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)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (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 (enExample)
EP (1) EP2406379A4 (enExample)
JP (1) JP2012520085A (enExample)
KR (1) KR20110128345A (enExample)
CN (1) CN102625842A (enExample)
AU (1) AU2010223888A1 (enExample)
CA (1) CA2755245A1 (enExample)
WO (1) WO2010105277A1 (enExample)

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WO2011113054A2 (en) * 2010-03-12 2011-09-15 Aurasense Llc Crosslinked polynucleotide structure
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
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ES3005233T3 (en) 2010-10-01 2025-03-14 Modernatx Inc Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
JP2014511687A (ja) 2011-03-31 2014-05-19 モデルナ セラピューティクス インコーポレイテッド 工学操作された核酸の送達および製剤
WO2012159164A1 (en) * 2011-05-23 2012-11-29 Phylogica Limited Method of determining, identifying or isolating cell-penetrating peptides
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP2763701B1 (en) 2011-10-03 2018-12-19 Moderna Therapeutics, Inc. Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
JP2015501844A (ja) 2011-12-16 2015-01-19 モデルナ セラピューティクス インコーポレイテッドModerna Therapeutics,Inc. 修飾ヌクレオシド、ヌクレオチドおよび核酸組成物
KR20140123054A (ko) * 2011-12-23 2014-10-21 씨엘에스엔 래버러토리스, 인코퍼레이티드 생물학적 활성 rna의 전달을 위한 조성물 및 방법
WO2013101690A1 (en) * 2011-12-29 2013-07-04 modeRNA Therapeutics Modified mrnas encoding cell-penetrating polypeptides
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
WO2013151663A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of membrane proteins
ES2921623T3 (es) 2012-11-26 2022-08-30 Modernatx Inc ARN modificado terminalmente
US9909123B2 (en) * 2013-03-15 2018-03-06 Techulon Inc. Antisense molecules for treatment of Staphylococcus aureus infection
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
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
WO2015023797A1 (en) 2013-08-13 2015-02-19 Northwestern University Lipophilic nanoparticles for drug delivery
WO2015048744A2 (en) 2013-09-30 2015-04-02 Moderna Therapeutics, Inc. Polynucleotides encoding immune modulating polypeptides
KR20160067219A (ko) 2013-10-03 2016-06-13 모더나 세라퓨틱스, 인코포레이티드 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드
KR102490462B1 (ko) 2013-10-28 2023-01-19 유니버시티 오브 피츠버그-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션 종양분해성 hsv 벡터
WO2015120541A1 (en) * 2014-02-14 2015-08-20 Symvivo Corporation Nucleic acid vectors and uses thereof
EP3164113B1 (en) 2014-06-04 2019-03-27 Exicure, Inc. Multivalent delivery of immune modulators by liposomal spherical nucleic acids for prophylactic or therapeutic applications
WO2016028940A1 (en) 2014-08-19 2016-02-25 Northwestern University Protein/oligonucleotide core-shell nanoparticle therapeutics
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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
ES2919953T3 (es) 2016-01-27 2022-07-29 Oncorus Inc Vectores víricos oncolíticos y usos de los mismos
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
WO2017161043A1 (en) 2016-03-16 2017-09-21 The J. David Gladstone Institutes Methods and compositions for treating obesity and/or diabetes and for identifying candidate treatment agents
CN109983121A (zh) 2016-06-30 2019-07-05 昂克诺斯公司 治疗性多肽的假型化溶瘤病毒递送
RU2771110C2 (ru) 2017-07-26 2022-04-26 Онкорус, Инк. Онколитические вирусные векторы и их применение
EP3731850A4 (en) 2017-12-29 2021-12-01 Oncorus, Inc. ONCOLYTIC VIRUS DELIVERY OF THERAPEUTIC POLYPEPTIDES
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
JP2022550130A (ja) 2019-09-26 2022-11-30 プレジデント アンド フェローズ オブ ハーバード カレッジ 最小アレスチンドメイン含有蛋白質1(arrdc1)コンストラクト
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
US20100233141A1 (en) 2010-09-16
KR20110128345A (ko) 2011-11-29
JP2012520085A (ja) 2012-09-06
AU2010223888A1 (en) 2011-10-06
WO2010105277A1 (en) 2010-09-16
CN102625842A (zh) 2012-08-01
US8569065B2 (en) 2013-10-29
US20140005254A1 (en) 2014-01-02
EP2406379A1 (en) 2012-01-18
EP2406379A4 (en) 2012-09-26

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Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20150313

FZDE Discontinued

Effective date: 20170921