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)
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

Claims (15)

  1. 式(IV)の構造を有する化合物:
    Figure 2020169171
    (式中、
    Figure 2020169171
    環Qは、
    Figure 2020169171
    であり;、環Pはアリールまたはヘテロアリールであり;
    Xは、−O−であり
    Zは、−CRであり、RはHであり;
    Wは、置換または無置換のC〜Cアルキレンであり;
    Rは、Hであり;
    11、R12、R13、R14、R16およびR17は、H、F、−OR、置換または無置換のC〜Cアルキル、置換または無置換のC〜Cフルオロアルキル、および置換または無置換のC〜Cヘテロアルキルからなる群からそれぞれ独立して選択され、ここで、アルキルは、ヒドロキシ、アミノ、メトキシ、置換または無置換のモノ−C〜Cアルキルアミノ、または置換または無置換のジ−C〜Cアルキルアミノにより置換されていてもよく;
    15およびR18は、同一であって、Hおよび−CH からなる群から選択され;
    は、H、D、置換または無置換のC〜Cアルキル、−CD、置換または無置換のC〜Cハロアルキル、置換または無置換のC〜Cヘテロアルキル、置換または無置換のC〜Cシクロアルキル、置換または無置換のC〜Cヘテロシクロアルキル、置換または無置換のアリール、または置換または無置換のヘテロアリールであり;
    aおよびbは、それぞれ0であり;
    cおよびdは、それぞれ1である)であって、式(IV)の化合物が、少なくとも80%の立体化学純度を有する、式(IV)の構造を有する化合物、またはその薬学的に許容される塩もしくは溶媒和物。
  2. 環Pが、
    Figure 2020169171
    からなる群から選択されるヘテロアリールである、
    請求項1に記載の化合物、またはその薬学的に許容される塩もしくは溶媒和物。
  3. 環Pが、
    Figure 2020169171
    からなる群から選択されるヘテロアリールである、
    請求項1に記載の化合物、またはその薬学的に許容される塩もしくは溶媒和物。
  4. 15 がHであり、R 18 がHである、請求項1に記載の化合物、またはその薬学的に許容される塩もしくは溶媒和物。
  5. 15 が−CH であり、R 18 が−CH である、請求項1に記載の化合物、またはその薬学的に許容される塩もしくは溶媒和物。
  6. 11 、R 12 、R 13 、R 14 、R 16 およびR 17 は、それぞれHである、請求項1に記載の化合物、またはその薬学的に許容される塩もしくは溶媒和物。
  7. Figure 2020169171
    pは1、2、または3である、請求項1に記載の化合物、またはその薬学的に許容される塩もしくは溶媒和物。
  8. RがHであり;R15およびR18がHであり;およびpが2または3である、請求項7に記載の化合物、またはその薬学的に許容される塩もしくは溶媒和物。
  9. RがHであり;R15およびR18がHであり;およびpが2である、請求項7に記載の化合物、またはその薬学的に許容される塩もしくは溶媒和物。
  10. RがHであり;R15およびR18がHであり;およびpが3である、請求項7に記載の化合物、またはその薬学的に許容される塩もしくは溶媒和物。
  11. RがHであり;R15およびR18がCHであり;およびpが2または3である、請求項7に記載の化合物、またはその薬学的に許容される塩もしくは溶媒和物。
  12. RがHであり;R15およびR18がCHであり;およびpが2である、請求項7に記載の化合物、またはその薬学的に許容される塩もしくは溶媒和物。
  13. RがHであり;R15およびR18がCHであり;およびpが3である、請求項7に記載の化合物、またはその薬学的に許容される塩もしくは溶媒和物。
  14. 請求項1〜13のいずれか1項に記載の化合物、またはその薬学的に許容される塩もしくは溶媒和物、および薬学的に許容される担体または賦形剤を含む医薬組成物。
  15. 疾患または状態の処置のための医薬の調製における、請求項1〜13のいずれか1項に記載の化合物、またはその薬学的に許容される塩もしくは溶媒和物の使用。
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
US201762562927P 2017-09-25 2017-09-25
US201762562948P 2017-09-25 2017-09-25
US62/562,948 2017-09-25
US62/562,927 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 (ja) 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) US11162101B2 (ja)
EP (2) EP3661509A4 (ja)
JP (2) JP7312749B2 (ja)
KR (2) KR102636383B1 (ja)
CN (2) CN111499615B (ja)
AU (2) AU2018309187C1 (ja)
BR (2) BR112020002270A2 (ja)
CA (1) CA3072205A1 (ja)
GB (2) GB2587457B (ja)
IL (2) IL272413B (ja)
MX (2) MX2020001425A (ja)
MY (1) MY201938A (ja)
RU (1) RU2020105929A (ja)
SG (2) SG11202000980QA (ja)
WO (1) WO2019028440A1 (ja)
ZA (2) ZA202000695B (ja)

Families Citing this family (61)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2879995T3 (es) 2015-12-10 2021-11-23 Ptc Therapeutics Inc Métodos para el tratamiento de la enfermedad de Huntington
