WO2022060944A1 - Compositions pour moduler l'épissage - Google Patents

Compositions pour moduler l'épissage Download PDF

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Publication number
WO2022060944A1
WO2022060944A1 PCT/US2021/050613 US2021050613W WO2022060944A1 WO 2022060944 A1 WO2022060944 A1 WO 2022060944A1 US 2021050613 W US2021050613 W US 2021050613W WO 2022060944 A1 WO2022060944 A1 WO 2022060944A1
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WO
WIPO (PCT)
Prior art keywords
substituted
unsubstituted
compound
hydrogen
alkyl
Prior art date
Application number
PCT/US2021/050613
Other languages
English (en)
Inventor
Michael Luzzio
Daniel Brian HORNE
Brian Lucas
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 WO2022060944A1 publication Critical patent/WO2022060944A1/fr

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Classifications

    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C07D451/14—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
    • C07D519/00—Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups C07D453/00 or C07D455/00

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

Abstract

L'invention concerne des composés modulateurs d'épissage à petites molécules qui modulent l'épissage de l'ARNm, tels que le pré-ARNm, codés par des gènes, ainsi que des procédés d'utilisation des composés modulateurs d'épissage à petites molécules pour moduler l'épissage et traiter des maladies et des états pathologiques.
PCT/US2021/050613 2020-09-16 2021-09-16 Compositions pour moduler l'épissage WO2022060944A1 (fr)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US202063079449P 2020-09-16 2020-09-16
US202063079430P 2020-09-16 2020-09-16
US202063079471P 2020-09-16 2020-09-16
US202063079451P 2020-09-16 2020-09-16
US63/079,471 2020-09-16
US63/079,430 2020-09-16
US63/079,451 2020-09-16
US63/079,449 2020-09-16

Publications (1)

Publication Number Publication Date
WO2022060944A1 true WO2022060944A1 (fr) 2022-03-24

Family

ID=80775608

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2021/050613 WO2022060944A1 (fr) 2020-09-16 2021-09-16 Compositions pour moduler l'épissage

Country Status (1)

Country Link
WO (1) WO2022060944A1 (fr)

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019028440A1 (fr) * 2017-08-04 2019-02-07 Skyhawk Therapeutics, Inc. Méthodes et compositions permettant de moduler l'épissageé
EA201800367A1 (ru) * 2015-12-10 2019-02-28 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Способы лечения болезни хантингтона
WO2020005873A1 (fr) * 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Composés hétérocycliques et hétéroaryles pour le traitement de la maladie de huntington
WO2020163401A1 (fr) * 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Procédés et compositions pour moduler l'épissage
WO2020163405A1 (fr) * 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Procédés et compositions pour moduler l'épissage

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EA201800367A1 (ru) * 2015-12-10 2019-02-28 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Способы лечения болезни хантингтона
WO2019028440A1 (fr) * 2017-08-04 2019-02-07 Skyhawk Therapeutics, Inc. Méthodes et compositions permettant de moduler l'épissageé
WO2020005873A1 (fr) * 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Composés hétérocycliques et hétéroaryles pour le traitement de la maladie de huntington
WO2020163401A1 (fr) * 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Procédés et compositions pour moduler l'épissage
WO2020163405A1 (fr) * 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Procédés et compositions pour moduler l'épissage

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