WO2009148605A3 - Procédés pour traiter une hypercholestérolémie - Google Patents

Procédés pour traiter une hypercholestérolémie Download PDF

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Publication number
WO2009148605A3
WO2009148605A3 PCT/US2009/003398 US2009003398W WO2009148605A3 WO 2009148605 A3 WO2009148605 A3 WO 2009148605A3 US 2009003398 W US2009003398 W US 2009003398W WO 2009148605 A3 WO2009148605 A3 WO 2009148605A3
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WO
WIPO (PCT)
Prior art keywords
methods
antisense compounds
antisense
disclosed
individual
Prior art date
Application number
PCT/US2009/003398
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English (en)
Other versions
WO2009148605A2 (fr
Inventor
Susan M. Freier
Rosanne M. Crooke
Mark J. Graham
Kristina L. Lemonidis
Sanjay Bhanot
Diane Tribble
Andrew T. Watt
Original Assignee
Isis 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 Isis Pharmaceuticals, Inc. filed Critical Isis Pharmaceuticals, Inc.
Publication of WO2009148605A2 publication Critical patent/WO2009148605A2/fr
Publication of WO2009148605A3 publication Critical patent/WO2009148605A3/fr

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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
    • C12N15/1137—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 against enzymes
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/6454—Dibasic site splicing serine proteases, e.g. kexin (3.4.21.61); furin (3.4.21.75) and other proprotein convertases
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • 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
    • C12N2310/34—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

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

L'invention porte sur des composés antisens et sur des procédés pour diminuer le cholestérol associé aux lipoprotéines basse densité (LDL-C) chez un individu ayant un taux de LDL-C élevé. De plus, l'invention porte sur des composés antisens et sur des procédés pour traiter, prévenir ou améliorer l’hypercholestérolémie et/ou l’athérosclérose. L'invention porte en outre sur des composés antisens et sur des procédés pour diminuer un risque de coronaropathie. De tels procédés comprennent l'administration, à un individu ayant besoin d'un traitement, d'un composé antisens ciblé vers un acide nucléique PCSK9. Les composés antisens administrés comprennent des oligonucléotides antisens « gapmères ».
PCT/US2009/003398 2008-06-04 2009-06-04 Procédés pour traiter une hypercholestérolémie WO2009148605A2 (fr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US5870708P 2008-06-04 2008-06-04
US61/058,707 2008-06-04
US5916908P 2008-06-05 2008-06-05
US61/059,169 2008-06-05

Publications (2)

Publication Number Publication Date
WO2009148605A2 WO2009148605A2 (fr) 2009-12-10
WO2009148605A3 true WO2009148605A3 (fr) 2010-04-01

Family

ID=41203920

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2009/003398 WO2009148605A2 (fr) 2008-06-04 2009-06-04 Procédés pour traiter une hypercholestérolémie

Country Status (1)

Country Link
WO (1) WO2009148605A2 (fr)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9127276B2 (en) 2013-05-01 2015-09-08 Isis Pharmaceuticals, Inc. Conjugated antisense compounds and their use

