RU2020105929A - Способы и композиции для модулирования сплайсинга - Google Patents

Способы и композиции для модулирования сплайсинга Download PDF

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RU2020105929A
RU2020105929A RU2020105929A RU2020105929A RU2020105929A RU 2020105929 A RU2020105929 A RU 2020105929A RU 2020105929 A RU2020105929 A RU 2020105929A RU 2020105929 A RU2020105929 A RU 2020105929A RU 2020105929 A RU2020105929 A RU 2020105929A
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substituted
unsubstituted
alkyl
heteroaryl
compound according
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RU2020105929A
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Russian (ru)
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Майкл Луццио
Кэтлин МАККАРТИ
Уилльям ХЭЙНИ
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Скайхоук Терапьютикс, Инк.
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/50—Pyridazines; Hydrogenated pyridazines
    • A61K31/501—Pyridazines; Hydrogenated pyridazines not condensed and containing further heterocyclic rings
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    • A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
    • A61K31/5375—1,4-Oxazines, e.g. morpholine
    • A61K31/5383—1,4-Oxazines, e.g. morpholine ortho- or peri-condensed with heterocyclic ring systems
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    • C07D451/02—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing not further condensed 8-azabicyclo [3.2.1] octane or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane; Cyclic acetals thereof
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    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D451/00—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof
    • C07D451/02—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing not further condensed 8-azabicyclo [3.2.1] octane or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane; Cyclic acetals thereof
    • C07D451/04—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing not further condensed 8-azabicyclo [3.2.1] octane or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane; Cyclic acetals thereof with hetero atoms directly attached in position 3 of the 8-azabicyclo [3.2.1] octane or in position 7 of the 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring system
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    • C07D451/02—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing not further condensed 8-azabicyclo [3.2.1] octane or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane; Cyclic acetals thereof
    • C07D451/04—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing not further condensed 8-azabicyclo [3.2.1] octane or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane; Cyclic acetals thereof with hetero atoms directly attached in position 3 of the 8-azabicyclo [3.2.1] octane or in position 7 of the 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring system
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    • C07D451/14—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing 9-azabicyclo [3.3.1] nonane ring systems, e.g. granatane, 2-aza-adamantane; Cyclic acetals thereof
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    • C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
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    • C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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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RU2020105929A 2017-08-04 2018-08-03 Способы и композиции для модулирования сплайсинга RU2020105929A (ru)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762541202P 2017-08-04 2017-08-04
