CN114007614A - 用于调节剪接的方法和组合物 - Google Patents

用于调节剪接的方法和组合物 Download PDF

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Publication number
CN114007614A
CN114007614A CN202080026934.6A CN202080026934A CN114007614A CN 114007614 A CN114007614 A CN 114007614A CN 202080026934 A CN202080026934 A CN 202080026934A CN 114007614 A CN114007614 A CN 114007614A
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China
Prior art keywords
substituted
unsubstituted
pharmaceutically acceptable
hydrogen
alkyl
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Pending
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CN202080026934.6A
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English (en)
Chinese (zh)
Inventor
M·卢奇欧
B·卢卡斯
D·B·霍内
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Skihawk Therapy
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Skihawk Therapy
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Priority to CN202411409049.2A priority Critical patent/CN119320388A/zh
Publication of CN114007614A publication Critical patent/CN114007614A/zh
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D403/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00
    • C07D403/14—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing three or more hetero rings
    • 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
    • C07D471/08—Bridged systems
    • 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
    • C07D451/06—Oxygen atoms
    • 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
    • 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
    • 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
    • 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
    • C07D491/08—Bridged 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
    • C07B—GENERAL METHODS OF ORGANIC CHEMISTRY; APPARATUS THEREFOR
    • C07B2200/00—Indexing scheme relating to specific properties of organic compounds
    • C07B2200/05—Isotopically modified compounds, e.g. labelled

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Engineering & Computer Science (AREA)
  • Emergency Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
CN202080026934.6A 2019-02-04 2020-02-03 用于调节剪接的方法和组合物 Pending CN114007614A (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN202411409049.2A CN119320388A (zh) 2019-02-04 2020-02-03 用于调节剪接的方法和组合物

Applications Claiming Priority (19)

Application Number Priority Date Filing Date Title
US201962800779P 2019-02-04 2019-02-04
US201962800691P 2019-02-04 2019-02-04
US201962800720P 2019-02-04 2019-02-04
US62/800,720 2019-02-04
US62/800,691 2019-02-04
US62/800,779 2019-02-04
US201962801206P 2019-02-05 2019-02-05
US201962801236P 2019-02-05 2019-02-05
US201962801208P 2019-02-05 2019-02-05
US201962801212P 2019-02-05 2019-02-05
US201962801231P 2019-02-05 2019-02-05
US201962801387P 2019-02-05 2019-02-05
US62/801,208 2019-02-05
US62/801,231 2019-02-05
US62/801,387 2019-02-05
US62/801,206 2019-02-05
US62/801,236 2019-02-05
US62/801,212 2019-02-05
PCT/US2020/016460 WO2020163248A1 (en) 2019-02-04 2020-02-03 Methods and compositions for modulating splicing

Related Child Applications (1)

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CN202411409049.2A Division CN119320388A (zh) 2019-02-04 2020-02-03 用于调节剪接的方法和组合物

Publications (1)

Publication Number Publication Date
CN114007614A true CN114007614A (zh) 2022-02-01

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CN202080026934.6A Pending CN114007614A (zh) 2019-02-04 2020-02-03 用于调节剪接的方法和组合物
CN202411409049.2A Pending CN119320388A (zh) 2019-02-04 2020-02-03 用于调节剪接的方法和组合物

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EP (1) EP3920917A4 (https=)
JP (1) JP7682096B2 (https=)
KR (1) KR102925387B1 (https=)
CN (2) CN114007614A (https=)
WO (1) WO2020163248A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2026067767A1 (zh) * 2024-09-30 2026-04-02 普罗思特(南京)生物科技有限公司 取代的哒嗪衍生物及其应用

