JP2025041652A5 - - Google Patents

Info

Publication number
JP2025041652A5
JP2025041652A5 JP2024209791A JP2024209791A JP2025041652A5 JP 2025041652 A5 JP2025041652 A5 JP 2025041652A5 JP 2024209791 A JP2024209791 A JP 2024209791A JP 2024209791 A JP2024209791 A JP 2024209791A JP 2025041652 A5 JP2025041652 A5 JP 2025041652A5
Authority
JP
Japan
Prior art keywords
sfg
sfu
sfa
composition
modified nucleoside
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
JP2024209791A
Other languages
English (en)
Japanese (ja)
Other versions
JP2025041652A (ja
Filing date
Publication date
Priority claimed from PCT/US2019/027109 external-priority patent/WO2019200185A1/en
Priority claimed from PCT/US2019/031672 external-priority patent/WO2019217784A1/en
Priority claimed from PCT/US2019/065058 external-priority patent/WO2020118246A1/en
Application filed filed Critical
Publication of JP2025041652A publication Critical patent/JP2025041652A/ja
Publication of JP2025041652A5 publication Critical patent/JP2025041652A5/ja
Pending legal-status Critical Current

Links

JP2024209791A 2018-12-06 2024-12-02 オリゴヌクレオチド組成物及びその方法 Pending JP2025041652A (ja)

Applications Claiming Priority (12)

Application Number Priority Date Filing Date Title
US201862776432P 2018-12-06 2018-12-06
US62/776,432 2018-12-06
USPCT/US2019/027109 2019-04-11
PCT/US2019/027109 WO2019200185A1 (en) 2018-04-12 2019-04-11 Oligonucleotide compositions and methods of use thereof
PCT/US2019/031672 WO2019217784A1 (en) 2018-05-11 2019-05-10 Oligonucleotide compositions and methods of use thereof
USPCT/US2019/031672 2019-05-10
US201962916192P 2019-10-16 2019-10-16
US201962916194P 2019-10-16 2019-10-16
US62/916,194 2019-10-16
US62/916,192 2019-10-16
PCT/US2019/065058 WO2020118246A1 (en) 2018-12-06 2019-12-06 Oligonucleotide compositions and methods thereof
JP2021531966A JP2022513719A (ja) 2018-12-06 2019-12-06 オリゴヌクレオチド組成物及びその方法

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
JP2021531966A Division JP2022513719A (ja) 2018-12-06 2019-12-06 オリゴヌクレオチド組成物及びその方法

Publications (2)

Publication Number Publication Date
JP2025041652A JP2025041652A (ja) 2025-03-26
JP2025041652A5 true JP2025041652A5 (https=) 2025-10-17

Family

ID=70973999

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021531966A Ceased JP2022513719A (ja) 2018-12-06 2019-12-06 オリゴヌクレオチド組成物及びその方法
JP2024209791A Pending JP2025041652A (ja) 2018-12-06 2024-12-02 オリゴヌクレオチド組成物及びその方法

Family Applications Before (1)

Application Number Title Priority Date Filing Date
JP2021531966A Ceased JP2022513719A (ja) 2018-12-06 2019-12-06 オリゴヌクレオチド組成物及びその方法

Country Status (8)

Country Link
US (1) US20220186217A1 (https=)
EP (1) EP3891284A4 (https=)
JP (2) JP2022513719A (https=)
CN (1) CN113383078A (https=)
AU (1) AU2019392928A1 (https=)
CA (1) CA3122271A1 (https=)
SG (1) SG11202105626TA (https=)
WO (1) WO2020118246A1 (https=)

