CA2755245A1 - Compositions and methods for the delivery of biologically active rnas - Google Patents

Compositions and methods for the delivery of biologically active rnas Download PDF

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Publication number
CA2755245A1
CA2755245A1 CA2755245A CA2755245A CA2755245A1 CA 2755245 A1 CA2755245 A1 CA 2755245A1 CA 2755245 A CA2755245 A CA 2755245A CA 2755245 A CA2755245 A CA 2755245A CA 2755245 A1 CA2755245 A1 CA 2755245A1
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CA
Canada
Prior art keywords
sequence
rna
sequences
biologically active
cell
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
CA2755245A
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English (en)
French (fr)
Inventor
Kevin Polach
Jason Fewell
Khursheed Anwer
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.)
Egen Inc
Original Assignee
Egen 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 Egen Inc filed Critical Egen Inc
Publication of CA2755245A1 publication Critical patent/CA2755245A1/en
Abandoned legal-status Critical Current

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    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • 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
    • 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
    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
CA2755245A 2009-03-13 2010-03-15 Compositions and methods for the delivery of biologically active rnas Abandoned CA2755245A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US16028709P 2009-03-13 2009-03-13
US16028809P 2009-03-13 2009-03-13
US61/160,287 2009-03-13
US61/160,288 2009-03-13
PCT/US2010/027365 WO2010105277A1 (en) 2009-03-13 2010-03-15 Compositions and methods for the delivery of biologically active rnas

Publications (1)

Publication Number Publication Date
CA2755245A1 true CA2755245A1 (en) 2010-09-16

Family

ID=42728852

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2755245A Abandoned CA2755245A1 (en) 2009-03-13 2010-03-15 Compositions and methods for the delivery of biologically active rnas

Country Status (8)

Country Link
US (2) US8569065B2 (OSRAM)
EP (1) EP2406379A4 (OSRAM)
JP (1) JP2012520085A (OSRAM)
KR (1) KR20110128345A (OSRAM)
CN (1) CN102625842A (OSRAM)
AU (1) AU2010223888A1 (OSRAM)
CA (1) CA2755245A1 (OSRAM)
WO (1) WO2010105277A1 (OSRAM)

