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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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)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Diabetes (AREA)
  • Biophysics (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Hematology (AREA)
  • Emergency Medicine (AREA)
  • Endocrinology (AREA)
  • Neurology (AREA)
  • Rheumatology (AREA)
  • Pain & Pain Management (AREA)
  • Virology (AREA)
  • Obesity (AREA)
  • Neurosurgery (AREA)
  • Heart & Thoracic Surgery (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 (https=)
EP (2) EP2657342B1 (https=)
JP (1) JP5583667B2 (https=)
AU (1) AU2009279458B2 (https=)
CA (1) CA2731521C (https=)
IL (1) IL210841A (https=)
WO (1) WO2010017544A2 (https=)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CN103957997B (zh) * 2011-10-25 2018-09-21 奥麒个人护理产品公司 含有顺序发酵或同时发酵的提取物的组合物
US9651549B2 (en) * 2012-07-13 2017-05-16 Genisphere, Llc Lateral flow assays using DNA dendrimers
JP2016513089A (ja) * 2013-02-01 2016-05-12 ジェニスフィア・エルエルシー 細胞への遺伝子送達のための機能性ｄｎａデンドリマー
WO2014153394A1 (en) 2013-03-21 2014-09-25 Genisphere, Llc Cellular delivery of dna intercalating agents
CN107530439B (zh) * 2015-04-17 2021-06-18 詹尼斯费尔公司 用于治疗癌症的对人抗原R表达的siRNA抑制
WO2017143171A1 (en) * 2016-02-19 2017-08-24 Genisphere Llc Nucleic acid carriers and therapeutic methods of use
IL260983B (en) * 2016-02-19 2022-07-01 Genisphere Llc Nucleic acid carriers and methods of medical use
JP2019513371A (ja) 2016-04-01 2019-05-30 アビディティー バイオサイエンシーズ エルエルシー 核酸ポリペプチド組成物とその使用
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
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
EP4022058A2 (en) 2019-08-30 2022-07-06 Sixfold Bioscience Ltd. Compositions for transfer of cargo to cells
PH12021553295A1 (en) * 2019-12-23 2022-08-01 Illumina Cambridge Ltd Nanoparticle with single site for template polynucleotide attachment
US11555190B2 (en) 2020-03-19 2023-01-17 Avidity Biosciences, Inc. Compositions and methods of treating Facioscapulohumeral muscular dystrophy
WO2021220053A2 (en) 2020-04-27 2021-11-04 Sixfold Bioscience Ltd. Compositions containing nucleic acid nanoparticles with modular functionality
KR20230035237A (ko) 2020-05-12 2023-03-13 일루미나, 인코포레이티드 재조합 말단 데옥시뉴클레오티딜 트랜스퍼라제를 사용하여 변형된 염기를 갖는 핵산의 생성
MX2023004104A (es) * 2020-10-09 2023-06-29 Sumitomo Pharma Co Ltd Conjugado de acido oligonucleico.
MX2024003266A (es) 2021-09-16 2024-04-03 Avidity Biosciences Inc Composiciones y metodos de tratamiento de la distrofia muscular facioescapulohumeral.
EP4703381A1 (en) 2024-09-03 2026-03-04 Institució Catalana de Recerca i Estudis Avançats Antibodies against icam-1 and applications thereof

