JP2020530778A - カプセル封入ポリヌクレオチド及び使用方法 - Google Patents

カプセル封入ポリヌクレオチド及び使用方法 Download PDF

Info

Publication number
JP2020530778A
JP2020530778A JP2020523236A JP2020523236A JP2020530778A JP 2020530778 A JP2020530778 A JP 2020530778A JP 2020523236 A JP2020523236 A JP 2020523236A JP 2020523236 A JP2020523236 A JP 2020523236A JP 2020530778 A JP2020530778 A JP 2020530778A
Authority
JP
Japan
Prior art keywords
mir
recombinant dna
dna molecule
virus
polynucleotide
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
JP2020523236A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020530778A5 (https=
Inventor
ミッチェル エイチ． ファイナー，
ミッチェル エイチ． ファイナー，
エドワード ケネディ，
エドワード ケネディ，
ロレーナ ラーナー，
ロレーナ ラーナー，
Original Assignee
オンコラス， インコーポレイテッド
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, 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 JP2020530778A publication Critical patent/JP2020530778A/ja
Publication of JP2020530778A5 publication Critical patent/JP2020530778A5/ja
Priority to JP2023166265A priority Critical patent/JP2023165916A/ja
Pending legal-status Critical Current

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
    • C12N15/87—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
    • 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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • 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
    • C12N2770/32023—Virus like particles [VLP]
    • 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
    • C12N2770/32032—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
    • C12N2770/32041—Use of virus, viral particle or viral elements as a vector
    • C12N2770/32043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Peptides Or Proteins (AREA)
JP2020523236A 2017-07-14 2018-07-13 カプセル封入ポリヌクレオチド及び使用方法 Pending JP2020530778A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023166265A JP2023165916A (ja) 2017-07-14 2023-09-27 カプセル封入ポリヌクレオチド及び使用方法

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762532886P 2017-07-14 2017-07-14
US62/532,886 2017-07-14
US201862648651P 2018-03-27 2018-03-27
US62/648,651 2018-03-27
PCT/US2018/042136 WO2019014623A1 (en) 2017-07-14 2018-07-13 Encapsulated polynucleotides and methods of use

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023166265A Division JP2023165916A (ja) 2017-07-14 2023-09-27 カプセル封入ポリヌクレオチド及び使用方法

Publications (2)

Publication Number Publication Date
JP2020530778A true JP2020530778A (ja) 2020-10-29
JP2020530778A5 JP2020530778A5 (https=) 2021-08-12

Family

ID=65002120

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020523236A Pending JP2020530778A (ja) 2017-07-14 2018-07-13 カプセル封入ポリヌクレオチド及び使用方法
JP2023166265A Pending JP2023165916A (ja) 2017-07-14 2023-09-27 カプセル封入ポリヌクレオチド及び使用方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2023166265A Pending JP2023165916A (ja) 2017-07-14 2023-09-27 カプセル封入ポリヌクレオチド及び使用方法

Country Status (13)

Country Link
US (1) US20200224220A1 (https=)
EP (1) EP3652325A4 (https=)
JP (2) JP2020530778A (https=)
KR (1) KR20200036873A (https=)
CN (1) CN111212914A (https=)
AU (1) AU2018301701A1 (https=)
BR (1) BR112020000839A2 (https=)
CA (1) CA3069821A1 (https=)
IL (1) IL271969A (https=)
MX (1) MX2020000495A (https=)
RU (1) RU2020106730A (https=)
SG (1) SG11202000312UA (https=)
WO (1) WO2019014623A1 (https=)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023243187A1 (ja) 2022-06-14 2023-12-21 キユーピー株式会社 脂質ナノ粒子及びその製造方法
JP2024503623A (ja) * 2021-01-06 2024-01-26 オンコラス， インコーポレイテッド カプセル化ｒｎａポリヌクレオチド及び使用方法

