CA2872304C - Organic compositions to treat kras-related diseases - Google Patents

Organic compositions to treat kras-related diseases Download PDF

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Publication number
CA2872304C
CA2872304C CA2872304A CA2872304A CA2872304C CA 2872304 C CA2872304 C CA 2872304C CA 2872304 A CA2872304 A CA 2872304A CA 2872304 A CA2872304 A CA 2872304A CA 2872304 C CA2872304 C CA 2872304C
Authority
CA
Canada
Prior art keywords
strand
rnai agent
kras
modified
seq
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.)
Active
Application number
CA2872304A
Other languages
English (en)
French (fr)
Other versions
CA2872304A1 (en
Inventor
Brian Bettencourt
Stuart Milstein
Ivanka Toudjarska
Earl MCDONALD
Michael SCHLABACH Jr.
Frank P. Stegmeier
Markus Warmuth
Kalyani GAMPA
Dieter Huesken
Mark Stump
Jan Weiler
Zainab JAGANI
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.)
Arrowhead Pharmaceuticals Inc
Original Assignee
Arrowhead 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 Arrowhead Pharmaceuticals Inc filed Critical Arrowhead Pharmaceuticals Inc
Priority to CA3173610A priority Critical patent/CA3173610A1/en
Publication of CA2872304A1 publication Critical patent/CA2872304A1/en
Application granted granted Critical
Publication of CA2872304C publication Critical patent/CA2872304C/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549Sugars, nucleosides, nucleotides or nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • A61P35/02Antineoplastic agents specific for leukemia
    • 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
    • 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
    • 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
    • C12N15/1135Non-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 oncogenes or tumor suppressor genes
    • 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
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/344Position-specific modifications, e.g. on every purine, at the 3'-end
    • 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
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Oncology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicinal Preparation (AREA)
CA2872304A 2012-05-02 2013-04-30 Organic compositions to treat kras-related diseases Active CA2872304C (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CA3173610A CA3173610A1 (en) 2012-05-02 2013-04-30 Organic compositions to treat kras-related diseases

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261641588P 2012-05-02 2012-05-02
US61/641,588 2012-05-02
PCT/US2013/038847 WO2013166004A2 (en) 2012-05-02 2013-04-30 Organic compositions to treat kras-related diseases

Related Child Applications (1)

Application Number Title Priority Date Filing Date
CA3173610A Division CA3173610A1 (en) 2012-05-02 2013-04-30 Organic compositions to treat kras-related diseases

Publications (2)

Publication Number Publication Date
CA2872304A1 CA2872304A1 (en) 2013-11-07
CA2872304C true CA2872304C (en) 2022-12-06

Family

ID=48366565

Family Applications (2)

Application Number Title Priority Date Filing Date
CA2872304A Active CA2872304C (en) 2012-05-02 2013-04-30 Organic compositions to treat kras-related diseases
CA3173610A Pending CA3173610A1 (en) 2012-05-02 2013-04-30 Organic compositions to treat kras-related diseases

Family Applications After (1)

Application Number Title Priority Date Filing Date
CA3173610A Pending CA3173610A1 (en) 2012-05-02 2013-04-30 Organic compositions to treat kras-related diseases

Country Status (12)