Family Cites Families (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NL154600B (nl) 1971-02-10 1977-09-15 Organon Nv Werkwijze voor het aantonen en bepalen van specifiek bindende eiwitten en hun corresponderende bindbare stoffen.
NL154598B (nl) 1970-11-10 1977-09-15 Organon Nv Werkwijze voor het aantonen en bepalen van laagmoleculire verbindingen en van eiwitten die deze verbindingen specifiek kunnen binden, alsmede testverpakking.
NL154599B (nl) 1970-12-28 1977-09-15 Organon Nv Werkwijze voor het aantonen en bepalen van specifiek bindende eiwitten en hun corresponderende bindbare stoffen, alsmede testverpakking.
US3901654A (en) 1971-06-21 1975-08-26 Biological Developments Receptor assays of biologically active compounds employing biologically specific receptors
US3853987A (en) 1971-09-01 1974-12-10 W Dreyer Immunological reagent and radioimmuno assay
US3867517A (en) 1971-12-21 1975-02-18 Abbott Lab Direct radioimmunoassay for antigens and their antibodies
NL171930C (nl) 1972-05-11 1983-06-01 Akzo Nv Werkwijze voor het aantonen en bepalen van haptenen, alsmede testverpakkingen.
US3850578A (en) 1973-03-12 1974-11-26 H Mcconnell Process for assaying for biologically active molecules
US3935074A (en) 1973-12-17 1976-01-27 Syva Company Antibody steric hindrance immunoassay with two antibodies
US3996345A (en) 1974-08-12 1976-12-07 Syva Company Fluorescence quenching with immunological pairs in immunoassays
US4034074A (en) 1974-09-19 1977-07-05 The Board Of Trustees Of Leland Stanford Junior University Universal reagent 2-site immunoradiometric assay using labelled anti (IgG)
US3984533A (en) 1975-11-13 1976-10-05 General Electric Company Electrophoretic method of detecting antigen-antibody reaction
US4098876A (en) 1976-10-26 1978-07-04 Corning Glass Works Reverse sandwich immunoassay
US4879219A (en) 1980-09-19 1989-11-07 General Hospital Corporation Immunoassay utilizing monoclonal high affinity IgM antibodies
US5011771A (en) 1984-04-12 1991-04-30 The General Hospital Corporation Multiepitopic immunometric assay
US4666828A (en) 1984-08-15 1987-05-19 The General Hospital Corporation Test for Huntington's disease
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4801531A (en) 1985-04-17 1989-01-31 Biotechnology Research Partners, Ltd. Apo AI/CIII genomic polymorphisms predictive of atherosclerosis
US5175270A (en) 1986-09-10 1992-12-29 Polyprobe, Inc. Reagents for detecting and assaying nucleic acid sequences
US5272057A (en) 1988-10-14 1993-12-21 Georgetown University Method of detecting a predisposition to cancer by the use of restriction fragment length polymorphism of the gene for human poly (ADP-ribose) polymerase
US5192659A (en) 1989-08-25 1993-03-09 Genetype Ag Intron sequence analysis method for detection of adjacent and remote locus alleles as haplotypes
US5281521A (en) 1992-07-20 1994-01-25 The Trustees Of The University Of Pennsylvania Modified avidin-biotin technique
US6117631A (en) 1996-10-29 2000-09-12 Polyprobe, Inc. Detection of antigens via oligonucleotide antibody conjugates
EP1005573B1 (en) 1997-07-29 2006-05-24 Polyprobe, Inc. Dendritic nucleic acids exhibiting maximal self-assembly
US20070173473A1 (en) * 2001-05-18 2007-07-26 Sirna Therapeutics, Inc. RNA interference mediated inhibition of proprotein convertase subtilisin Kexin 9 (PCSK9) gene expression using short interfering nucleic acid (siNA)
US20040023248A1 (en) * 2001-12-07 2004-02-05 Whitehead Institiute For Biomedical Research Methods and reagents for improving nucleic acid detection
EP1530581A4 (en) * 2002-06-12 2006-08-30 Datascope Investment Corp POLYMERIC MARKING MOLECULES
US7223544B2 (en) * 2003-06-27 2007-05-29 Cornell Research Foundation, Inc. Nucleic acid-engineered materials
KR101147147B1 (ko) * 2004-04-01 2012-05-25 머크 샤프 앤드 돔 코포레이션 Ｒｎａ 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드
WO2005113571A2 (en) * 2004-05-13 2005-12-01 The University Of North Carolina At Chapel Hill Methods for the delivery of oligomeric compounds
US8067572B2 (en) * 2005-05-25 2011-11-29 The University Of York Hybrid interfering RNA
US20100136614A1 (en) * 2005-10-18 2010-06-03 Dan Luo Dendrimer-like modular delivery vector
WO2008147526A1 (en) * 2007-05-23 2008-12-04 The Trustees Of The University Of Pennsylvania Targeted carriers for intracellular drug delivery
US9029524B2 (en) * 2007-12-10 2015-05-12 California Institute Of Technology Signal activated RNA interference

Also Published As

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

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