JPWO2020163541A5 - - Google Patents

Download PDF

Info

Publication number
JPWO2020163541A5
JPWO2020163541A5 JP2021545878A JP2021545878A JPWO2020163541A5 JP WO2020163541 A5 JPWO2020163541 A5 JP WO2020163541A5 JP 2021545878 A JP2021545878 A JP 2021545878A JP 2021545878 A JP2021545878 A JP 2021545878A JP WO2020163541 A5 JPWO2020163541 A5 JP WO2020163541A5
Authority
JP
Japan
Prior art keywords
substituted
unsubstituted
pharmaceutically acceptable
compound
acceptable salt
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
JP2021545878A
Other languages
English (en)
Japanese (ja)
Other versions
JP7551629B2 (ja
JP2022520051A5 (https=
JP2022520051A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/016894 external-priority patent/WO2020163541A1/en
Publication of JP2022520051A publication Critical patent/JP2022520051A/ja
Publication of JPWO2020163541A5 publication Critical patent/JPWO2020163541A5/ja
Publication of JP2022520051A5 publication Critical patent/JP2022520051A5/ja
Application granted granted Critical
Publication of JP7551629B2 publication Critical patent/JP7551629B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2021545878A 2019-02-05 2020-02-05 スプライシングを調節するための方法および組成物 Active JP7551629B2 (ja)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201962801381P 2019-02-05 2019-02-05
US201962801475P 2019-02-05 2019-02-05
US201962801390P 2019-02-05 2019-02-05
US201962801386P 2019-02-05 2019-02-05
US62/801,381 2019-02-05
US62/801,390 2019-02-05
US62/801,386 2019-02-05
US62/801,475 2019-02-05
PCT/US2020/016894 WO2020163541A1 (en) 2019-02-05 2020-02-05 Methods and compositions for modulating splicing

Publications (4)

Publication Number Publication Date
JP2022520051A JP2022520051A (ja) 2022-03-28
JPWO2020163541A5 true JPWO2020163541A5 (https=) 2023-02-10
JP2022520051A5 JP2022520051A5 (https=) 2023-02-10
JP7551629B2 JP7551629B2 (ja) 2024-09-17

Family

ID=71947942

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021545878A Active JP7551629B2 (ja) 2019-02-05 2020-02-05 スプライシングを調節するための方法および組成物

Country Status (6)

Country Link
US (1) US12509460B2 (https=)
EP (1) EP3920926A4 (https=)
JP (1) JP7551629B2 (https=)
KR (1) KR102925382B1 (https=)
CN (1) CN114126613A (https=)
WO (1) WO2020163541A1 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2020163405A1 (en) 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
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
JP7603595B2 (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
TW202131920A (zh) 2019-11-01 2021-09-01 瑞士商諾華公司 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途
CA3182912A1 (en) 2020-05-13 2021-11-18 Chdi Foundation, Inc. Htt modulators for treating huntington's disease
IL301964A (en) 2020-10-08 2023-06-01 Redona Therapeutics Inc History of benzo[H]quinazoline-4-amine and thiano[2,3-H]quinazoline-4-amine for cancer treatment
MX2024005933A (es) 2021-11-17 2024-08-06 Chdi Foundation Inc Moduladores de htt pata tratar la enfermedad de huntington.
KR20260044269A (ko) 2023-06-30 2026-04-01 스카이호크 테라퓨틱스, 인코포레이티드 헌팅턴병 치료를 위한 스플라이싱 조절제인 6-(6-(((1r,2r,3s,5s)-2-플루오로-9-아자바이시클로[3.3.1]노난-3-일)(메틸)아미노)피리다진-3-일)-2-메틸벤조[d]옥사졸-5-올의 결정질 형태
AR133109A1 (es) 2023-06-30 2025-08-27 Skyhawk Therapeutics Inc Composiciones para modular el empalme
TW202539670A (zh) * 2024-02-23 2025-10-16 大陸商海思科醫藥集團股份有限公司 Braf抑制劑的製備方法
WO2025255218A1 (en) 2024-06-05 2025-12-11 Skyhawk Therapeutics, Inc. Compositions and methods for modulating splicing

Family Cites Families (155)

