100A1 (en) 2017-09-07 2018-09-06 COMPOSITIONS AND METHODS FOR TARGETED CODING ADMINISTRATION, EXPRESSION AND MODULATION OF RIBONUCLEIC ACIDS IN TISSUE
PCT/GB2019/050454 WO2019158955A1 (en) 2018-02-19 2019-02-19 Compositions and methods for organ-protective expression and modulation of coding ribonucleic acids

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JP2024015220A Division JP2024059656A (ja) 2017-09-07 2024-02-02 コード化リボ核酸の器官保護発現および調節のための組成物および方法

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JP2021514190A true JP2021514190A (ja) 2021-06-10
JP2021514190A5 JP2021514190A5 (https=) 2022-02-17

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US (1) US20200376142A1 (https=)
EP (2) EP4008333A1 (https=)
JP (1) JP2021514190A (https=)
KR (1) KR20200123139A (https=)
CN (1) CN111954535A (https=)
AU (1) AU2019221826A1 (https=)
CA (1) CA3091543A1 (https=)
WO (1) WO2019158955A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023526905A (ja) * 2020-04-30 2023-06-26 ハンジョウ・コンバード・カンパニー,リミテッド マイクロrnaで調節及び制御可能な単離された組換え腫瘍溶解性ポックスウイルス及びその使用

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201714430D0 (en) 2017-09-07 2017-10-25 Micol Romain Compositions and processes for targeted delivery and expression and modulation of therapeutic components in tissue
JP7539723B2 (ja) 2020-02-21 2024-08-26 コンバインド セラピューティクス インコーポレイテッド コード化リボ核酸の器官保護発現及び調節のための組成物並びに方法
MX2023001187A (es) * 2020-07-31 2023-04-04 Combined Therapeutics Inc Composiciones y métodos para una vacunación mejorada.
CN114908089B (zh) * 2021-02-08 2024-03-15 上海细胞治疗集团有限公司 3’utr的构建方法和应用
EP4334446A1 (en) 2021-05-03 2024-03-13 CureVac SE Improved nucleic acid sequence for cell type specific expression
EP4241790A1 (en) * 2022-03-07 2023-09-13 InnaTher Gene Therapy S.à.r.l. Expression system for the treatment of cancer
EP4241791A1 (en) * 2022-03-07 2023-09-13 InnaTher Gene Therapy S.à.r.l. Combined gene and radio therapy for the treatment of cancer
CN116334183B (zh) * 2023-02-18 2025-10-21 四川大学华西医院 基于多重信号放大和金属离子选择性识别现象的尿液中双miRNA的检测方法及其应用
CN116240270A (zh) * 2023-03-06 2023-06-09 西北农林科技大学 一种动物驯服行为相关rrm1基因突变体的应用
CN117530932B (zh) * 2024-01-08 2024-04-26 中国药科大学 一种抑制肿瘤生长及肺转移的人工外泌体及其应用

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015501844A (ja) * 2011-12-16 2015-01-19 モデルナ セラピューティクス インコーポレイテッドModerna Therapeutics,Inc. 修飾ヌクレオシド、ヌクレオチドおよび核酸組成物
WO2016040395A1 (en) * 2014-09-08 2016-03-17 Massachusetts Institute Of Technology Rna-based logic circuits with rna binding proteins, aptamers and small molecules
WO2017132552A1 (en) * 2016-01-27 2017-08-03 Oncorus, Inc. Oncolytic viral vectors and uses thereof
JP2017523777A (ja) * 2014-07-17 2017-08-24 モデルナティエックス インコーポレイテッドModernaTX,Inc. ポリヌクレオチドの末端修飾
WO2017201325A1 (en) * 2016-05-18 2017-11-23 Modernatx, Inc. Combinations of mrnas encoding immune modulating polypeptides and uses thereof

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009012263A2 (en) * 2007-07-18 2009-01-22 The Trustees Of Columbia University In The City Of New York Tissue-specific micrornas and compositions and uses thereof
EP3269395A1 (en) 2008-11-07 2018-01-17 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
CA3285826A1 (en) 2012-02-22 2026-03-02 The Trustees Of The University Of Pennsylvania Compositions and methods for generating a persisting population of t cells useful for the treatment of cancer
BR112016009465A8 (pt) * 2013-10-28 2018-01-30 Univ Pittsburgh Commonwealth Sys Higher Education vetor de hsv oncolítico.

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015501844A (ja) * 2011-12-16 2015-01-19 モデルナ セラピューティクス インコーポレイテッドModerna Therapeutics,Inc. 修飾ヌクレオシド、ヌクレオチドおよび核酸組成物
JP2017523777A (ja) * 2014-07-17 2017-08-24 モデルナティエックス インコーポレイテッドModernaTX,Inc. ポリヌクレオチドの末端修飾
WO2016040395A1 (en) * 2014-09-08 2016-03-17 Massachusetts Institute Of Technology Rna-based logic circuits with rna binding proteins, aptamers and small molecules
WO2017132552A1 (en) * 2016-01-27 2017-08-03 Oncorus, Inc. Oncolytic viral vectors and uses thereof
WO2017201325A1 (en) * 2016-05-18 2017-11-23 Modernatx, Inc. Combinations of mrnas encoding immune modulating polypeptides and uses thereof

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023526905A (ja) * 2020-04-30 2023-06-26 ハンジョウ・コンバード・カンパニー,リミテッド マイクロrnaで調節及び制御可能な単離された組換え腫瘍溶解性ポックスウイルス及びその使用
JP7699838B2 (ja) 2020-04-30 2025-06-30 ハンジョウ・コンバード・カンパニー,リミテッド マイクロrnaで調節及び制御可能な単離された組換え腫瘍溶解性ポックスウイルス及びその使用

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EP4008333A1 (en) 2022-06-08
KR20200123139A (ko) 2020-10-28
US20200376142A1 (en) 2020-12-03
AU2019221826A1 (en) 2020-09-17
CA3091543A1 (en) 2019-08-22
WO2019158955A1 (en) 2019-08-22
WO2019158955A9 (en) 2020-02-20
CN111954535A (zh) 2020-11-17
EP3755344A1 (en) 2020-12-30

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