MX2017009295A - Compuestos para mejorar el empalme del acido ribonucleico mensajero (arnm). - Google Patents

Compuestos para mejorar el empalme del acido ribonucleico mensajero (arnm).

Info

Publication number
MX2017009295A
MX2017009295A MX2017009295A MX2017009295A MX2017009295A MX 2017009295 A MX2017009295 A MX 2017009295A MX 2017009295 A MX2017009295 A MX 2017009295A MX 2017009295 A MX2017009295 A MX 2017009295A MX 2017009295 A MX2017009295 A MX 2017009295A
Authority
MX
Mexico
Prior art keywords
compounds
mrna splicing
improving mrna
improving
methods
Prior art date
Application number
MX2017009295A
Other languages
English (en)
Inventor
Zhang Wei
Johnson Graham
Marugan Juan
A Slaugenhaupt Susan
D Paquette William
Original Assignee
Massachusetts Gen Hospital
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 Massachusetts Gen Hospital filed Critical Massachusetts Gen Hospital
Publication of MX2017009295A publication Critical patent/MX2017009295A/es

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D473/00—Heterocyclic compounds containing purine ring systems
    • C07D473/26—Heterocyclic compounds containing purine ring systems with an oxygen, sulphur, or nitrogen atom directly attached in position 2 or 6, but not in both
    • C07D473/32—Nitrogen atom
    • C07D473/34—Nitrogen atom attached in position 6, e.g. adenine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A61K31/52—Purines, e.g. adenine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D405/00—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom
    • C07D405/02—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings
    • C07D405/12—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings linked by a chain containing hetero atoms as chain links
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D417/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00
    • C07D417/02—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00 containing two hetero rings
    • C07D417/12—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00 containing two hetero rings linked by a chain containing hetero atoms as chain links
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D471/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
    • C07D471/04—Ortho-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D473/00—Heterocyclic compounds containing purine ring systems
    • C07D473/02—Heterocyclic compounds containing purine ring systems with oxygen, sulphur, or nitrogen atoms directly attached in positions 2 and 6
    • C07D473/16—Heterocyclic compounds containing purine ring systems with oxygen, sulphur, or nitrogen atoms directly attached in positions 2 and 6 two nitrogen atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
    • C07D487/04—Ortho-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D495/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms
    • C07D495/02—Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms in which the condensed system contains two hetero rings
    • C07D495/04—Ortho-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D513/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for in groups C07D463/00, C07D477/00 or C07D499/00 - C07D507/00
    • C07D513/02—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for in groups C07D463/00, C07D477/00 or C07D499/00 - C07D507/00 in which the condensed system contains two hetero rings
    • C07D513/04—Ortho-condensed systems

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Neurosurgery (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)

Abstract

Se proveen compuestos útiles para mejorar el empalme del ARNm en una célula. Los compuestos ejemplares provistos en la presente son útiles para mejorar el empalme de ARNm en genes que comprenden por lo menos un exón que termina en la secuencia de nucleótidos CAA. También se proveen métodos para preparar los compuestos y métodos de tratamiento de enfermedades del sistema nervioso central.
MX2017009295A 2015-01-16 2016-01-15 Compuestos para mejorar el empalme del acido ribonucleico mensajero (arnm). MX2017009295A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562104547P 2015-01-16 2015-01-16
US201562180380P 2015-06-16 2015-06-16
PCT/US2016/013553 WO2016115434A1 (en) 2015-01-16 2016-01-15 Compounds for improving mrna splicing

Publications (1)

Publication Number Publication Date
MX2017009295A true MX2017009295A (es) 2018-03-07

Family

ID=56406434

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2017009295A MX2017009295A (es) 2015-01-16 2016-01-15 Compuestos para mejorar el empalme del acido ribonucleico mensajero (arnm).
MX2022015737A MX2022015737A (es) 2015-01-16 2017-07-14 Compuestos para mejorar el empalme del acido ribonucleico mensajero (arnm).

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2022015737A MX2022015737A (es) 2015-01-16 2017-07-14 Compuestos para mejorar el empalme del acido ribonucleico mensajero (arnm).