US62/541,202 2017-08-04
US201762562948P 2017-09-25 2017-09-25
US201762562927P 2017-09-25 2017-09-25
US62/562,927 2017-09-25
US62/562,948 2017-09-25
PCT/US2018/045282 WO2019028440A1 (en) 2017-08-04 2018-08-03 METHODS AND COMPOSITIONS FOR MODULATING SPLICING

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RU2020105929A true RU2020105929A (ru) 2021-09-06

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US (8) US11008572B2 (enExample)
EP (2) EP3661509A4 (enExample)
JP (2) JP7312749B2 (enExample)
KR (2) KR102636383B1 (enExample)
CN (2) CN111499615B (enExample)
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IL (2) IL273741B (enExample)
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MY (1) MY201938A (enExample)
RU (1) RU2020105929A (enExample)
SG (2) SG11202000980QA (enExample)
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Families Citing this family (75)

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Publication number Priority date Publication date Assignee Title
KR20220100719A (ko) 2015-12-10 2022-07-15 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료 또는 개선을 위한 조성물
CA3043755A1 (en) 2016-11-28 2018-05-31 Ptc Therapeutics, Inc. Methods for modulating rna splicing
AU2018282154B2 (en) 2017-06-05 2022-04-07 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
KR20200017476A (ko) 2017-06-14 2020-02-18 피티씨 테라퓨틱스, 인크. Rna 스플라이싱의 변경 방법
WO2019005993A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. METHODS OF TREATING HUNTINGTON'S DISEASE
CA3067591A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. Methods for treating huntington's disease
KR102636383B1 (ko) 2017-08-04 2024-02-14 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
AU2019243048A1 (en) * 2018-03-27 2020-10-15 Ptc Therapeutics, Inc. Compounds for treating Huntington's disease
KR20200142039A (ko) 2018-04-10 2020-12-21 스카이호크 테라퓨틱스, 인코포레이티드 암 치료용 화합물
WO2020005882A1 (en) 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
BR112020026534A2 (pt) 2018-06-27 2021-03-23 Ptc Therapeutics, Inc. Compostos de heteroarila para o tratamento da doença de huntington
IL279688B2 (en) 2018-06-27 2025-01-01 Ptc Therapeutics Inc Heterocyclic and heteroaryl compounds for the treatment of Huntington's disease
CA3110211A1 (en) 2018-08-20 2020-02-27 Rogcon, Inc. Antisense oligonucleotides targeting scn2a for the treatment of scn1a encephalopathies
US20220090087A1 (en) * 2019-01-23 2022-03-24 The Florey Institute Of Neuroscience And Mental Health Antisense oligonucleotides targeting scn2a retained introns
CN119320388A (zh) 2019-02-04 2025-01-17 斯基霍克疗法公司 用于调节剪接的方法和组合物
JP2022519637A (ja) * 2019-02-04 2022-03-24 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物
EP3920919A4 (en) * 2019-02-05 2023-03-29 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICE
KR102913697B1 (ko) * 2019-02-05 2026-01-15 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
EP3920918A4 (en) * 2019-02-05 2022-11-16 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATION OF SPLICING
CN113677342A (zh) * 2019-02-05 2021-11-19 斯基霍克疗法公司 用于调节剪接的方法和组合物
JP7551629B2 (ja) * 2019-02-05 2024-09-17 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物
JP7603594B2 (ja) * 2019-02-06 2024-12-20 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物
WO2020163544A1 (en) * 2019-02-06 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
JP2022525417A (ja) * 2019-03-15 2022-05-13 スカイホーク・セラピューティクス・インコーポレーテッド 異常スプライシングを修正するための組成物および方法
