JP2018528783A5 - - Google Patents

Download PDF

Info

Publication number
JP2018528783A5
JP2018528783A5 JP2018515456A JP2018515456A JP2018528783A5 JP 2018528783 A5 JP2018528783 A5 JP 2018528783A5 JP 2018515456 A JP2018515456 A JP 2018515456A JP 2018515456 A JP2018515456 A JP 2018515456A JP 2018528783 A5 JP2018528783 A5 JP 2018528783A5
Authority
JP
Japan
Prior art keywords
duplex
modified
modified oligonucleotide
extrahepatic
section
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2018515456A
Other languages
English (en)
Japanese (ja)
Other versions
JP2018528783A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2016/053836 external-priority patent/WO2017053999A1/en
Publication of JP2018528783A publication Critical patent/JP2018528783A/ja
Publication of JP2018528783A5 publication Critical patent/JP2018528783A5/ja
Pending legal-status Critical Current

Links

JP2018515456A 2015-09-25 2016-09-26 コンジュゲートアンチセンス化合物及びその使用 Pending JP2018528783A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562233292P 2015-09-25 2015-09-25
US62/233,292 2015-09-25
PCT/US2016/053836 WO2017053999A1 (en) 2015-09-25 2016-09-26 Conjugated antisense compounds and their use

Publications (2)

Publication Number Publication Date
JP2018528783A JP2018528783A (ja) 2018-10-04
JP2018528783A5 true JP2018528783A5 (enExample) 2019-11-07

Family

ID=58387451

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2018515456A Pending JP2018528783A (ja) 2015-09-25 2016-09-26 コンジュゲートアンチセンス化合物及びその使用

Country Status (4)

Country Link
US (2) US11116843B2 (enExample)
EP (1) EP3353306A4 (enExample)
JP (1) JP2018528783A (enExample)
WO (1) WO2017053999A1 (enExample)

Families Citing this family (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7043078B2 (ja) 2016-09-23 2022-03-29 国立大学法人 東京医科歯科大学 血液脳関門通過型ヘテロ2本鎖核酸
EP3662060A2 (en) 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
CA3099522C (en) * 2017-09-19 2024-10-08 Univ Alberta GAPMERES AND METHODS OF USING THESE FOR THE TREATMENT OF MUSCULAR DYSTROPHY
JP2021506239A (ja) * 2017-12-14 2021-02-22 アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. 複合アンチセンス化合物及びその使用
CA3088112A1 (en) 2018-01-12 2019-07-18 Bristol-Myers Squibb Company Antisense oligonucleotides targeting alpha-synuclein and uses thereof
KR102839166B1 (ko) 2018-01-12 2025-07-28 브리스톨-마이어스 스큅 컴퍼니 알파-시누클레인을 표적화하는 안티센스 올리고뉴클레오티드 및 그의 용도
IL320427A (en) 2018-02-21 2025-06-01 Bristol Myers Squibb Co CAMK2D antisense oligonucleotides and their uses
EP3766972B1 (en) 2018-03-14 2025-04-23 Institute of Science Tokyo Nucleic acid complex
WO2019182294A1 (ko) * 2018-03-19 2019-09-26 주식회사 케이티 비디오 신호 처리 방법 및 장치
WO2019181946A1 (ja) 2018-03-19 2019-09-26 国立大学法人東京医科歯科大学 毒性が軽減した核酸
WO2019182109A1 (ja) * 2018-03-22 2019-09-26 国立大学法人東京医科歯科大学 ヘテロ核酸のbbb通過脂質リガンド
US20220064636A1 (en) * 2018-10-05 2022-03-03 Ionis Pharmaceuticals, Inc. Modified oligomeric compounds and uses thereof
CN120290561A (zh) * 2019-03-29 2025-07-11 田边三菱制药株式会社 用于调节dux4的表达的化合物、方法和医药组合物
CN113677374A (zh) * 2019-04-08 2021-11-19 国立大学法人东京医科齿科大学 肌疾病治疗用药物组合物
JP7737667B2 (ja) * 2019-09-18 2025-09-11 国立大学法人東京科学大学 核酸複合体
WO2021070959A1 (ja) * 2019-10-11 2021-04-15 国立大学法人東京医科歯科大学 修飾ヘテロ核酸
EP4055166A2 (en) * 2019-11-06 2022-09-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
BR112022013686A2 (pt) * 2020-01-10 2022-09-06 Dyne Therapeutics Inc Complexos de direcionamento a músculo e usos dos mesmos para tratar distrofia miotônica
CN115516092B (zh) * 2020-09-30 2025-05-16 亿腾医药(苏州)有限公司 血管生成素样3(ANGPTL3)的siRNA及其用途
WO2022106695A1 (en) 2020-11-23 2022-05-27 Alpha Anomeric Sas Nucleic acid duplexes
WO2023178144A2 (en) 2022-03-16 2023-09-21 Empirico Inc. Galnac compositions for improving sirna bioavailability
AU2023245603A1 (en) 2022-03-28 2024-11-07 Empirico Inc. Modified oligonucleotides
US20250304957A1 (en) 2022-05-13 2025-10-02 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
WO2024238385A2 (en) 2023-05-12 2024-11-21 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
TW202508607A (zh) * 2023-05-19 2025-03-01 大陸商北京炫景瑞醫藥科技有限公司 雙股寡核苷酸及其共軛物和用途
WO2024257861A1 (ja) * 2023-06-16 2024-12-19 日産化学株式会社 脂質結合オリゴヌクレオチド又はその複合体

