JP5988581B2 - アンチセンスオリゴヌクレオチドを用いる多発性硬化症の治療方法 - Google Patents

アンチセンスオリゴヌクレオチドを用いる多発性硬化症の治療方法 Download PDF

Info

Publication number
JP5988581B2
JP5988581B2 JP2011516297A JP2011516297A JP5988581B2 JP 5988581 B2 JP5988581 B2 JP 5988581B2 JP 2011516297 A JP2011516297 A JP 2011516297A JP 2011516297 A JP2011516297 A JP 2011516297A JP 5988581 B2 JP5988581 B2 JP 5988581B2
Authority
JP
Japan
Prior art keywords
pharmaceutical composition
multiple sclerosis
oligonucleotide
human patients
group
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.)
Expired - Fee Related
Application number
JP2011516297A
Other languages
English (en)
Japanese (ja)
Other versions
JP2011525531A5 (enExample
JP2011525531A (ja
Inventor
エティ・クリンガー
ショッシー・テッスラー
フセイン・ハラック
ジョージ・タチャス
マーク・ポール・ダイアモンド
Original Assignee
アンチセンス セラピューティクス リミテッド
アンチセンス セラピューティクス リミテッド
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 アンチセンス セラピューティクス リミテッド, アンチセンス セラピューティクス リミテッド filed Critical アンチセンス セラピューティクス リミテッド
Publication of JP2011525531A publication Critical patent/JP2011525531A/ja
Publication of JP2011525531A5 publication Critical patent/JP2011525531A5/ja
Application granted granted Critical
Publication of JP5988581B2 publication Critical patent/JP5988581B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1138Non-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 against receptors or cell surface proteins
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/06Immunosuppressants, e.g. drugs for graft rejection
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Transplantation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
JP2011516297A 2008-06-23 2009-06-23 アンチセンスオリゴヌクレオチドを用いる多発性硬化症の治療方法 Expired - Fee Related JP5988581B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US13297308P 2008-06-23 2008-06-23
US61/132,973 2008-06-23
PCT/US2009/003760 WO2010008474A2 (en) 2008-06-23 2009-06-23 Methods for treating multiple sclerosis using antisense oligonucleotides

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2014208153A Division JP6131230B2 (ja) 2008-06-23 2014-10-09 アンチセンスオリゴヌクレオチドを用いる多発性硬化症の治療方法

Publications (3)

Publication Number Publication Date
JP2011525531A JP2011525531A (ja) 2011-09-22
JP2011525531A5 JP2011525531A5 (enExample) 2012-08-09
JP5988581B2 true JP5988581B2 (ja) 2016-09-07

Family

ID=41550900

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2011516297A Expired - Fee Related JP5988581B2 (ja) 2008-06-23 2009-06-23 アンチセンスオリゴヌクレオチドを用いる多発性硬化症の治療方法
JP2014208153A Expired - Fee Related JP6131230B2 (ja) 2008-06-23 2014-10-09 アンチセンスオリゴヌクレオチドを用いる多発性硬化症の治療方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2014208153A Expired - Fee Related JP6131230B2 (ja) 2008-06-23 2014-10-09 アンチセンスオリゴヌクレオチドを用いる多発性硬化症の治療方法

Country Status (8)

Country Link
US (2) US8415314B2 (enExample)
EP (2) EP2318424B1 (enExample)
JP (2) JP5988581B2 (enExample)
AU (1) AU2009271678B2 (enExample)
CA (1) CA2728562C (enExample)
DK (2) DK2937358T3 (enExample)
ES (2) ES2532404T3 (enExample)
WO (1) WO2010008474A2 (enExample)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2532404T3 (es) 2008-06-23 2015-03-26 Antisense Therapeutics Ltd Métodos para tratar la esclerosis múltiple usando oligonucleótidos antisentido
AU2011301712C1 (en) * 2010-09-17 2017-01-05 Antisense Therapeutics Ltd Method for reducing the number of certain circulating leukocytes in a human subject
EP3294889B1 (en) * 2015-05-11 2021-01-06 Murdoch University Multiple sclerosis treatment
AU2018286483C1 (en) * 2017-06-16 2024-10-31 Antisense Therapeutics Ltd Methods for treating multiple sclerosis using antisense oligonucleotides
KR20210018820A (ko) * 2018-05-04 2021-02-18 안티센스 테라퓨틱스 엘티디 치료 용도 및 방법
WO2020223762A1 (en) * 2019-05-06 2020-11-12 Antisense Therapeutics Ltd Methods for treating muscular dystrophy using inhibitory oligonucleotides to cd49d