WO2020210537A1 (en) 2019-04-09 2020-10-15 Envisagenics, Inc. Cancer-specific molecules and methods of use thereof
CN109988163A (zh) * 2019-04-15 2019-07-09 宁夏医科大学 一种异丙基托品醇异构体的制备分离方法
CN114245794B (zh) 2019-05-13 2024-09-13 Ptc医疗公司 用于治疗亨廷顿氏病的化合物
UY38687A (es) 2019-05-17 2023-05-15 Novartis Ag Inhibidores del inflamasoma nlrp3, composiciones, combinaciones de los mismos y métodos para su uso
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
AU2020319168B2 (en) 2019-07-25 2024-02-29 Novartis Ag Regulatable expression systems
WO2021071982A1 (en) * 2019-10-08 2021-04-15 Skyhawk Therapeutics, Inc. Compounds for modulating splicing
EP4051280A1 (en) 2019-11-01 2022-09-07 Novartis AG The use of a splicing modulator for a treatment slowing progression of huntington's disease
US20230148184A1 (en) * 2020-02-28 2023-05-11 Remix Therapeutics Inc. Compounds and methods for modulating splicing
JP2023515618A (ja) 2020-02-28 2023-04-13 リミックス セラピューティクス インコーポレイテッド 核酸スプライシングを調節するためのピリダジン誘導体
US20240226098A1 (en) * 2020-02-28 2024-07-11 Remix Therapeutics Inc. Compounds and methods for modulating splicing
CA3169697A1 (en) 2020-02-28 2021-09-02 Dominic Reynolds Thiophenyl derivatives useful for modulating nucleic acid splicing
US20230167093A1 (en) 2020-04-08 2023-06-01 Remix Therapeutics Inc. Compounds and methods for modulating splicing
KR20230004575A (ko) 2020-04-08 2023-01-06 레믹스 테라퓨틱스 인크. 스플라이싱을 조절하기 위한 화합물 및 방법
CA3182912A1 (en) 2020-05-13 2021-11-18 Chdi Foundation, Inc. Htt modulators for treating huntington's disease
US20240239811A1 (en) 2020-07-02 2024-07-18 Remix Therapeutics Inc. 5-(5-(piperidin-4-yl)thieno[3,2-c]pyrazol-2-yl]indazole derivatives and related compounds as modulators for splicing nucleic acids and for the t
EP4175956A1 (en) 2020-07-02 2023-05-10 Remix Therapeutics Inc. 2-(indazol-5-yl)-6-(piperidin-4-yl)-1,7-naphthyridine derivatives and related compounds as modulators for splicing nucleic acids and for the treatment of proliferative diseases
WO2022031998A2 (en) * 2020-08-05 2022-02-10 Skyhawk Therapeutics, Inc. Process for the preparation of intermediates useful in the preparation of compounds that modulate splicing
WO2022032007A2 (en) * 2020-08-05 2022-02-10 Skyhawk Therapeutics, Inc. Process for the preparation of intermediates useful in the preparation of compounds that modulate splicing
EP4192454B1 (en) * 2020-08-05 2026-04-22 Skyhawk Therapeutics, Inc. Azabicyclo-amino-triazine compounds for modulating splicing for the treatment of neurological diseases
CN114113621A (zh) * 2020-08-25 2022-03-01 张曼 尿液鸟嘌呤核苷酸结合蛋白亚基α-13及其多肽片段在烧伤中的应用
WO2022060944A1 (en) * 2020-09-16 2022-03-24 Skyhawk Therapeutics, Inc. Compositions for modulating splicing
WO2022060943A1 (en) * 2020-09-16 2022-03-24 Skyhawk Therapeutics, Inc. Compositions for modulating splicing
WO2022060951A1 (en) * 2020-09-16 2022-03-24 Skyhawk Therapeutics, Inc. Compositions for modulating splicing
US20240018522A1 (en) * 2020-10-26 2024-01-18 Remix Therapeutics Inc. Oligonucleotides useful for modulation of splicing
WO2022109022A1 (en) * 2020-11-17 2022-05-27 Skyhawk Therapeutics, Inc. Rna-targeting splicing modifiers for treatment of mda5-associated conditions and diseases
BR112023025318A2 (pt) * 2021-06-04 2024-02-27 Hoffmann La Roche Derivados de triazina e seu uso no tratamento do câncer
US20240368163A1 (en) 2021-08-30 2024-11-07 Remix Therapeutics Inc. Compounds and methods for modulating splicing
MX2024002554A (es) 2021-08-30 2024-07-02 Remix Therapeutics Inc Compuestos y metodos para modular el empalme.
CR20240112A (es) 2021-08-30 2024-08-07 Remix Therapeutics Inc Compuestos y métodos para modular splicing
