JP2018511555A - リポジストロフィー集団におけるアポリポタンパク質Ｃ−ＩＩＩ（ＡｐｏＣＩＩＩ）発現の調節 - Google Patents

リポジストロフィー集団におけるアポリポタンパク質Ｃ−ＩＩＩ（ＡｐｏＣＩＩＩ）発現の調節 Download PDF

Info

Publication number
JP2018511555A
JP2018511555A JP2017538667A JP2017538667A JP2018511555A JP 2018511555 A JP2018511555 A JP 2018511555A JP 2017538667 A JP2017538667 A JP 2017538667A JP 2017538667 A JP2017538667 A JP 2017538667A JP 2018511555 A JP2018511555 A JP 2018511555A
Authority
JP
Japan
Prior art keywords
certain embodiments
lipodystrophy
animal
apociii
compound
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
JP2017538667A
Other languages
English (en)
Japanese (ja)
Other versions
JP2018511555A5 (enExample
Inventor
ディジェニオ，アンドレス
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Ionis Pharmaceuticals Inc
Original Assignee
Ionis Pharmaceuticals Inc
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 Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of JP2018511555A publication Critical patent/JP2018511555A/ja
Publication of JP2018511555A5 publication Critical patent/JP2018511555A5/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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Dermatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
JP2017538667A 2015-02-27 2016-02-26 リポジストロフィー集団におけるアポリポタンパク質Ｃ−ＩＩＩ（ＡｐｏＣＩＩＩ）発現の調節 Pending JP2018511555A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562126439P 2015-02-27 2015-02-27
US62/126,439 2015-02-27
PCT/US2016/019728 WO2016138355A1 (en) 2015-02-27 2016-02-26 Modulation of apolipoprotein c-iii (apociii) expression in lipodystrophy populations

Publications (2)

Publication Number Publication Date
JP2018511555A true JP2018511555A (ja) 2018-04-26
JP2018511555A5 JP2018511555A5 (enExample) 2020-06-18

Family

ID=56789093

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2017538667A Pending JP2018511555A (ja) 2015-02-27 2016-02-26 リポジストロフィー集団におけるアポリポタンパク質Ｃ−ＩＩＩ（ＡｐｏＣＩＩＩ）発現の調節

Country Status (13)

Country Link
US (2) US20180245076A1 (enExample)
EP (1) EP3270931A4 (enExample)
JP (1) JP2018511555A (enExample)
KR (1) KR20170122769A (enExample)
CN (1) CN107405358A (enExample)
AU (1) AU2016222548A1 (enExample)
BR (1) BR112017015307A2 (enExample)
CA (1) CA2977971A1 (enExample)
HK (1) HK1248522A1 (enExample)
IL (1) IL253346A0 (enExample)
MX (1) MX2017011009A (enExample)
RU (1) RU2737719C2 (enExample)
WO (1) WO2016138355A1 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2019002818A (es) * 2016-09-12 2019-08-29 Aegerion Pharmaceuticals Inc Metodos para detectar anticuerpos neutralizantes anti-leptina.
AU2017368050A1 (en) 2016-11-29 2019-06-20 Puretech Lyt, Inc. Exosomes for delivery of therapeutic agents
WO2022072244A1 (en) * 2020-10-02 2022-04-07 Ionis Pharmaceuticals, Inc. Methods for reducing apociii expression
JP2024525800A (ja) * 2021-07-16 2024-07-12 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060264395A1 (en) * 2003-04-16 2006-11-23 Crooke Rosanne M Modulation of apolipoprotein c-III expression
JP2014516516A (ja) * 2011-04-27 2014-07-17 アイシス ファーマシューティカルズ， インコーポレーテッド アポリポタンパク質ｃｉｉｉ（ａｐｏｃｉｉｉ）発現の調節
WO2014127268A2 (en) * 2013-02-14 2014-08-21 Isis Pharmaceuticals, Inc. Modulation of apolipoprotein c-iii (apociii) expression in lipoprotein lipase deficient (lpld) populations

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL2670486T3 (pl) * 2011-01-31 2016-08-31 Cadila Healthcare Ltd Leczenie lipodystrofii
US9909124B2 (en) * 2013-06-21 2018-03-06 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating apolipoprotein C-III expression for improving a diabetic profile
US20150045431A1 (en) * 2013-08-06 2015-02-12 Amarin Pharmaceuticals Ireland Limited Methods of treating a cardiovascular disorder in a subject on apo-c3 modulating therapy

