EP3920919A4 - Verfahren und zusammensetzungen zur modulierung der spleissung - Google Patents

Verfahren und zusammensetzungen zur modulierung der spleissung 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Withdrawn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
English (en)
French (fr)
Other versions
EP3920919A1 (de
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/de
Publication of EP3920919A4 publication Critical patent/EP3920919A4/de
Withdrawn legal-status Critical Current

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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D451/00Heterocyclic 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
    • C07D451/14Heterocyclic 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/439Heterocyclic 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/468-Azabicyclo [3.2.1] octane; Derivatives thereof, e.g. atropine, cocaine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/53Heterocyclic 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D451/00Heterocyclic 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
    • C07D451/02Heterocyclic 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
    • C07D451/04Heterocyclic 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D491/00Heterocyclic 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
    • C07D491/02Heterocyclic 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
    • C07D491/04Ortho-condensed systems
    • C07D491/056Ortho-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 Verfahren und zusammensetzungen zur modulierung der spleissung Withdrawn EP3920919A4 (de)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962801506P 2019-02-05 2019-02-05
US201962801486P 2019-02-05 2019-02-05
US201962801502P 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 (de) 2021-12-15
EP3920919A4 true EP3920919A4 (de) 2023-03-29

Family

ID=71947086

Family Applications (1)

Application Number Title Priority Date Filing Date
EP20753044.5A Withdrawn EP3920919A4 (de) 2019-02-05 2020-02-04 Verfahren und zusammensetzungen zur modulierung der spleissung

Country Status (5)

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

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102650496B1 (ko) 2018-10-31 2024-03-26 길리애드 사이언시즈, 인코포레이티드 Hpk1 억제제로서의 치환된 6-아자벤즈이미다졸 화합물
CN117105933A (zh) 2018-10-31 2023-11-24 吉利德科学公司 具有hpk1抑制活性的取代的6-氮杂苯并咪唑化合物
JP2022521467A (ja) 2019-02-05 2022-04-08 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
JP2022519323A (ja) 2019-02-06 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
WO2020237025A1 (en) 2019-05-23 2020-11-26 Gilead Sciences, Inc. Substituted exo-methylene-oxindoles which are hpk1/map4k1 inhibitors
CN114650822A (zh) 2019-11-01 2022-06-21 诺华股份有限公司 剪接调节子用于减慢亨廷顿氏病进展的治疗的用途
JP2023532623A (ja) 2020-05-13 2023-07-31 シーエイチディーアイ ファウンデーション,インコーポレーテッド ハンチントン病を処置するためのhttモジュレータ
WO2022060944A1 (en) * 2020-09-16 2022-03-24 Skyhawk Therapeutics, Inc. Compositions for modulating splicing

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
CN107428729B (zh) * 2015-02-09 2021-07-16 豪夫迈·罗氏有限公司 用于治疗癌症的化合物
CN112272666A (zh) * 2018-04-10 2021-01-26 斯基霍克疗法公司 用于治疗癌症的化合物

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
EP3920919A1 (de) 2021-12-15
CN113645970A (zh) 2021-11-12
WO2020163401A1 (en) 2020-08-13
JP2022519312A (ja) 2022-03-22
KR20210134657A (ko) 2021-11-10

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