IL273741B - Methods and compositions for fusion modulation - Google Patents

Methods and compositions for fusion modulation

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Publication number
IL273741B
IL273741B IL273741A IL27374120A IL273741B IL 273741 B IL273741 B IL 273741B IL 273741 A IL273741 A IL 273741A IL 27374120 A IL27374120 A IL 27374120A IL 273741 B IL273741 B IL 273741B
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IL
Israel
Prior art keywords
substituted
unsubstituted
alkyl
ring
heteroaryl
Prior art date
Application number
IL273741A
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English (en)
Hebrew (he)
Other versions
IL273741A (en
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Skyhawk Therapeutics Inc
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Publication date
Application filed by Skyhawk Therapeutics Inc filed Critical Skyhawk Therapeutics Inc
Publication of IL273741A publication Critical patent/IL273741A/en
Publication of IL273741B publication Critical patent/IL273741B/en

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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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    • C07D403/10—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 linked by a carbon chain containing aromatic rings
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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
    • A61K31/50—Pyridazines; Hydrogenated pyridazines
    • A61K31/501—Pyridazines; Hydrogenated pyridazines not condensed and containing further heterocyclic rings
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    • A61K31/5375—1,4-Oxazines, e.g. morpholine
    • A61K31/5383—1,4-Oxazines, e.g. morpholine ortho- or peri-condensed with heterocyclic ring systems
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    • 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
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    • 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
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    • 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
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    • 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
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    • 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
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    • C07D498/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and oxygen atoms as the only ring hetero atoms
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/333—Modified A

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IL273741A 2017-08-04 2018-08-03 Methods and compositions for fusion modulation IL273741B (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762541202P 2017-08-04 2017-08-04
US201762562948P 2017-09-25 2017-09-25
US201762562927P 2017-09-25 2017-09-25
PCT/US2018/045282 WO2019028440A1 (en) 2017-08-04 2018-08-03 METHODS AND COMPOSITIONS FOR MODULATING SPLICING

Publications (2)

Publication Number Publication Date
IL273741A IL273741A (en) 2020-05-31
IL273741B true IL273741B (en) 2022-09-01

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IL273741A IL273741B (en) 2017-08-04 2018-08-03 Methods and compositions for fusion modulation
IL272413A IL272413B (en) 2017-08-04 2018-08-03 Methods and compositions for fusion modulation

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US (8) US11162101B2 (enExample)
EP (2) EP3661509A4 (enExample)
JP (2) JP7312749B2 (enExample)
KR (2) KR102636383B1 (enExample)
CN (2) CN111499615B (enExample)
AU (2) AU2018309187C1 (enExample)
CA (1) CA3072205A1 (enExample)
GB (2) GB2587457B (enExample)
IL (2) IL273741B (enExample)
MX (2) MX2020001425A (enExample)
MY (1) MY201938A (enExample)
RU (1) RU2020105929A (enExample)
SG (2) SG10202002990XA (enExample)
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MX391850B (es) 2015-12-10 2025-03-19 Ptc Therapeutics Inc Compuestos para usarse en el tratamiento o mejoramiento de la enfermedad de huntington
MX2019005588A (es) 2016-11-28 2019-10-15 Ptc Therapeutics Inc Metodos para modular corte y empalme de arn.
CA3066224A1 (en) 2017-06-05 2018-12-13 Ptc Therapeutics Inc. Compounds for treating huntington's disease
KR20200017476A (ko) 2017-06-14 2020-02-18 피티씨 테라퓨틱스, 인크. Rna 스플라이싱의 변경 방법
MX2019015580A (es) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
US11382918B2 (en) 2017-06-28 2022-07-12 Ptc Therapeutics, Inc. Methods for treating Huntington's Disease
MY201938A (en) 2017-08-04 2024-03-25 Skyhawk Therapeutics Inc Methods and compositions for modulating splicing
JP7399870B2 (ja) 2018-03-27 2023-12-18 ピーティーシー セラピューティクス， インコーポレイテッド ハンチントン病を処置するための化合物
WO2019199972A1 (en) 2018-04-10 2019-10-17 Skyhawk Therapeutics, Inc. Compounds for the treatment of cancer
US11685746B2 (en) 2018-06-27 2023-06-27 Ptc Therapeutics, Inc. Heteroaryl compounds for treating Huntington's disease
KR20210038845A (ko) 2018-06-27 2021-04-08 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료를 위한 헤테로아릴 화합물
HUE068060T2 (hu) 2018-06-27 2024-12-28 Ptc Therapeutics Inc Heterociklikus és heteroaril vegyületek Huntington-kór kezelésére
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