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

Info

Publication number
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
Authority
JP
Japan
Prior art keywords
deuterated
over
antisense oligonucleotide
smad7 antisense
smad7
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2017540330A
Other languages
English (en)
Japanese (ja)
Other versions
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
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by 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 filed Critical 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
Publication of JP2017532982A publication Critical patent/JP2017532982A/ja
Publication of JP2017532982A5 publication Critical patent/JP2017532982A5/ja
Pending legal-status Critical Current

Links

Classifications

    • 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

Landscapes

  • 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)

Families Citing this family (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2017004973A (es) 2014-10-17 2017-12-07 Nogra Pharma Ltd Metodos y composiciones para tratar a un sujeto con un oligonucleotido antisentido de smad7.
MA41271A (fr) * 2014-12-26 2017-10-31 Celgene Alpine Invest Company Ii Llc Méthodes d'utilisation d'oligonucléotides antisens ciblant smad7
JP2018529726A (ja) * 2015-09-30 2018-10-11 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 Ｔｌｒ調節因子及び使用方法
WO2017147276A1 (en) 2016-02-23 2017-08-31 Celgene Corporation Methods of treating intestinal fibrosis using smad7 inhibition
JP2019522972A (ja) 2016-07-05 2019-08-22 バイオマリン テクノロジーズ ベー．フェー． 遺伝性障害の治療のための改善された特徴を有する二環式スキャフォールド部分を含むプレｍＲＮＡスプライススイッチング又は調節オリゴヌクレオチド
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
WO2020089325A1 (en) 2018-11-02 2020-05-07 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
AU2023385245A1 (en) 2022-11-24 2025-06-19 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of hereditary hfe-hemochromatosis
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
WO2024175550A1 (en) 2023-02-20 2024-08-29 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
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

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

Similar Documents

Publication Publication Date Title
JP2017532982A (ja) Ｓｍａｄ７アンチセンスオリゴヌクレオチドの同位体置換体
JP2022519019A (ja) オリゴヌクレオチド組成物及びその方法
US7893224B2 (en) Oligonucleotides comprising a ligand tethered to a modified or non-natural nucleobase
JP2017532982A5 (enExample)
JP2002510319A (ja) オリゴヌクレオチドの消化管を介したデリバリーのための組成物及び方法
JP2020528910A (ja) オリゴヌクレオチド化合物の調製方法
JP7022069B2 (ja) ラパドシン（Ｒａｐａｄｏｃｉｎｓ）、受動拡散型ヌクレオシド輸送体１阻害剤及びその使用
Marafini et al. Antisense approach to inflammatory bowel disease: prospects and challenges
JP2011515357A5 (enExample)
IL294400A (en) Prmt5 inhibitor for use in a method of treating psoriasis and other autoimmune conditions
US10100317B2 (en) Compositions of matter that reduce pain, shock, and inflammation by blocking linoleic acid metabolites and uses thereof
RS60645B1 (sr) Postupci za pripremu polinukleotida upotrebom kompozicija multivalentne katjonske soli
CN109789155B (zh) 含有地西他滨、5-氮杂胞苷和四氢尿苷的组合物以及其用途
JP2016533391A (ja) オリゴヌクレオチドについてのホスホロジアミデート骨格結合
WO2024208357A1 (zh) 调控CIDEB mRNA表达的dsRNA分子
Mäkilä et al. Noninvasive and quantitative monitoring of the distributions and kinetics of microRNA-targeting molecules in vivo by positron emission tomography
US20230183814A1 (en) Predictive biomarkers for onvansertib treatment
CN120441636B (zh) 寡核苷酸化合物、药物及其应用
JP7707184B2 (ja) 修飾短干渉ｒｎａ組成物、及びがんの処置におけるそれらの使用
RU2803235C1 (ru) Способ лечения псориаза и других аутоиммунных состояний с помощью ингибитора prmt5
Agrawal et al. Comparative pharmacokinetics of antisense oligonucleotides
WO2022250072A1 (ja) 心疾患治療薬
DE69835525T2 (de) Verminderungsregulation der genexpression durch kolorektale verabreichung von synthetischen oligonukleotiden
CN120958131A (zh) 用于治疗储袋炎的smad7抑制性反义寡核苷酸(aso)及其使用方法
TW202516006A (zh) ＲａｓＧＲＰ４的反義寡核苷酸

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20171012

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20171012

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20180329

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20180918

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20190416