Country Status (16)

Country Link
US (3) US10676475B2 (es)
EP (2) EP4115882A1 (es)
AU (2) AU2016206589B2 (es)
BR (1) BR112017015019A2 (es)
CA (2) CA2973949C (es)
CL (2) CL2017001823A1 (es)
DK (1) DK3244891T3 (es)
EA (1) EA037663B1 (es)
ES (1) ES2928714T3 (es)
HU (1) HUE059891T2 (es)
IL (2) IL253291B (es)
MX (2) MX2017009295A (es)
PL (1) PL3244891T3 (es)
PT (1) PT3244891T (es)
TW (2) TWI727939B (es)
WO (1) WO2016115434A1 (es)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EA037663B1 (ru) * 2015-01-16 2021-04-28 Те Дженерал Хоспитал Корпорейшн СОЕДИНЕНИЯ ДЛЯ УЛУЧШЕНИЯ СПЛАЙСИНГА мРНК
WO2017100726A1 (en) 2015-12-10 2017-06-15 Ptc Therapeutics, Inc. Methods for treatng huntington's disease
CN110300590A (zh) * 2016-10-21 2019-10-01 林伯士拉克许米公司 Tyk2抑制剂及其用途
EP3583951A4 (en) 2017-02-20 2021-05-05 Kyoto University PHARMACEUTICAL COMPOSITION AND TREATMENT METHODS FOR GENETIC DISEASES ASSOCIATED WITH SPICE ANOMALIES
AU2018282154B2 (en) 2017-06-05 2022-04-07 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
CA3067592A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. Methods for treating huntington's disease
WO2019005980A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. METHODS OF TREATING HUNTINGTON'S DISEASE
SG11202009212WA (en) 2018-03-27 2020-10-29 Ptc Therapeutics Inc Compounds for treating huntington's disease
KR20210042265A (ko) 2018-06-27 2021-04-19 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료를 위한 헤테로사이클릭 및 헤테로아릴 화합물
CA3114722A1 (en) * 2018-10-01 2020-04-09 Genzyme Corporation Thieno[3,2-b]pyridine derivatives as udp glycosyltransferase inhibitors and methods of use
MX2021009667A (es) * 2019-02-13 2021-12-10 Ptc Therapeutics Inc Compuestos de tioeno[3,2-b] piridin-7-amina para tratar la disautonomía familiar.
WO2020167624A1 (en) 2019-02-13 2020-08-20 Ptc Therapeutics, Inc. Pyrrolo[2,3-d]pyrimidine compounds for treating familial dysautonomia
WO2020180770A1 (en) * 2019-03-01 2020-09-10 Revolution Medicines, Inc. Bicyclic heterocyclyl compounds and uses thereof
CN113767100A (zh) * 2019-03-01 2021-12-07 锐新医药公司 双环杂芳基化合物及其用途
CN114901662A (zh) 2019-11-08 2022-08-12 锐新医药公司 双环杂芳基化合物及其用途
CN115209955A (zh) 2019-12-12 2022-10-18 Ptc医疗公司 治疗家族性自主神经功能障碍的化合物
WO2021138678A1 (en) 2020-01-02 2021-07-08 The General Hospital Corporation Rna splicing modulation
CN111777612B (zh) * 2020-06-05 2022-04-19 广东达元绿洲食品安全科技股份有限公司 一种6-苄基腺嘌呤半抗原、人工抗原及其在免疫检测中的应用
WO2022169864A1 (en) * 2021-02-05 2022-08-11 Ptc Therapeutics Inc. Methods for treating spinocerebellar ataxia type 3
CN113151296B (zh) * 2021-03-22 2022-09-13 云南中烟工业有限责任公司 一种烟草热激蛋白相关的基因及其应用