WO2018098446A1 (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
EA202090034A1 (ru) 2017-06-14 2020-04-16 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Способы модификации сплайсинга рнк
WO2019005980A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. METHODS OF TREATING HUNTINGTON'S DISEASE
BR112019027719A2 (pt) 2017-06-28 2020-07-28 Ptc Therapeutics, Inc. métodos para tratar a doença de huntington
GB2587457B (en) 2017-08-04 2022-06-01 Skyhawk Therapeutics Inc Methods and compositions for modulating splicing
KR20210005559A (ko) * 2018-03-27 2021-01-14 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료 화합물
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
US11685746B2 (en) 2018-06-27 2023-06-27 Ptc Therapeutics, Inc. Heteroaryl compounds for treating Huntington's disease
AU2019294478B2 (en) 2018-06-27 2023-03-23 Ptc Therapeutics, Inc. Heterocyclic and heteroaryl compounds for treating Huntington's disease
JP7476199B2 (ja) 2018-08-20 2024-04-30 ロジコン, インコーポレイテッド Scn1a脳症の治療のためのscn2aを標的とするアンチセンスオリゴヌクレオチド
WO2020154462A1 (en) * 2019-01-23 2020-07-30 Rogcon U.R., Inc. Antisense oligonucleotides targeting scn2a retained introns
EP3938352A4 (en) * 2019-02-04 2022-11-09 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICE
EP3920919A4 (en) * 2019-02-05 2023-03-29 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICE
KR20210135239A (ko) * 2019-02-05 2021-11-12 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
WO2020163409A1 (en) * 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
KR20210135241A (ko) 2019-02-05 2021-11-12 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
JP2022520051A (ja) * 2019-02-05 2022-03-28 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
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
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
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
MX2022005254A (es) 2019-11-01 2022-06-29 Novartis Ag El uso de un modulador de empalme para un tratamiento que retrasa la progresion de la enfermedad de huntington.
IL295953A (en) * 2020-02-28 2022-10-01 Remix Therapeutics Inc Pyridazine derivatives regulate nucleic acid splicing
IL295955A (en) * 2020-02-28 2022-10-01 Remix Therapeutics Inc Compounds and Methods for Splicing Regulation
EP4110775A1 (en) 2020-02-28 2023-01-04 Remix Therapeutics Inc. Thiophenyl derivatives useful for modulating nucleic acid splicing
US20230365566A1 (en) 2020-02-28 2023-11-16 Remix Therapeutics Inc. Heterocyclic amides and their use for modulating splicing
BR112022020337A2 (pt) 2020-04-08 2022-12-13 Remix Therapeutics Inc Compostos e métodos para modulação de splicing
WO2021207532A1 (en) 2020-04-08 2021-10-14 Remix Therapeutics Inc. Compounds and methods for modulating splicing
MX2022013856A (es) 2020-05-13 2023-04-05 Chdi Foundation Inc Moduladores htt para tratar la enfermedad de huntington.
