CA2731521C - Long-acting dna dendrimers and methods thereof - Google Patents

Long-acting dna dendrimers and methods thereof Download PDF

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Publication number
CA2731521C
CA2731521C CA2731521A CA2731521A CA2731521C CA 2731521 C CA2731521 C CA 2731521C CA 2731521 A CA2731521 A CA 2731521A CA 2731521 A CA2731521 A CA 2731521A CA 2731521 C CA2731521 C CA 2731521C
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dna
dendrimer
another embodiment
dna dendrimer
sirna
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CA2731521A
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French (fr)
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CA2731521A1 (en
Inventor
James M. Kadushin
Robert C. Getts
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Code Biotherapeutics Inc
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Genisphere LLC
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • 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
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04—Antibacterial agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/12—Antihypertensives
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Diabetes (AREA)
  • Oncology (AREA)
  • Hematology (AREA)
  • Communicable Diseases (AREA)
  • Cardiology (AREA)
  • Obesity (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Rheumatology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Virology (AREA)
  • Pain & Pain Management (AREA)
  • Endocrinology (AREA)
CA2731521A 2008-08-08 2009-08-10 Long-acting dna dendrimers and methods thereof Active CA2731521C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US18831808P 2008-08-08 2008-08-08
US61/188,318 2008-08-08
PCT/US2009/053264 WO2010017544A2 (en) 2008-08-08 2009-08-10 Long-acting dna dendrimers and methods thereof

Publications (2)

Publication Number Publication Date
CA2731521A1 CA2731521A1 (en) 2010-02-11
CA2731521C true CA2731521C (en) 2018-04-10

Family

ID=41664237

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2731521A Active CA2731521C (en) 2008-08-08 2009-08-10 Long-acting dna dendrimers and methods thereof

Country Status (7)

Country Link
US (1) US20120122800A1 (enExample)
EP (2) EP2657342B1 (enExample)
JP (1) JP5583667B2 (enExample)
AU (1) AU2009279458B2 (enExample)
CA (1) CA2731521C (enExample)
IL (1) IL210841A (enExample)
WO (1) WO2010017544A2 (enExample)

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US20110206611A1 (en) 2010-02-24 2011-08-25 Genisphere, Llc DNA Dendrimers as Thermal Ablation Devices
US20140228234A1 (en) 2011-06-15 2014-08-14 Naomi B. Zak Target-recognition compositions comprising novel synthetic conjugates for trapping and diagnosis of a target molecule
HK1201486A1 (en) * 2011-10-25 2015-09-04 Arch Personal Care Products, L.P. Composition containing an extract of a sequential or simultaneous fermentation
US9651549B2 (en) * 2012-07-13 2017-05-16 Genisphere, Llc Lateral flow assays using DNA dendrimers
CN105073141A (zh) * 2013-02-01 2015-11-18 詹尼斯费尔公司 用于将基因递送到细胞的功能化dna树状聚合物
EP2976109B1 (en) 2013-03-21 2018-07-25 Genisphere, LLC Cellular delivery of dna intercalating agents
EP3289084B1 (en) * 2015-04-17 2020-09-16 Genisphere, LLC siRNA INHIBITION OF HUMAN ANTIGEN R EXPRESSION FOR TREATMENT OF CANCER
JP7366541B2 (ja) * 2016-02-19 2023-10-23 コード バイオセラピューティクス インコーポレイテッド 核酸キャリア及び治療上の使用方法
EP3416691A4 (en) * 2016-02-19 2020-02-26 Genisphere, LLC SUPPORTS OF NUCLEIC ACIDS AND THEIR THERAPEUTIC METHODS OF USE
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
CN118667812A (zh) 2017-12-06 2024-09-20 艾维迪提生物科学公司 治疗肌萎缩和强直性肌营养不良的组合物和方法
US20190269728A1 (en) 2018-03-02 2019-09-05 Sixfold Bioscience Ltd. Compositions for delivery of cargo to cells
KR102544292B1 (ko) * 2018-05-11 2023-06-15 주고꾸 도료 가부시키가이샤 방오 도료 조성물, 방오 도막, 방오 도막 부착 기재 및 방오 방법
US20230183746A1 (en) 2019-08-30 2023-06-15 Sixfold Bioscience Ltd. Compositions for transfer of cargo to cells
IL289295B2 (en) * 2019-12-23 2025-07-01 Illumina Cambridge Ltd A nanoparticle with a single site for attaching a polynucleotide template
EP4121063A4 (en) 2020-03-19 2024-07-03 Avidity Biosciences, Inc. COMPOSITIONS AND METHODS FOR TREATING FACIO-SCAPULO-HUMERAL MUSCULAR DYSTROPHY
EP4143316A2 (en) 2020-04-27 2023-03-08 Sixfold Bioscience Ltd. Compositions containing nucleic acid nanoparticles with modular functionality
CN115916967A (zh) 2020-05-12 2023-04-04 伊鲁米纳公司 使用重组末端脱氧核苷酸转移酶生成具有经修饰的碱基的核酸
AU2021355846A1 (en) * 2020-10-09 2023-06-08 Sumitomo Pharma Co., Ltd. Oligonucleic acid conjugate
KR20240055874A (ko) 2021-09-16 2024-04-29 어비디티 바이오사이언시스 인크. 안면견갑상완 근이영양증을 치료하는 조성물 및 방법

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Also Published As

Publication number Publication date
JP2011530537A (ja) 2011-12-22
JP5583667B2 (ja) 2014-09-03
EP2320953B1 (en) 2015-07-15
IL210841A0 (en) 2011-04-28
WO2010017544A3 (en) 2011-03-24
CA2731521A1 (en) 2010-02-11
EP2320953A2 (en) 2011-05-18
WO2010017544A2 (en) 2010-02-11
EP2657342B1 (en) 2015-11-18
AU2009279458B2 (en) 2015-07-02
IL210841A (en) 2016-05-31
US20120122800A1 (en) 2012-05-17
AU2009279458A1 (en) 2010-02-11
EP2657342A1 (en) 2013-10-30

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