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6242591B1 (en) * 1997-10-15 2001-06-05 Isis Pharmaceuticals, Inc. Synthesis of sulfurized 2'-substituted oligonucleotides
US5968826A (en) * 1998-10-05 1999-10-19 Isis Pharmaceuticals Inc. Antisense inhibition of integrin α4 expression
WO2005108610A2 (en) * 2004-04-05 2005-11-17 The Government Of The United States Of America As Represented By The Secretary, Department Of Health And Human Services Methods for the selection of subjects for multiple sclerosis therapy
JP2008516991A (ja) * 2004-10-20 2008-05-22 アンチセンス セラピューティクス リミテッド インテグリンα4発現のアンチセンス調節
ES2532404T3 (es) 2008-06-23 2015-03-26 Antisense Therapeutics Ltd Métodos para tratar la esclerosis múltiple usando oligonucleótidos antisentido

Also Published As

Publication number Publication date
JP2015044824A (ja) 2015-03-12
EP2318424B1 (en) 2015-02-25
DK2937358T3 (da) 2019-01-02
AU2009271678B2 (en) 2013-09-19
EP2318424A4 (en) 2012-12-26
JP6131230B2 (ja) 2017-05-17
WO2010008474A2 (en) 2010-01-21
JP2011525531A (ja) 2011-09-22
ES2532404T3 (es) 2015-03-26
WO2010008474A3 (en) 2010-04-22
AU2009271678A1 (en) 2010-01-21
US8415314B2 (en) 2013-04-09
DK2318424T3 (en) 2015-04-07
WO2010008474A4 (en) 2011-01-27
EP2937358B1 (en) 2018-09-19
US20100119480A1 (en) 2010-05-13
EP2937358A1 (en) 2015-10-28
CA2728562C (en) 2018-10-23
US20130345293A1 (en) 2013-12-26
CA2728562A1 (en) 2010-01-21
ES2699891T3 (es) 2019-02-13
EP2318424A2 (en) 2011-05-11
US8759314B2 (en) 2014-06-24

Similar Documents

Publication Publication Date Title
JP6131230B2 (ja) アンチセンスオリゴヌクレオチドを用いる多発性硬化症の治療方法
EP2791335B1 (en) Chimeric double-stranded nucleic acid
WO2024243323A1 (en) Treatment of multiple sclerosis with anti-cd40l antibodies
AU2018286483B2 (en) Methods for treating multiple sclerosis using antisense oligonucleotides
CN108473989A (zh) Il-34反义寡核苷酸以及使用其的方法
KR20220061915A (ko) Cd49d에 대한 억제성 올리고뉴클레오티드를 사용하는 근이영양증의 치료 방법
AU2018421460B2 (en) Therapeutic uses and methods
US20250179503A1 (en) Methods of using oligomeric compounds to treat scn2a-related disorders
KR20210015652A (ko) 뇌전증 및 이와 동반하는 인지기능 장애의 진단, 예방 및 치료를 위한 mlc1의 용도
US20250283083A1 (en) Compositions and methods for splicing modulation of unc13a
WO2022158608A1 (ja) 良性成人型家族性ミオクローヌスてんかんの予防又は治療剤
AU2019377275A1 (en) Method for reducing the risk of a cardiovascular event with conjugated antisense compounds targeting apo(a)

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20120620

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20120620

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20131126

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20140225

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20140304

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20140609

A711 Notification of change in applicant

Free format text: JAPANESE INTERMEDIATE CODE: A711

Effective date: 20141009

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20160428

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20160809

R150 Certificate of patent or registration of utility model

Ref document number: 5988581

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

LAPS Cancellation because of no payment of annual fees