Families Citing this family (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8093222B2 (en) 2006-11-27 2012-01-10 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
TW200838551A (en) 2006-11-27 2008-10-01 Isis Pharmaceuticals Inc Methods for treating hypercholesterolemia
BRPI0923225A2 (pt) 2008-12-02 2016-10-04 Chiralgen Ltd metodo para sintese de acidos nucleicos modificados no atomo de fosforo
KR101885383B1 (ko) 2009-07-06 2018-08-03 웨이브 라이프 사이언시스 리미티드 신규한 핵산 프로드러그 및 그의 사용 방법
WO2011009697A1 (fr) 2009-07-21 2011-01-27 Santaris Pharma A/S Oligomères anti-sens ciblant pcsk9
CN106146591B (zh) 2010-01-08 2020-07-31 Ionis制药公司 血管生成素样3表达的调节
JP5875006B2 (ja) * 2010-08-31 2016-03-02 国立大学法人大阪大学 オリゴヌクレオチド、およびオリゴヌクレオチドを有効成分として含有する脂質異常症治療剤
JP5868324B2 (ja) 2010-09-24 2016-02-24 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ 不斉補助基
CN103796657B (zh) 2011-07-19 2017-07-11 波涛生命科学有限公司 合成官能化核酸的方法
EP2873674B1 (fr) 2012-07-13 2020-05-06 Shin Nippon Biomedical Laboratories, Ltd. Adjuvant d'acide nucléique chiral
SG11201500239VA (en) 2012-07-13 2015-03-30 Wave Life Sciences Japan Asymmetric auxiliary group
EP4219516A3 (fr) 2012-07-13 2024-01-10 Wave Life Sciences Ltd. Controle chiral
PL2920304T3 (pl) 2012-11-15 2019-07-31 Roche Innovation Center Copenhagen A/S Koniugaty oligonukleotydowe
EP2951305B1 (fr) 2013-01-30 2018-08-15 F.Hoffmann-La Roche Ag Conjugués glucidiques d'oligonucléotides d'acides nucléiques bloqués
CN103314925A (zh) * 2013-06-21 2013-09-25 四川大学华西医院 一种恒河猴2型糖尿病模型的制备方法
PT3013959T (pt) * 2013-06-27 2020-02-04 Roche Innovation Ct Copenhagen As Oligómeros antissentido e conjugados dirigidos para pcsk9
WO2015108047A1 (fr) 2014-01-15 2015-07-23 株式会社新日本科学 Adjuvant d'acide nucléique chiral possédant une activité d'induction d'immunité, et activateur d'induction d'immunité
JPWO2015108048A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
US10322173B2 (en) 2014-01-15 2019-06-18 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
KR102423317B1 (ko) 2014-01-16 2022-07-22 웨이브 라이프 사이언시스 리미티드 키랄 디자인
US10570169B2 (en) 2014-05-22 2020-02-25 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
KR20190065341A (ko) 2016-10-06 2019-06-11 아이오니스 파마수티컬즈, 인코포레이티드 올리고머 화합물들의 접합 방법
WO2018102397A1 (fr) 2016-11-29 2018-06-07 PureTech Health LLC Exosomes destinés à l'administration d'agents thérapeutiques
JOP20190215A1 (ar) 2017-03-24 2019-09-19 Ionis Pharmaceuticals Inc مُعدّلات التعبير الوراثي عن pcsk9
CN106947825B (zh) * 2017-04-25 2020-04-28 华南理工大学 用于食蟹猴起源（亚群）鉴定的snp标记及其应用
EP4268817A1 (fr) * 2020-07-01 2023-11-01 Min Chen Utilisation d'un inhibiteur de pcsk9 dans la préparation d'un médicament destiné au traitement de multiples maladies
CN113730593B (zh) * 2021-08-02 2024-01-26 成都凌泰氪生物技术有限公司 一种增强核酸药物缓释能力的方法

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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WO2007131237A2 (fr) * 2006-05-05 2007-11-15 Isis Pharmaceuticals, Inc. Composés et procédés permettant de moduler l'expression de la protéine ptp1b
WO2008043753A2 (fr) * 2006-10-09 2008-04-17 Santaris Pharma A/S Composés antagonistes de l'arn pour la modulation de pcsk9
WO2008066776A2 (fr) * 2006-11-27 2008-06-05 Isis Pharmaceuticals, Inc. Procédés pour traiter l'hypercholestérolémie

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003011887A2 (fr) * 2001-08-01 2003-02-13 Isis Pharmaceuticals, Inc. Modulation antisens de l'expression de l'apolipoproteine b
WO2007131237A2 (fr) * 2006-05-05 2007-11-15 Isis Pharmaceuticals, Inc. Composés et procédés permettant de moduler l'expression de la protéine ptp1b
WO2008043753A2 (fr) * 2006-10-09 2008-04-17 Santaris Pharma A/S Composés antagonistes de l'arn pour la modulation de pcsk9
WO2008066776A2 (fr) * 2006-11-27 2008-06-05 Isis Pharmaceuticals, Inc. Procédés pour traiter l'hypercholestérolémie

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
GRAHAM MARK J ET AL: "Antisense inhibition of proprotein convertase subtilisin/kexin type 9 reduces serum LDL in hyperlipidemic mice", JOURNAL OF LIPID RESEARCH, vol. 48, no. 4, April 2007 (2007-04-01), pages 763 - 767, XP002565438, ISSN: 0022-2275 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9127276B2 (en) 2013-05-01 2015-09-08 Isis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
US9181549B2 (en) 2013-05-01 2015-11-10 Isis Pharmaceuticals, Inc. Conjugated antisense compounds and their use

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