JP2024503623A - カプセル化ｒｎａポリヌクレオチド及び使用方法 - Google Patents

カプセル化ｒｎａポリヌクレオチド及び使用方法 Download PDF

Info

Publication number
JP2024503623A
JP2024503623A JP2023541041A JP2023541041A JP2024503623A JP 2024503623 A JP2024503623 A JP 2024503623A JP 2023541041 A JP2023541041 A JP 2023541041A JP 2023541041 A JP2023541041 A JP 2023541041A JP 2024503623 A JP2024503623 A JP 2024503623A
Authority
JP
Japan
Prior art keywords
sequence
lnp
lipid
cancer
mir
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.)
Pending
Application number
JP2023541041A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2022150485A5 (https=
JP2024503623A5 (https=
Inventor
ロレーナ ラーナー，
エドワード エム． ケネディ，
クリストフ クエバ，
ジェシカ デターリング，
ジェフリー デイビッド ブライアント，
チー－イン フー，
トゥーバ エー． チーマ，
ショーン エセックス，
Original Assignee
オンコラス， 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 オンコラス， インコーポレイテッド filed Critical オンコラス， インコーポレイテッド
Publication of JP2024503623A publication Critical patent/JP2024503623A/ja
Publication of JPWO2022150485A5 publication Critical patent/JPWO2022150485A5/ja
Publication of JP2024503623A5 publication Critical patent/JP2024503623A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/768—Oncolytic viruses not provided for in groups A61K35/761 - A61K35/766
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal 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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules 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
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C12N2770/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011—Details
    • C12N2770/32011—Picornaviridae
    • C12N2770/32021—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • C12N2770/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011—Details
    • C12N2770/32011—Picornaviridae
    • C12N2770/32051—Methods of production or purification of viral material
    • 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
    • C12N2770/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011—Details
    • C12N2770/32011—Picornaviridae
    • C12N2770/32061—Methods of inactivation or attenuation
    • C12N2770/32062—Methods of inactivation or attenuation by genetic engineering
    • 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
    • C12N2770/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011—Details
    • C12N2770/32011—Picornaviridae
    • C12N2770/32311—Enterovirus
    • C12N2770/32321—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • C12N2770/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011—Details
    • C12N2770/32011—Picornaviridae
    • C12N2770/32311—Enterovirus
    • C12N2770/32332—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • 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
    • C12N2770/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011—Details
    • C12N2770/32011—Picornaviridae
    • C12N2770/32311—Enterovirus
    • C12N2770/32351—Methods of production or purification of viral material
    • 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
    • C12N2770/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011—Details
    • C12N2770/32011—Picornaviridae
    • C12N2770/32311—Enterovirus
    • C12N2770/32361—Methods of inactivation or attenuation
    • C12N2770/32362—Methods of inactivation or attenuation by genetic engineering
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Virology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Microbiology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Mycology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Oncology (AREA)
  • Biophysics (AREA)
  • Dispersion Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Optics & Photonics (AREA)
  • Nanotechnology (AREA)
  • Immunology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
JP2023541041A 2021-01-06 2022-01-06 カプセル化ｒｎａポリヌクレオチド及び使用方法 Pending JP2024503623A (ja)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US202163134376P 2021-01-06 2021-01-06
US63/134,376 2021-01-06
US202163147959P 2021-02-10 2021-02-10
US63/147,959 2021-02-10
US202163181917P 2021-04-29 2021-04-29
US202163181663P 2021-04-29 2021-04-29
US202163181899P 2021-04-29 2021-04-29
US63/181,899 2021-04-29
US63/181,663 2021-04-29
US63/181,917 2021-04-29
PCT/US2022/011450 WO2022150485A1 (en) 2021-01-06 2022-01-06 Encapsulated rna polynucleotides and methods of use

Publications (3)

Publication Number Publication Date
JP2024503623A true JP2024503623A (ja) 2024-01-26
JPWO2022150485A5 JPWO2022150485A5 (https=) 2025-02-03
JP2024503623A5 JP2024503623A5 (https=) 2025-02-03

Family

ID=82357635

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023541041A Pending JP2024503623A (ja) 2021-01-06 2022-01-06 カプセル化ｒｎａポリヌクレオチド及び使用方法

Country Status (7)

Country Link
US (1) US20240115636A1 (https=)
EP (1) EP4274591A4 (https=)
JP (1) JP2024503623A (https=)
KR (1) KR20230143141A (https=)
AU (1) AU2022206676A1 (https=)
CA (1) CA3204244A1 (https=)
WO (1) WO2022150485A1 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023091490A1 (en) * 2021-11-16 2023-05-25 Senda Biosciences, Inc. Novel ionizable lipids and lipid nanoparticles and methods of using the same
WO2023223183A1 (en) * 2022-05-16 2023-11-23 Crispr Therapeutics Ag Picornaviral vectors for gene editing
WO2024031078A2 (en) * 2022-08-05 2024-02-08 Elevatebio Technologies, Inc. Chimeric oncolytic viruses with tropism for poliovirus receptor
EP4612284A2 (en) 2022-11-04 2025-09-10 Life Edit Therapeutics, Inc. Evolved adenine deaminases and rna-guided nuclease fusion proteins with internal insertion sites and methods of use
WO2024162304A1 (ja) 2023-01-31 2024-08-08 アステラス製薬株式会社 環状アミンを有するカルバモイル脂質又はウレア脂質、それを含む脂質ナノ粒子、及び医薬組成物
WO2024178397A2 (en) 2023-02-24 2024-08-29 Elevatebio Technologies, Inc. Modified immune effector cells and methods of use
WO2024226832A1 (en) * 2023-04-25 2024-10-31 Orgenesis Inc. Ribonucleases for treating viral infections
WO2025022367A2 (en) 2023-07-27 2025-01-30 Life Edit Therapeutics, Inc. Rna-guided nucleases and active fragments and variants thereof and methods of use
WO2025083619A1 (en) 2023-10-18 2025-04-24 Life Edit Therapeutics, Inc. Rna-guided nucleases and acive fragments and variants thereof and methods of use
TW202546224A (zh) 2024-02-12 2025-12-01 美商生命編輯治療學公司 新穎的ｒｎａ引導之核酸酶及用於聚合酶編輯之蛋白質
WO2026003754A1 (en) 2024-06-25 2026-01-02 Life Edit Therapeutics, Inc. Novel reverse transcriptases and uses thereof
WO2026006491A1 (en) * 2024-06-25 2026-01-02 Cornell University Senecavirus a (sva) vaccine compositions and methods thereof