* 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
ATE420351T1 (de) 2001-08-31 2009-01-15 Gen Probe Inc Sonden mit affinitätsverschiebung zur quantifizierung von analytenpolynukleotiden
US6992087B2 (en) 2001-11-21 2006-01-31 Pfizer Inc Substituted aryl 1,4-pyrazine derivatives
US7135484B2 (en) 2002-08-14 2006-11-14 Abbott Laboratories Azabicyclic compounds are central nervous system active agents
US7220741B2 (en) 2002-09-30 2007-05-22 Neurosearch A/S 1,4-diazabicycloalkane derivatives, their preparation and use
MXPA05006861A (es) 2003-02-27 2005-12-12 Neurosearch As Derivados arildiazabiciclicos novedosos.
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
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
US7879882B2 (en) 2004-10-07 2011-02-01 Merck Sharp & Dohme Corp. Thiazolyl mglur5 antagonists and methods for their use
EA012181B1 (ru) 2004-10-18 2009-08-28 Амген, Инк. Соединения тиадиазола и их применение
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
DE602006010991D1 (de) 2005-01-26 2010-01-21 Schering Corp 3-(indazol-5-yl)-(1,2,4)triazinderivate und verwandte verbindungen als proteinkinaseinhibitoren zur behandlung von krebs
CA2606288A1 (en) 2005-04-18 2006-10-26 Neurogen Corporation Subtituted 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
UA92083C2 (ru) 2006-05-19 2010-09-27 Эбботт Леборетриз Азабициклические алкановые производные, замещенные конденсированным бициклогетероциклом
US8158619B2 (en) 2006-05-30 2012-04-17 Neurosearch A/S 1,4-diaza-bicyclo[3.2.2]nonyl oxadiazolyl compounds 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
AU2008317566B2 (en) 2007-10-26 2014-05-01 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
BRPI0820418A2 (pt) 2007-11-21 2015-05-19 Abbott Lab "derivados de alcano azabicíclico substituído com biarila como moduladores da atividade do receptor nicotínico de acetilcolina"
WO2009116353A1 (ja) 2008-03-18 2009-09-24 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
US8309577B2 (en) 2008-04-23 2012-11-13 Bristol-Myers Squibb Company Quinuclidine compounds as α-7 nicotinic acetylcholine receptor ligands
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
CA2740193A1 (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
US20120149756A1 (en) 2009-04-10 2012-06-14 Associatin Institut de Myologie Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease
PT3305302T (pt) 2009-06-17 2018-12-14 Biogen Ma Inc Composições e métodos de modulação de excisões de smn2 em um sujeito
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
SI2531492T1 (sl) 2010-02-05 2016-08-31 Heptares Therapeutics Limited Derivati 1,2,4-triazin-4-amina
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
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
BR112013002112B1 (pt) 2010-07-29 2021-04-06 Rigel Pharmaceuticals, Inc. Composto, composição farmacêutica, e, uso de um composto, ou de um respectivo sal farmaceuticamente aceitável, ou de uma composição
CN103732258A (zh) 2011-03-14 2014-04-16 Nse产品公司 用于促进细胞纯化的口服制剂
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
WO2013063458A2 (en) 2011-10-27 2013-05-02 Mayo Foundation For Medical Education And Research Inhibiting g protein coupled receptor 6 kinase polypeptides
MX352861B (es) 2011-12-30 2017-12-13 Ptc Therapeutics Inc Compuestos para tratar la atrofia muscular espinal.
MX357834B (es) 2012-01-26 2018-07-26 Ptc Therapeutics Inc Compuestos de tipo isocumarina sustituidos para tratar la atrofia muscular espinal.
WO2013117693A1 (en) 2012-02-10 2013-08-15 Glaxosmithkline Intellectual Property Development Limited Pde4 inhibitor for treating huntington's disease
EP2812004B1 (en) 2012-02-10 2018-06-27 PTC Therapeutics, Inc. Compounds for treating spinal muscular atrophy
CA2865957C (en) 2012-03-01 2020-02-11 F. Hoffmann-La Roche Ag Compounds for treating spinal muscular atrophy
CA2868026C (en) 2012-03-23 2021-02-16 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
ES2762326T5 (es) 2012-10-15 2023-04-27 Ionis Pharmaceuticals Inc Métodos para modular la expresión de C9ORF72
CN105263492B (zh) 2013-01-15 2018-04-10 诺华有限公司 α7烟碱型乙酰胆碱受体激动剂在治疗发作性睡病中的应用
EP2945633B1 (en) 2013-01-15 2021-06-30 Novartis AG Use of alpha 7 nicotinic acetylcholine receptor agonists
BR112015016992A8 (pt) 2013-01-15 2018-01-23 Novartis Ag uso de agonistas do receptor alfa 7 nicotínico de acetilcolina
US9040712B2 (en) 2013-01-23 2015-05-26 Novartis Ag Thiadiazole analogs thereof and methods for treating SMN-deficiency-related-conditions
CA2913785C (en) 2013-06-25 2021-08-31 F. Hoffmann-La Roche Ag Compounds for treating spinal muscular atrophy
EP4105208A1 (en) 2013-07-31 2022-12-21 Novartis AG 1,4-disubstituted pyridazine derivatives and their use for treating smn-deficiency-related conditions
EP3035935B1 (en) 2013-08-19 2020-03-11 F. Hoffmann-La Roche AG Compounds for use in the prophylaxis and treatment of cancer
US20230009804A1 (en) 2013-08-23 2023-01-12 Ateb, Inc. Machine-learning techniques for generating entity instructions
WO2015031036A1 (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
JP6643545B2 (ja) 2014-03-14 2020-02-12 ラクオリア創薬株式会社 Trpm8拮抗剤としてのアザスピロ誘導体
WO2015143245A1 (en) 2014-03-19 2015-09-24 Isis Pharmaceuticals, Inc. Methods for modulating ataxin 2 expression
WO2015190921A2 (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
WO2016037039A1 (en) 2014-09-04 2016-03-10 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
EP3256126B1 (en) * 2015-02-09 2024-03-27 F. Hoffmann-La Roche AG Compounds for the treatment of cancer
EP3053577A1 (en) 2015-02-09 2016-08-10 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
AR105392A1 (es) 2015-07-20 2017-09-27 Genzyme Corp Inhibidores del receptor del factor estimulador de colonias 1 (csf-1r)