EP4395890A1 (en) 2021-08-30 2024-07-10 Remix Therapeutics Inc. Compounds and methods for modulating splicing
EP4395889A1 (en) 2021-08-30 2024-07-10 Remix Therapeutics Inc. Compounds and methods for modulating splicing
MX2024003519A (es) 2021-09-24 2024-04-01 Alnylam Pharmaceuticals Inc Composiciones de agentes de acido ribonucleico de interferencia (arni) de proteina tau asociada a microtubulos (mapt) y sus metodos de uso.
TW202330552A (zh) 2021-10-13 2023-08-01 美商雷密克斯醫療公司 調節剪接之化合物及方法
WO2023064880A1 (en) 2021-10-13 2023-04-20 Remix Therapeutics Inc. Compounds and methods for modulating nucleic acid splicing
WO2023091456A1 (en) 2021-11-17 2023-05-25 Chdi Foundation, Inc. N-(2h-indazol-5-yl)pyrazine-2-carboxamide derivatives and similar compounds as htt modulators for the treatment of huntington's disease
IL312843A (en) * 2021-11-18 2024-07-01 Rgenta Therapeutics Inc Heterocyclic amides and methods of using the same
CN114225051B (zh) * 2021-12-16 2024-05-10 中国人民解放军空军军医大学 一种用于治疗银屑病的药物及其应用
WO2023133229A2 (en) 2022-01-05 2023-07-13 Remix Therapeutics Inc. Compounds and methods for modulating splicing
TW202346305A (zh) 2022-01-05 2023-12-01 美商雷密克斯醫療公司 用於調節剪切之化合物及方法
US20250092065A1 (en) 2022-01-05 2025-03-20 Remix Therapeutics Inc. 2-(indazol-5-yl)-6-(piperidin-4-yl)-1,7-naphthyridine derivatives and related compounds as modulators for splicing nucleic acids and for the treatment of proliferative diseases
CN114350813B (zh) * 2022-03-17 2022-08-02 广州达安临床检验中心有限公司 Tmem236基因在制备用于诊断直肠癌新辅助放疗抵抗标记物中的用途
CN114592006A (zh) * 2022-04-29 2022-06-07 昆明理工大学 Memo1基因的新用途
KR102470669B1 (ko) * 2022-06-03 2022-11-25 충남대학교 산학협력단 스플라이싱-스위치 올리고뉴클레오티드를 유효성분으로 포함하는 폴리프로모 1 유전자의 스플라이싱 조절용 조성물
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
UY40374A (es) 2022-08-03 2024-02-15 Novartis Ag Inhibidores de inflamasoma nlrp3
TW202432551A (zh) * 2022-10-26 2024-08-16 日商安斯泰來製藥股份有限公司 稠環嗒嗪衍生物
US20240150759A1 (en) * 2022-11-04 2024-05-09 The Board Of Trustees Of The Leland Stanford Junior University Splicing Modulators for the Treatment of Timothy Syndrome
CN121099993A (zh) * 2023-02-15 2025-12-09 海鞘治疗公司 Htt反式剪接分子
WO2025247338A1 (zh) * 2024-05-30 2025-12-04 纽欧申医药(上海)有限公司 哒嗪环类化合物、其药物组合物及其应用
CN119569728B (zh) * 2024-12-02 2025-09-30 重庆飞因科生物科技有限公司 吡啶并吡咯类衍生物及其在制备富亮氨酸重复激酶2抑制剂中的应用

Family Cites Families (133)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4873192A (en) 1987-02-17 1989-10-10 The United States Of America As Represented By The Department Of Health And Human Services Process for site specific mutagenesis without phenotypic selection
NZ227684A (en) 1988-01-30 1991-07-26 Merck Sharp & Dohme Pyrazine, pyridazine, and pyrimidine derivatives and pharmaceutical compositions
GB9005737D0 (en) 1990-03-14 1990-05-09 Beecham Group Plc Novel compounds
FR2676444B1 (fr) 1991-05-16 1995-03-10 Sanofi Elf Nouveaux derives d'amino-3 pyridazines actifs sur le systeme nerveux central, procede de preparation et compositions pharmaceutiques en contenant.
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
US20030105320A1 (en) 2001-08-31 2003-06-05 Becker Michael M. Affinity-shifted probes for quantifying analyte polynucleotides
US7135484B2 (en) 2002-08-14 2006-11-14 Abbott Laboratories Azabicyclic compounds are central nervous system active agents
ATE353899T1 (de) 2002-09-30 2007-03-15 Neurosearch As Neue 1,4-diazabicycloalkanderivate, deren herstellung und verwendung
US8014953B2 (en) 2003-08-08 2011-09-06 The Regents Of The University Of California RNA surveillance among curated proteins
US7399765B2 (en) 2003-09-19 2008-07-15 Abbott Laboratories Substituted diazabicycloalkane derivatives