JP2023532332A (ja) 2020-07-02 2023-07-27 リミックス セラピューティクス インコーポレイテッド 核酸のスプライシング及び増殖性疾患の処置のためのモジュレーターとしての2-(インダゾール-5-イル)-6-(ピペリジン-4-イル)-1,7-ナフチリジン誘導体及び関連する化合物
CA3182952A1 (en) 2020-07-02 2022-01-06 Dominic Reynolds 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 treatment of proliferative diseases
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
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
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
WO2022093835A1 (en) * 2020-10-26 2022-05-05 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
WO2023034827A1 (en) 2021-08-30 2023-03-09 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2023034811A1 (en) 2021-08-30 2023-03-09 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2023034812A1 (en) 2021-08-30 2023-03-09 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
WO2023034833A1 (en) 2021-08-30 2023-03-09 Remix Therapeutics Inc. Compounds and methods for modulating splicing
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
WO2023064879A1 (en) 2021-10-13 2023-04-20 Remix Therapeutics Inc. Compounds and methods for modulating nucleic acid splicing
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
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
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
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

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
ATE420351T1 (de) 2001-08-31 2009-01-15 Gen Probe Inc Sonden mit affinitätsverschiebung zur quantifizierung von analytenpolynukleotiden
US7135484B2 (en) 2002-08-14 2006-11-14 Abbott Laboratories Azabicyclic compounds are central nervous system active agents
DE60311853T2 (de) 2002-09-30 2007-06-21 Neurosearch A/S 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
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
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
JP4931823B2 (ja) 2004-10-18 2012-05-16 アムジエン・インコーポレーテツド チアジアゾール化合物及び使用方法
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
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
WO2006113704A2 (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
MX2008014672A (es) 2006-05-19 2009-03-09 Abbott Lab Derivados de alcano azabiciclico sustituidos con bicicloheterociclo fusionado activos en el sistema nervioso central.
CA2653769A1 (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
HUE028662T2 (en) 2007-10-26 2016-12-28 Academisch Ziekenhuis Leiden Preparations and methods for controlling muscle disorders
EP2231657A1 (en) 2007-11-21 2010-09-29 Abbott Laboratories Biaryl substituted azabicyclic alkane derivatives as nicotinic acetylcholine receptor activity modulators
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
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
EP2367824B1 (en) 2008-12-23 2016-03-23 AbbVie Inc. Anti-viral derivatives of pyrimidine
US9464287B2 (en) 2009-03-16 2016-10-11 Curna, Inc. Treatment of nuclear factor (erythroid-derived 2)-like 2 (NRF2) related diseases by inhibition of natural antisense transcript to NRF2
CN102625840A (zh) 2009-04-10 2012-08-01 肌肉学研究协会 用于治疗疾病的三环dna反义寡核苷酸、组合物和方法
KR20190037378A (ko) 2009-06-17 2019-04-05 바이오젠 엠에이 인코포레이티드 대상에게서 smn2 스플라이싱을 조정하기 위한 조성물 및 방법
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
US8940762B2 (en) * 2010-01-20 2015-01-27 Abbvie Inc. Methods for treating pain
SI2531492T1 (sl) 2010-02-05 2016-08-31 Heptares Therapeutics Limited Derivati 1,2,4-triazin-4-amina
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
AR081331A1 (es) 2010-04-23 2012-08-08 Cytokinetics Inc Amino- pirimidinas composiciones de las mismas y metodos para el uso de los mismos
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
