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MX2020001425A - Metodos y composiciones para modular el empalme. - Google Patents

Metodos y composiciones para modular el empalme.

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Publication number
MX2020001425A
MX2020001425A MX2020001425A MX2020001425A MX2020001425A MX 2020001425 A MX2020001425 A MX 2020001425A MX 2020001425 A MX2020001425 A MX 2020001425A MX 2020001425 A MX2020001425 A MX 2020001425A MX 2020001425 A MX2020001425 A MX 2020001425A
Authority
MX
Mexico
Prior art keywords
methods
compositions
splicing
modulating splicing
mrna
Prior art date
Application number
MX2020001425A
Other languages
English (en)
Inventor
William Haney
Michael LUZZIO
Kathleen Mccarthy
Original Assignee
Skyhawk Therapeutics Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Skyhawk Therapeutics Inc filed Critical Skyhawk Therapeutics Inc
Publication of MX2020001425A publication Critical patent/MX2020001425A/es

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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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    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • 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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    • 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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Abstract

En este documento se describen compuestos moduladores de empalme de molécula pequeña que modulan el empalme de ARNm, tal como pre-ARNm, codificado por genes, y métodos de uso de los compuestos moduladores de empalme de molécula pequeña para modular el empalme y tratar enfermedades y afecciones.
MX2020001425A 2017-08-04 2018-08-03 Metodos y composiciones para modular el empalme. MX2020001425A (es)

Applications Claiming Priority (4)

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

Publications (1)

Publication Number Publication Date
MX2020001425A true MX2020001425A (es) 2020-08-06

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MX2020001425A MX2020001425A (es) 2017-08-04 2018-08-03 Metodos y composiciones para modular el empalme.
MX2020005748A MX2020005748A (es) 2017-08-04 2020-02-04 Metodos y composiciones para modular el empalme.

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US (8) US11162101B2 (es)
EP (2) EP3661509A4 (es)
JP (2) JP7312749B2 (es)
KR (2) KR102636383B1 (es)
CN (2) CN111499615B (es)
AU (2) AU2018309187C1 (es)
CA (1) CA3072205A1 (es)
GB (2) GB2579747B8 (es)
IL (2) IL273741B (es)
MX (2) MX2020001425A (es)
MY (1) MY201938A (es)
RU (1) RU2020105929A (es)
SG (2) SG11202000980QA (es)
WO (1) WO2019028440A1 (es)
ZA (2) ZA202000695B (es)