Families Citing this family (55)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11201500239VA (en) 2012-07-13 2015-03-30 Wave Life Sciences Japan Asymmetric auxiliary group
JP6453212B2 (ja) 2012-07-13 2019-01-16 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． キラル制御
ES2917473T3 (es) 2014-01-16 2022-07-08 Wave Life Sciences Ltd Diseño quiral
RU2708237C2 (ru) 2014-08-22 2019-12-05 Общество с ограниченной ответственностью "НооГен" Модифицированные олигонуклеотиды и способ их получения
CN108135921B (zh) 2015-07-22 2023-10-17 波涛生命科学有限公司 寡核苷酸组合物及其方法
CA3015823A1 (en) 2016-03-13 2017-09-21 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis
CN109562122A (zh) 2016-06-03 2019-04-02 波涛生命科学有限公司 寡核苷酸、组合物及其方法
EP3475424A1 (en) 2016-06-22 2019-05-01 ProQR Therapeutics II B.V. Single-stranded rna-editing oligonucleotides
US10941402B2 (en) 2016-09-01 2021-03-09 Proqr Therapeutics Ii B.V. Chemically modified single-stranded RNA-editing oligonucleotides
MA46905A (fr) 2016-11-23 2019-10-02 Wave Life Sciences Ltd Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides
US11603532B2 (en) 2017-06-02 2023-03-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
US11597927B2 (en) 2017-06-02 2023-03-07 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
US11718638B2 (en) 2017-06-21 2023-08-08 Wave Life Sciences Ltd. Compounds, compositions and methods for synthesis
TW201920672A (zh) 2017-08-08 2019-06-01 新加坡商波濤生命科學有限公司 寡核苷酸組合物及其使用方法
CN111108096A (zh) 2017-09-18 2020-05-05 波涛生命科学有限公司 寡核苷酸制备技术
EP3694530A4 (en) 2017-10-12 2021-06-30 Wave Life Sciences Ltd. COMPOSITIONS OF OLIGONUCLEOTIDES AND RELATED PROCESSES
IL277889B2 (en) 2018-04-12 2025-01-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods of using them
EP3790596A4 (en) 2018-05-11 2022-04-06 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
EP3829595A4 (en) 2018-08-02 2022-08-24 Dyne Therapeutics, Inc. MUSCLE-DIRECTED COMPLEXES AND USES THEREOF IN THE TREATMENT OF DYSTROPHINOPATHIES
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
CN113748116A (zh) * 2019-03-20 2021-12-03 波涛生命科学有限公司 可用于寡核苷酸制备的技术
WO2021113270A1 (en) 2019-12-02 2021-06-10 Shape Therapeutics Inc. Therapeutic editing
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
EP4476202A1 (en) * 2022-02-11 2024-12-18 Wave Life Sciences Ltd. Stereoselective technologies for chiral compounds
US20250154504A1 (en) 2022-02-14 2025-05-15 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
AR128918A1 (es) * 2022-03-30 2024-06-26 Biomarin Pharm Inc Oligonucleótidos que omiten el exón 51 de distrofina y sus aplicaciones
EP4555086A1 (en) 2022-07-15 2025-05-21 ProQR Therapeutics II B.V. Oligonucleotides for adar-mediated rna editing and use thereof
WO2024013360A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Chemically modified oligonucleotides for adar-mediated rna editing
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes
AR131145A1 (es) 2022-11-24 2025-02-19 Proqr Therapeutics Ii Bv Oligonucleótidos antisentido para el tratamiento de hemocromatosis hfe hereditaria
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
CA3276262A1 (en) 2022-12-09 2024-06-13 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of cardiovascular disease
GB202300865D0 (en) 2023-01-20 2023-03-08 Proqr Therapeutics Ii Bv Delivery of oligonucleotides
EP4669753A1 (en) 2023-02-20 2025-12-31 ProQR Therapeutics II B.V. Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease
GB202304363D0 (en) 2023-03-24 2023-05-10 Proqr Therapeutics Ii Bv Chemically modified antisense oligonucleotides for use in RNA editing
WO2024206175A1 (en) 2023-03-24 2024-10-03 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurological disorders
AU2024246572A1 (en) 2023-03-27 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of liver disease
WO2024256620A1 (en) 2023-06-16 2024-12-19 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurodegenerative disease
WO2025051946A1 (en) 2023-09-07 2025-03-13 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of metabolic disorders
WO2025072879A1 (en) * 2023-09-28 2025-04-03 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
WO2025072881A2 (en) * 2023-09-28 2025-04-03 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
WO2025104239A1 (en) 2023-11-16 2025-05-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of classic galactosemia
WO2025132708A1 (en) 2023-12-20 2025-06-26 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of huntington's disease
GB202404661D0 (en) 2024-04-02 2024-05-15 Proqr Therapeutics Ii Bv Antisense oligoncleotides for the treatment of liver disease
WO2025213142A2 (en) * 2024-04-05 2025-10-09 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
WO2025224230A1 (en) 2024-04-25 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of fatty liver disease
GB202410081D0 (en) 2024-07-11 2024-08-28 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of cardiovascular disease
WO2026022136A1 (en) 2024-07-23 2026-01-29 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of metabolic disorders
WO2026060374A2 (en) 2024-09-16 2026-03-19 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurological disorders
WO2026068781A1 (en) 2024-09-30 2026-04-02 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of liver disease
WO2026080323A1 (en) 2024-10-09 2026-04-16 Quralis Corporation Treatment of neurological diseases using modulators of unc13a gene transcripts