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
EP1937066A4 (en) 2005-08-18 2008-12-24 Alnylam Pharmaceuticals Inc METHOD AND COMPOSITIONS FOR THE TREATMENT OF NERVOUS DISEASES
AU2008340354B2 (en) * 2007-12-04 2014-04-17 Alnylam Pharmaceuticals, Inc. Folate-iRNA conjugates
US20100048676A1 (en) 2008-08-04 2010-02-25 University Of Rochester Non-androgen dependent roles for androgen receptor in liver cancer
US20100222417A1 (en) 2008-11-26 2010-09-02 Alnylam Pharmaceuticals Compostions and methods for enhancing oligonucleotide delivery across and into epithelial tissues
PT2396038E (pt) * 2009-02-12 2016-02-19 Curna Inc Tratamento das doenças associadas com o factor neurotrófico derivado do cérebro (bdnf) por inibição do produto antisenso natural da transcrição para bdnf
KR20180105730A (ko) 2010-07-19 2018-09-28 아이오니스 파마수티컬즈, 인코포레이티드 근육긴장성 이영양증-단백질 키나제(dmpk) 발현의 조절 방법
EP3533873A1 (en) * 2011-09-14 2019-09-04 Translate Bio MA, Inc. Multimeric oligonucleotide compounds
EP2791335B1 (en) 2011-12-16 2018-11-14 National University Corporation Tokyo Medical and Dental University Chimeric double-stranded nucleic acid
US9175291B2 (en) 2012-10-11 2015-11-03 Isis Pharmaceuticals Inc. Modulation of androgen receptor expression
CA2901983A1 (en) 2013-03-01 2014-09-04 National University Corporation Tokyo Medical And Dental University Chimeric single-stranded antisense polynucleotides and double-stranded antisense agent
KR102482890B1 (ko) 2013-05-01 2022-12-30 아이오니스 파마수티컬즈, 인코포레이티드 아포지질단백질 (a) 발현을 조절하는 조성물 및 방법
EP3004347B1 (en) 2013-05-30 2018-09-26 National University Corporation Tokyo Medical and Dental University Double-stranded agents for delivering therapeutic oligonucleotides
WO2014203518A1 (en) 2013-06-16 2014-12-24 National University Corporation Tokyo Medical And Dental University Double-stranded antisense nucleic acid with exon-skipping effect
TW201536329A (zh) 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法
US12139542B2 (en) * 2014-05-27 2024-11-12 Trustees Of Boston University Inhibitors of aortic carboxypeptidase-like protein (ACLP)
EP3180426B1 (en) * 2014-08-17 2019-12-25 The Broad Institute, Inc. Genome editing using cas9 nickases
US20210052631A1 (en) 2015-09-25 2021-02-25 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use

Similar Documents

Publication Publication Date Title
JP2018528783A5 (enExample)
US11116843B2 (en) Conjugated antisense compounds and their use
RU2501803C2 (ru) Композиции и способы модуляции экспрессии рецептора фактора роста фибробластов 4 (fgfr4)
JP5763539B2 (ja) 5’及び2’ビス置換ヌクレオシド及びそれから製造されるオリゴマー化合物
US9428750B2 (en) Oligomeric compounds comprising tricyclic nucleosides and methods for their use
RU2015151199A (ru) Сопряженные антисмысловые соединения и их применение
CN110846316B (zh) 在基因沉默中具有降低的脱靶效应的una寡聚物
CN114025848A (zh) 用于调节剪接和翻译的方法和组合物
JP2021072862A (ja) B型肝炎感染の治療用のPAPD5及びPAPD7 mRNAを低減させるための核酸分子
US20160186175A1 (en) Bicyclic morpholino compounds and oligomeric compounds prepared therefrom
US9249178B2 (en) Tricyclic nucleosides and oligomeric compounds prepared therefrom
KR20200104347A (ko) 포스포로디티오에이트 인터뉴클레오시드 연결을 포함하는 갭머 올리고뉴클레오티드
EP3516062A1 (en) Myostatin irna compositions and methods of use thereof
US20160186174A1 (en) Substituted morpholino compounds analogs thereof and oligomeric compounds prepared therefrom
WO2021157730A1 (ja) 核酸医薬とその使用
US20220098595A1 (en) Nr2e3 expression reducing oligonucleotides, compositions containing the same, and methods of their use
JPWO2021030213A5 (enExample)
US20230089502A1 (en) Products and compositions
JP6934695B2 (ja) 核酸医薬とその使用
JP2023531249A (ja) Fubp1発現を調節するための増強されたオリゴヌクレオチド
CN117813383B (zh) 用于抑制CIDEB基因表达的siRNA、药物及其应用
EP3914713A1 (en) Nrl expression reducing oligonucleotides, compositions containing the same, and methods of their use
US20250368998A1 (en) Nucleotide analogues, and preparation and application thereof
WO2020044349A1 (en) Compounds, cojugates and compositions for use in the methods for trans-membrane delivery of molecules
DE102022124232A1 (de) Antisense-Oligonukleotide für die Behandlung des Joubert-Syndroms