Families Citing this family (72)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EA201800367A1 (ru) 2015-12-10 2019-02-28 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Способы лечения болезни хантингтона
EP3544435A4 (en) 2016-11-28 2020-11-04 PTC Therapeutics, Inc. RNA SPLICE MODULATION PROCESSES
JP7376471B2 (ja) 2017-06-05 2023-11-08 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 ハンチントン病を処置するための化合物
WO2018232039A1 (en) 2017-06-14 2018-12-20 Ptc Therapeutics, Inc. Methods for modifying rna splicing
US11395822B2 (en) 2017-06-28 2022-07-26 Ptc Therapeutics, Inc. Methods for treating Huntington's disease
BR112019027717A2 (pt) 2017-06-28 2020-07-28 Ptc Therapeutics, Inc. métodos para tratar a doença de huntington
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
WO2019199972A1 (en) 2018-04-10 2019-10-17 Skyhawk Therapeutics, Inc. Compounds for the treatment of cancer
BR112020026534A2 (pt) 2018-06-27 2021-03-23 Ptc Therapeutics, Inc. Compostos de heteroarila para o tratamento da doença de huntington
WO2020005882A1 (en) 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
SI3814357T1 (sl) 2018-06-27 2024-09-30 Ptc Therapeutics, Inc. Heterociklične in heteroarilne spojine za zdravljenje Huntingtonove bolezni
WO2020041348A1 (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
CN113692402A (zh) * 2019-02-04 2021-11-23 斯基霍克疗法公司 用于调节剪接的方法和组合物
EP3920919A4 (en) * 2019-02-05 2023-03-29 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICE
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
JP7603592B2 (ja) * 2019-02-05 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
KR20210135240A (ko) * 2019-02-05 2021-11-12 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
CN114126613A (zh) * 2019-02-05 2022-03-01 斯基霍克疗法公司 用于调节剪接的方法和组合物
EP3920928A4 (en) * 2019-02-06 2022-09-28 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATION OF SPLICING
CN119638696A (zh) * 2019-02-06 2025-03-18 斯基霍克疗法公司 用于调节剪接的方法和组合物
WO2020190793A1 (en) * 2019-03-15 2020-09-24 Skyhawk Therapeutics, Inc. Compositions and methods for correction of aberrant splicing
JP7545411B2 (ja) 2019-04-09 2024-09-04 エンビサジェニックス， インコーポレイテッド がん特異的分子およびその使用の方法
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
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
WO2021014428A1 (en) 2019-07-25 2021-01-28 Novartis Ag Regulatable expression systems
WO2021071982A1 (en) * 2019-10-08 2021-04-15 Skyhawk Therapeutics, Inc. Compounds for modulating splicing
WO2021084495A1 (en) 2019-11-01 2021-05-06 Novartis Ag The use of a splicing modulator for a treatment slowing progression of huntington's disease
WO2021174163A1 (en) 2020-02-28 2021-09-02 Remix Therapeutics Inc. Fused bicyclic compounds useful for modulating nucleic acid splicing
US20230148184A1 (en) * 2020-02-28 2023-05-11 Remix Therapeutics Inc. Compounds and methods for modulating splicing
EP4110775A1 (en) 2020-02-28 2023-01-04 Remix Therapeutics Inc. Thiophenyl derivatives useful for modulating nucleic acid splicing
CA3169667A1 (en) * 2020-02-28 2021-09-02 Dominic Reynolds Pyridazine derivatives for modulating nucleic acid splicing
WO2021207530A1 (en) 2020-04-08 2021-10-14 Remix Therapeutics Inc. Compounds and methods for modulating splicing
CN115551593A (zh) 2020-04-08 2022-12-30 雷密克斯医疗公司 用于调节剪接的化合物和方法
EP4149937A1 (en) 2020-05-13 2023-03-22 CHDI Foundation, Inc. Htt modulators for treating huntington's disease
MX2023000167A (es) 2020-07-02 2023-05-03 Remix Therapeutics Inc Derivados de 5-[5-(piperidin-4-il)tieno[3,2-c]pirazol-2-il]indazol y compuestos relacionados como moduladores para el corte y empalme de ácidos nucleicos y para el tratamiento de enfermedades proliferativas.
AU2021299515A1 (en) 2020-07-02 2023-02-02 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
EP4192454A4 (en) * 2020-08-05 2024-09-11 Skyhawk Therapeutics, Inc. COMPOSITIONS FOR MODULATION OF SPLICING
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
