JP2020169171A5 - - Google Patents

Download PDF

Info

Publication number
JP2020169171A5
JP2020169171A5 JP2020078335A JP2020078335A JP2020169171A5 JP 2020169171 A5 JP2020169171 A5 JP 2020169171A5 JP 2020078335 A JP2020078335 A JP 2020078335A JP 2020078335 A JP2020078335 A JP 2020078335A JP 2020169171 A5 JP2020169171 A5 JP 2020169171A5
Authority
JP
Japan
Prior art keywords
pharmaceutically acceptable
solvate
substituted
acceptable salt
compound according
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.)
Granted
Application number
JP2020078335A
Other languages
English (en)
Japanese (ja)
Other versions
JP7135026B2 (ja
JP2020169171A (ja
Filing date
Publication date
Application filed filed Critical
Publication of JP2020169171A publication Critical patent/JP2020169171A/ja
Publication of JP2020169171A5 publication Critical patent/JP2020169171A5/ja
Application granted granted Critical
Publication of JP7135026B2 publication Critical patent/JP7135026B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2020078335A 2017-08-04 2020-04-27 スプライシングをモジュレートする方法および組成物 Active JP7135026B2 (ja)

Applications Claiming Priority (6)

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

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
JP2020529113A Division JP7312749B2 (ja) 2017-08-04 2018-08-03 スプライシングをモジュレートする方法および組成物

Publications (3)

Publication Number Publication Date
JP2020169171A JP2020169171A (ja) 2020-10-15
JP2020169171A5 true JP2020169171A5 (US07846941-20101207-C00217.png) 2021-09-16
JP7135026B2 JP7135026B2 (ja) 2022-09-12

Family

ID=65233135

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020529113A Active JP7312749B2 (ja) 2017-08-04 2018-08-03 スプライシングをモジュレートする方法および組成物
JP2020078335A Active JP7135026B2 (ja) 2017-08-04 2020-04-27 スプライシングをモジュレートする方法および組成物

Family Applications Before (1)

Application Number Title Priority Date Filing Date
JP2020529113A Active JP7312749B2 (ja) 2017-08-04 2018-08-03 スプライシングをモジュレートする方法および組成物

Country Status (16)

Country Link
US (8) US11008572B2 (US07846941-20101207-C00217.png)
EP (2) EP3689863A1 (US07846941-20101207-C00217.png)
JP (2) JP7312749B2 (US07846941-20101207-C00217.png)
KR (2) KR102636383B1 (US07846941-20101207-C00217.png)
CN (2) CN111499615B (US07846941-20101207-C00217.png)
AU (2) AU2018309187C1 (US07846941-20101207-C00217.png)
BR (2) BR112020002270A2 (US07846941-20101207-C00217.png)
CA (1) CA3072205A1 (US07846941-20101207-C00217.png)
GB (2) GB2587457B (US07846941-20101207-C00217.png)
IL (2) IL273741B (US07846941-20101207-C00217.png)
MX (2) MX2020001425A (US07846941-20101207-C00217.png)
MY (1) MY201938A (US07846941-20101207-C00217.png)
RU (1) RU2020105929A (US07846941-20101207-C00217.png)
SG (2) SG11202000980QA (US07846941-20101207-C00217.png)
WO (1) WO2019028440A1 (US07846941-20101207-C00217.png)
ZA (2) ZA202000695B (US07846941-20101207-C00217.png)