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019210394A1 (en) * 2018-04-29 2019-11-07 Precision Nanosystems Inc. Compositions for transfecting resistant cell types
WO2020142725A1 (en) * 2019-01-04 2020-07-09 Oncorus, Inc. Encapsulated rna polynucleotides and methods of use
WO2020210901A1 (en) * 2019-04-15 2020-10-22 Precision Nanosystems Inc. Nonviral modification of t cell gene expression
JP2020530778A (ja) * 2017-07-14 2020-10-29 オンコラス， インコーポレイテッド カプセル封入ポリヌクレオチド及び使用方法
JP2020532528A (ja) * 2017-08-31 2020-11-12 モデルナティエックス インコーポレイテッドＭｏｄｅｒｎａＴＸ，Ｉｎｃ． 脂質ナノ粒子の生成方法

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006074526A1 (en) * 2005-01-17 2006-07-20 Viralytics Limited Method and composition for treatment of neoplasms
HRP20220652T1 (hr) * 2015-12-10 2022-06-24 Modernatx, Inc. Pripravci i postupci unosa terapijskih sredstava

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020530778A (ja) * 2017-07-14 2020-10-29 オンコラス， インコーポレイテッド カプセル封入ポリヌクレオチド及び使用方法
JP2020532528A (ja) * 2017-08-31 2020-11-12 モデルナティエックス インコーポレイテッドＭｏｄｅｒｎａＴＸ，Ｉｎｃ． 脂質ナノ粒子の生成方法
WO2019210394A1 (en) * 2018-04-29 2019-11-07 Precision Nanosystems Inc. Compositions for transfecting resistant cell types
WO2020142725A1 (en) * 2019-01-04 2020-07-09 Oncorus, Inc. Encapsulated rna polynucleotides and methods of use
WO2020210901A1 (en) * 2019-04-15 2020-10-22 Precision Nanosystems Inc. Nonviral modification of t cell gene expression

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
YADAVA KUMAR SUNIL ET AL.: "Low temperature, easy scaling up method for development of smart nanostructure hybrid lipid capsules", COLLOIDS AND SURFACES B: BIOINTERFACES , vol. Vol.190, 110927, JPN6025047184, 2 March 2020 (2020-03-02), pages 1 - 9, ISSN: 0005733160 *

Also Published As

Publication number Publication date
KR20230143141A (ko) 2023-10-11
US20240115636A1 (en) 2024-04-11
EP4274591A1 (en) 2023-11-15
AU2022206676A9 (en) 2024-05-09
AU2022206676A1 (en) 2023-08-24
CA3204244A1 (en) 2022-07-14
WO2022150485A1 (en) 2022-07-14
EP4274591A4 (en) 2024-12-11

Similar Documents

Publication Publication Date Title
JP2024503623A (ja) カプセル化ｒｎａポリヌクレオチド及び使用方法
JP2022516318A (ja) カプセル化ポリヌクレオチド及び使用方法
JP2022537154A (ja) 細胞治療のための環状ｒｎａ
CN112912509B (zh) T细胞的活化/增殖方法
US20260041643A1 (en) Stealth lipid nanoparticle compositions for cell targeting
US20230416308A1 (en) Encapsulated rna replicons and methods of use
KR20240035821A (ko) 단일 사슬 가변 단편(scFv) 변형 지질 나노입자 조성물 및 이의 용도
AU2022420620A1 (en) Circular polyribonucleotides encoding antifusogenic polypeptides
JP2025507571A (ja) チェックポイントがんワクチンをコードするｍＲＮＡ及びその使用
WO2024031078A2 (en) Chimeric oncolytic viruses with tropism for poliovirus receptor
US20250281556A1 (en) Production of rna polynucleotides encoding picornavirus
CN116916943A (zh) 包封的rna多核苷酸和使用方法
RU2806549C2 (ru) Способ активации/пролиферации t-клеток
WO2026055547A1 (en) Dna compositions and related methods
WO2025184694A1 (en) Nanoparticles and uses thereof
WO2025006684A1 (en) Circular polyribonucleotides encoding antifusogenic polypeptides

Legal Events

Date Code Title Description
A711 Notification of change in applicant

Free format text: JAPANESE INTERMEDIATE CODE: A711

Effective date: 20241119

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241225

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20241225

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20251118

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20260218