WO2022060951A1 (en) * 2020-09-16 2022-03-24 Skyhawk Therapeutics, Inc. Compositions for modulating splicing
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
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
US20240400584A1 (en) 2021-08-30 2024-12-05 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2023034827A1 (en) 2021-08-30 2023-03-09 Remix Therapeutics Inc. Compounds and methods for modulating splicing
TW202319047A (zh) 2021-08-30 2023-05-16 美商雷密克斯醫療公司 調節剪接之化合物及用途
CA3230256A1 (en) 2021-08-30 2023-03-09 Dominic Reynolds Compounds and methods for modulating splicing
WO2023034836A1 (en) 2021-08-30 2023-03-09 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
TW202333689A (zh) 2021-10-13 2023-09-01 美商雷密克斯醫療公司 調節剪接之化合物及方法
CA3238090A1 (en) 2021-11-17 2023-05-25 Chdi Foundation, Inc. Htt modulators for treating huntington's disease
CN118786117A (zh) * 2021-11-18 2024-10-15 亚根达医疗公司 杂环酰胺及其使用方法
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
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
WO2023133229A2 (en) 2022-01-05 2023-07-13 Remix Therapeutics Inc. Compounds and methods for modulating splicing
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
EP4610258A1 (en) * 2022-10-26 2025-09-03 Tenvie Therapeutics, Inc. Fused pyridazine derivative
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
AU2024223513A1 (en) * 2023-02-15 2025-09-18 Ascidian Therapeutics, Inc. Htt trans-splicing molecules
CN121443613A (zh) * 2024-05-30 2026-01-30 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
CA2457527A1 (en) 2001-08-31 2003-03-13 Gen-Probe Incorporated 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
ES2280836T3 (es) 2002-09-30 2007-09-16 Neurosearch A/S Nuevos derivados de 1,4-diazabicicloalcano, su preparacion y uso.
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
US7309699B2 (en) 2003-12-22 2007-12-18 Abbott Laboratories 3-Quinuclidinyl amino-substituted biaryl derivatives
US7160876B2 (en) 2003-12-22 2007-01-09 Abbott Laboratories Fused bicycloheterocycle substituted quinuclidine derivatives
US7655657B2 (en) 2003-12-22 2010-02-02 Abbott Laboratories Fused bicycloheterocycle substituted quinuclidine derivatives
US7309487B2 (en) 2004-02-09 2007-12-18 George Inana Methods and compositions for detecting and treating retinal diseases
AR049401A1 (es) 2004-06-18 2006-07-26 Novartis Ag Aza-biciclononanos
GB0415746D0 (en) 2004-07-14 2004-08-18 Novartis Ag Organic compounds
EP1893608B1 (en) 2004-10-07 2011-08-17 Merck Sharp & Dohme Corp. Thiazolyl mglur5 antagonists and methods for their use
ES2400689T3 (es) 2004-10-18 2013-04-11 Amgen, Inc Compuestos de tiadiazol y métodos de uso
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
WO2006081230A2 (en) 2005-01-26 2006-08-03 Schering Corporation 3-(indazol-5-yl)-(1,2, 4) triazine derivatives and related compounds as protein kinase inhibitors for the treatment of cancer
TW200716594A (en) 2005-04-18 2007-05-01 Neurogen Corp Substituted heteroaryl CB1 antagonists
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
DK2018380T3 (da) * 2006-05-19 2012-01-23 Abbott Lab CNS-aktiverede kondenserede bi-heterocyklisk-substituerede azabicykliske alkanderivater
AU2007267175A1 (en) 2006-05-30 2007-12-06 Neurosearch A/S Novel 1,4-diaza-bicyclo[3.2.2]nonyl oxadiazolyl derivatives and their medical use
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
PL2203173T3 (pl) 2007-10-26 2016-06-30 Academisch Ziekenhuis Leiden Środki i sposoby przeciwdziałania zaburzeniom mięśni
EP2280010B1 (en) 2007-11-21 2012-12-19 Abbott Laboratories Biaryl substituted azabicyclic alkane derivatives as nicotinic acetylcholine receptor activity modulators
US8437067B2 (en) 2008-03-18 2013-05-07 Konica Minolta Holdings, Inc. Electrochemical display element
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
RU2505540C2 (ru) 2008-12-23 2014-01-27 Эббви Инк. Антивирусные соединения
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