Families Citing this family (64)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL281633B (en) 2015-12-10 2022-07-01 Ptc Therapeutics Inc Methods for treating Huntington's disease
EP3544435A4 (en) 2016-11-28 2020-11-04 PTC Therapeutics, Inc. RNA SPLICE MODULATION PROCESSES
EP3634953B1 (en) 2017-06-05 2024-01-03 PTC Therapeutics, Inc. Compounds for treating huntington's disease
CN111372611A (zh) 2017-06-14 2020-07-03 Ptc医疗公司 修饰rna剪接的方法
CA3067591A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. Methods for treating huntington's disease
US11395822B2 (en) 2017-06-28 2022-07-26 Ptc Therapeutics, Inc. Methods for treating Huntington's disease
CN111499615B (zh) 2017-08-04 2024-02-02 斯基霍克疗法公司 用于调节剪接的方法和组合物
CA3094703A1 (en) 2018-03-27 2019-10-03 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
CN112272666A (zh) 2018-04-10 2021-01-26 斯基霍克疗法公司 用于治疗癌症的化合物
WO2020005873A1 (en) 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heterocyclic and heteroaryl compounds for treating huntington's disease
US11685746B2 (en) 2018-06-27 2023-06-27 Ptc Therapeutics, Inc. Heteroaryl compounds for treating Huntington's disease
EP3840733A4 (en) 2018-08-20 2022-07-20 Rogcon, Inc. ANTISENSE OLIGONUCLEOTIDES DIRECTED TO SCN2A FOR THE TREATMENT OF SCN1A ENCEPHALOPATHIES
EP3969469A4 (en) * 2019-01-23 2022-11-23 The Florey Institute of Neuroscience and Mental Health ANTISENSE OLIGONUCLEOTIDES TARGETING PRESERVED SCN2A 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
JP2022523148A (ja) * 2019-02-05 2022-04-21 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
JP2022519312A (ja) * 2019-02-05 2022-03-22 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
EP3920915A4 (en) 2019-02-05 2022-10-05 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICE
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
KR20210135240A (ko) * 2019-02-05 2021-11-12 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
EP3921311A4 (en) * 2019-02-06 2022-11-09 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICE
EP3920928A4 (en) * 2019-02-06 2022-09-28 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATION OF SPLICING
CN113840602A (zh) * 2019-03-15 2021-12-24 斯基霍克疗法公司 用于校正异常剪接的组合物和方法
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
US11129829B2 (en) * 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
KR20220035937A (ko) 2019-07-25 2022-03-22 노파르티스 아게 조절 가능한 발현 시스템
WO2021071982A1 (en) * 2019-10-08 2021-04-15 Skyhawk Therapeutics, Inc. Compounds for modulating splicing
CN114650822A (zh) 2019-11-01 2022-06-21 诺华股份有限公司 剪接调节子用于减慢亨廷顿氏病进展的治疗的用途
US20230142338A1 (en) 2020-02-28 2023-05-11 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
AU2021228767A1 (en) * 2020-02-28 2022-09-29 Remix Therapeutics Inc. Heterocyclic amides and their use for modulating splicing
WO2021174167A1 (en) 2020-02-28 2021-09-02 Remix Therapeutics Inc. Compounds and methods for modulating splicing
JP2023520925A (ja) 2020-04-08 2023-05-22 リミックス セラピューティクス インコーポレイテッド スプライシングを調節するための化合物及び方法
KR20220167298A (ko) 2020-04-08 2022-12-20 레믹스 테라퓨틱스 인크. 스플라이싱을 조절하기 위한 화합물 및 방법
CN116997548A (zh) 2020-05-13 2023-11-03 Chdi基金会股份有限公司 用于治疗亨廷顿病的htt调节剂
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.
TW202216710A (zh) 2020-07-02 2022-05-01 美商雷密克斯醫療公司 調節剪接之化合物及方法
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
CA3188104A1 (en) * 2020-08-05 2022-02-10 Michael Luzzio Compositions for modulating splicing
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
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
WO2022060943A1 (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
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
EP4396177A1 (en) 2021-08-30 2024-07-10 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2023034836A1 (en) 2021-08-30 2023-03-09 Remix Therapeutics Inc. Compounds and methods for modulating splicing
TW202328135A (zh) 2021-08-30 2023-07-16 美商雷密克斯醫療公司 調節剪接之化合物及用途
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
WO2023034833A1 (en) 2021-08-30 2023-03-09 Remix Therapeutics Inc. Compounds and methods for modulating splicing
EP4416156A1 (en) 2021-10-13 2024-08-21 Remix Therapeutics Inc. Compounds and methods for modulating nucleic acid splicing
KR20240102967A (ko) 2021-10-13 2024-07-03 레믹스 테라퓨틱스 인크. 스플라이싱을 조절하기 위한 화합물 및 방법
AU2022390051A1 (en) * 2021-11-18 2024-05-30 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
WO2023133217A1 (en) 2022-01-05 2023-07-13 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
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
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
WO2024090469A1 (ja) * 2022-10-26 2024-05-02 アステラス製薬株式会社 縮環ピリダジン誘導体
WO2024173719A1 (en) * 2023-02-15 2024-08-22 Ascidian Therapeutics, Inc. Htt trans-splicing molecules