US7655657B2 (en) 2003-12-22 2010-02-02 Abbott Laboratories Fused bicycloheterocycle substituted quinuclidine derivatives
US7160876B2 (en) 2003-12-22 2007-01-09 Abbott Laboratories Fused bicycloheterocycle substituted quinuclidine derivatives
US7309699B2 (en) 2003-12-22 2007-12-18 Abbott Laboratories 3-Quinuclidinyl amino-substituted biaryl derivatives
US7309487B2 (en) 2004-02-09 2007-12-18 George Inana Methods and compositions for detecting and treating retinal diseases
PE20060437A1 (es) 2004-06-18 2006-06-08 Novartis Ag COMPUESTOS AZA-BICICLONONANOS COMO LIGANDOS COLINERGICOS DE nAChR
GB0415746D0 (en) 2004-07-14 2004-08-18 Novartis Ag Organic compounds
CN101223166B (zh) 2004-10-07 2011-10-19 默沙东公司 噻唑基mglur5拮抗剂及其应用方法
CN101389335A (zh) 2004-10-18 2009-03-18 安姆根有限公司 噻二唑化合物和使用方法
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
CN101146796A (zh) 2005-01-26 2008-03-19 先灵公司 作为蛋白激酶抑制剂用于治疗癌症的3-(吲唑-5-基)-(1,2,4)三嗪衍生物及相关化合物
AR053712A1 (es) 2005-04-18 2007-05-16 Neurogen Corp Heteroarilos sustituidos, antagonistas de cb1 (receptor 1 canabinoide)
US7598052B2 (en) 2005-10-11 2009-10-06 The Regents Of The University Of Michigan Expression profile of thyroid cancer
US8603457B2 (en) 2005-12-02 2013-12-10 University Of Rochester Nonsense suppression and genetic codon alteration by targeted modification
US7834178B2 (en) 2006-03-01 2010-11-16 Bristol-Myers Squibb Company Triazine 11-beta hydroxysteroid dehydrogenase type 1 inhibitors
RS52062B (sr) 2006-05-19 2012-06-30 Abbott Laboratories Cns aktivni azabiciklični alkanski derivati koji su supstituisani spojenim bicikloheterociklom
ATE463497T1 (de) 2006-05-30 2010-04-15 Neurosearch As Neues 1,4-diaza-bicycloä3.2.2ünonyl-oxadiazolyl- derivat und seine medizinische verwendung
US8314119B2 (en) 2006-11-06 2012-11-20 Abbvie Inc. Azaadamantane derivatives and methods of use
US8343941B2 (en) 2007-03-30 2013-01-01 Rutgers, The State University Of New Jersey Compositions and methods for gene silencing
US8283116B1 (en) 2007-06-22 2012-10-09 Ptc Therapeutics, Inc. Methods of screening for compounds for treating spinal muscular atrophy using SMN mRNA translation regulation
DE102007032507A1 (de) * 2007-07-12 2009-04-02 Merck Patent Gmbh Pyridazinonderivate
EP2203173B1 (en) 2007-10-26 2015-12-23 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
US7902222B2 (en) 2007-11-21 2011-03-08 Abbott Laboratories Biaryl substituted azabicyclic alkane derivatives
JP5177219B2 (ja) 2008-03-18 2013-04-03 コニカミノルタホールディングス株式会社 電気化学的表示素子
WO2009151546A2 (en) 2008-05-27 2009-12-17 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
US8133724B2 (en) 2008-09-17 2012-03-13 University Of Maryland, Baltimore Human androgen receptor alternative splice variants as biomarkers and therapeutic targets
WO2010075376A2 (en) 2008-12-23 2010-07-01 Abbott Laboratories Anti-viral compounds
EP2408919B1 (en) 2009-03-16 2017-10-18 CuRNA, Inc. Treatment of nuclear factor (erythroid-derived 2)-like 2 (nrf2) related diseases by inhibition of natural antisense transcript to nrf2
JP2012523225A (ja) 2009-04-10 2012-10-04 アソシアシオン・アンスティテュ・ドゥ・ミオロジー 疾患の処置のためのトリシクロ−ｄｎａアンチセンスオリゴヌクレオチド、組成物及び方法
CN115227709A (zh) 2009-06-17 2022-10-25 冷泉港实验室 用于在对象中调节smn2剪接的组合物和方法
JO3250B1 (ar) 2009-09-22 2018-09-16 Novartis Ag إستعمال منشطات مستقبل نيكوتينيك أسيتيل كولين ألفا 7
EP2305679A1 (en) 2009-09-28 2011-04-06 GenKyoTex SA Pyrazoline dione derivatives as nadph oxidase inhibitors
AR079724A1 (es) * 2010-01-20 2012-02-15 Abbott Lab Metodos para el tratamiento del dolor
DK2531492T3 (en) 2010-02-05 2016-07-04 Heptares Therapeutics Ltd 1,2,4-triazin-4-amine derivatives
AR081331A1 (es) 2010-04-23 2012-08-08 Cytokinetics Inc Amino- pirimidinas composiciones de las mismas y metodos para el uso de los mismos