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060264395A1 (en) * 2003-04-16 2006-11-23 Crooke Rosanne M Modulation of apolipoprotein c-III expression
JP2014516516A (ja) * 2011-04-27 2014-07-17 アイシス ファーマシューティカルズ， インコーポレーテッド アポリポタンパク質ｃｉｉｉ（ａｐｏｃｉｉｉ）発現の調節
WO2014127268A2 (en) * 2013-02-14 2014-08-21 Isis Pharmaceuticals, Inc. Modulation of apolipoprotein c-iii (apociii) expression in lipoprotein lipase deficient (lpld) populations

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
AM. J. MED., vol. 108, JPN6020003804, 2000, pages 143 - 152, ISSN: 0004354029 *
J. CLIN. ENDOCRINOL. METAB., vol. 86, JPN6020003808, 2001, pages 296 - 302, ISSN: 0004205537 *
JOURNAL OF HUMAN GENETICS, vol. 59, JPN6020036542, 2014, pages 16 - 23, ISSN: 0004354030 *

Also Published As

Publication number Publication date
RU2017133142A3 (enExample) 2019-08-12
AU2016222548A1 (en) 2017-07-27
US20180245076A1 (en) 2018-08-30
EP3270931A1 (en) 2018-01-24
CN107405358A (zh) 2017-11-28
RU2017133142A (ru) 2019-03-28
EP3270931A4 (en) 2018-10-03
US20200095581A1 (en) 2020-03-26
HK1248522A1 (zh) 2018-10-19
MX2017011009A (es) 2017-10-20
KR20170122769A (ko) 2017-11-06
BR112017015307A2 (pt) 2018-01-16
IL253346A0 (en) 2017-09-28
CA2977971A1 (en) 2016-09-01
RU2737719C2 (ru) 2020-12-02
WO2016138355A1 (en) 2016-09-01

Similar Documents

Publication Publication Date Title
JP6681422B2 (ja) リポタンパク質リパーゼ欠損（ｌｐｌｄ）集団におけるアポリポタンパク質ｃ−ｉｉｉ（ａｐｏｃｉｉｉ）発現の調節
JP6472820B2 (ja) アポリポタンパク質（ａ）発現を調節するための方法および組成物
JP6203707B2 (ja) アポリポタンパク質ｃｉｉｉ（ａｐｏｃｉｉｉ）発現の調節
US20180312840A1 (en) Compounds and methods for modulating apolipoprotein c-iii expression for improving a diabetic profile
US20200095581A1 (en) Modulation of apolipoprotein c-iii (apociii) expression in lipodystrophy populations
HK40000607B (en) Modulation of apolipoprotein c-iii (apociii) expression in lipoprotein lipase deficient (lpld) populations
HK1217441B (en) Modulation of apolipoprotein c-iii (apociii) expression in lipoprotein lipase deficient (lpld) populations

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20190225

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20190225

A524 Written submission of copy of amendment under article 19 pct

Free format text: JAPANESE INTERMEDIATE CODE: A524

Effective date: 20190318

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20200203

A524 Written submission of copy of amendment under article 19 pct

Free format text: JAPANESE INTERMEDIATE CODE: A524

Effective date: 20200507

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20200928

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210128

C60 Trial request (containing other claim documents, opposition documents)

Free format text: JAPANESE INTERMEDIATE CODE: C60

Effective date: 20210128

C11 Written invitation by the commissioner to file amendments

Free format text: JAPANESE INTERMEDIATE CODE: C11

Effective date: 20210209

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20210322

C21 Notice of transfer of a case for reconsideration by examiners before appeal proceedings

Free format text: JAPANESE INTERMEDIATE CODE: C21

Effective date: 20210323

A912 Re-examination (zenchi) completed and case transferred to appeal board

Free format text: JAPANESE INTERMEDIATE CODE: A912

Effective date: 20210430

C211 Notice of termination of reconsideration by examiners before appeal proceedings

Free format text: JAPANESE INTERMEDIATE CODE: C211

Effective date: 20210507

C22 Notice of designation (change) of administrative judge

Free format text: JAPANESE INTERMEDIATE CODE: C22

Effective date: 20220131

C22 Notice of designation (change) of administrative judge

Free format text: JAPANESE INTERMEDIATE CODE: C22

Effective date: 20220406

C13 Notice of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: C13

Effective date: 20220613

C28A Non-patent document cited

Free format text: JAPANESE INTERMEDIATE CODE: C2838

Effective date: 20220613

C23 Notice of termination of proceedings

Free format text: JAPANESE INTERMEDIATE CODE: C23

Effective date: 20221017

C03 Trial/appeal decision taken

Free format text: JAPANESE INTERMEDIATE CODE: C03

Effective date: 20221110

C30A Notification sent

Free format text: JAPANESE INTERMEDIATE CODE: C3012

Effective date: 20221110