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
WO2003020952A2 (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
NZ538512A (en) 2002-09-30 2006-12-22 Neurosearch As 1,4-diazabicycloalkane derivatives, their preparation and use in treatment of disorders responsive to modulation of cholmergic and/or monoamine receptors
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
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
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
JP5635727B2 (ja) 2004-10-07 2014-12-03 メルク・シャープ・アンド・ドーム・コーポレーションMerck Sharp & Dohme Corp. チアゾリルmglur5アンタゴニスト及びそれらの使用のための方法
CA2583217C (en) 2004-10-18 2011-05-31 Amgen Inc. 1,3,4-thiadiazole compounds as protein kinase inhibitors
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
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
CN102516241B (zh) * 2006-05-19 2015-07-29 艾伯维巴哈马有限公司 Cns活性的稠合的双环杂环取代的氮杂双环烷烃衍生物
ZA200807756B (en) 2006-05-30 2009-11-25 Neurosearch As 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
CN101896186A (zh) 2007-10-26 2010-11-24 莱顿教学医院 对抗肌肉病症的方式和方法
RU2010125121A (ru) 2007-11-21 2011-12-27 Эббот Лэборетриз (Us) Биарилзамещенные производные азабициклических алканов
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
WO2010075376A2 (en) 2008-12-23 2010-07-01 Abbott Laboratories Anti-viral compounds
WO2010107733A2 (en) 2009-03-16 2010-09-23 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
DK3449926T3 (da) 2009-06-17 2019-11-11 Biogen Ma Inc Sammensætninger og fremgangsmåder til modulering af smn2-splejsning hos et individ
TWI558398B (zh) 2009-09-22 2016-11-21 諾華公司 菸鹼乙醯膽鹼受體α7活化劑之用途
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
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
US9133123B2 (en) 2010-04-23 2015-09-15 Cytokinetics, Inc. Certain amino-pyridines and amino-triazines, compositions thereof, and methods for their use
AR081331A1 (es) 2010-04-23 2012-08-08 Cytokinetics Inc Amino- pirimidinas composiciones de las mismas y metodos para el uso de los mismos
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
WO2011163636A2 (en) 2010-06-24 2011-12-29 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
EP2686021B1 (en) 2011-03-14 2018-08-15 NSE Products, Inc. Oral formulations for promoting cellular purification
US9464065B2 (en) 2011-03-24 2016-10-11 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 POLYPEPTIDES FROM 6-G PROTEIN-COUPLED RECEPTOR RECEPTORS
CA2861609C (en) 2011-12-30 2021-02-16 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
US9399649B2 (en) 2012-01-26 2016-07-26 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
PE20142364A1 (es) 2012-02-10 2015-01-10 Hoffmann La Roche Compuestos para tratar atrofia muscular espinal
KR102099997B1 (ko) 2012-03-01 2020-04-13 피티씨 테라퓨틱스, 인크. 척수성 근위축증을 치료하기 위한 화합물
EA028382B1 (ru) 2012-03-23 2017-11-30 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Соединения для лечения спинальной мышечной атрофии
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
ES2762326T5 (es) 2012-10-15 2023-04-27 Ionis Pharmaceuticals Inc Métodos para modular la expresión de C9ORF72
WO2014111751A1 (en) * 2013-01-15 2014-07-24 Novartis Ag Use of alpha 7 nicotinic receptor agonists for the treatment of narcolepsy
BR112015016992A8 (pt) * 2013-01-15 2018-01-23 Novartis Ag uso de agonistas do receptor alfa 7 nicotínico de acetilcolina
BR112015016995B8 (pt) * 2013-01-15 2022-07-26 Novartis Ag Uso de agonistas do receptor alfa 7 nicotínico de acetilcolina
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
WO2014209841A2 (en) 2013-06-25 2014-12-31 F. Hoffmann-La Roche Ag Compounds for treating spinal muscular atrophy
EA030631B1 (ru) 2013-07-31 2018-09-28 Новартис Аг 1,4-дизамещенные аналоги пиридазинхинолина и способы лечения состояний, связанных с smn-дефицитом
CA2915764A1 (en) 2013-08-19 2015-02-26 F. Hoffmann-La Roche Ag Screening method
WO2015031036A1 (en) 2013-08-26 2015-03-05 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
EP3116858B1 (en) 2014-03-14 2020-11-11 RaQualia Pharma Inc. Azaspiro derivatives as trpm8 antagonists
US10006027B2 (en) 2014-03-19 2018-06-26 Ionis Pharmaceuticals, Inc. Methods for modulating Ataxin 2 expression
