CA2625473A1 - Compounds and methods for peptide ribonucleic acid condensate particles for rna therapeutics - Google Patents

Compounds and methods for peptide ribonucleic acid condensate particles for rna therapeutics Download PDF

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Publication number
CA2625473A1
CA2625473A1 CA002625473A CA2625473A CA2625473A1 CA 2625473 A1 CA2625473 A1 CA 2625473A1 CA 002625473 A CA002625473 A CA 002625473A CA 2625473 A CA2625473 A CA 2625473A CA 2625473 A1 CA2625473 A1 CA 2625473A1
Authority
CA
Canada
Prior art keywords
compound
peptide
sirna
seq
particles
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.)
Abandoned
Application number
CA002625473A
Other languages
English (en)
French (fr)
Inventor
Roger C. Adami
Tianying Zhu
Kunyuan Cui
Michael E. Houston, Jr.
Lishan Chen
Yuching Chen
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.)
Marina Biotech Inc
Original Assignee
Individual
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 Individual filed Critical Individual
Publication of CA2625473A1 publication Critical patent/CA2625473A1/en
Abandoned legal-status Critical Current

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Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/001Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof by chemical synthesis
    • C07K14/003Peptide-nucleic acids (PNAs)
    • 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
    • A61K47/62Medicinal 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 a protein, peptide or polyamino acid
    • A61K47/64Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/645Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • A61K47/6455Polycationic oligopeptides, polypeptides or polyamino acids, e.g. for complexing nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/02Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • A61P31/16Antivirals for RNA viruses for influenza or rhinoviruses
    • 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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N15/1136Non-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 growth factors, growth regulators, cytokines, lymphokines or hormones
    • 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
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • 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
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/10Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10TECHNICAL SUBJECTS COVERED BY FORMER USPC
    • Y10TTECHNICAL SUBJECTS COVERED BY FORMER US CLASSIFICATION
    • Y10T428/00Stock material or miscellaneous articles
    • Y10T428/29Coated or structually defined flake, particle, cell, strand, strand portion, rod, filament, macroscopic fiber or mass thereof
    • Y10T428/2982Particulate matter [e.g., sphere, flake, etc.]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Rheumatology (AREA)
  • Endocrinology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Pain & Pain Management (AREA)
  • Pulmonology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
CA002625473A 2005-10-14 2006-10-13 Compounds and methods for peptide ribonucleic acid condensate particles for rna therapeutics Abandoned CA2625473A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US72721605P 2005-10-14 2005-10-14
US60/727,216 2005-10-14
US73366405P 2005-11-04 2005-11-04
US60/733,664 2005-11-04
US82587806P 2006-09-15 2006-09-15
US60/825,878 2006-09-15
PCT/US2006/040174 WO2007047482A2 (en) 2005-10-14 2006-10-13 Compounds and methods for peptide ribonucleic acid condensate particles for rna therapeutics

Publications (1)

Publication Number Publication Date
CA2625473A1 true CA2625473A1 (en) 2007-04-26

Family

ID=37963128

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002625473A Abandoned CA2625473A1 (en) 2005-10-14 2006-10-13 Compounds and methods for peptide ribonucleic acid condensate particles for rna therapeutics

Country Status (9)

Country Link
US (2) US20100129460A1 (enExample)
EP (1) EP1934360A2 (enExample)
JP (3) JP5536334B2 (enExample)
KR (1) KR20080061397A (enExample)
CN (1) CN101331231B (enExample)
AU (1) AU2006304291A1 (enExample)
CA (1) CA2625473A1 (enExample)
MX (1) MX2008004899A (enExample)
WO (1) WO2007047482A2 (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AR069704A1 (es) 2007-12-18 2010-02-10 Alcon Res Ltd Sistema de administracion de rnai de interferencia y usos del mismo
US9089610B2 (en) * 2008-08-19 2015-07-28 Nektar Therapeutics Complexes of small-interfering nucleic acids
WO2010059829A2 (en) * 2008-11-19 2010-05-27 Mdrna, Inc. Compositions and methods for triggered release rna therapeutics
WO2013020986A1 (de) 2011-08-08 2013-02-14 Universität Regensburg Polyanion-nanokomplexe für therapeutische anwendungen
EP2623978A1 (en) 2012-02-03 2013-08-07 Charité - Universitätsmedizin Berlin CD8+ T-cell subsets as markers for prediction of delayed fracture healing
US20140335192A1 (en) * 2013-05-09 2014-11-13 Kipperman, RM, Ch 7 BK Trustee for Traversa Therap Delivery of rna interfering agents
AU2016335158A1 (en) * 2015-10-05 2018-04-12 Devgen Nv Methods of preserving the biological activity of ribonucleic acids
EP4005634A4 (en) * 2019-07-24 2023-11-08 Tohoku University Chimeric molecule, pharmaceutical composition, method for cleaving target nucleic acid, and kit for target nucleic acid cleavage or diagnosis