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3041340A (en) 1962-06-26 Method of preparing substituted
US3037980A (en) * 1955-08-18 1962-06-05 Burroughs Wellcome Co Pyrrolopyrimidine vasodilators and method of making them
GB9613736D0 (en) 1996-07-01 1996-09-04 Hancock Tony Transport cradle for a skid-supported helicopter
HUP0100931A3 (en) 1998-02-26 2002-08-28 Aventis Pharma Inc 6,9-disubstituted 2-[trans-(-aminocyclohexyl)amino]purines and pharmaceutical compositions containing them
CZ294535B6 (cs) 2001-08-02 2005-01-12 Ústav Experimentální Botaniky Avčr Heterocyklické sloučeniny na bázi N6-substituovaného adeninu, způsoby jejich přípravy, jejich použití pro přípravu léčiv, kosmetických přípravků a růstových regulátorů, farmaceutické přípravky, kosmetické přípravky a růstové regulátory tyto sloučeniny obsahující
WO2005003290A2 (en) 2002-09-10 2005-01-13 The Regents Of The University Of California Methods and devices for determining a cell characteristic, and applications employing same
AU2004278773B2 (en) 2003-10-03 2011-05-19 The General Hospital Corporation Methods for altering mRNA splicing and treating familial dysautonomia and other mechanistically related disorders
US20050187389A1 (en) 2004-01-13 2005-08-25 Ambit Biosciences Corporation Pyrrolopyrimidine derivatives and analogs and their use in the treatment and prevention of diseases
GB0526246D0 (en) 2005-12-22 2006-02-01 Novartis Ag Organic compounds
CN101379061B (zh) 2006-02-02 2016-06-15 千禧药品公司 E1活化酶抑制剂
EP1889847A1 (en) * 2006-07-10 2008-02-20 DeveloGen Aktiengesellschaft Pyrrolopyrimidines for pharmaceutical compositions
NZ579834A (en) 2007-03-23 2012-03-30 Amgen Inc Heterocyclic compounds as selective inhibitors of PI3K activity
GB0706632D0 (en) 2007-04-04 2007-05-16 Cyclacel Ltd New purine derivatives
CA2709784A1 (en) 2007-12-21 2009-07-09 University Of Rochester Method for altering the lifespan of eukaryotic organisms
AU2009238590A1 (en) * 2008-04-22 2009-10-29 Portola Pharmaceuticals, Inc. Inhibitors of protein kinases
BRPI0919488A2 (pt) * 2008-09-30 2015-12-01 Astrazeneca Ab composto, uso de um composto, método para tratar câncer em um animal de sangue quente, composição farmacêutica, e, processo para preparar um composto
EP2393814A1 (en) * 2009-02-09 2011-12-14 SuperGen, Inc. Pyrrolopyrimidinyl axl kinase inhibitors
WO2010118367A2 (en) 2009-04-10 2010-10-14 Progenics Pharmaceuticals, Inc. Antiviral pyrimidines
WO2011041655A1 (en) * 2009-10-01 2011-04-07 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Quinazolin-4-amine derivatives; and methods of use
JP2011136925A (ja) * 2009-12-28 2011-07-14 Dainippon Sumitomo Pharma Co Ltd 含窒素二環性化合物
US20130072506A1 (en) 2011-09-16 2013-03-21 Lenka ZAHAJSKA 6,8-disubstituted purine compositions
US9278987B2 (en) * 2011-11-18 2016-03-08 Sarepta Therapeutics, Inc. Functionally-modified oligonucleotides and subunits thereof
CZ306894B6 (cs) 2013-02-08 2017-08-30 Univerzita PalackĂ©ho v Olomouci 2-Substituované-6-biarylmethylamino-9-cyklopentyl-9H-purinové deriváty, jejich použití jako léčiva a farmaceutické přípravky tyto sloučeniny obsahující
WO2014124458A1 (en) 2013-02-11 2014-08-14 The Regents Of The University Of California Compositions and methods for treating neurodegenerative diseases
US9938279B2 (en) 2013-04-09 2018-04-10 Energenesis Biomedical Co., Ltd Method for treating disease or condition susceptible to amelioration by AMPK activators and compounds of formula which are useful to activate AMP-activated protein kinase (AMPK)
EP3020829B1 (en) 2013-07-12 2021-11-17 Kyoto University Pharmaceutical composition capable of inhibiting abnormal splicing causative of onset or progress of a genetic disease
AU2015217152A1 (en) 2014-02-11 2016-08-11 Mitokinin, Inc. Compositions and methods using the same for treatment of neurodegenerative and mitochondrial disease
WO2016011394A1 (en) 2014-07-18 2016-01-21 The General Hospital Corporation Imaging agents for neural flux
EA037663B1 (ru) * 2015-01-16 2021-04-28 Те Дженерал Хоспитал Корпорейшн СОЕДИНЕНИЯ ДЛЯ УЛУЧШЕНИЯ СПЛАЙСИНГА мРНК