AU2015272127A1 (en) 2014-06-10 2017-01-19 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
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
BR112017015273A2 (pt) 2015-01-20 2018-01-09 Merial Inc. compostos e composições antihelmínticos e método de utilizações dos mesmos
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
EP4249472A3 (en) * 2015-05-30 2023-12-13 PTC Therapeutics, Inc. Methods for modulating rna splicing
SG10201912699RA (en) 2015-07-20 2020-02-27 Genzyme Corp Colony stimulating factor-1 receptor (csf-1r) inhibitors
WO2017041036A1 (en) 2015-09-02 2017-03-09 Pathways Bioscience, Llc Compositions and methods for treatment or prevention of oral mucositis
ES2814251T3 (es) 2015-09-24 2021-03-26 Glaxosmithkline Ip No 2 Ltd Moduladores de la indolamina 2,3-dioxigenasa
US11413278B2 (en) 2015-10-08 2022-08-16 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
EP3183347A4 (en) * 2015-10-17 2018-04-18 Lifesplice Pharma LLC Splice modulating oligonucleotides and methods of use thereof
WO2017080967A1 (en) 2015-11-12 2017-05-18 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
IL281633B (en) 2015-12-10 2022-07-01 Ptc Therapeutics Inc Methods for treating Huntington's disease
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
US11135221B2 (en) 2015-12-23 2021-10-05 Moonshot Pharma Llc Methods for inducing an immune response
AU2017207991A1 (en) 2016-01-15 2018-08-02 Ohio State University Pyridazine derivatives as EAAT2 activators
SG11201806544XA (en) 2016-02-01 2018-08-30 Arrakis Therapeutics Inc Compounds and methods of treating rna-mediated diseases
CA3015345A1 (en) 2016-02-23 2017-08-31 Indiana University Research & 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
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
KR20210082548A (ko) 2016-06-13 2021-07-05 스칼러 락, 인크. 미오스타틴 억제제의 용도 및 조합 요법
MX2018016038A (es) * 2016-07-01 2019-05-13 Arrakis Therapeutics Inc Compuestos y metodos para modular la funcion del acido ribonucleico (arn).
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 SYSTEM LEVEL ANALYSIS OF CANCER GENOME ATLAS 32 CANCERS (TCGA) REVEALING PATTERNS OF RNAT FRAGMENTATION DEPENDING ON DISEASE AND HIGHLY SELECTIVE ASSOCIATIONS WITH RNA MESSENGER AND REPEAT ELEMENTS
CN111372611A (zh) * 2017-06-14 2020-07-03 Ptc医疗公司 修饰rna剪接的方法
CA3067591A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. Methods for treating huntington's disease
US11395822B2 (en) 2017-06-28 2022-07-26 Ptc Therapeutics, Inc. Methods for treating Huntington's disease
CN111499615B (zh) * 2017-08-04 2024-02-02 斯基霍克疗法公司 用于调节剪接的方法和组合物
EP3686196B1 (en) 2017-09-20 2024-06-12 Hangzhou Innogate Pharma Co., Ltd. Polycyclic compound acting as ido inhibitor and/or ido-hdac dual inhibitor
WO2019075265A1 (en) 2017-10-12 2019-04-18 Revolution Medicines, Inc. PYRIDINE, PYRAZINE AND TRIAZINE COMPOUNDS AS ALLOSTERIC INHIBITORS OF SHP2
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
CA3094703A1 (en) 2018-03-27 2019-10-03 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
CN112272666A (zh) 2018-04-10 2021-01-26 斯基霍克疗法公司 用于治疗癌症的化合物
MX2020014116A (es) 2018-06-27 2021-06-15 Reborna Biosciences Inc Agente profilactico o terapeutico para atrofia muscular espinal.
US11685746B2 (en) 2018-06-27 2023-06-27 Ptc Therapeutics, Inc. Heteroaryl compounds for treating Huntington's disease
WO2020005873A1 (en) 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heterocyclic and 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
US11987568B2 (en) 2018-08-03 2024-05-21 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
US20230142338A1 (en) 2020-02-28 2023-05-11 Remix Therapeutics Inc. Thiophenyl derivatives useful for modulating nucleic acid splicing
WO2021174167A1 (en) 2020-02-28 2021-09-02 Remix Therapeutics Inc. Compounds and methods for modulating splicing
CA3169667A1 (en) 2020-02-28 2021-09-02 Dominic Reynolds Pyridazine derivatives for modulating nucleic acid splicing
AU2021228767A1 (en) 2020-02-28 2022-09-29 Remix Therapeutics Inc. Heterocyclic amides and their use for modulating splicing
KR20220167298A (ko) 2020-04-08 2022-12-20 레믹스 테라퓨틱스 인크. 스플라이싱을 조절하기 위한 화합물 및 방법
JP2023520925A (ja) 2020-04-08 2023-05-22 リミックス セラピューティクス インコーポレイテッド スプライシングを調節するための化合物及び方法