US20120149756A1 (en) 2009-04-10 2012-06-14 Associatin Institut de Myologie Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease
CN115227710A (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
WO2011090669A1 (en) 2010-01-20 2011-07-28 Abbott Laboratories Methods for treating pain
SG183146A1 (en) 2010-02-05 2012-09-27 Heptares Therapeutics Ltd 1,2,4-triazine-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
US9133123B2 (en) 2010-04-23 2015-09-15 Cytokinetics, Inc. Certain amino-pyridines and amino-triazines, compositions thereof, and methods for their use
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
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
JP6069661B2 (ja) 2010-06-24 2017-02-01 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 種々のアミロイドβペプチドアロフォームレベルの調節における組成物及びその使用
KR101764952B1 (ko) 2010-07-29 2017-08-03 리겔 파마슈티칼스, 인크. Ａｍｐｋ-활성화 헤테로시클릭 화합물 및 그의 사용 방법
KR102090836B1 (ko) 2011-03-14 2020-03-18 엔에스이 프로덕츠, 인크. 세포 정제를 촉진하기 위한 경구 제제
WO2012129562A2 (en) 2011-03-24 2012-09-27 The Scripps Research Institute Compounds and methods for inducing chondrogenesis
WO2013019938A1 (en) 2011-08-02 2013-02-07 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
WO2013101974A1 (en) 2011-12-30 2013-07-04 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
CA2862084C (en) 2012-01-26 2021-05-11 Ptc Therapeutics, Inc. 1h-isochromen-1-one derivatives and compositions thereof for treating spinal muscular atrophy
EP3406252B1 (en) 2012-02-10 2020-05-13 PTC Therapeutics, Inc. Compounds 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
CN104302181B (zh) 2012-03-01 2017-09-15 Ptc医疗公司 用于治疗脊髓性肌萎缩的化合物
KR102109992B1 (ko) 2012-03-23 2020-05-13 피티씨 테라퓨틱스, 인크. 척수성 근위축증을 치료하기 위한 화합물
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
DK2906696T4 (da) 2012-10-15 2023-02-27 Ionis Pharmaceuticals Inc Fremgangsmåder til modulering af c9orf72-ekspression
JP2016508159A (ja) * 2013-01-15 2016-03-17 ノバルティス アーゲー アルファ７ニコチン性アセチルコリン受容体アゴニストの使用
CA2898043C (en) * 2013-01-15 2019-08-06 Novartis Ag Use of alpha 7 nicotinic receptor agonists for the treatment of narcolepsy
WO2014111838A1 (en) * 2013-01-15 2014-07-24 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
RU2673542C2 (ru) 2013-06-25 2018-11-28 Ф. Хоффманн-Ля Рош Аг Соединения для лечения спинальной мышечной атрофии
KR20210130843A (ko) 2013-07-31 2021-11-01 노파르티스 아게 1,4-이치환된 피리다진 유사체 및 smn-결핍-관련 상태를 치료하기 위한 그의 용도
WO2015024876A2 (en) 2013-08-19 2015-02-26 F. Hoffmann-La Roche Ag Screening method
US9884865B2 (en) 2013-08-26 2018-02-06 Purdue Pharma L.P. Azaspiro[4.5] decane derivatives and use thereof
US9982260B2 (en) 2013-09-30 2018-05-29 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
RU2683309C2 (ru) 2014-03-14 2019-03-28 Раквалиа Фарма Инк. Азаспиропроизводные в качестве антагонистов trpm8
WO2015143245A1 (en) 2014-03-19 2015-09-24 Isis Pharmaceuticals, Inc. Methods for modulating ataxin 2 expression
CA2950876A1 (en) 2014-06-10 2015-12-17 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
AU2016209321B2 (en) 2015-01-20 2019-05-09 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
JP6884102B2 (ja) 2015-02-09 2021-06-09 エフ・ホフマン−ラ・ロシュ・アクチェンゲゼルシャフト がんの治療のための化合物
EP4249472A3 (en) * 2015-05-30 2023-12-13 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
US10358427B2 (en) 2015-09-24 2019-07-23 Glaxosmithkline Intellectual Property Development Limited Modulators of indoleamine 2,3-dioxygenase
WO2017062751A1 (en) 2015-10-08 2017-04-13 The Regents Of The University Of California Compounds and methods for promoting stress resistance
US10196639B2 (en) 2015-10-09 2019-02-05 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
JP6659841B2 (ja) 2015-11-12 2020-03-04 エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ 脊髄性筋萎縮症を処置するための組成物
WO2017100276A1 (en) 2015-12-08 2017-06-15 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
EA201800367A1 (ru) * 2015-12-10 2019-02-28 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Способы лечения болезни хантингтона