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6617442B1 (en) * 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
AU2002362312A1 (en) * 2001-09-17 2003-04-01 Mayo Foundation For Medical Education And Research Cobalamin mediated delivery of nucleic acids, analogs and derivatives thereof
AU2008317566B2 (en) * 2007-10-26 2014-05-01 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
JP5786109B2 (ja) * 2008-10-27 2015-09-30 プロセンサ テクノロジーズ ベー．フェー． Ｄｕｃｈｅｎｎｅ型筋ジストロフィーｍＲＮＡ前駆体のエクソン４５の効率的なスキッピングのための方法及び手段
WO2011109427A2 (en) * 2010-03-01 2011-09-09 Alnylam Pharmaceuticals, Inc. Improving the biological activity of sirna through modulation of its thermodynamic profile
JP6453212B2 (ja) * 2012-07-13 2019-01-16 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． キラル制御
ES2917473T3 (es) * 2014-01-16 2022-07-08 Wave Life Sciences Ltd Diseño quiral
SMT201900169T1 (it) * 2014-03-12 2019-05-10 Nippon Shinyaku Co Ltd Acido nucleico antisenso
RU2708237C2 (ru) * 2014-08-22 2019-12-05 Общество с ограниченной ответственностью "НооГен" Модифицированные олигонуклеотиды и способ их получения
EP3858993A1 (en) * 2015-10-09 2021-08-04 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
JP7511326B2 (ja) * 2015-10-09 2024-07-05 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド組成物およびその方法
CA3029772A1 (en) * 2016-07-05 2018-01-11 Biomarin Technologies B.V. Pre-mrna splice switching or modulating oligonucleotides comprising bicyclic scaffold moieties, with improved characteristics for the treatment of genetic disorders

Similar Documents

Publication Publication Date Title
JP2025041652A5 (https=)
JPWO2019217784A5 (https=)
JPWO2020118246A5 (https=)
JP2023145620A5 (https=)
JP2024056718A5 (https=)
Agrawal et al. Modified oligonucleotides as therapeutic and diagnostic agents
JP7792371B2 (ja) ＧａｌＮＡｃ誘導体
JP2024099745A5 (https=)
JP2020522265A5 (https=)
JP2023078293A5 (https=)
JPWO2021071788A5 (https=)
JP2021521140A5 (https=)
JP2018525357A5 (https=)
JP2020524485A5 (https=)
JPWO2022270585A5 (https=)
JPWO2020227691A5 (https=)
JPWO2020160336A5 (https=)
JP2019525918A5 (https=)
JP5995855B2 (ja) 塩基修飾オリゴヌクレオチド
JP2021523720A5 (https=)
FI4426270T3 (fi) Lipidi-nanopartikkelit oligonukleotidien kuljetusta varten
JP2022501024A5 (https=)
JPWO2019215333A5 (https=)
JPWO2023014938A5 (https=)
JP2022180420A5 (https=)