AR081626A1 (es) 2010-04-23 2012-10-10 Cytokinetics Inc Compuestos amino-piridazinicos, composiciones farmaceuticas que los contienen y uso de los mismos para tratar trastornos musculares cardiacos y esqueleticos
EP2560488B1 (en) 2010-04-23 2015-10-28 Cytokinetics, Inc. Certain amino-pyridines and amino-triazines, compositions thereof, and methods for their use
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
EP2585455B1 (en) 2010-06-24 2019-08-07 The Regents of the University of California Compounds and uses thereof in modulating levels of various amyloid beta peptide alloforms
KR101764952B1 (ko) 2010-07-29 2017-08-03 리겔 파마슈티칼스, 인크. Ａｍｐｋ-활성화 헤테로시클릭 화합물 및 그의 사용 방법
JP6625792B2 (ja) 2011-03-14 2019-12-25 エヌエスイー プロダクツ インコーポレイテッド 細胞浄化を促進するための経口製剤
WO2012129562A2 (en) 2011-03-24 2012-09-27 The Scripps Research Institute Compounds and methods for inducing chondrogenesis
US9447075B2 (en) 2011-08-02 2016-09-20 The Brigham And Women's Hospital, Inc. Pyridazine derivatives as EAAT2 activators
EP2771069A4 (en) 2011-10-27 2015-08-26 Mayo Foundation INHIBITION OF G-PROTEIN-COUPLED RECEPTOR-6-KINASE POLYPEPTIDE
JP6193881B2 (ja) 2011-12-30 2017-09-06 ピーティーシー セラピューティクス， インコーポレイテッド 脊髄性筋萎縮症を治療するための化合物
CA2862084C (en) 2012-01-26 2021-05-11 Ptc Therapeutics, Inc. 1h-isochromen-1-one derivatives and compositions thereof for treating spinal muscular atrophy
WO2013117693A1 (en) 2012-02-10 2013-08-15 Glaxosmithkline Intellectual Property Development Limited Pde4 inhibitor for treating huntington's disease
ES2697174T3 (es) 2012-02-10 2019-01-22 Ptc Therapeutics Inc Compuestos para tratar atrofia muscular espinal
KR102099997B1 (ko) 2012-03-01 2020-04-13 피티씨 테라퓨틱스, 인크. 척수성 근위축증을 치료하기 위한 화합물
US9914722B2 (en) 2012-03-23 2018-03-13 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
US8729263B2 (en) * 2012-08-13 2014-05-20 Novartis Ag 1,4-disubstituted pyridazine analogs there of and methods for treating SMN-deficiency-related conditions
AU2013204200B2 (en) 2012-10-11 2016-10-20 Brandeis University Treatment of amyotrophic lateral sclerosis
US9963699B2 (en) 2012-10-15 2018-05-08 Ionis Pharmaceuticals, Inc. Methods for modulating C9ORF72 expression
KR101879919B1 (ko) * 2013-01-15 2018-07-18 노파르티스 아게 알파 7 니코틴성 아세틸콜린 수용체 작용물질의 용도
CA2898043C (en) * 2013-01-15 2019-08-06 Novartis Ag Use of alpha 7 nicotinic receptor agonists for the treatment of narcolepsy
EP2945633B1 (en) * 2013-01-15 2021-06-30 Novartis AG Use of alpha 7 nicotinic acetylcholine receptor agonists
ITMI20130063A1 (it) * 2013-01-17 2014-07-18 Valier Alberto Dispositivo manuale per la raccolta dal suolo di frutti o altri oggetti
US9040712B2 (en) 2013-01-23 2015-05-26 Novartis Ag Thiadiazole analogs thereof and methods for treating SMN-deficiency-related-conditions
AR096684A1 (es) 2013-06-25 2016-01-27 Ptc Therapeutics Inc Compuestos para tratar atrofia muscular espinal
KR102318727B1 (ko) 2013-07-31 2021-10-28 노파르티스 아게 1,4-이치환된 피리다진 유사체 및 smn-결핍-관련 상태를 치료하기 위한 그의 용도
CN105392790B (zh) 2013-08-19 2019-04-19 豪夫迈·罗氏有限公司 4H-吡啶并[1,2-a]嘧啶-4-酮化合物制备用于预防或治疗癌症的药物的用途
CA2922567A1 (en) 2013-08-26 2015-03-05 Purdue Pharma L.P. Azaspiro[4.5]decane derivatives and use thereof
EP3052654A4 (en) 2013-09-30 2017-05-03 The Regents of the University of California Identification of structurally similar small molecules that enhance therapeutic exon skipping
US11162096B2 (en) 2013-10-14 2021-11-02 Ionis Pharmaceuticals, Inc Methods for modulating expression of C9ORF72 antisense transcript
WO2015136947A1 (en) 2014-03-14 2015-09-17 Raqualia Pharma Inc. Azaspiro derivatives as trpm8 antagonists
