BR112021009379A2 - anelossomos para entrega de modalidades terapêuticas secretadas - Google Patents

anelossomos para entrega de modalidades terapêuticas secretadas Download PDF

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Publication number
BR112021009379A2
BR112021009379A2 BR112021009379-6A BR112021009379A BR112021009379A2 BR 112021009379 A2 BR112021009379 A2 BR 112021009379A2 BR 112021009379 A BR112021009379 A BR 112021009379A BR 112021009379 A2 BR112021009379 A2 BR 112021009379A2
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BR
Brazil
Prior art keywords
nucleic acid
sequence
genetic element
orf1
acid sequence
Prior art date
Application number
BR112021009379-6A
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English (en)
Portuguese (pt)
Inventor
Erica Gabrielle Weinstein
Avak Kahvejian
Simon Delagrave
Lawrence Yozwiak Nathan
Kevin James Lebo
Fernando Martin Diaz
Dhananjay Maniklal Nawandar
D. Tedstone Ryan
David Pitts Jared
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Flagship Pioneering Innovations V, Inc
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Publication date
Application filed by Flagship Pioneering Innovations V, Inc filed Critical Flagship Pioneering Innovations V, Inc
Publication of BR112021009379A2 publication Critical patent/BR112021009379A2/pt

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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
    • C12N15/63Introduction 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
    • C12N15/86Viral vectors
    • 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
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/46Ingredients of undetermined constitution or reaction products thereof, e.g. skin, bone, milk, cotton fibre, eggshell, oxgall or plant extracts
    • 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
    • A61K48/0008Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51Nanocapsules; Nanoparticles
    • A61K9/5107Excipients; Inactive ingredients
    • A61K9/5176Compounds of unknown constitution, e.g. material from plants or animals
    • A61K9/5184Virus capsids or envelopes enclosing drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • 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
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/00022New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/00023Virus like particles [VLP]
    • 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
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/00041Use of virus, viral particle or viral elements as a vector
    • C12N2750/00042Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • 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
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/00041Use of virus, viral particle or viral elements as a vector
    • C12N2750/00043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Botany (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
BR112021009379-6A 2018-12-12 2019-12-12 anelossomos para entrega de modalidades terapêuticas secretadas BR112021009379A2 (pt)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862778869P 2018-12-12 2018-12-12
US201862778866P 2018-12-12 2018-12-12
US62/778,869 2018-12-12
US62/778,866 2018-12-12
PCT/US2019/065919 WO2020123773A2 (en) 2018-12-12 2019-12-12 Anellosomes for delivering secreted therapeutic modalities

Publications (1)

Publication Number Publication Date
BR112021009379A2 true BR112021009379A2 (pt) 2021-08-17

Family

ID=69160343

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021009379-6A BR112021009379A2 (pt) 2018-12-12 2019-12-12 anelossomos para entrega de modalidades terapêuticas secretadas

Country Status (11)

Country Link
US (2) US20230048858A1 (https=)
EP (1) EP3894568A2 (https=)
JP (2) JP2022512395A (https=)
KR (1) KR20210131309A (https=)
CN (1) CN113631717A (https=)
AU (1) AU2019396520A1 (https=)
BR (1) BR112021009379A2 (https=)
CA (1) CA3119531A1 (https=)
IL (1) IL283778A (https=)
MX (1) MX2021006943A (https=)
WO (1) WO2020123773A2 (https=)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018201160A1 (en) 2017-04-28 2018-11-01 Agrospheres, Inc. Compositions and methods for enzyme immobilization
CA3073449A1 (en) 2017-09-25 2019-03-28 Agrospheres, Inc. Compositions and methods for scalable production and delivery of biologicals
MX2021006977A (es) * 2018-12-12 2021-10-13 Flagship Pioneering Innovations V Inc Anellosomas y metodos de uso.
CA3119814A1 (en) * 2018-12-12 2020-06-18 Flagship Pioneering Innovations V, Inc. Anellosomes for delivering protein replacement therapeutic modalities
MX2022016475A (es) * 2020-06-17 2023-04-11 Flagship Pioneering Innovations V Inc Metodos de identificacion y caracterizacion de anellovirus y usos de los mismos.
WO2022094238A2 (en) * 2020-10-29 2022-05-05 Flagship Pioneering Innovations V, Inc. Chicken anemia virus (cav)-based vectors
EP4288115A4 (en) * 2021-02-08 2025-06-18 Flagship Pioneering Innovations V, Inc. Hybrid aav-anellovectors
EP4337235A4 (en) * 2021-05-12 2025-07-02 Flagship Pioneering Innovations V Inc ANELLOVECTORS AND METHODS OF USE
IL311987A (en) * 2021-10-12 2024-06-01 Flagship Pioneering Innovations V Inc New analvector compositions and methods
CN118284411A (zh) * 2021-10-12 2024-07-02 旗舰先锋创新V股份有限公司 新颖的指环载体组合物和方法
TW202430215A (zh) 2022-12-14 2024-08-01 美商旗艦先鋒創新有限責任(Vii)公司 用於將治療劑遞送至骨之組成物和方法
TW202529819A (zh) 2023-10-13 2025-08-01 美商旗艦先鋒創新公司 組織特異性指環載體(anellovector)遞送
CN117695410B (zh) * 2024-02-06 2024-05-03 中国人民解放军军事科学院军事医学研究院 CRISPR/Cas9纳米抗菌剂及其制备方法

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6395472B1 (en) * 1999-02-05 2002-05-28 Abbott Laboratories Methods of utilizing the TT virus
JP7103750B2 (ja) * 2013-12-12 2022-07-20 ザ・ブロード・インスティテュート・インコーポレイテッド ゲノム編集のためのCRISPR-Cas系及び組成物の送達、使用及び治療適用
CN108291216B (zh) * 2015-09-24 2022-09-16 宾夕法尼亚州大学信托人 用于治疗补体介导的疾病的组合物和方法
IL271275B1 (en) * 2017-06-13 2026-04-01 Flagship Pioneering Innovations V Inc Preparations containing croons and their uses
MX2020003888A (es) * 2017-10-18 2020-11-06 Regenxbio Inc Anticuerpos terapeuticos postraduccionalmente modificados completamente humanos.

Also Published As

Publication number Publication date
WO2020123773A2 (en) 2020-06-18
US20230048858A1 (en) 2023-02-16
US20260009050A1 (en) 2026-01-08
KR20210131309A (ko) 2021-11-02
WO2020123773A9 (en) 2020-08-27
IL283778A (en) 2021-07-29
JP2022512395A (ja) 2022-02-03
CN113631717A (zh) 2021-11-09
JP2024100936A (ja) 2024-07-26
CA3119531A1 (en) 2020-06-18
WO2020123773A3 (en) 2020-07-23
AU2019396520A1 (en) 2021-05-27
EP3894568A2 (en) 2021-10-20
MX2021006943A (es) 2021-11-17

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