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11612625B2 (en) 2017-07-26 2023-03-28 Oncorus, Inc. Oncolytic viral vectors and uses thereof
NZ762100A (en) 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
SG11202006457YA (en) 2018-01-05 2020-08-28 Ottawa Hospital Res Inst Modified orthopoxvirus vectors
CN111867609A (zh) * 2018-03-12 2020-10-30 梅约医学教育与研究基金会 用感染性核酸治疗癌症
EP3833766A1 (en) 2018-08-09 2021-06-16 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
HRP20240586T1 (hr) 2018-10-09 2024-07-19 The University Of British Columbia Sastavi i sustavi koji sadrže transfekcijske sposobne vezikle bez organskih otapala i deterdženta i s njim povezanim postupcima
MX2021005448A (es) * 2018-11-13 2021-08-11 Oncorus Inc Polinucleotidos encapsulados y metodos de uso.
JP2022516318A (ja) * 2019-01-04 2022-02-25 オンコラス， インコーポレイテッド カプセル化ポリヌクレオチド及び使用方法
WO2020185298A1 (en) * 2019-03-14 2020-09-17 Massachusetts Institute Of Technology Engineered herpes simplex virus-1 (hsv-1) vectors and uses thereof
US20220267799A1 (en) * 2019-08-05 2022-08-25 Virogin Biotech Canada Ltd Genetically modified enterovirus vectors
CA3157063A1 (en) * 2019-10-10 2021-04-15 Oncorus, Inc. Dual viruses and dual oncolytic viruses and methods of treatment
CA3180557A1 (en) * 2020-05-29 2021-12-02 Lorena Lerner Encapsulated rna replicons and methods of use
US20240261381A1 (en) * 2020-06-11 2024-08-08 Etherna Immunotherapies Nv Lipid nanoparticles
US20230242994A1 (en) * 2020-07-10 2023-08-03 Shanghai Miran Biotech Co., Ltd. Fluorescent cross-linked rnase h mutant conjugate, mirna combination and application thereof
CN113368261A (zh) * 2021-06-17 2021-09-10 苏州大学 一种非病毒载体及其制备方法与应用
WO2023069948A1 (en) 2021-10-18 2023-04-27 Flagship Pioneering Innovations Vii, Llc Dna compositions and related methods
WO2023225371A1 (en) * 2022-05-20 2023-11-23 Virogin Biotech Canada Ltd Genetically modified enterovirus vectors with enhanced genomic stability
CN118634238A (zh) * 2022-06-27 2024-09-13 浙江大学 抗口腔肿瘤药物活性成分及其用途

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050214923A1 (en) * 2004-03-25 2005-09-29 Yu Dechao Pan cancer oncolytic vectors and methods of use thereof
JP2006525815A (ja) * 2003-05-28 2006-11-16 ウィスコンシン・アラムナイ・リサーチ・ファウンデイション Ｐｏｌｉｉプロモーターおよびリボザイムを有する組換えインフルエンザベクター
JP2007506434A (ja) * 2003-09-26 2007-03-22 ノバルティス アクチエンゲゼルシャフト ＳｅｎｅｃａＶａｌｌｅｙウイルスベースの組成物および疾患の処置方法
WO2014171526A1 (ja) * 2013-04-17 2014-10-23 国立大学法人九州大学 遺伝子改変コクサッキーウイルス

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB0810912D0 (en) * 2008-06-13 2008-07-23 Inst Animal Health Ltd Vector
CN102227503B (zh) * 2008-09-26 2015-10-21 托卡根公司 重组载体
JP6025567B2 (ja) * 2009-12-16 2016-11-16 カッパーアールエヌエー，インコーポレイテッド 膜結合転写因子ペプチダーゼ、部位１（ｍｂｔｐｓ１）に対する天然アンチセンス転写物の阻害によるｍｂｔｐｓ１関連性疾患の治療
WO2013083753A2 (en) * 2011-12-07 2013-06-13 Institut Pasteur Identification of a swine parecho-like virus and applications
CA2868238A1 (en) * 2012-04-18 2013-10-24 Ramot At Tel Aviv University Ltd. Lipidated glycosaminoglycan particles for the delivery of nucleic acids
US10513711B2 (en) * 2014-08-13 2019-12-24 Dupont Us Holding, Llc Genetic targeting in non-conventional yeast using an RNA-guided endonuclease
AU2016274655B2 (en) * 2015-06-10 2021-06-17 NeoTrail Therapeutics, Inc. HPV vaccines
WO2017096201A1 (en) * 2015-12-02 2017-06-08 Memorial Sloan-Kettering Cancer Center Seneca valley virus (svv) cellular receptor targeted oncotherapy

