KR102556825B1 - Smn2 조정을 위한 조성물 및 방법 - Google Patents

Smn2 조정을 위한 조성물 및 방법 Download PDF

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KR102556825B1
KR102556825B1 KR1020197004350A KR20197004350A KR102556825B1 KR 102556825 B1 KR102556825 B1 KR 102556825B1 KR 1020197004350 A KR1020197004350 A KR 1020197004350A KR 20197004350 A KR20197004350 A KR 20197004350A KR 102556825 B1 KR102556825 B1 KR 102556825B1
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oligonucleotide
certain embodiments
artificial sequence
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KR20190027907A (ko
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프랑크 리고
타자 피. 프라카쉬
푸닛 피. 세트
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아이오니스 파마수티컬즈, 인코포레이티드
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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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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
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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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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Psychiatry (AREA)
  • Pain & Pain Management (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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KR1020197004350A 2016-07-15 2017-07-17 Smn2 조정을 위한 조성물 및 방법 Active KR102556825B1 (ko)

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)

Publication Number Publication Date
KR20190027907A KR20190027907A (ko) 2019-03-15
KR102556825B1 true KR102556825B1 (ko) 2023-07-17

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KR1020197004350A Active KR102556825B1 (ko) 2016-07-15 2017-07-17 Smn2 조정을 위한 조성물 및 방법

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US (3) US20190323006A1 (https=)
EP (2) EP3484524B1 (https=)
JP (3) JP7197463B2 (https=)
KR (1) KR102556825B1 (https=)
CN (1) CN109844115B (https=)
AU (2) AU2017297622A1 (https=)
BR (1) BR112019000356A2 (https=)
CA (1) CA3030864A1 (https=)
CL (2) CL2019000115A1 (https=)
CO (1) CO2019001236A2 (https=)
DK (1) DK3484524T3 (https=)
ES (1) ES2935658T3 (https=)
FI (1) FI3484524T3 (https=)
HR (1) HRP20221378T1 (https=)
HU (1) HUE060831T2 (https=)
IL (2) IL308256A (https=)
LT (1) LT3484524T (https=)
MX (1) MX2019000619A (https=)
NZ (1) NZ750713A (https=)
PE (1) PE20190513A1 (https=)
PL (1) PL3484524T3 (https=)
PT (1) PT3484524T (https=)
RS (1) RS63928B1 (https=)
SG (1) SG11201900238UA (https=)
SI (1) SI3484524T1 (https=)
SM (1) SMT202200489T1 (https=)
WO (1) WO2018014041A2 (https=)

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EP2983676B1 (en) * 2013-04-12 2019-03-20 The Curators of the University of Missouri Smn2 element 1 antisense compositions and methods and uses thereof
BR112017004056A2 (pt) 2014-09-12 2017-12-05 Biogen Ma Inc composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo
US10053465B2 (en) 2015-08-26 2018-08-21 Incyte Corporation Pyrrolopyrimidine derivatives as TAM inhibitors
MD3436461T2 (ro) 2016-03-28 2024-05-31 Incyte Corp Compuși pirolotriazină ca inhibitori TAM
SI3687996T1 (sl) 2017-09-27 2022-04-29 Incyte Corporation Soli pirolotriazinskih derivatov, ki so uporabne kot zaviralci TAM
MD3813800T2 (ro) 2018-06-29 2025-10-31 Incyte Corp Formulări de inhibitor al AXL/MER
AU2020316997A1 (en) * 2019-07-19 2022-03-10 Biogen Ma Inc. Methods of treating or preventing spinal muscular atrophy
US20220280548A1 (en) * 2019-08-15 2022-09-08 Biogen Ma Inc. Combination therapy for spinal muscular atrophy
US20220370491A1 (en) * 2019-09-18 2022-11-24 National University Corporation Tokyo Medical And Dental University Nucleic acid complex
WO2021174036A1 (en) * 2020-02-28 2021-09-02 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating scn1a expression
CR20220485A (es) * 2020-02-28 2022-11-10 Ionis Pharmaceuticals Inc Compuestos y métodos para modular smn2
EP4114401A1 (en) 2020-03-06 2023-01-11 Incyte Corporation Combination therapy comprising axl/mer and pd-1/pd-l1 inhibitors
KR20240032184A (ko) * 2021-04-22 2024-03-11 몰레큘라 악시옴, 엘엘씨 Sos 유전자 발현을 조절하기 위한 조성물 및 방법
FR3128873B1 (fr) 2021-11-10 2026-01-16 Biophytis Phytoecdysones et/ou dérivés de 20-hydroxyecdysone en combinaison avec un principe actif visant à restaurer l’expression SMN pour leur utilisation dans le traitement de l’amyotrophie spinale

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