AU2022398247A1 - Compounds and methods for modulating splicing - Google Patents

Compounds and methods for modulating splicing Download PDF

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Publication number
AU2022398247A1
AU2022398247A1 AU2022398247A AU2022398247A AU2022398247A1 AU 2022398247 A1 AU2022398247 A1 AU 2022398247A1 AU 2022398247 A AU2022398247 A AU 2022398247A AU 2022398247 A AU2022398247 A AU 2022398247A AU 2022398247 A1 AU2022398247 A1 AU 2022398247A1
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Australia
Prior art keywords
seq
compound
heteroaryl
formula
alkyl
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AU2022398247A
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English (en)
Inventor
Anant A. AGRAWAL
Allen T. Hopper
Benoît MOREAU
Sudeep PRAJAPATI
Dominic Reynolds
Michael W. SEILER
Peter Smith
Frederic VAILLANCOURT
Stepan Vyskocil
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Remix Therapeutics Inc
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Remix Therapeutics Inc
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Publication of AU2022398247A1 publication Critical patent/AU2022398247A1/en
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    • 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
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/50—Pyridazines; Hydrogenated pyridazines
    • A61K31/5025—Pyridazines; Hydrogenated pyridazines ortho- or peri-condensed with heterocyclic ring systems
    • 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
    • A61K31/4353—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 ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/437—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 ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • A61K31/4523—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
    • A61K31/454—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. pimozide, domperidone
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    • 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
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • A61K31/4523—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
    • A61K31/4545—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring hetero atom, e.g. pipamperone, anabasine
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    • 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
    • A61K31/47—Quinolines; Isoquinolines
    • A61K31/472—Non-condensed isoquinolines, e.g. papaverine
    • A61K31/4725—Non-condensed isoquinolines, e.g. papaverine containing further heterocyclic rings
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/496—Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene or sparfloxacin
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/4985—Pyrazines or piperazines ortho- or peri-condensed with heterocyclic ring systems
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    • C07D401/14—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
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    • 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
    • C07D403/02—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 two hetero rings
    • C07D403/04—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 two hetero rings directly linked by a ring-member-to-ring-member bond
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    • C07D413/04—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing two hetero rings directly linked by a ring-member-to-ring-member bond
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  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
AU2022398247A 2021-11-24 2022-11-23 Compounds and methods for modulating splicing Pending AU2022398247A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163283127P 2021-11-24 2021-11-24
US63/283,127 2021-11-24
PCT/US2022/050914 WO2023097007A1 (en) 2021-11-24 2022-11-23 Compounds and methods for modulating splicing

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Publication Number Publication Date
AU2022398247A1 true AU2022398247A1 (en) 2024-06-13

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ID=84602127

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AU2022398247A Pending AU2022398247A1 (en) 2021-11-24 2022-11-23 Compounds and methods for modulating splicing

Country Status (8)

Country Link
US (1) US20250082633A1 (https=)
EP (1) EP4436961A1 (https=)
JP (1) JP2024541481A (https=)
KR (1) KR20240149387A (https=)
CN (1) CN118804912A (https=)
AU (1) AU2022398247A1 (https=)
CA (1) CA3239110A1 (https=)
WO (1) WO2023097007A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20250353841A1 (en) * 2022-05-20 2025-11-20 Sarah Elizabeth Huff Heterocyclic compounds for treating huntington's disease
WO2025111409A1 (en) * 2023-11-21 2025-05-30 Biogen Ma Inc. Heterocyclic compounds of formula (i) for use in the treatment of spinal muscular atrophy via modulation of smn2 splicing

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
UY34750A (es) * 2012-04-20 2013-11-29 Gilead Sciences Inc ?compuestos para el tratamiento del hiv, composiciones,métodos de preparación, intermediarios y métodos terapéuticos?.
EP3119763A1 (en) * 2014-03-18 2017-01-25 Iteos Therapeutics Novel 3-indol substituted derivatives, pharmaceutical compositions and methods for use
EP3523289A1 (en) * 2016-10-07 2019-08-14 Araxes Pharma LLC Heterocyclic compounds as inhibitors of ras and methods of use thereof
JP2023520916A (ja) * 2020-04-08 2023-05-22 リミックス セラピューティクス インコーポレイテッド スプライシングを調整するための化合物及び方法
KR20230005210A (ko) * 2020-04-08 2023-01-09 레믹스 테라퓨틱스 인크. 스플라이싱을 조절하기 위한 화합물 및 방법

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CA3239110A1 (en) 2023-06-01
US20250082633A1 (en) 2025-03-13
EP4436961A1 (en) 2024-10-02
KR20240149387A (ko) 2024-10-14
JP2024541481A (ja) 2024-11-08
WO2023097007A1 (en) 2023-06-01
CN118804912A (zh) 2024-10-18

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