Similar Documents

Publication Publication Date Title
JP2020169171A5 (US07846941-20101207-C00217.png)
JP2020530487A5 (US07846941-20101207-C00217.png)
JP2020507589A5 (US07846941-20101207-C00217.png)
JP2008505157A5 (US07846941-20101207-C00217.png)
JP2009536660A5 (US07846941-20101207-C00217.png)
JP2005533040A5 (US07846941-20101207-C00217.png)
JP2021046404A5 (US07846941-20101207-C00217.png)
JP2021500394A5 (US07846941-20101207-C00217.png)
JP2005523922A5 (US07846941-20101207-C00217.png)
JP2016121196A5 (US07846941-20101207-C00217.png)
JP2018515495A5 (US07846941-20101207-C00217.png)
JP2019524883A5 (US07846941-20101207-C00217.png)
JP2016510323A5 (US07846941-20101207-C00217.png)
JP2011509309A5 (US07846941-20101207-C00217.png)
JP2020529994A5 (US07846941-20101207-C00217.png)
JP2013010792A5 (US07846941-20101207-C00217.png)
RU2005128190A (ru) Ингибиторы фактора подавления миграции макрофагов (фпм) и способы их идентификации
JP2008511601A5 (US07846941-20101207-C00217.png)
JP2014503574A5 (US07846941-20101207-C00217.png)
JP2006514012A5 (US07846941-20101207-C00217.png)
JP6644685B2 (ja) ポマリドミドの調製およびその精製のための改善された方法
JP2017522304A5 (US07846941-20101207-C00217.png)
JP2007514691A5 (US07846941-20101207-C00217.png)
JP2011509302A5 (US07846941-20101207-C00217.png)
JP2019527724A5 (US07846941-20101207-C00217.png)