[go: up one dir, main page]

EP3920919A4 - Procédés et compositions pour moduler l'épissage - Google Patents

Procédés et compositions pour moduler l'épissage Download PDF

Info

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Withdrawn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)
English (en)
Other versions
EP3920919A1 (fr
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/fr
Publication of EP3920919A4 publication Critical patent/EP3920919A4/fr
Withdrawn legal-status Critical Current

Links

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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (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 Procédés et compositions pour moduler l'épissage Withdrawn EP3920919A4 (fr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962801506P 2019-02-05 2019-02-05
US201962801502P 2019-02-05 2019-02-05
US201962801486P 2019-02-05 2019-02-05
PCT/US2020/016671 WO2020163401A1 (fr) 2019-02-05 2020-02-04 Procédés et compositions pour moduler l'épissage

Publications (2)

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

Family

ID=71947086

Family Applications (1)

Application Number Title Priority Date Filing Date
EP20753044.5A Withdrawn EP3920919A4 (fr) 2019-02-05 2020-02-04 Procédés et compositions pour moduler l'épissage

Country Status (5)

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

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11071730B2 (en) 2018-10-31 2021-07-27 Gilead Sciences, Inc. Substituted 6-azabenzimidazole compounds
PL3873903T3 (pl) 2018-10-31 2024-05-20 Gilead Sciences, Inc. Podstawione związki 6-azabenzimidazolu jako inhibitory hpk1
CN114007613A (zh) 2019-02-05 2022-02-01 斯基霍克疗法公司 用于调节剪接的方法和组合物
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
EP3972695A1 (fr) 2019-05-23 2022-03-30 Gilead Sciences, Inc. Exo-méthylène-oxindoles substitués qui sont des inhibiteurs de hpk1/map4k1
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
MX2022013856A (es) 2020-05-13 2023-04-05 Chdi Foundation Inc Moduladores htt para tratar la enfermedad de huntington.
WO2022060944A1 (fr) * 2020-09-16 2022-03-24 Skyhawk Therapeutics, Inc. Compositions pour moduler l'épissage
JP2024540477A (ja) 2021-11-17 2024-10-31 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 ハンチントン病を治療するためのｈｔｔモジュレータとしてのｎ－（２ｈ－インダゾール－５－イル）ピラジン－２－カルボキサミド誘導体及び類似化合物

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007137030A2 (fr) * 2006-05-19 2007-11-29 Abbott Laboratories Dérivés d'alcanes azabicycliques substitués à bicyclohétérocycle fusionné
WO2011095625A1 (fr) * 2010-02-05 2011-08-11 Heptares Therapeutics Limited Dérivés de 1,2,4-triazine-4-amine
WO2017100726A1 (fr) * 2015-12-10 2017-06-15 Ptc Therapeutics, Inc. Méthodes de traitement de la maladie de huntington
WO2018232039A1 (fr) * 2017-06-14 2018-12-20 Ptc Therapeutics, Inc. Procédés de modification de l'épissage de l'arn
WO2019028440A1 (fr) * 2017-08-04 2019-02-07 Skyhawk Therapeutics, Inc. Méthodes et compositions permettant de moduler l'épissageé
WO2020163382A1 (fr) * 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Procédés et compositions pour moduler l'épissage
WO2020163248A1 (fr) * 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Procédés et compositions pour moduler l'épissage

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6884102B2 (ja) * 2015-02-09 2021-06-09 エフ・ホフマン−ラ・ロシュ・アクチェンゲゼルシャフト がんの治療のための化合物
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 (fr) * 2006-05-19 2007-11-29 Abbott Laboratories Dérivés d'alcanes azabicycliques substitués à bicyclohétérocycle fusionné
WO2011095625A1 (fr) * 2010-02-05 2011-08-11 Heptares Therapeutics Limited Dérivés de 1,2,4-triazine-4-amine
WO2017100726A1 (fr) * 2015-12-10 2017-06-15 Ptc Therapeutics, Inc. Méthodes de traitement de la maladie de huntington
WO2018232039A1 (fr) * 2017-06-14 2018-12-20 Ptc Therapeutics, Inc. Procédés de modification de l'épissage de l'arn
WO2019028440A1 (fr) * 2017-08-04 2019-02-07 Skyhawk Therapeutics, Inc. Méthodes et compositions permettant de moduler l'épissageé
WO2020163382A1 (fr) * 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Procédés et compositions pour moduler l'épissage
WO2020163248A1 (fr) * 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Procédés et compositions pour moduler l'épissage

Non-Patent Citations (1)

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

Also Published As

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

Similar Documents

Publication Publication Date Title
EP3920918A4 (fr) Procédés et compositions pour moduler l&#39;épissage
EP3661509A4 (fr) Méthodes et compositions permettant de moduler l&#39;épissageé
EP3920915A4 (fr) Procédés et compositions pour moduler l&#39;épissage
EP3920919A4 (fr) Procédés et compositions pour moduler l&#39;épissage
EP3920916A4 (fr) Procédés et compositions pour moduler l&#39;épissage
EP3920920A4 (fr) Procédés et compositions pour moduler l&#39;épissage
IL287398A (en) Methods and preparations for modulating composition and translation
IL290595A (en) Compositions and methods for modulating protein fusion and expression
EP3921311A4 (fr) Procédés et compositions pour moduler l&#39;épissage
EP3585171A4 (fr) Compositions et procédés associés destinés à la modulation d&#39;endosymbiontes
EP3920910A4 (fr) Procédés et compositions pour moduler l&#39;épissage
EP3938352A4 (fr) Procédés et compositions pour moduler l&#39;épissage
EP3911682A4 (fr) Compositions et procédés de modulation de l&#39;internalisation cellulaire
EP3870173A4 (fr) Modulateurs et inhibiteurs de wdr5
EP4025957B8 (fr) Modulateur électro-optique
EP4192454A4 (fr) Compositions pour moduler l&#39;épissage
EP3976026A4 (fr) Compositions et procédés de modulation du comportement cognitif
HK40070060A (en) Methods and compositions for modulating splicing and translation
HK40076949A (en) Compositions and methods for modulating splicing and protein expression
HK40106316A (zh) 用於调节ube3a-ats的化合物和方法
HK40082816A (en) Compositions and methods for modulating factor viii function
HK40029842A (en) Methods and compositions for modulating splicing
HK40071079A (en) Methods and compositions for modulating splicing of alternative introns
HK40109289A (en) Compositions and methods for modulating mrna splicing
HK40077811A (en) Compounds and methods useful for modulating gene splicing

Legal Events

Date Code Title Description
STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: THE INTERNATIONAL PUBLICATION HAS BEEN MADE

PUAI Public reference made under article 153(3) epc to a published international application that has entered the european phase

Free format text: ORIGINAL CODE: 0009012

STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: REQUEST FOR EXAMINATION WAS MADE

17P Request for examination filed

Effective date: 20210824

AK Designated contracting states

Kind code of ref document: A1

Designated state(s): AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR

DAV Request for validation of the european patent (deleted)
DAX Request for extension of the european patent (deleted)
A4 Supplementary search report drawn up and despatched

Effective date: 20230223

RIC1 Information provided on ipc code assigned before grant

Ipc: A61P 25/00 20060101ALI20230217BHEP

Ipc: A61K 31/53 20060101ALI20230217BHEP

Ipc: A61K 31/46 20060101ALI20230217BHEP

Ipc: A61K 31/439 20060101AFI20230217BHEP

STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: THE APPLICATION IS DEEMED TO BE WITHDRAWN

18D Application deemed to be withdrawn

Effective date: 20230901