ES2823350T3 (es) 2010-07-29 2021-05-06 Rigel Pharmaceuticals Inc Compuestos heterocíclicos que activan AMPK y métodos de uso de los mismos
EP2686021B1 (en) 2011-03-14 2018-08-15 NSE Products, Inc. Oral formulations for promoting cellular purification
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
CA2853722A1 (en) 2011-10-27 2013-05-02 Mayo Foundation For Medical Education And Research Inhibiting g protein coupled receptor 6 kinase polypeptides
KR102057351B1 (ko) 2011-12-30 2019-12-18 피티씨 테라퓨틱스, 인크. 척수성 근위축증을 치료하기 위한 화합물
JP6193888B2 (ja) 2012-01-26 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
WO2013117693A1 (en) 2012-02-10 2013-08-15 Glaxosmithkline Intellectual Property Development Limited Pde4 inhibitor for treating huntington's disease
DK2812004T3 (en) 2012-02-10 2018-10-15 Ptc Therapeutics Inc COMPOUNDS FOR TREATMENT OF SPINAL MUSCLE DROPHY
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
MY174339A (en) * 2012-08-13 2020-04-09 Novartis Ag 1,4-disubstituted pyridazine analogs and methods for treating smn-deficiency-related conditions
AU2013204200B2 (en) 2012-10-11 2016-10-20 Brandeis University Treatment of amyotrophic lateral sclerosis
EP2906696B2 (en) 2012-10-15 2022-12-14 Ionis Pharmaceuticals, Inc. Methods for modulating c9orf72 expression
JP6137336B2 (ja) * 2013-01-15 2017-05-31 ノバルティス アーゲー ナルコレプシーの処置のためのアルファ7ニコチン性受容体アゴニストの使用
WO2014111837A1 (en) * 2013-01-15 2014-07-24 Novartis Ag Use of alpha 7 nicotinic acetylcholine receptor agonists
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
EP3013345B1 (en) 2013-06-25 2017-11-22 F. Hoffmann-La Roche AG Compounds for treating spinal muscular atrophy
PL3027600T3 (pl) 2013-07-31 2022-08-08 Novartis Ag 1,4-dwupodstawione pochodne pirydazyny i ich zastosowanie do leczenia stanów związanych z niedoborem smn
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
CN105612152A (zh) 2013-08-26 2016-05-25 普渡制药公司 氮杂螺[4.5]癸烷衍生物及其用途
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
US10006027B2 (en) 2014-03-19 2018-06-26 Ionis 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
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
MX2017009408A (es) 2015-01-20 2017-10-02 Merial Inc Compuestos antihelminticos, composiciones y metodo para usar los mismos.
EP3053577A1 (en) 2015-02-09 2016-08-10 F. Hoffmann-La Roche AG Compounds for the treatment of cancer
EP3256126B1 (en) * 2015-02-09 2024-03-27 F. Hoffmann-La Roche AG Compounds for the treatment of cancer
EP3310169B1 (en) * 2015-05-30 2023-05-17 PTC Therapeutics, Inc. Methods for modulating rna splicing
PE20180573A1 (es) 2015-07-20 2018-04-04 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
KR20180056729A (ko) 2015-09-24 2018-05-29 글락소스미스클라인 인털렉츄얼 프로퍼티 (넘버 2) 리미티드 인돌아민 2,3-디옥시게나제의 조절제
WO2017062751A1 (en) 2015-10-08 2017-04-13 The Regents Of The University Of California Compounds and methods for promoting stress resistance
JP6923517B2 (ja) 2015-10-09 2021-08-18 ユニバーシティ・オブ・サザンプトン 遺伝子発現の調節及び脱制御されたタンパク質発現のスクリーニング
EP3183347A4 (en) * 2015-10-17 2018-04-18 Lifesplice Pharma LLC Splice modulating oligonucleotides and methods of use thereof
AU2016353961B2 (en) 2015-11-12 2019-08-29 F. Hoffmann-La Roche Ag Compositions for treating spinal muscular atrophy
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
ES2879995T3 (es) 2015-12-10 2021-11-23 Ptc Therapeutics Inc Métodos para el tratamiento de la enfermedad de Huntington
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
WO2017112956A1 (en) 2015-12-23 2017-06-29 Moonshot Pharma Llc Methods for inducing an immune response
US10787433B2 (en) 2016-01-15 2020-09-29 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).