WO2015143245A1 (en) 2014-03-19 2015-09-24 Isis Pharmaceuticals, Inc. Methods for modulating ataxin 2 expression
US10724092B2 (en) 2014-06-10 2020-07-28 Erasmus University Medical Center Rotterdam Methods for characterizing alternatively or aberrantly spliced mRNA isoforms
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
WO2016027168A2 (en) * 2014-08-20 2016-02-25 Lifesplice Pharma Llc Splice modulating oligonucleotides and methods of use thereof
US20170355956A1 (en) 2014-09-04 2017-12-14 Rutgers, The State University Of New Jersey Compositions for increasing survival of motor neuron protein (smn) levels in target cells and methods of use thereof for the treatment of spinal muscular atrophy
WO2016064895A1 (en) 2014-10-20 2016-04-28 The Brigham And Women's Hospital, Inc. Targeting apolipoprotein e (apoe) in neurologic disease
US10053697B1 (en) 2015-01-09 2018-08-21 The Board Of Trustees Of The Leland Stanford Junior University Programmable alternative splicing devices and uses thereof
EP3247701B1 (en) 2015-01-20 2021-09-22 Boehringer Ingelheim Animal Health USA Inc. Anthelmintic compounds, compositions and method of using thereof
EP3053577A1 (en) 2015-02-09 2016-08-10 F. Hoffmann-La Roche AG Compounds for the treatment of cancer
WO2016128343A1 (en) 2015-02-09 2016-08-18 F. Hoffmann-La Roche Ag Compounds for the treatment of cancer
US10668171B2 (en) * 2015-05-30 2020-06-02 Ptc Therapeutics, Inc. Methods for modulating RNA splicing
CN107922396B (zh) 2015-07-20 2022-08-05 建新公司 集落刺激因子-1受体(csf-1r)抑制剂
US20180271803A1 (en) 2015-09-02 2018-09-27 Pathways Bioscience, Llc Compositions and methods for treatment or prevention of oral mucositis
RU2018112749A (ru) 2015-09-24 2019-10-24 ГлаксоСмитКлайн Интеллекчуал Проперти (N 2) Лимитед Модуляторы индоламин-2,3-диоксигеназы
US11413278B2 (en) 2015-10-08 2022-08-16 The Regents Of The University Of California Compounds and methods for promoting stress resistance
AU2016334804B2 (en) 2015-10-09 2022-03-31 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
AU2016353961B2 (en) 2015-11-12 2019-08-29 F. Hoffmann-La Roche Ag Compositions for treating spinal muscular atrophy
US20180344680A1 (en) 2015-12-08 2018-12-06 The Arizona Board Of Regents On Behalf Of The University Of Arizona Compositions and methods for treatment, amelioration, and prevention of diabetes-related skin ulcers
KR20220100719A (ko) 2015-12-10 2022-07-15 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료 또는 개선을 위한 조성물
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
WO2017112954A1 (en) 2015-12-23 2017-06-29 Moonshot Pharma Llc Methods for inducing an immune response by promoting premature termination codon read-through
WO2017123991A1 (en) 2016-01-15 2017-07-20 The Brigham And Women's Hospital, Inc. Pyridazine derivatives as eaat2 activators
JP2019511562A (ja) 2016-02-01 2019-04-25 アラーキス セラピューティクス， インコーポレイテッド Ｒｎａ媒介疾患を処置する化合物および方法
US9895358B2 (en) 2016-02-23 2018-02-20 Indiana University Research And Technology Corporation Combination therapies for treatment of spinal muscular atrophy
US11674952B2 (en) 2016-02-24 2023-06-13 The Rockefeller University Embryonic cell-based therapeutic candidate screening systems, models for Huntington's Disease and uses thereof
US11066456B2 (en) 2016-02-25 2021-07-20 Washington University Compositions comprising TREM2 and methods of use thereof
US20170268066A1 (en) 2016-03-15 2017-09-21 Chalmers Ventures Ab Cancer biomarkers
CA3019240A1 (en) 2016-03-29 2017-10-05 Merck Patent Gmbh N1-(3,3,3-trifluoro-2-hydroxy-2-methylpropionyl)-piperidine derivatives as inhibitors of pyruvate dehydrogenase kinase