Also Published As

Publication number Publication date
EA037663B1 (ru) 2021-04-28
ES2928714T3 (es) 2022-11-22
TWI727939B (zh) 2021-05-21
CA3199430A1 (en) 2016-07-21
AU2016206589B2 (en) 2020-09-10
US20200283441A1 (en) 2020-09-10
HUE059891T2 (hu) 2023-01-28
CL2019002167A1 (es) 2019-12-13
AU2016206589A1 (en) 2017-07-27
US11702417B2 (en) 2023-07-18
EP3244891A1 (en) 2017-11-22
PL3244891T3 (pl) 2022-12-27
IL270945A (en) 2020-01-30
AU2020277224A1 (en) 2020-12-24
EP4115882A1 (en) 2023-01-11
EA201791624A1 (ru) 2018-06-29
DK3244891T3 (da) 2022-10-24
EP3244891B9 (en) 2022-11-16
CL2017001823A1 (es) 2018-04-13
US20230279007A1 (en) 2023-09-07
EP3244891B1 (en) 2022-07-20
US20180118748A1 (en) 2018-05-03
US10676475B2 (en) 2020-06-09
IL253291A0 (en) 2017-09-28
ES2928714T9 (es) 2022-11-29
EP3244891A4 (en) 2018-12-19
TW202204345A (zh) 2022-02-01
AU2020277224B2 (en) 2022-12-22
CA2973949C (en) 2023-07-11
PT3244891T (pt) 2022-10-20
MX2022015737A (es) 2023-01-18
CA2973949A1 (en) 2016-07-21
BR112017015019A2 (pt) 2018-01-30
IL253291B (en) 2021-10-31
TW201636341A (zh) 2016-10-16
WO2016115434A1 (en) 2016-07-21

Similar Documents

Publication Publication Date Title
MX2022015737A (es) Compuestos para mejorar el empalme del acido ribonucleico mensajero (arnm).
IL260959A (en) Gene therapy for the treatment of mucoid polysaccharomyces type i
IL262304A (en) Methods for delivering single-stranded RNA
HK1243764A1 (zh) 用於富集循環腫瘤dna的方法
IL265953A (en) Gene insertion is targeted for improved immune cell therapy
EA201891192A1 (ru) Средства и способы лечения миопатий, связанных с титином, и других титинопатий
IL286868A (en) Compounds and methods that monitor the expression of genes in the central nervous system
AU2019268074A1 (en) Methods for treating hepcidin-mediated disorders
IL262211B1 (en) Gene therapy for the treatment of type ii mucositis
GB2593353B (en) Methods for targeted insertion of DNA in genes
EP3283502A4 (en) METHODS FOR REACTIVATION OF GENES ON INACTIVE X CHROMOSOME
HK1254397A1 (zh) 通過t細胞療法治療cmv視網膜炎的方法
EP3321354A4 (en) Method for inducing oligodendrocyte precursor cells from oct4-induced human somatic cells through direct reprogramming
TW201613975A (en) Methods for treating multiple myeloma
HK1253299A1 (zh) 一種通過基因治療治療眼疾病的改進方法
MX2018005872A (es) Profarmacos de acido nucleico.
MX2022001801A (es) Secuencias recombinantes de cromosoma b de maiz y sus usos.
SG11201704744QA (en) Methods and kits for purifying plasmid dna
EP3159403A4 (en) Method for preparing dendritic cells with increased specific gene expression, and composition for treating or preventing autoimmune diseases, containing dendritic cells prepared using same
EP3393479A4 (en) METHOD FOR THE TREATMENT OF CANCER BY INHIBITION OF DNA REPAIR PROTEINS USING ANTISENSE-BASED THERAPIES
SG11202106810UA (en) Methods for targeted complementary dna enrichment
IL279257A (en) Method of enhancing glucose levels in the central nervous system
IL267231A (en) Methods of treating diseases associated with ilc2 cells
IL262951A (en) Methods of drug therapy associated with ilc3 cells
EA202190500A3 (ru) СОЕДИНЕНИЯ ДЛЯ УЛУЧШЕНИЯ СПЛАЙСИНГА мРНК