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TWI721623B (zh) 2018-10-31 2021-03-11 美商基利科學股份有限公司 經取代之6-氮雜苯并咪唑化合物
TW202136261A (zh) 2018-10-31 2021-10-01 美商基利科學股份有限公司 經取代之6-氮雜苯并咪唑化合物
WO2020163405A1 (en) 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
JP7551629B2 (ja) 2019-02-05 2024-09-17 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
WO2020163401A1 (en) * 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
JP7603594B2 (ja) * 2019-02-06 2024-12-20 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
JP7603595B2 (ja) * 2019-02-06 2024-12-20 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
US11453681B2 (en) 2019-05-23 2022-09-27 Gilead Sciences, Inc. Substituted eneoxindoles and uses thereof
TW202131920A (zh) 2019-11-01 2021-09-01 瑞士商諾華公司 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途
US11618751B1 (en) 2022-03-25 2023-04-04 Ventus Therapeutics U.S., Inc. Pyrido-[3,4-d]pyridazine amine derivatives useful as NLRP3 derivatives
CA3182912A1 (en) 2020-05-13 2021-11-18 Chdi Foundation, Inc. Htt modulators for treating huntington's disease
MX2023001558A (es) * 2020-08-05 2023-04-26 Skyhawk Therapeutics Inc Composiciones para modular el corte y empalme.
IL301964A (en) 2020-10-08 2023-06-01 Redona Therapeutics Inc History of benzo[H]quinazoline-4-amine and thiano[2,3-H]quinazoline-4-amine for cancer treatment
US11319319B1 (en) 2021-04-07 2022-05-03 Ventus Therapeutics U.S., Inc. Compounds for inhibiting NLRP3 and uses thereof
US20240239758A1 (en) 2021-04-28 2024-07-18 Astellas Pharma Inc. Substituted triazine compound
KR20240019083A (ko) * 2021-06-04 2024-02-14 에프. 호프만-라 로슈 아게 트리아진 유도체 및 암 치료에서 이의 용도
MX2024005933A (es) 2021-11-17 2024-08-06 Chdi Foundation Inc Moduladores de htt pata tratar la enfermedad de huntington.
US20260015353A1 (en) * 2022-07-21 2026-01-15 Hoffmann-La Roche Inc. Novel compounds
US12331048B2 (en) 2022-10-31 2025-06-17 Ventus Therapeutics U.S., Inc. Pyrido-[3,4-d]pyridazine amine derivatives useful as NLRP3 inhibitors

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EP0327155A2 (en) * 1988-01-30 1989-08-09 Merck Sharp & Dohme Ltd. Pyrazines, pyrimidines and pyridazines, useful in the treatment of senile dementia
US20100305089A1 (en) * 2006-05-19 2010-12-02 Abbott Laboratories Fused Bicycloheterocycle Substituted Azabicyclic Alkane Derivatives
WO2018232039A1 (en) * 2017-06-14 2018-12-20 Ptc Therapeutics, Inc. Methods for modifying rna splicing

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GB9005737D0 (en) * 1990-03-14 1990-05-09 Beecham Group Plc Novel compounds
EA202092001A1 (ru) * 2018-03-27 2021-01-29 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Соединения для лечения болезни гентингтона

Patent Citations (3)

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Publication number Priority date Publication date Assignee Title
EP0327155A2 (en) * 1988-01-30 1989-08-09 Merck Sharp & Dohme Ltd. Pyrazines, pyrimidines and pyridazines, useful in the treatment of senile dementia
US20100305089A1 (en) * 2006-05-19 2010-12-02 Abbott Laboratories Fused Bicycloheterocycle Substituted Azabicyclic Alkane Derivatives
WO2018232039A1 (en) * 2017-06-14 2018-12-20 Ptc Therapeutics, Inc. Methods for modifying rna splicing

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2026067767A1 (zh) * 2024-09-30 2026-04-02 普罗思特(南京)生物科技有限公司 取代的哒嗪衍生物及其应用

Also Published As

Publication number Publication date
KR102925387B1 (ko) 2026-02-10
EP3920917A4 (en) 2022-09-28
WO2020163248A1 (en) 2020-08-13
EP3920917A1 (en) 2021-12-15
US20220048902A1 (en) 2022-02-17
CN119320388A (zh) 2025-01-17
KR20210124362A (ko) 2021-10-14
JP7682096B2 (ja) 2025-05-23
JP2022519636A (ja) 2022-03-24

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