JP2017532982A - Ｓｍａｄ７アンチセンスオリゴヌクレオチドの同位体置換体 - Google Patents

Ｓｍａｄ７アンチセンスオリゴヌクレオチドの同位体置換体 Download PDF

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JP2017532982A
JP2017532982A JP2017540330A JP2017540330A JP2017532982A JP 2017532982 A JP2017532982 A JP 2017532982A JP 2017540330 A JP2017540330 A JP 2017540330A JP 2017540330 A JP2017540330 A JP 2017540330A JP 2017532982 A JP2017532982 A JP 2017532982A
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deuterated
over
antisense oligonucleotide
smad7 antisense
smad7
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JP2017532982A5 (https=
Inventor
アンソニー シカラ ピーター
アンソニー シカラ ピーター
Original Assignee
セルジーン アルパイン インベストメント カンパニー Ｉｉ， エルエルシー
セルジーン アルパイン インベストメント カンパニー Ｉｉ， エルエルシー
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Publication of JP2017532982A publication Critical patent/JP2017532982A/ja
Publication of JP2017532982A5 publication Critical patent/JP2017532982A5/ja
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/04—Drugs for disorders of the alimentary tract or the digestive system for ulcers, gastritis or reflux esophagitis, e.g. antacids, inhibitors of acid secretion, mucosal protectants
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2017540330A 2014-10-17 2014-10-17 Ｓｍａｄ７アンチセンスオリゴヌクレオチドの同位体置換体 Pending JP2017532982A (ja)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
PCT/IB2014/065421 WO2015011694A2 (en) 2014-10-17 2014-10-17 Isotopologues of smad7 antisense oligonucleotides

Publications (2)

Publication Number Publication Date
JP2017532982A true JP2017532982A (ja) 2017-11-09
JP2017532982A5 JP2017532982A5 (https=) 2018-05-31

Family

ID=51947403

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2017540330A Pending JP2017532982A (ja) 2014-10-17 2014-10-17 Ｓｍａｄ７アンチセンスオリゴヌクレオチドの同位体置換体

Country Status (4)

Country Link
US (1) US20170247695A1 (https=)
EP (1) EP3207135A2 (https=)
JP (1) JP2017532982A (https=)
WO (1) WO2015011694A2 (https=)

Families Citing this family (29)

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WO2016059239A1 (en) 2014-10-17 2016-04-21 Nogra Pharma Limited Methods and compositions for treating a subject with a smad7 antisense oligonucleotide
MA41271A (fr) * 2014-12-26 2017-10-31 Celgene Alpine Invest Company Ii Llc Méthodes d'utilisation d'oligonucléotides antisens ciblant smad7
MX2018003699A (es) * 2015-09-30 2018-08-01 Celgene Alpine Invest Company Ii Llc Moduladores de tlr y metodos de uso.
JP2019505598A (ja) 2016-02-23 2019-02-28 セルジーン アルパイン インベストメント カンパニー Ｉｉ， エルエルシー Ｓｍａｄ７の阻害を用いて、腸線維症を治療する方法
CA3029772A1 (en) 2016-07-05 2018-01-11 Biomarin Technologies B.V. Pre-mrna splice switching or modulating oligonucleotides comprising bicyclic scaffold moieties, with improved characteristics for the treatment of genetic disorders
EP3658155A4 (en) * 2017-07-28 2021-06-30 Nogra Pharma Limited PROCESS FOR THE PREPARATION OF OLIGONUCLEOTIDIC COMPOUNDS
US10517889B2 (en) 2017-09-08 2019-12-31 Ionis Pharmaceuticals, Inc. Modulators of SMAD7 expression
IL282818B1 (en) 2018-11-02 2026-04-01 Biomarin Tech Bv Bispecific antisense oligonucleotides for dystrophin exon skipping
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes
AR131145A1 (es) 2022-11-24 2025-02-19 Proqr Therapeutics Ii Bv Oligonucleótidos antisentido para el tratamiento de hemocromatosis hfe hereditaria
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
CA3276262A1 (en) 2022-12-09 2024-06-13 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of cardiovascular disease
GB202300865D0 (en) 2023-01-20 2023-03-08 Proqr Therapeutics Ii Bv Delivery of oligonucleotides
EP4669753A1 (en) 2023-02-20 2025-12-31 ProQR Therapeutics II B.V. Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease
WO2024206175A1 (en) 2023-03-24 2024-10-03 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurological disorders
GB202304363D0 (en) 2023-03-24 2023-05-10 Proqr Therapeutics Ii Bv Chemically modified antisense oligonucleotides for use in RNA editing
AU2024246572A1 (en) 2023-03-27 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of liver disease
WO2024256620A1 (en) 2023-06-16 2024-12-19 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurodegenerative disease
WO2025051946A1 (en) 2023-09-07 2025-03-13 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of metabolic disorders
WO2025104239A1 (en) 2023-11-16 2025-05-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of classic galactosemia
WO2025132708A1 (en) 2023-12-20 2025-06-26 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of huntington's disease
GB202404661D0 (en) 2024-04-02 2024-05-15 Proqr Therapeutics Ii Bv Antisense oligoncleotides for the treatment of liver disease
GB202405143D0 (en) 2024-04-11 2024-05-29 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of poly-q disease
WO2025224230A1 (en) 2024-04-25 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of fatty liver disease
GB202410081D0 (en) 2024-07-11 2024-08-28 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of cardiovascular disease
WO2026022136A1 (en) 2024-07-23 2026-01-29 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of metabolic disorders
WO2026060374A2 (en) 2024-09-16 2026-03-19 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurological disorders
WO2026068781A1 (en) 2024-09-30 2026-04-02 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of liver disease
WO2026080897A1 (en) 2024-10-11 2026-04-16 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of chronic pain

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2006521815A (ja) * 2003-04-02 2006-09-28 ジュリアーニ インターナショナル リミテッド Ｓｍａｄ７に対するアンチセンスオリゴヌクレオチド（ＯＤＮ）及びその医療分野での使用
JP2012508705A (ja) * 2008-11-13 2012-04-12 ジュリアーニ インターナショナル リミテッド アンチセンス組成物およびその作製および使用
WO2014022566A2 (en) * 2012-07-31 2014-02-06 Ased, Llc Synthesis of deuterated ribo nucleosides, n-protected phosphoramidites, and oligonucleotides

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US6159697A (en) 2000-01-19 2000-12-12 Isis Pharmaceuticals, Inc. Antisense modulation of Smad7 expression
US8288414B2 (en) 2007-09-12 2012-10-16 Deuteria Pharmaceuticals, Inc. Deuterium-enriched lenalidomide

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2006521815A (ja) * 2003-04-02 2006-09-28 ジュリアーニ インターナショナル リミテッド Ｓｍａｄ７に対するアンチセンスオリゴヌクレオチド（ＯＤＮ）及びその医療分野での使用
JP2012508705A (ja) * 2008-11-13 2012-04-12 ジュリアーニ インターナショナル リミテッド アンチセンス組成物およびその作製および使用
WO2014022566A2 (en) * 2012-07-31 2014-02-06 Ased, Llc Synthesis of deuterated ribo nucleosides, n-protected phosphoramidites, and oligonucleotides

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
WORLD J. GASTROENTEROL., 2012, VOL. 18, NO. 40, PP. 5664-5668, JPN6018036306, ISSN: 0004014784 *

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EP3207135A2 (en) 2017-08-23
WO2015011694A3 (en) 2015-11-12
WO2015011694A2 (en) 2015-01-29
US20170247695A1 (en) 2017-08-31

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