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB9218164D0 (en) * 1992-08-26 1992-10-14 Applied Research Systems Virus and substances related thereto
US5679559A (en) * 1996-07-03 1997-10-21 University Of Utah Research Foundation Cationic polymer and lipoprotein-containing system for gene delivery
AU2281201A (en) * 1999-12-29 2001-07-09 A. James Mixson Histidine copolymer and methods for using same
AU2850001A (en) * 2000-02-07 2001-08-20 Transgene S.A. Non-naturally occurring nucleic acid compositions, their use for the preparationof formulations useful for transfecting nucleic acid into cells and applicati ons
WO2001095946A2 (en) * 2000-06-14 2001-12-20 Transgene S.A. Combination product for carrying out a cytotoxic treatment in a mammal
WO2002027031A2 (en) * 2000-09-28 2002-04-04 Cellomics, Inc. Methods and reagents for live-cell gene expression quantification
JP2004035409A (ja) * 2002-05-15 2004-02-05 Geneshuttle Biopharm Inc ベクターとしての使用のための新規融合タンパク質
US20040019008A1 (en) * 2002-05-28 2004-01-29 Lewis David L. Compositions and processes using siRNA, amphipathic compounds and polycations
US20030224055A1 (en) * 2002-05-28 2003-12-04 Lewis David L. Compositions and processes for inhibiting gene expression using polynucleotides
FR2841137B1 (fr) * 2002-06-20 2004-08-13 Bioalliance Pharma Systeme de vectorisation comprenant des nanoparticules de taille homogene d'au moins un polymere et d'au moins un polysaccharide charge positivement
WO2004028471A2 (en) * 2002-09-28 2004-04-08 Massachusetts Institute Of Technology Influenza therapeutic
AU2003219576A1 (en) * 2003-04-03 2004-10-25 Korea Advanced Institute Of Science And Technology Conjugate for gene transfer comprising oligonucleotide and hydrophilic polymer, polyelectrolyte complex micelles formed from the conjugate, and methods for preparation thereof
US20050136437A1 (en) * 2003-08-25 2005-06-23 Nastech Pharmaceutical Company Inc. Nanoparticles for delivery of nucleic acids and stable double-stranded RNA
WO2005056752A2 (en) * 2003-10-24 2005-06-23 Gencia Corporation Methods and compositions for delivering polynucleotides
WO2005071116A1 (en) * 2004-01-22 2005-08-04 University Of Massachusetts Modulation of hsv infection
US8691781B2 (en) * 2004-11-05 2014-04-08 Sirnaomics, Inc. Compositions for treating respiratory viral infections and their use
CN101060849A (zh) * 2004-11-17 2007-10-24 巴尔的摩马里兰大学 作为有效的siRNA载体的高分支HK肽
SG161267A1 (en) * 2005-04-12 2010-05-27 Intradigm Corp Composition and methods of rnai therapeutics for treatment of cancer and other neovascularization diseases
US9505867B2 (en) * 2005-05-31 2016-11-29 Ecole Polytechmique Fédérale De Lausanne Triblock copolymers for cytoplasmic delivery of gene-based drugs
US20070213257A1 (en) * 2005-08-12 2007-09-13 Nastech Pharmaceutical Company Inc. Compositions and methods for complexes of nucleic acids and peptides

Also Published As

Publication number Publication date
CN101331231B (zh) 2012-11-21
EP1934360A2 (en) 2008-06-25
HK1130506A1 (en) 2009-12-31
US20130072424A1 (en) 2013-03-21
KR20080061397A (ko) 2008-07-02
WO2007047482A2 (en) 2007-04-26
CN101331231A (zh) 2008-12-24
US20100129460A1 (en) 2010-05-27
JP2016073290A (ja) 2016-05-12
JP2014110796A (ja) 2014-06-19
WO2007047482A3 (en) 2007-11-29
AU2006304291A1 (en) 2007-04-26
JP5536334B2 (ja) 2014-07-02
WO2007047482B1 (en) 2008-01-17
MX2008004899A (es) 2008-09-04
JP2009511600A (ja) 2009-03-19

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EEER Examination request
FZDE Discontinued

Effective date: 20160921