AU2017223706A1 (en) 2016-02-23 2018-09-20 Indiana University Research & Technology Corporation Combination therapies for treatment of spinal muscular 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
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
US10577360B2 (en) 2016-04-12 2020-03-03 Esteve Pharmaceuticals, S.A. Arylamide derivatives having multimodal activity against pain
HUE051480T2 (hu) 2016-06-13 2021-03-01 Scholar Rock Inc Miosztatin-inhibitorok alkalmazása és kombinációs terápiák
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
WO2018098446A1 (en) 2016-11-28 2018-05-31 Ptc Therapeutics, Inc Methods for modulating rna splicing
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
WO2018204412A1 (en) 2017-05-01 2018-11-08 Thomas Jefferson University Systems-level analysis of tcga cancers reveals disease trna fragmentation patterns and associations with messenger rnas and repeat
EA202090034A1 (ru) 2017-06-14 2020-04-16 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Способы модификации сплайсинга рнк
BR112019027719A2 (pt) 2017-06-28 2020-07-28 Ptc Therapeutics, Inc. métodos para tratar a doença de huntington
WO2019005980A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. METHODS OF TREATING HUNTINGTON'S DISEASE
GB2587457B (en) 2017-08-04 2022-06-01 Skyhawk Therapeutics Inc Methods and compositions for modulating splicing
EP3686196A4 (en) 2017-09-20 2021-03-31 Hangzhou Innogate Pharma Co., Ltd. POLYCYCLIC CONNECTION AS IDO INHIBITOR AND / OR IDO HDAC DOUBLE INHIBITOR
SG11202002941WA (en) 2017-10-12 2020-04-29 Revolution Medicines Inc Pyridine, pyrazine, and triazine compounds as allosteric shp2 inhibitors
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
US10905707B2 (en) 2017-11-10 2021-02-02 University Of Massachusetts Compositions and methods for the treatment of expanded repeat-associated disorders
KR20210005559A (ko) 2018-03-27 2021-01-14 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료 화합물
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
MX2020014315A (es) 2018-06-27 2021-05-27 Ptc Therapeutics Inc Compuestos de heteroarilo para tratar la enfermedad de huntington.
CA3105174A1 (en) 2018-06-27 2020-01-02 Reborna Biosciences, Inc. Prophylactic or therapeutic agent for spinal muscular atrophy
US11685746B2 (en) 2018-06-27 2023-06-27 Ptc Therapeutics, Inc. Heteroaryl compounds for treating Huntington's disease
AU2019294478B2 (en) 2018-06-27 2023-03-23 Ptc Therapeutics, Inc. Heterocyclic and 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
US11129829B2 (en) * 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
US20230365566A1 (en) 2020-02-28 2023-11-16 Remix Therapeutics Inc. Heterocyclic amides and their use for modulating splicing
EP4110775A1 (en) 2020-02-28 2023-01-04 Remix Therapeutics Inc. Thiophenyl derivatives useful for modulating nucleic acid splicing
IL295955A (en) 2020-02-28 2022-10-01 Remix Therapeutics Inc Compounds and Methods for Splicing Regulation
IL295953A (en) 2020-02-28 2022-10-01 Remix Therapeutics Inc Pyridazine derivatives regulate nucleic acid splicing
BR112022020337A2 (pt) 2020-04-08 2022-12-13 Remix Therapeutics Inc Compostos e métodos para modulação de splicing
WO2021207532A1 (en) 2020-04-08 2021-10-14 Remix Therapeutics Inc. Compounds and methods for modulating splicing

Similar Documents

Publication Publication Date Title
JP2020169171A5 (ja)
JP2020530487A5 (ja)
JP2020507589A5 (ja)
JP2008505157A5 (ja)
JP2009536660A5 (ja)
JP2005533040A5 (ja)
JP2005523922A5 (ja)
JP2016121196A5 (ja)
JP2018515495A5 (ja)
JP2021500394A5 (ja)
JP2016510323A5 (ja)
JP2021046404A5 (ja)
JP2019524883A5 (ja)
JP2011509309A5 (ja)
JP2013010792A5 (ja)
RU2005128190A (ru) Ингибиторы фактора подавления миграции макрофагов (фпм) и способы их идентификации
JP2008511601A5 (ja)
JP2006514012A5 (ja)
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
JP2020529994A5 (ja)
JP2017522304A5 (ja)
JP2019526596A5 (ja)
JP2007514691A5 (ja)
JP2020532545A5 (ja)
JP2011509302A5 (ja)