US10940161B2 (en) 2016-04-04 2021-03-09 University Of Florida Research Foundation, Incorporated Manipulation of EIF3 to modulate repeat associated non-ATG (RAN) translation
EP3442960A1 (en) 2016-04-12 2019-02-20 Esteve Pharmaceuticals, S.A. Arylamide derivatives having multimodal activity against pain
KR20210082548A (ko) 2016-06-13 2021-07-05 스칼러 락, 인크. 미오스타틴 억제제의 용도 및 조합 요법
CN109563515A (zh) * 2016-07-01 2019-04-02 阿拉基斯医疗公司 化合物和调节rna功能的方法
CA3043755A1 (en) 2016-11-28 2018-05-31 Ptc Therapeutics, Inc. Methods for modulating rna splicing
EP3583951B1 (en) 2017-02-20 2026-03-25 Kyoto University Pharmaceutical composition and treatment method for genetic disease associated with splicing abnormalities
US11715549B2 (en) 2017-05-01 2023-08-01 Thomas Jefferson University Systems-level analysis of 32 TCGA cancers reveals disease-dependent tRNA fragmentation patterns and very selective associations with messenger RNAs and repeat elements
KR20200017476A (ko) * 2017-06-14 2020-02-18 피티씨 테라퓨틱스, 인크. Rna 스플라이싱의 변경 방법
WO2019005993A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. METHODS OF TREATING HUNTINGTON'S DISEASE
CA3067591A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. Methods for treating huntington's disease
KR102636383B1 (ko) 2017-08-04 2024-02-14 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
US11266640B2 (en) 2017-09-20 2022-03-08 Hangzhou Innogate Pharma Co., Ltd. Polycyclic compound acting as IDO inhibitor and/or IDO-HDAC dual inhibitor
SG11202002941WA (en) 2017-10-12 2020-04-29 Revolution Medicines Inc Pyridine, pyrazine, and triazine compounds as allosteric shp2 inhibitors
EP3716767A4 (en) 2017-10-24 2021-11-24 Sangamo Therapeutics, Inc. METHODS AND COMPOSITIONS FOR TREATMENT OF RARE DISEASES
EP3479845A1 (en) 2017-11-06 2019-05-08 Stalicla S.A. Challenge test for diagnosing subtype of autism spectrum disease
WO2019094694A1 (en) 2017-11-10 2019-05-16 University Of Massachusetts Compositions and methods for the treatment of expanded repeat-associated disorders
AU2019243048A1 (en) 2018-03-27 2020-10-15 Ptc Therapeutics, Inc. Compounds for treating Huntington's disease
KR20200142039A (ko) 2018-04-10 2020-12-21 스카이호크 테라퓨틱스, 인코포레이티드 암 치료용 화합물
WO2020005882A1 (en) 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
BR112020026534A2 (pt) 2018-06-27 2021-03-23 Ptc Therapeutics, Inc. Compostos de heteroarila para o tratamento da doença de huntington
CA3105174A1 (en) 2018-06-27 2020-01-02 Reborna Biosciences, Inc. Prophylactic or therapeutic agent for spinal muscular atrophy
IL279688B2 (en) 2018-06-27 2025-01-01 Ptc Therapeutics Inc Heterocyclic and heteroaryl compounds for the treatment of Huntington's disease
WO2020028814A1 (en) 2018-08-03 2020-02-06 H. Lee Moffitt Cancer Center And Research Institute, Inc. Allosteric inhibitor of wee1 kinase
TW202028183A (zh) 2018-10-10 2020-08-01 大陸商江蘇豪森藥業集團有限公司 含氮雜芳類衍生物調節劑、其製備方法和應用
US11129829B2 (en) * 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
CA3169697A1 (en) 2020-02-28 2021-09-02 Dominic Reynolds Thiophenyl derivatives useful for modulating nucleic acid splicing
JP2023515618A (ja) 2020-02-28 2023-04-13 リミックス セラピューティクス インコーポレイテッド 核酸スプライシングを調節するためのピリダジン誘導体
US20230148184A1 (en) 2020-02-28 2023-05-11 Remix Therapeutics Inc. Compounds and methods for modulating splicing
US20240226098A1 (en) 2020-02-28 2024-07-11 Remix Therapeutics Inc. Compounds and methods for modulating splicing
US20230167093A1 (en) 2020-04-08 2023-06-01 Remix Therapeutics Inc. Compounds and methods for modulating splicing
KR20230004575A (ko) 2020-04-08 2023-01-06 레믹스 테라퓨틱스 인크. 스플라이싱을 조절하기 위한 화합물 및 방법

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