MA53669A - Compositions et procédés d'administration d'acides nucléiques - Google Patents

Compositions et procédés d'administration d'acides nucléiques

Info

Publication number
MA53669A
MA53669A MA053669A MA53669A MA53669A MA 53669 A MA53669 A MA 53669A MA 053669 A MA053669 A MA 053669A MA 53669 A MA53669 A MA 53669A MA 53669 A MA53669 A MA 53669A
Authority
MA
Morocco
Prior art keywords
compositions
methods
nucleic acid
acid delivery
delivery
Prior art date
Application number
MA053669A
Other languages
English (en)
Inventor
Aaron Larsen
Melissa Moore
Jennifer Nelson
Original Assignee
Modernatx 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 Modernatx Inc filed Critical Modernatx Inc
Publication of MA53669A publication Critical patent/MA53669A/fr

Links

Classifications

    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67—General methods for enhancing the expression
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • 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
    • 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
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • C12N2310/11—Antisense
    • 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
    • C12N2310/15—Nucleic acids forming more than 2 strands, e.g. TFOs
    • C12N2310/152—Nucleic acids forming more than 2 strands, e.g. TFOs on a single-stranded target, e.g. fold-back TFOs
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2320/31—Combination therapy

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
MA053669A 2018-09-20 2019-09-20 Compositions et procédés d'administration d'acides nucléiques MA53669A (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201862734082P 2018-09-20 2018-09-20

Publications (1)

Publication Number Publication Date
MA53669A true MA53669A (fr) 2021-07-28

Family

ID=69887851

Family Applications (1)

Application Number Title Priority Date Filing Date
MA053669A MA53669A (fr) 2018-09-20 2019-09-20 Compositions et procédés d'administration d'acides nucléiques

Country Status (4)

Country Link
US (1) US20220298516A1 (fr)
EP (1) EP3852814A4 (fr)
MA (1) MA53669A (fr)
WO (1) WO2020061397A1 (fr)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11584932B2 (en) 2016-11-01 2023-02-21 The Research Foundation For The State University Of New York 5-halouracil-modified microRNAs and their use in the treatment of cancer
WO2021262919A2 (fr) 2020-06-26 2021-12-30 The Research Foundation For The State University Of New York Microarn modifiés par 5-halogéno-uracile et leur utilisation dans le traitement du cancer
JP2024518546A (ja) * 2021-05-12 2024-05-01 ザ ブロード インスティテュート，インコーポレーテッド 修飾されたｍＲＮＡ、修飾された非コードＲＮＡ、およびその使用
WO2023141474A1 (fr) * 2022-01-18 2023-07-27 The Broad Institute, Inc. Arnm poly-queue et poly-coiffe et ses utilisations
JP2025519910A (ja) * 2022-06-24 2025-06-26 ザ・ブロード・インスティテュート・インコーポレイテッド キャップされた環状ｒｎａ分子を調製するための組成物及び方法
EP4655398A1 (fr) * 2023-01-27 2025-12-03 Eleven Therapeutics Ltd Molécules polynucléotidiques artificielles pour une stabilité et une traduction améliorées
WO2025017029A1 (fr) * 2023-07-17 2025-01-23 Eleven Therapeutics Ltd Blocs de construction non naturels pour une traduction et une stabilité améliorées d'arnm

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2105364A1 (fr) * 1993-09-01 1995-03-02 Eric T. Kool Oligonucleotides en forme de trefle, renfermant des domaines de fixation parallele et antiparallele
WO2013185067A1 (fr) * 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Polynucléotides résistant à la nucléase et leurs utilisations
CA2921556A1 (fr) * 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Compositions et procedes pour la modulation d'arn
WO2015196118A1 (fr) * 2014-06-19 2015-12-23 Moderna Therapeutics, Inc. Molécules d'acide nucléique différentes et utilisations

Also Published As

Publication number Publication date
WO2020061397A1 (fr) 2020-03-26
US20220298516A1 (en) 2022-09-22
EP3852814A1 (fr) 2021-07-28
EP3852814A4 (fr) 2022-09-14

Similar Documents

Publication Publication Date Title
EP3846822A4 (fr) Compositions et procédés pour l'administration spécifique d'un organe d'acides nucléiques
MA53669A (fr) Compositions et procédés d'administration d'acides nucléiques
EP3846857A4 (fr) Compositions et procédés pour la délivrance spécifique d'organe d'acides nucléiques
MA53919A (fr) Constructions d'acides nucléiques et procédés d'utilisation
EP3880717A4 (fr) Compositions et procédés d'administration de polypeptides effecteurs crispr/cas
EP3317414A4 (fr) Compositions et procédés d'administration d'outils d'édition de gène au moyen de vésicules de polymère
EP3500585A4 (fr) Produits d'acides nucléiques et leurs procédés d'administration
MA51796A (fr) Procédés et compositions pour l'administration de protéines thérapeutiques
EP3554555A4 (fr) Compositions et procédés d'administration de charge d'acide nucléique et/ou de protéine
EP3452597A4 (fr) Procédés et compositions d'agents biologiquement actifs
EP3684397A4 (fr) Systèmes, procédés et compositions pour l'édition ciblée d'acides nucléiques
EP3790596A4 (fr) Compositions d'oligonucléotides et leurs procédés d'utilisation
EP3775203A4 (fr) Compositions d'oligonucléotides et leurs procédés d'utilisation
EP3813853A4 (fr) Compositions pour l'administration de médicaments et leurs méthodes d'utilisation
EP3982932A4 (fr) Compositions et procédés pour véhicules d'administration biologiques
EP3596213A4 (fr) Compositions et procédés d'amplification d'expression génique
EP3512554A4 (fr) Compositions plaquettaires et procédés pour l'administration d'agents thérapeutiques
EP3337515A4 (fr) Composés et procédés d'administration transmembranaire de molécules
EP3898942A4 (fr) Compositions de nanoparticules pour l'administration efficace d'acides nucléiques et leurs procédés de fabrication et d'utilisation
MA47824A (fr) Administration polyanionique d'acides nucléiques
EP3291765A4 (fr) Procédés d'administration d'un agent à l'oeil
EP3356522A4 (fr) Compositions contenant un exosome chargé d'une protéine, et procédés de préparation et d'administration correspondants
EP3880688A4 (fr) Préparations et compositions d'oligosaccharides
EP3801732A4 (fr) Procédés et systèmes d'administration de médicament
EP3472354A4 (fr) Réactions d'acides nucléiques et procédés et compositions associés