CL2017001823A1 - Compuestos para mejorar el empalme de marn - Google Patents

Compuestos para mejorar el empalme de marn

Info

Publication number
CL2017001823A1
CL2017001823A1 CL2017001823A CL2017001823A CL2017001823A1 CL 2017001823 A1 CL2017001823 A1 CL 2017001823A1 CL 2017001823 A CL2017001823 A CL 2017001823A CL 2017001823 A CL2017001823 A CL 2017001823A CL 2017001823 A1 CL2017001823 A1 CL 2017001823A1
Authority
CL
Chile
Prior art keywords
compounds
marn
improve
splicing
methods
Prior art date
Application number
CL2017001823A
Other languages
English (en)
Inventor
Wei Zhang
Juan MARUGAN
Susan A Slaugenhaupt
Graham Johnson
William D Paquette
Original Assignee
Massachusetts Gen Hospital
United States Dept Of Health And Human Services
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, United States Dept Of Health And Human Services filed Critical Massachusetts Gen Hospital
Publication of CL2017001823A1 publication Critical patent/CL2017001823A1/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

<p>SE PROPORCIONAN COMPUESTOS EFICACES PARA MEJORAR EL EMPALME DE MARN EN UNA CÉLULA. COMPUESTOS EJEMPLARES QUE SE PROPORCIONAN EN LA PRESENTE SON EFICACES PARA MEJORAR EL EMPALME DE MARN EN GENES QUE COMPRENDEN AL MENOS UN EXÓN QUE TERMINA EN LA SECUENCIA DE NUCLEÓTIDOS CAA. SE PROPORCIONAN MÉTODOS PARA PREPARAR LOS COMPUESTOS Y MÉTODOS PARA TRATAR ENFERMEDADES DEL SISTEMA NERVIOSO CENTRAL.</p>
CL2017001823A 2015-01-16 2017-07-13 Compuestos para mejorar el empalme de marn CL2017001823A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562104547P 2015-01-16 2015-01-16
US201562180380P 2015-06-16 2015-06-16

Publications (1)

Publication Number Publication Date
CL2017001823A1 true CL2017001823A1 (es) 2018-04-13

Family

ID=56406434

Family Applications (2)

Application Number Title Priority Date Filing Date
CL2017001823A CL2017001823A1 (es) 2015-01-16 2017-07-13 Compuestos para mejorar el empalme de marn
CL2019002167A CL2019002167A1 (es) 2015-01-16 2019-08-01 Compuesto de la fórmula (ic) o una sal farmacéuticamente aceptable del mismo, o una composición farmacéutica que comprende dicho compuesto o una sal farmacéuticamente aceptable del mismo, y al menos un portador farmacéuticamente aceptable, y su uso en el tratamiento de disautonomía familiar al mejorar el empalme de marn. (divisional solicitud 201701823.)

Family Applications After (1)

Application Number Title Priority Date Filing Date
CL2019002167A CL2019002167A1 (es) 2015-01-16 2019-08-01 Compuesto de la fórmula (ic) o una sal farmacéuticamente aceptable del mismo, o una composición farmacéutica que comprende dicho compuesto o una sal farmacéuticamente aceptable del mismo, y al menos un portador farmacéuticamente aceptable, y su uso en el tratamiento de disautonomía familiar al mejorar el empalme de marn. (divisional solicitud 201701823.)

Country Status (16)

Country Link
US (3) US10676475B2 (es)
EP (2) EP4115882A1 (es)
AU (2) AU2016206589B2 (es)
BR (1) BR112017015019A2 (es)
CA (2) CA3199430A1 (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) TW202204345A (es)
WO (1) WO2016115434A1 (es)

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Also Published As

Publication number Publication date
WO2016115434A1 (en) 2016-07-21
PL3244891T3 (pl) 2022-12-27
IL253291A0 (en) 2017-09-28
TW202204345A (zh) 2022-02-01
TW201636341A (zh) 2016-10-16
US20200283441A1 (en) 2020-09-10
AU2016206589A1 (en) 2017-07-27
CA3199430A1 (en) 2016-07-21
US20230279007A1 (en) 2023-09-07
BR112017015019A2 (pt) 2018-01-30
EP4115882A1 (en) 2023-01-11
ES2928714T9 (es) 2022-11-29
EP3244891A1 (en) 2017-11-22
EP3244891B1 (en) 2022-07-20
MX2022015737A (es) 2023-01-18
CA2973949C (en) 2023-07-11
US10676475B2 (en) 2020-06-09
CL2019002167A1 (es) 2019-12-13
PT3244891T (pt) 2022-10-20
TWI727939B (zh) 2021-05-21
ES2928714T3 (es) 2022-11-22
AU2020277224A1 (en) 2020-12-24
EA037663B1 (ru) 2021-04-28
HUE059891T2 (hu) 2023-01-28
US11702417B2 (en) 2023-07-18
EP3244891B9 (en) 2022-11-16
EP3244891A4 (en) 2018-12-19
EA201791624A1 (ru) 2018-06-29
MX2017009295A (es) 2018-03-07
DK3244891T3 (da) 2022-10-24
IL253291B (en) 2021-10-31
AU2016206589B2 (en) 2020-09-10
IL270945A (en) 2020-01-30
AU2020277224B2 (en) 2022-12-22
CA2973949A1 (en) 2016-07-21
US20180118748A1 (en) 2018-05-03

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