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US9593347B2 (en) 2010-04-16 2017-03-14 University of Pittsburgh—of the Commonwealth System of Higher Education Identification of mutations in herpes simplex virus envelope glycoproteins that enable or enhance vector retargeting to novel non-HSV receptors
EP3578205A1 (en) 2010-08-06 2019-12-11 ModernaTX, Inc. A pharmaceutical formulation comprising engineered nucleic acids and medical use thereof
CN103429606A (zh) 2010-10-01 2013-12-04 现代治疗公司 设计核酸及其使用方法
CA2831613A1 (en) 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
EP2714971A4 (en) * 2011-05-23 2015-01-21 Phylogica Ltd METHOD FOR DETERMINING, IDENTIFYING AND INSULATING CELL-PENETRATING PEPTIDES
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
KR20190099538A (ko) 2011-10-03 2019-08-27 모더나 세라퓨틱스, 인코포레이티드 변형된 뉴클레오사이드, 뉴클레오타이드, 및 핵산, 및 이들의 용도
EP4144378A1 (en) 2011-12-16 2023-03-08 ModernaTX, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
CN104169419A (zh) * 2011-12-23 2014-11-26 艾根股份有限公司 用于输送生物活性rna的组合物和方法
US20140371302A1 (en) * 2011-12-29 2014-12-18 Modema Therapeutics, Inc. Modified mrnas encoding cell-penetrating polypeptides
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
AU2013243954A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
DK2922554T3 (en) 2012-11-26 2022-05-23 Modernatx Inc Terminalt modificeret rna
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
WO2014144423A2 (en) * 2013-03-15 2014-09-18 Techulon Inc. Antisense molecules for treatment of staphylococcus aureus infection
WO2015002956A1 (en) * 2013-07-01 2015-01-08 Ohio State Innovation Foundation Exosome delivery system
KR20160042917A (ko) 2013-07-25 2016-04-20 엑시큐어, 인크. 예방 및 치료 용도를 위한 면역자극제로서의 구형 핵산-기재 구축물
US10568898B2 (en) 2013-08-13 2020-02-25 Northwestern University Lipophilic nanoparticles for drug delivery
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
BR112016007255A2 (pt) 2013-10-03 2017-09-12 Moderna Therapeutics Inc polinucleotídeos que codificam receptor de lipoproteína de baixa densidade
EP3184641B1 (en) * 2013-10-28 2020-07-08 University of Pittsburgh - Of the Commonwealth System of Higher Education Oncolytic hsv vector
US10066240B2 (en) * 2014-02-14 2018-09-04 Symvivo Corporation Nucleic acid vectors and uses thereof
US10434064B2 (en) 2014-06-04 2019-10-08 Exicure, Inc. Multivalent delivery of immune modulators by liposomal spherical nucleic acids for prophylactic or therapeutic applications
CN106794256B (zh) 2014-08-19 2021-04-30 西北大学 蛋白质/寡核苷酸核-壳纳米颗粒治疗剂
WO2016057549A1 (en) 2014-10-06 2016-04-14 Aurasense Therapeutics, Llc Anti-tnf compounds
US9816080B2 (en) * 2014-10-31 2017-11-14 President And Fellows Of Harvard College Delivery of CAS9 via ARRDC1-mediated microvesicles (ARMMs)
US10078092B2 (en) 2015-03-18 2018-09-18 Northwestern University Assays for measuring binding kinetics and binding capacity of acceptors for lipophilic or amphiphilic molecules
SG11201806134SA (en) 2016-01-27 2018-08-30 Oncorus Inc Oncolytic viral vectors and uses thereof
US11639371B2 (en) * 2016-02-17 2023-05-02 The Chinese University Of Hong Kong Peptidylic inhibitors of nucleolin (NCL) targeting CAG-repeat RNA toxicity and methods for reducing polyQ-mediated toxicity in polyQ diseases
JP2019515654A (ja) 2016-03-16 2019-06-13 ザ ジェイ. デヴィッド グラッドストーン インスティテューツ 肥満及び/又は糖尿病を処置するための方法及び組成物、並びに候補処置薬剤を識別するための方法及び組成物
CA3029426A1 (en) 2016-06-30 2018-01-04 Oncorus, Inc. Pseudotyped oncolytic viral delivery of therapeutic polypeptides
US11612625B2 (en) 2017-07-26 2023-03-28 Oncorus, Inc. Oncolytic viral vectors and uses thereof
JP2021508477A (ja) 2017-12-29 2021-03-11 オンコラス, インコーポレイテッド 治療用ポリペプチドの腫瘍溶解性ウイルス送達
WO2020010083A1 (en) * 2018-07-05 2020-01-09 The Regents Of The University Of California Compositions and methods for delivery of rna to a cell
AU2020353149A1 (en) 2019-09-26 2022-04-14 President And Fellows Of Harvard College Minimal arrestin domain containing protein 1 (ARRDC1) constructs
WO2023244760A1 (en) * 2022-06-15 2023-12-21 Duke University Protein library display systems and methods thereof

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Also Published As

Publication number Publication date
CN102625842A (zh) 2012-08-01
WO2010105277A1 (en) 2010-09-16
US20100233141A1 (en) 2010-09-16
KR20110128345A (ko) 2011-11-29
US20140005254A1 (en) 2014-01-02
EP2406379A1 (en) 2012-01-18
AU2010223888A1 (en) 2011-10-06
US8569065B2 (en) 2013-10-29
EP2406379A4 (en) 2012-09-26
JP2012520085A (ja) 2012-09-06

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EEER Examination request

Effective date: 20150313

FZDE Discontinued

Effective date: 20170921