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2006525815A (ja) * 2003-05-28 2006-11-16 ウィスコンシン・アラムナイ・リサーチ・ファウンデイション Ｐｏｌｉｉプロモーターおよびリボザイムを有する組換えインフルエンザベクター
JP2007506434A (ja) * 2003-09-26 2007-03-22 ノバルティス アクチエンゲゼルシャフト ＳｅｎｅｃａＶａｌｌｅｙウイルスベースの組成物および疾患の処置方法
US20050214923A1 (en) * 2004-03-25 2005-09-29 Yu Dechao Pan cancer oncolytic vectors and methods of use thereof
WO2014171526A1 (ja) * 2013-04-17 2014-10-23 国立大学法人九州大学 遺伝子改変コクサッキーウイルス

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
PLOS ONE, vol. Vol. 8, Issue 1, e55228, JPN7023001265, 2013, ISSN: 0005199486 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024503623A (ja) * 2021-01-06 2024-01-26 オンコラス， インコーポレイテッド カプセル化ｒｎａポリヌクレオチド及び使用方法
WO2023243187A1 (ja) 2022-06-14 2023-12-21 キユーピー株式会社 脂質ナノ粒子及びその製造方法

Also Published As

Publication number Publication date
EP3652325A4 (en) 2021-09-15
MX2020000495A (es) 2020-08-20
JP2023165916A (ja) 2023-11-17
CN111212914A (zh) 2020-05-29
IL271969A (en) 2020-02-27
BR112020000839A2 (pt) 2020-07-21
SG11202000312UA (en) 2020-02-27
CA3069821A1 (en) 2019-01-17
US20200224220A1 (en) 2020-07-16
EP3652325A1 (en) 2020-05-20
KR20200036873A (ko) 2020-04-07
AU2018301701A1 (en) 2020-02-27
WO2019014623A1 (en) 2019-01-17
RU2020106730A (ru) 2021-08-16

Similar Documents

Publication Publication Date Title
JP2020530778A (ja) カプセル封入ポリヌクレオチド及び使用方法
Shahryari et al. Engineering gene therapy: advances and barriers
KR20210113260A (ko) 캡슐화된 rna 폴리뉴클레오타이드 및 사용 방법
JP2022507269A (ja) カプセル化ポリヌクレオチド及び使用方法
JP7428664B2 (ja) 合成肝臓指向性アデノ随伴ウイルスカプシドおよびその使用
BR112020013319A2 (pt) vetores de dna de extremidade fechada obteníveis a partir de síntese livre de células e processo para obter vetores de cedna
JP2024503623A (ja) カプセル化ｒｎａポリヌクレオチド及び使用方法
JP7583732B2 (ja) フェニルアラニンヒドロキシラーゼ（ｐａｈ）治療薬を発現するための非ウイルス性ｄｎａベクターおよびその使用
CN113874513A (zh) 非病毒dna载体及其用于表达fviii治疗剂的用途
JP2023528300A (ja) 封入されたｒｎａレプリコンおよび使用方法
CA3157063A1 (en) Dual viruses and dual oncolytic viruses and methods of treatment
JP2022523806A (ja) 閉端ｄｎａ（ｃｅｄｎａ）および免疫調節化合物
KR20250129819A (ko) 핵산, 이온화 가능한 지질, 스테롤, 지질에 고정된 중합체, 및 헬퍼 지질을 포함하는 지질 나노입자 및 이의 용도
HK40029850A (en) Encapsulated polynucleotides and methods of use
CN119233824A (zh) 编码小核糖核酸病毒的rna多核苷酸的产生
Idrees et al. Sodium Channelopathies and Novel Viral/non-viral Vectors for their Gene Therapy
Jolly10 et al. Late Breaking Abstracts: Presented at the American Society of Gene & Cell Therapy’s 16th Annual Meeting, May 15–18, 2013, Salt Lake City, Utah
Enhancer-Blocking RNA ViRus VectoRs

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210630

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20210630

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20220531

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220627

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20220907

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20221115

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20221221

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230330

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20230619

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20230816

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20231120