JP2022514501A - 細胞内治療モダリティを送達するためのアネロソーム - Google Patents

細胞内治療モダリティを送達するためのアネロソーム Download PDF

Info

Publication number
JP2022514501A
JP2022514501A JP2021533523A JP2021533523A JP2022514501A JP 2022514501 A JP2022514501 A JP 2022514501A JP 2021533523 A JP2021533523 A JP 2021533523A JP 2021533523 A JP2021533523 A JP 2021533523A JP 2022514501 A JP2022514501 A JP 2022514501A
Authority
JP
Japan
Prior art keywords
nucleic acid
anerosome
sequence
synthetic
orf1
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.)
Withdrawn
Application number
JP2021533523A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2020123753A5 (enExample
Inventor
エリカ ガブリエル ワインスタイン,
アバク カーベジアン,
サイモン デラグレイブ,
ネイサン ローレンス ヨズウィアク,
ケビン ジェイムズ レボ,
フェルナンド マーティン ディアズ,
ダナンジャイ マニクラール ナワンダル,
ライアン ディー. テッドストーン,
ジャレド デイビッド ピッツ,
Original Assignee
フラッグシップ パイオニアリング イノベーションズ ブイ, インコーポレイテッド
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 フラッグシップ パイオニアリング イノベーションズ ブイ, インコーポレイテッド filed Critical フラッグシップ パイオニアリング イノベーションズ ブイ, インコーポレイテッド
Publication of JP2022514501A publication Critical patent/JP2022514501A/ja
Publication of JPWO2020123753A5 publication Critical patent/JPWO2020123753A5/ja
Priority to JP2024075358A priority Critical patent/JP2024102242A/ja
Withdrawn legal-status Critical Current

Links

Images

Classifications

    • 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
    • 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
    • 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
    • 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

Landscapes

  • 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)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Botany (AREA)
  • Optics & Photonics (AREA)
  • Nanotechnology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2021533523A 2018-12-12 2019-12-12 細胞内治療モダリティを送達するためのアネロソーム Withdrawn JP2022514501A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024075358A JP2024102242A (ja) 2018-12-12 2024-05-07 細胞内治療モダリティを送達するためのアネロソーム

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862778851P 2018-12-12 2018-12-12
US201862778866P 2018-12-12 2018-12-12
US62/778,866 2018-12-12
US62/778,851 2018-12-12
PCT/US2019/065874 WO2020123753A2 (en) 2018-12-12 2019-12-12 Anellosomes for delivering intracellular therapeutic modalities

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024075358A Division JP2024102242A (ja) 2018-12-12 2024-05-07 細胞内治療モダリティを送達するためのアネロソーム

Publications (2)

Publication Number Publication Date
JP2022514501A true JP2022514501A (ja) 2022-02-14
JPWO2020123753A5 JPWO2020123753A5 (enExample) 2022-12-20

Family

ID=69160334

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021533523A Withdrawn JP2022514501A (ja) 2018-12-12 2019-12-12 細胞内治療モダリティを送達するためのアネロソーム
JP2024075358A Pending JP2024102242A (ja) 2018-12-12 2024-05-07 細胞内治療モダリティを送達するためのアネロソーム

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024075358A Pending JP2024102242A (ja) 2018-12-12 2024-05-07 細胞内治療モダリティを送達するためのアネロソーム

Country Status (11)

Country Link
US (1) US20220040117A1 (enExample)
EP (1) EP3894567A2 (enExample)
JP (2) JP2022514501A (enExample)
KR (1) KR20210131308A (enExample)
CN (1) CN114127302A (enExample)
AU (1) AU2019396516A1 (enExample)
BR (1) BR112021009282A2 (enExample)
CA (1) CA3119339A1 (enExample)
IL (1) IL283771A (enExample)
MX (1) MX2021006941A (enExample)
WO (1) WO2020123753A2 (enExample)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020123795A2 (en) * 2018-12-12 2020-06-18 Flagship Pioneering Innovations V, Inc. Anellosomes for delivering protein replacement therapeutic modalities
MX2021006977A (es) * 2018-12-12 2021-10-13 Flagship Pioneering Innovations V Inc Anellosomas y metodos de uso.
WO2021252955A1 (en) * 2020-06-12 2021-12-16 Flagship Pioneering Innovations V, Inc. Tandem anellovirus constructs
US20250059559A1 (en) * 2021-12-15 2025-02-20 Flagship Pioneering Innovations V, Inc. Surface-modified viral particles and modular viral particles
US20240415978A1 (en) * 2023-06-14 2024-12-19 Flagship Pioneering Innovations V, Inc. Anellovectors for delivery of effectors to the central nervous system
WO2025080985A1 (en) 2023-10-13 2025-04-17 Flagship Pioneering Innovations V, Inc. Tissue specific anellovector delivery

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2003525583A (ja) * 1999-02-05 2003-09-02 アボット・ラボラトリーズ Ttウイルスの使用法

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20200038236A (ko) * 2017-06-13 2020-04-10 플래그쉽 파이어니어링 이노베이션스 브이, 인크. 큐론을 포함하는 조성물 및 그의 용도

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2003525583A (ja) * 1999-02-05 2003-09-02 アボット・ラボラトリーズ Ttウイルスの使用法

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
EUR RESPIR J, vol. 42, JPN6024024624, 2013, pages 470 - 479, ISSN: 0005357535 *
J VIROLOGY, vol. 78, JPN6023043858, 2004, pages 10820 - 10824, ISSN: 0005357533 *
JOURNAL OF VIROLOGY, vol. 85, no. 14, JPN6024024625, 2011, pages 7284 - 7295, ISSN: 0005357534 *
VIROLOGY, vol. 321, JPN6023043859, 2004, pages 341 - 348, ISSN: 0005357532 *

Also Published As

Publication number Publication date
KR20210131308A (ko) 2021-11-02
CA3119339A1 (en) 2020-06-18
CN114127302A (zh) 2022-03-01
JP2024102242A (ja) 2024-07-30
MX2021006941A (es) 2021-11-17
EP3894567A2 (en) 2021-10-20
BR112021009282A2 (pt) 2021-08-17
WO2020123753A3 (en) 2020-07-23
US20220040117A1 (en) 2022-02-10
WO2020123753A2 (en) 2020-06-18
IL283771A (en) 2021-07-29
AU2019396516A1 (en) 2021-05-27

Similar Documents

Publication Publication Date Title
US11446344B1 (en) Anellovirus compositions and methods of use
US20230279423A1 (en) Compositions comprising curons and uses thereof
JP2022512395A (ja) 分泌治療モダリティを送達するためのアネロソーム
JP2022513797A (ja) アネロソーム及び使用の方法
JP2022513459A (ja) タンパク質代替治療モダリティを送達するためのアネロソーム
JP2022514501A (ja) 細胞内治療モダリティを送達するためのアネロソーム
CN116034160A (zh) 杆状病毒表达系统
WO2021252955A1 (en) Tandem anellovirus constructs

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20221212

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20221212

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20231107

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240205

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240403

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240507

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20240627

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241028

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20241225

A761 Written withdrawal of application

Free format text: JAPANESE INTERMEDIATE CODE: A761

Effective date: 20250218