Country Link
US (2) US20150238515A1 (OSRAM)
EP (3) EP3272868A1 (OSRAM)
JP (1) JP2015517466A (OSRAM)
KR (1) KR20150004414A (OSRAM)
CN (2) CN104583406A (OSRAM)
AU (5) AU2013256471A1 (OSRAM)
BR (1) BR112014027337A2 (OSRAM)
CA (2) CA2872304C (OSRAM)
EA (1) EA201492004A1 (OSRAM)
HK (1) HK1206387A1 (OSRAM)
MX (1) MX2014013367A (OSRAM)
WO (1) WO2013166004A2 (OSRAM)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW201620525A (zh) * 2014-03-14 2016-06-16 波士頓生醫公司 使k-ras靜默之非對稱干擾rna組成物及其使用方法
MX377654B (es) 2014-05-14 2025-03-11 Targimmune Tharapeutics Ag Vectores mejorados de polietilenimina polietilenglicol.
US11180759B2 (en) * 2016-01-19 2021-11-23 The University Of North Carolina At Chapel Hill Methods and compositions using RNA interference and antisense oligonucleotides for inhibition of KRAS
MA45470A (fr) * 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques kras et leurs utilisations
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
KR102543645B1 (ko) * 2016-07-20 2023-06-16 주식회사 지씨지놈 유전성 발달장애 진단용 조성물 및 이의 용도
JP7506601B2 (ja) 2017-12-06 2024-06-26 アビディティー バイオサイエンシーズ,インク. 筋萎縮症と筋緊張性ジストロフィーを処置するための組成物および方法
AU2020287343A1 (en) * 2019-06-05 2021-12-09 Southern Research Institute Thiarabine- and thiarabine prodrug-based treatments
EP4058152A4 (en) * 2019-11-14 2024-05-08 The Board of Regents of the University of Oklahoma PROSTATE CANCER TREATMENTS USING OLIGONUCLEOTIDES INTERFERENCE
CA3172111A1 (en) 2020-03-19 2021-09-23 Barbora MALECOVA Compositions and methods of treating facioscapulohumeral muscular dystrophy
EP4100530A4 (en) * 2020-04-07 2024-05-22 The University of North Carolina at Chapel Hill METHODS AND COMPOSITIONS USING RNA INTERFERENCE AND ANTISENSE OLIGONUCLEOTIDES FOR INHIBITION OF KRAS
WO2023034537A1 (en) * 2021-09-02 2023-03-09 Molecular Axiom, Llc Compositions and methods for modulating kras expression
CN118265544A (zh) 2021-09-16 2024-06-28 艾维迪提生物科学公司 治疗面肩肱型肌营养不良的组合物和方法
WO2024155770A2 (en) * 2023-01-18 2024-07-25 Molecular Axiom, Llc Compositions for modulating kras expression and uses thereof
EP4669752A1 (en) * 2023-02-22 2025-12-31 Altamira Therapeutics AG KRAS COMPOSITIONS AND INHIBITION METHODS FOR THE TREATMENT OF A DISEASE

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US6977244B2 (en) 1996-10-04 2005-12-20 Board Of Regents, The University Of Texas Systems Inhibition of Bcl-2 protein expression by liposomal antisense oligodeoxynucleotides
US5891467A (en) 1997-01-31 1999-04-06 Depotech Corporation Method for utilizing neutral lipids to modify in vivo release from multivesicular liposomes
EP2314700A1 (en) 1999-01-28 2011-04-27 Medical College of Georgia Research Institute, Inc Composition and method for in vivo and in vitro attenuation of gene expression using double stranded RNA
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
CA2403397A1 (en) 2000-03-16 2001-09-20 Genetica, Inc. Methods and compositions for rna interference
US6680068B2 (en) 2000-07-06 2004-01-20 The General Hospital Corporation Drug delivery formulations and targeting
US20040234586A1 (en) 2001-06-11 2004-11-25 Sylvain Meloche Compositions and methods for enhancing nucleic acid transfer into cells
ATE468861T1 (de) 2001-08-16 2010-06-15 Univ Pennsylvania Synthese und verwendung von reagenzien für die verbesserte dna-lipofektion und/oder prodrug- und arzneimitteltherapien mit langsamer freisetzung
ATE485031T1 (de) 2002-06-28 2010-11-15 Protiva Biotherapeutics Inc Verfahren und vorrichtung zur herstellung von liposomen
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Also Published As

Publication number Publication date
WO2013166004A2 (en) 2013-11-07
EP3736333A1 (en) 2020-11-11
JP2015517466A (ja) 2015-06-22
EP3272868A1 (en) 2018-01-24
HK1206387A1 (en) 2016-01-08
CN109481455A (zh) 2019-03-19
CA3173610A1 (en) 2013-11-07
KR20150004414A (ko) 2015-01-12
AU2017261481A1 (en) 2017-11-30
US20180153919A1 (en) 2018-06-07
AU2013256471A1 (en) 2014-11-13
WO2013166004A3 (en) 2014-01-03
CA2872304A1 (en) 2013-11-07
EA201492004A1 (ru) 2015-08-31
AU2020200247A1 (en) 2020-02-06
AU2016204832A1 (en) 2016-07-28
BR112014027337A2 (pt) 2017-07-18
US20150238515A1 (en) 2015-08-27
MX2014013367A (es) 2014-12-08
CN104583406A (zh) 2015-04-29
EP2844757A2 (en) 2015-03-11
AU2022202991A1 (en) 2022-05-26

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