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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Prior art keywords
dna
dendrimer
another embodiment
dna dendrimer
sirna
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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)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Diabetes (AREA)
  • Physics & Mathematics (AREA)
  • Hematology (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Endocrinology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Neurosurgery (AREA)
  • Emergency Medicine (AREA)
  • Neurology (AREA)
  • Rheumatology (AREA)
  • Pain & Pain Management (AREA)
  • Obesity (AREA)
  • Cardiology (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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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
WO2014121050A1 (en) * 2013-02-01 2014-08-07 Genisphere, Llc Functionalized dna dendrimers for gene delivery to cells
JP6346265B2 (ja) 2013-03-21 2018-06-20 ジェニスフィア・エルエルシー Ｄｎａインターカレーティング剤の細胞送達
EP3289084B1 (en) * 2015-04-17 2020-09-16 Genisphere, LLC siRNA INHIBITION OF HUMAN ANTIGEN R EXPRESSION FOR TREATMENT OF CANCER
JP2019513692A (ja) * 2016-02-19 2019-05-30 ジェニスフィア・エルエルシー 核酸キャリア及び治療上の使用方法
WO2017143156A1 (en) * 2016-02-19 2017-08-24 Genisphere Llc Nucleic acid carriers and therapeutic methods of use
KR102775461B1 (ko) 2016-04-01 2025-02-28 어비디티 바이오사이언시스 인크. 핵산-폴리펩타이드 조성물 및 이의 용도
CN118638787A (zh) 2017-12-06 2024-09-13 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
EP3759225A1 (en) 2018-03-02 2021-01-06 Sixfold Bioscience Ltd. Compositions for delivery of cargo to cells
JP7082659B2 (ja) * 2018-05-11 2022-06-08 中国塗料株式会社 防汚塗料組成物、防汚塗膜、防汚塗膜付き基材及び防汚方法
WO2021053405A2 (en) 2019-08-30 2021-03-25 Sixfold Bioscience Ltd. Compositions for transfer of cargo to cells
BR112021026769A2 (pt) * 2019-12-23 2022-07-26 Illumina Inc Nanopartícula com sítio único para fixação de polinucleotídeo-molde e métodos
TW202144572A (zh) 2020-03-19 2021-12-01 美商亞維代堤生物科學公司 治療臉肩胛肱骨肌肉失養症之組合物及方法
CN115997016A (zh) 2020-04-27 2023-04-21 西克斯福德生物科学有限公司 包含具有模块化官能团的核酸纳米颗粒的组合物
US12209266B2 (en) 2020-05-12 2025-01-28 Illumina, Inc. Generating nucleic acids with modified bases using recombinant terminal deoxynucleotidyl transferase
KR20230084520A (ko) * 2020-10-09 2023-06-13 스미토모 파마 가부시키가이샤 올리고 핵산 콘쥬게이트
EP4401792A4 (en) 2021-09-16 2025-12-03 Avidity Biosciences Inc COMPOSITIONS AND METHODS OF TREATMENT OF FACIO-SCAPULO-HUMERAL MUSCULAR DYSTROPHY
EP4703381A1 (en) 2024-09-03 2026-03-04 Institució Catalana de Recerca i Estudis Avançats Antibodies against icam-1 and applications thereof

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Also Published As

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

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