CN109844115B - 用于调节smn2的化合物和方法 - Google Patents

用于调节smn2的化合物和方法 Download PDF

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Publication number
CN109844115B
CN109844115B CN201780043645.5A CN201780043645A CN109844115B CN 109844115 B CN109844115 B CN 109844115B CN 201780043645 A CN201780043645 A CN 201780043645A CN 109844115 B CN109844115 B CN 109844115B
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modified
oligonucleotide
certain embodiments
artificial sequence
oligomeric compound
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CN109844115A (zh
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F·里戈
T·P·普拉卡什
P·P·塞思
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Ionis Pharmaceuticals Inc
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • A—HUMAN NECESSITIES
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    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
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CN201780043645.5A 2016-07-15 2017-07-17 用于调节smn2的化合物和方法 Active CN109844115B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662363195P 2016-07-15 2016-07-15
US62/363,195 2016-07-15
PCT/US2017/042463 WO2018014041A2 (en) 2016-07-15 2017-07-17 Compounds and methods for modulation of smn2

Publications (2)

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CN109844115A CN109844115A (zh) 2019-06-04
CN109844115B true CN109844115B (zh) 2023-06-30

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CN114456176B (zh) 2016-03-28 2024-08-30 因赛特公司 作为tam抑制剂的吡咯并三嗪化合物
KR102739325B1 (ko) 2017-09-27 2024-12-09 인사이트 코포레이션 Tam 억제제로서 유용한 피롤로트리아진 유도체의 염
US11241438B2 (en) 2018-06-29 2022-02-08 Incyte Corporation Formulations of an AXL/MER inhibitor
HUE064071T2 (hu) * 2019-07-19 2024-02-28 Biogen Ma Inc Eljárások a spinális izomatrófia kezelésére vagy megelõzésére
WO2021030766A1 (en) * 2019-08-15 2021-02-18 Biogen Ma Inc. Combination therapy for spinal muscular atrophy
JP7737667B2 (ja) * 2019-09-18 2025-09-11 国立大学法人東京科学大学 核酸複合体
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WO2021174036A1 (en) * 2020-02-28 2021-09-02 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating scn1a expression
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US20240182894A1 (en) * 2021-04-22 2024-06-06 Molecular Axiom, Llc Compositions and methods for modulating sos gene expressions
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