WO2017041036A1 (en) 2015-09-02 2017-03-09 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
EP3359685B1 (en) 2015-10-09 2026-01-28 University Of Southampton Modulation of gene expression for deregulated protein expression
EP3183347A4 (en) 2015-10-17 2018-04-18 Lifesplice Pharma LLC Splice modulating oligonucleotides and methods of use thereof
EA036399B1 (ru) 2015-11-12 2020-11-06 Ф. Хоффманн-Ля Рош Аг Композиции для лечения спинальной мышечной атрофии
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
JP2019502753A (ja) 2015-12-23 2019-01-31 ムーンショット ファーマ エルエルシー ナンセンス変異依存mRNA分解機構の抑制による免疫応答の誘導法
WO2017123991A1 (en) 2016-01-15 2017-07-20 The Brigham And Women's Hospital, Inc. Pyridazine derivatives as eaat2 activators
MX2018009325A (es) 2016-02-01 2019-05-15 Arrakis Therapeutics Inc Compuestos y metodos para tratar enfermedades mediadas por el acido ribonucleico (arn).
MX2018010109A (es) 2016-02-23 2018-12-17 Univ Indiana Res & Tech Corp Terapias de combinacion para tratamiento de atrofia muscular espinal.
CN109069870B (zh) 2016-02-24 2022-04-29 洛克菲勒大学 基于胚胎细胞的用于亨廷顿氏病的治疗候选物筛选系统、模型及它们的应用
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
JP6923551B2 (ja) 2016-03-29 2021-08-18 メルク パテント ゲゼルシャフト ミット ベシュレンクテル ハフツングMerck Patent Gesellschaft mit beschraenkter Haftung ピルバートデヒドロゲナーゼキナーゼのインヒビターとしてのn1−(3,3,3−トリフルオロ−2−ヒドロキシ−2−メチルプロピオニル)−ピペリジン誘導体
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
US10577360B2 (en) 2016-04-12 2020-03-03 Esteve Pharmaceuticals, S.A. Arylamide derivatives having multimodal activity against pain
AU2017283546C1 (en) 2016-06-13 2020-11-19 Scholar Rock, Inc. Use of myostatin inhibitors and combination therapies
SG11201811643TA (en) 2016-07-01 2019-01-30 Arrakis Therapeutics Inc Compounds and methods for modulating rna function
JP2019535789A (ja) 2016-11-28 2019-12-12 ピーティーシー セラピューティクス,インコーポレーテッド Rnaスプライシングを調節する方法
WO2018151326A1 (ja) 2017-02-20 2018-08-23 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
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
JP2020523365A (ja) * 2017-06-14 2020-08-06 ピーティーシー セラピューティクス,インコーポレーテッド Rnaスプライシングを改変する方法
CN111182898B (zh) 2017-06-28 2024-04-16 Ptc医疗公司 用于治疗亨廷顿氏病的方法
MX2019015580A (es) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
JP7312749B2 (ja) * 2017-08-04 2023-07-21 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 スプライシングをモジュレートする方法および組成物
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
MX2020003579A (es) 2017-10-12 2020-07-22 Revolution Medicines Inc Compuestos de piridina, pirazina, y triazina como inhibidores de shp2 alostericos.
CA3079727A1 (en) 2017-10-24 2019-05-02 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
EA202092001A1 (ru) 2018-03-27 2021-01-29 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Соединения для лечения болезни гентингтона
JP2021521200A (ja) * 2018-04-10 2021-08-26 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 癌の処置のための化合物
CN112469707B (zh) 2018-06-27 2024-06-21 利伯纳生物科学株式会社 脊髓性肌萎缩症的预防剂或治疗剂
HRP20240935T1 (hr) 2018-06-27 2024-11-22 Ptc Therapeutics, Inc. Heterociklični i heteroaril spojevi za liječenje huntingtonove bolesti
WO2020005877A1 (en) 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
EP3814360B8 (en) 2018-06-27 2024-11-06 PTC Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
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
WO2020163382A1 (en) 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
KR102925387B1 (ko) 2019-02-04 2026-02-10 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
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
EP3920910A4 (en) 2019-02-04 2022-11-09 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATION OF SPLICING
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
US20230008867A1 (en) 2019-02-05 2023-01-12 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
WO2020163406A1 (en) 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
WO2020163405A1 (en) 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
WO2020163401A1 (en) 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
JP2022519311A (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
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
JP7603595B2 (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
WO2020190793A1 (en) 2019-03-15 2020-09-24 Skyhawk Therapeutics, Inc. Compositions and methods for correction of aberrant splicing
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
WO2021071984A1 (en) 2019-10-08 2021-04-15 Skyhawk Therapeutics, Inc. Compounds for modulating splicing
WO2021071981A1 (en) 2019-10-08 2021-04-15 Skyhawk Therapeutics, Inc. Compounds for modulating splicing
WO2021174174A1 (en) 2020-02-28 2021-09-02 Remix Therapeutics Inc. Thiophenyl derivatives useful for modulating nucleic acid splicing
WO2021174176A1 (en) 2020-02-28 2021-09-02 Remix Therapeutics Inc. Pyridazine dervatives for modulating nucleic acid splicing
MX2022010681A (es) 2020-02-28 2023-03-21 Remix Therapeutics Inc Compuestos y metodos para modular el empalme.
CN115485025A (zh) 2020-02-28 2022-12-16 雷密克斯医疗公司 用于调节剪接的化合物和方法
WO2021207530A1 (en) 2020-04-08 2021-10-14 Remix Therapeutics Inc. Compounds and methods for modulating splicing
MX2022012678A (es) 2020-04-08 2023-01-11 Remix Therapeutics Inc Compuestos y metodos para modular el corte y empalme.
MX2023001558A (es) 2020-08-05 2023-04-26 Skyhawk Therapeutics Inc Composiciones para modular el corte y empalme.
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
WO2022060951A1 (en) 2020-09-16 2022-03-24 Skyhawk Therapeutics, Inc. Compositions for modulating splicing
US20240368163A1 (en) 2021-08-30 2024-11-07 Remix Therapeutics Inc. Compounds and methods for modulating splicing

