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 (enExample
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
Pending legal-status Critical Current

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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 (enExample) 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 (enExample)
EP (1) EP3207135A2 (enExample)
JP (1) JP2017532982A (enExample)
WO (1) WO2015011694A2 (enExample)

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KR20170069262A (ko) 2014-10-17 2017-06-20 노그라 파마 리미티드 Smad7 안티센스 올리고뉴클레오티드로 대상체를 치료하기 위한 방법 및 조성물
MA41271A (fr) * 2014-12-26 2017-10-31 Celgene Alpine Invest Company Ii Llc Méthodes d'utilisation d'oligonucléotides antisens ciblant smad7
CA3000195A1 (en) * 2015-09-30 2017-04-06 Celgene Alpine Investment Company Ii, Llc Tlr modulators and methods of use
WO2017147276A1 (en) 2016-02-23 2017-08-31 Celgene Corporation Methods of treating intestinal fibrosis using smad7 inhibition
CN109757108A (zh) 2016-07-05 2019-05-14 比奥马林技术公司 具有治疗遗传疾病的改善特征的包含双环支架部分的前体mRNA剪接转换或调节寡核苷酸
CN111148519A (zh) * 2017-07-28 2020-05-12 细胞基因公司 制备寡核苷酸化合物的方法
WO2019051173A1 (en) 2017-09-08 2019-03-14 Ionis Pharmaceuticals, Inc. MODULATORS OF SMAD7 EXPRESSION
EP3874044A1 (en) 2018-11-02 2021-09-08 BioMarin Technologies B.V. Bispecific antisense oligonucleotides for dystrophin exon skipping
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes
JP2025536808A (ja) 2022-11-24 2025-11-07 プロキューアール・セラピューティクス・セカンド・ベスローテン・フェンノートシャップ 遺伝性ｈｆｅヘモクロマトーシスの治療のためのアンチセンスオリゴヌクレオチド
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
JP2025540146A (ja) 2022-12-09 2025-12-11 プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ 心血管疾患の治療のためのアンチセンスオリゴヌクレオチド
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
CN121002181A (zh) 2023-03-27 2025-11-21 ProQR治疗上市公司Ⅱ 用于治疗肝脏疾病的反义寡核苷酸
TW202516003A (zh) 2023-06-16 2025-04-16 荷蘭商Ｐｒｏｑｒ治療上市公司Ｉｉ 用於治療神經退化性疾病之反義寡核苷酸
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

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

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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 *

Also Published As

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

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