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
US10787433B2 (en) 2016-01-15 2020-09-29 The Brigham And Women's Hospital, Inc. Pyridazine derivatives as EAAT2 activators
CA3012700A1 (en) * 2016-02-01 2017-08-10 Arrakis Therapeutics, Inc. Compounds and methods of treating rna-mediated diseases
EP3419981A4 (en) 2016-02-23 2019-09-18 Indiana University Research & Technology Corporation COMBINATION THERAPIES FOR THE TREATMENT OF SPINAL MUSCLE ATROPHY
WO2017147536A1 (en) 2016-02-24 2017-08-31 The Rockefeller University Embryonic cell-based therapeutic candidate screening systems, models for huntington's disease and uses thereof
WO2017147509A1 (en) 2016-02-25 2017-08-31 Marco Colonna Compositions comprising trem2 and methods of use thereof
US20170268066A1 (en) 2016-03-15 2017-09-21 Chalmers Ventures Ab Cancer biomarkers
CN108779093B (zh) 2016-03-29 2021-03-09 默克专利股份公司 作为丙酮酸脱氢酶激酶的抑制剂的n1-(3,3,3-三氟-2-羟基-2-甲基丙酰基)-哌啶衍生物
CA3019847A1 (en) 2016-04-04 2017-10-12 University Of Florida Research Foundation, Incorporated Manipulation of eif3 to modulate repeat associated non-atg (ran) translation
US10577360B2 (en) 2016-04-12 2020-03-03 Esteve Pharmaceuticals, S.A. Arylamide derivatives having multimodal activity against pain
NZ789269A (en) 2016-06-13 2026-01-30 Scholar Rock Inc Use of myostatin inhibitors and combination therapies
AU2017290894A1 (en) * 2016-07-01 2019-01-17 Arrakis Therapeutics, Inc. Compounds and methods for modulating RNA function
EP3544435A4 (en) 2016-11-28 2020-11-04 PTC Therapeutics, Inc. RNA SPLICE MODULATION PROCESSES
JP7129095B2 (ja) 2017-02-20 2022-09-01 国立大学法人京都大学 スプライシング異常に起因する遺伝性疾病のための医薬組成物及び治療方法
EP3628055A4 (en) 2017-05-01 2021-06-09 Thomas Jefferson University Systems-level analysis of tcga cancers reveals disease trna fragmentation patterns and associations with messenger rnas and repeat
WO2018232039A1 (en) 2017-06-14 2018-12-20 Ptc Therapeutics, Inc. Methods for modifying rna splicing
BR112019027717A2 (pt) 2017-06-28 2020-07-28 Ptc Therapeutics, Inc. métodos para tratar a doença de huntington
US11395822B2 (en) 2017-06-28 2022-07-26 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
CN109843872B (zh) 2017-09-20 2022-08-30 杭州英创医药科技有限公司 作为ido抑制剂和/或ido-hdac双重抑制剂的多环化合物
BR112020007058A2 (pt) 2017-10-12 2020-10-06 Revolution Medicines, Inc. compostos de piridina, pirazina, e triazina como inibidores de shp2 alostéricos
US20190167815A1 (en) 2017-10-24 2019-06-06 Sangamo Therapeutics, Inc. Methods and compositions for the 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
WO2019199972A1 (en) 2018-04-10 2019-10-17 Skyhawk Therapeutics, Inc. Compounds for the treatment of cancer
BR112020026534A2 (pt) 2018-06-27 2021-03-23 Ptc Therapeutics, Inc. Compostos de heteroarila para o tratamento da doença de huntington
SI3814357T1 (sl) 2018-06-27 2024-09-30 Ptc Therapeutics, Inc. Heterociklične in heteroarilne spojine za zdravljenje Huntingtonove bolezni
JOP20200338A1 (ar) 2018-06-27 2020-12-24 Reborna Biosciences Inc عامل وقائي أو علاجي للضمور العضلي نخاعي المنشأ
WO2020005882A1 (en) 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heteroaryl compounds for treating 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
CA3169667A1 (en) 2020-02-28 2021-09-02 Dominic Reynolds Pyridazine derivatives for modulating nucleic acid splicing
US20230148184A1 (en) 2020-02-28 2023-05-11 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2021174163A1 (en) 2020-02-28 2021-09-02 Remix Therapeutics Inc. Fused bicyclic compounds useful for modulating nucleic acid splicing
EP4110775A1 (en) 2020-02-28 2023-01-04 Remix Therapeutics Inc. Thiophenyl derivatives useful for modulating nucleic acid splicing
WO2021207530A1 (en) 2020-04-08 2021-10-14 Remix Therapeutics Inc. Compounds and methods for modulating splicing
CN115551593A (zh) 2020-04-08 2022-12-30 雷密克斯医疗公司 用于调节剪接的化合物和方法

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