Similar Documents

Publication Publication Date Title
JPWO2020163541A5 (https=)
JP2020530487A5 (https=)
JPWO2020163248A5 (https=)
RU2004117887A (ru) Ариланилиновые агонисты бета2 адренергических рецепторов
RU2003133448A (ru) 3,4-дизамещенные циклобутен-1,2-дионы как лиганды схс- хемокинового рецептора
RU2014134141A (ru) Соединения пуринона в качестве ингибиторов киназ
JP2004509113A5 (https=)
JP2007502845A5 (https=)
JP2014015465A5 (https=)
JP2005509024A5 (https=)
JP2013545816A5 (https=)
US5900432A (en) Therapeutic N-(4-benzoyl-2-methyl-phenyl)-3,3,3-trifluoro-2-hydroxy-2-methyl-propanamides
RU2005111975A (ru) Производные фенэтаноламина для лечения респираторных заболеваний
JP2008508192A5 (https=)
PT1708991E (pt) Derivados de sulfonamida para o tratamento de doenças
JP2005511545A5 (https=)
JPWO2020163405A5 (https=)
JPWO2020163544A5 (https=)
JP2013537193A5 (https=)
EP3239160A1 (en) Tizoxanide phosphate and alkane sulfonate and pharmaceutical applications thereof
JPWO2022047031A5 (https=)
JP2005533064A5 (https=)
JP2006500372A5 (https=)
JP2021532183A5 (https=)
JP2013544880A5 (https=)