EP3920919A4 - Methods and compositions for modulating splicing - Google Patents

Methods and compositions for modulating splicing Download PDF

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Publication number
EP3920919A4
EP3920919A4 EP20753044.5A EP20753044A EP3920919A4 EP 3920919 A4 EP3920919 A4 EP 3920919A4 EP 20753044 A EP20753044 A EP 20753044A EP 3920919 A4 EP3920919 A4 EP 3920919A4
Authority
EP
European Patent Office
Prior art keywords
compositions
methods
modulating splicing
splicing
modulating
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.)
Withdrawn
Application number
EP20753044.5A
Other languages
German (de)
French (fr)
Other versions
EP3920919A1 (en
Inventor
Michael Luzzio
Brian Lucas
Daniel Brian HORNE
Tiansheng Wang
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Skyhawk Therapeutics Inc
Original Assignee
Skyhawk Therapeutics Inc
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 Skyhawk Therapeutics Inc filed Critical Skyhawk Therapeutics Inc
Publication of EP3920919A1 publication Critical patent/EP3920919A1/en
Publication of EP3920919A4 publication Critical patent/EP3920919A4/en
Withdrawn legal-status Critical Current

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D451/00—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof
    • C07D451/14—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing 9-azabicyclo [3.3.1] nonane ring systems, e.g. granatane, 2-aza-adamantane; Cyclic acetals thereof
    • 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
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/439—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom the ring forming part of a bridged ring system, e.g. quinuclidine
    • 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
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/46—8-Azabicyclo [3.2.1] octane; Derivatives thereof, e.g. atropine, cocaine
    • 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
    • A61K31/53—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with three nitrogens as the only ring hetero atoms, e.g. chlorazanil, melamine
    • 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
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D451/00—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof
    • C07D451/02—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing not further condensed 8-azabicyclo [3.2.1] octane or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane; Cyclic acetals thereof
    • C07D451/04—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing not further condensed 8-azabicyclo [3.2.1] octane or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane; Cyclic acetals thereof with hetero atoms directly attached in position 3 of the 8-azabicyclo [3.2.1] octane or in position 7 of the 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D491/00—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00
    • C07D491/02—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00 in which the condensed system contains two hetero rings
    • C07D491/04—Ortho-condensed systems
    • C07D491/056—Ortho-condensed systems with two or more oxygen atoms as ring hetero atoms in the oxygen-containing ring

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Emergency Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
EP20753044.5A 2019-02-05 2020-02-04 Methods and compositions for modulating splicing Withdrawn EP3920919A4 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962801486P 2019-02-05 2019-02-05
US201962801502P 2019-02-05 2019-02-05
US201962801506P 2019-02-05 2019-02-05
PCT/US2020/016671 WO2020163401A1 (en) 2019-02-05 2020-02-04 Methods and compositions for modulating splicing

Publications (2)

Publication Number Publication Date
EP3920919A1 EP3920919A1 (en) 2021-12-15
EP3920919A4 true EP3920919A4 (en) 2023-03-29

Family

ID=71947086

Family Applications (1)

Application Number Title Priority Date Filing Date
EP20753044.5A Withdrawn EP3920919A4 (en) 2019-02-05 2020-02-04 Methods and compositions for modulating splicing

Country Status (5)

Country Link
EP (1) EP3920919A4 (en)
JP (1) JP2022519312A (en)
KR (1) KR20210134657A (en)
CN (1) CN113645970A (en)
WO (1) WO2020163401A1 (en)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020092528A1 (en) 2018-10-31 2020-05-07 Gilead Sciences, Inc. Substituted 6-azabenzimidazole compounds having hpk1 inhibitory activity
US11203591B2 (en) 2018-10-31 2021-12-21 Gilead Sciences, Inc. Substituted 6-azabenzimidazole compounds
WO2020163248A1 (en) 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
CN114126613A (en) 2019-02-05 2022-03-01 斯基霍克疗法公司 Methods and compositions for modulating splicing
EP3920915A4 (en) 2019-02-05 2022-10-05 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
CN113661162A (en) 2019-02-06 2021-11-16 斯基霍克疗法公司 Methods and compositions for modulating splicing
WO2020163544A1 (en) 2019-02-06 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
EP3972695A1 (en) 2019-05-23 2022-03-30 Gilead Sciences, Inc. Substituted exo-methylene-oxindoles which are hpk1/map4k1 inhibitors
AU2020377204A1 (en) 2019-11-01 2022-06-02 Novartis Ag The use of a splicing modulator for a treatment slowing progression of huntington's disease
BR112022023025A2 (en) 2020-05-13 2023-03-28 Chdi Foundation Inc HTT MODULATORS TO TREAT HUNTINGTON'S DISEASE
WO2022060944A1 (en) * 2020-09-16 2022-03-24 Skyhawk Therapeutics, Inc. Compositions for modulating splicing
AU2021356130A1 (en) 2020-10-08 2023-05-18 Redona Therapeutics, Inc. Benzo[h]quinazolin-4-amine and thieno[3,2-h]quinazolin-4-amine derivatives for the treatment of cancer
US12281116B2 (en) 2021-11-17 2025-04-22 Chdi Foundation, Inc. HTT modulators for treating Huntington's disease

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007137030A2 (en) * 2006-05-19 2007-11-29 Abbott Laboratories Cns active fused bicycloheterocycle substituted azabicyclic alkane derivatives
WO2011095625A1 (en) * 2010-02-05 2011-08-11 Heptares Therapeutics Limited 1,2,4-triazine-4-amine derivatives
WO2017100726A1 (en) * 2015-12-10 2017-06-15 Ptc Therapeutics, Inc. Methods for treatng huntington's disease
WO2018232039A1 (en) * 2017-06-14 2018-12-20 Ptc Therapeutics, Inc. Methods for modifying rna splicing
WO2019028440A1 (en) * 2017-08-04 2019-02-07 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
WO2020163248A1 (en) * 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
WO2020163382A1 (en) * 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016128343A1 (en) * 2015-02-09 2016-08-18 F. Hoffmann-La Roche Ag Compounds for the treatment of cancer
CN112272666A (en) * 2018-04-10 2021-01-26 斯基霍克疗法公司 Compounds for the treatment of cancer

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007137030A2 (en) * 2006-05-19 2007-11-29 Abbott Laboratories Cns active fused bicycloheterocycle substituted azabicyclic alkane derivatives
WO2011095625A1 (en) * 2010-02-05 2011-08-11 Heptares Therapeutics Limited 1,2,4-triazine-4-amine derivatives
WO2017100726A1 (en) * 2015-12-10 2017-06-15 Ptc Therapeutics, Inc. Methods for treatng huntington's disease
WO2018232039A1 (en) * 2017-06-14 2018-12-20 Ptc Therapeutics, Inc. Methods for modifying rna splicing
WO2019028440A1 (en) * 2017-08-04 2019-02-07 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
WO2020163248A1 (en) * 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
WO2020163382A1 (en) * 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
See also references of WO2020163401A1 *

Also Published As

Publication number Publication date
WO2020163401A1 (en) 2020-08-13
EP3920919A1 (en) 2021-12-15
KR20210134657A (en) 2021-11-10
CN113645970A (en) 2021-11-12
JP2022519312A (en) 2022-03-22

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