JP2021514207A - 改変ウイルスカプシド - Google Patents

改変ウイルスカプシド Download PDF

Info

Publication number
JP2021514207A
JP2021514207A JP2020566878A JP2020566878A JP2021514207A JP 2021514207 A JP2021514207 A JP 2021514207A JP 2020566878 A JP2020566878 A JP 2020566878A JP 2020566878 A JP2020566878 A JP 2020566878A JP 2021514207 A JP2021514207 A JP 2021514207A
Authority
JP
Japan
Prior art keywords
seq
viral
candidate
modified
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
JP2020566878A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021514207A5 (https=
Inventor
ビョークルン，トマス
デイヴィッドソン，マーカス
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Individual
Original Assignee
Individual
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 Individual filed Critical Individual
Publication of JP2021514207A publication Critical patent/JP2021514207A/ja
Publication of JP2021514207A5 publication Critical patent/JP2021514207A5/ja
Priority to JP2024014824A priority Critical patent/JP2024050766A/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1034—Isolating an individual clone by screening libraries
    • C12N15/1082—Preparation or screening gene libraries by chromosomal integration of polynucleotide sequences, HR-, site-specific-recombination, transposons, viral vectors
    • 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
    • C12N15/69—Increasing the copy number of the vector
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use 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)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Bioinformatics & Computational Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
JP2020566878A 2018-02-15 2019-02-14 改変ウイルスカプシド Pending JP2021514207A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024014824A JP2024050766A (ja) 2018-02-15 2024-02-02 改変ウイルスカプシド

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP18156932 2018-02-15
EP18156932.8 2018-02-15
PCT/EP2019/053610 WO2019158619A1 (en) 2018-02-15 2019-02-14 Modified viral capsids

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024014824A Division JP2024050766A (ja) 2018-02-15 2024-02-02 改変ウイルスカプシド

Publications (2)

Publication Number Publication Date
JP2021514207A true JP2021514207A (ja) 2021-06-10
JP2021514207A5 JP2021514207A5 (https=) 2022-02-22

Family

ID=61521290

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020566878A Pending JP2021514207A (ja) 2018-02-15 2019-02-14 改変ウイルスカプシド
JP2024014824A Pending JP2024050766A (ja) 2018-02-15 2024-02-02 改変ウイルスカプシド

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024014824A Pending JP2024050766A (ja) 2018-02-15 2024-02-02 改変ウイルスカプシド

Country Status (9)

Country Link
US (2) US20210107947A1 (https=)
EP (1) EP3752521A1 (https=)
JP (2) JP2021514207A (https=)
CN (1) CN112105630A (https=)
AU (2) AU2019220368B2 (https=)
BR (1) BR112020016666A2 (https=)
CA (1) CA3095660A1 (https=)
SG (1) SG11202009854XA (https=)
WO (1) WO2019158619A1 (https=)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023518809A (ja) * 2020-03-20 2023-05-08 ディグニティー ヘルス アデノ随伴ウイルスの改変方法及び分離方法
CN116390773A (zh) 2020-08-06 2023-07-04 应用医学研究基金会 用于通过基因疗法治疗诸如阿尔茨海默病的Tau蛋白病的病毒颗粒
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
CN115894659B (zh) * 2022-10-19 2023-11-10 上海优宁维生物科技股份有限公司 微管相关蛋白Tau抗原及其制备方法和应用
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
AU2024332181A1 (en) 2023-08-31 2026-02-12 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2025082618A1 (en) 2023-10-20 2025-04-24 Fuse Vectors Aps In vitro parvovirus capsid manufacturing
TW202545971A (zh) 2024-02-08 2025-12-01 美商戴諾治療公司 殼體多肽及其使用方法
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016134375A1 (en) * 2015-02-20 2016-08-25 University Of Iowa Research Foundation Methods and compositions for treating genetic eye diseases
JP2016533709A (ja) * 2013-09-26 2016-11-04 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド 標的遺伝子治療のための合成コンビナトリアルａａｖカプシドライブラリー
WO2017192750A1 (en) * 2016-05-04 2017-11-09 Oregon Health & Science University Recombinant adeno-associated viral vectors
JP2017534601A (ja) * 2014-10-03 2017-11-24 ユニバーシティ オブ マサチューセッツ 新規の高効率ライブラリーにより同定されるａａｖベクター
WO2018022905A2 (en) * 2016-07-29 2018-02-01 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU6368901A (en) * 2000-05-31 2001-12-11 Univ Saskatchewan Modified bovine adenovirus having altered tropism
SI1496944T1 (sl) * 2002-05-01 2009-02-28 Univ Florida Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinov
WO2015117010A2 (en) * 2014-01-31 2015-08-06 Temple University Of The Commonwealth System Of Higher Education Bag3 as a target for therapy of heart failure
AU2017341849B2 (en) * 2016-10-13 2024-03-21 University Of Massachusetts AAV capsid designs
WO2018119330A2 (en) * 2016-12-22 2018-06-28 Oregon Health & Science University Adeno associated viral vectors
EP3585883A4 (en) * 2017-02-21 2021-04-14 University of Florida Research Foundation, Incorporated MODIFIED AAV CAPSID PROTEINS AND USES THEREOF
CA3053154A1 (en) * 2017-06-30 2019-01-03 The Regents Of The University Of California Adeno-associated virus virions with variant capsids and methods of use thereof

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016533709A (ja) * 2013-09-26 2016-11-04 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド 標的遺伝子治療のための合成コンビナトリアルａａｖカプシドライブラリー
JP2017534601A (ja) * 2014-10-03 2017-11-24 ユニバーシティ オブ マサチューセッツ 新規の高効率ライブラリーにより同定されるａａｖベクター
WO2016134375A1 (en) * 2015-02-20 2016-08-25 University Of Iowa Research Foundation Methods and compositions for treating genetic eye diseases
WO2017192750A1 (en) * 2016-05-04 2017-11-09 Oregon Health & Science University Recombinant adeno-associated viral vectors
WO2018022905A2 (en) * 2016-07-29 2018-02-01 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
"A BARCODE-BASED IN VIVO SCREENING METHOD FOR CREATING NOVEL AAV SEROTYPES FOR CNS-DIRECTED GENE THER", MOLECULAR THERAPY, vol. Vol.25, No.5, Suppl. 1, 508, JPN5021006654, 2017, pages 233 - 234, ISSN: 0005318772 *
"DEVELOPMENT OF A UNIVERSAL AAV BARCODE-SEQ SYSTEM EXPRESSING RNA BARCODES", MOLECULAR THERAPY, vol. Vol.22, Suppl.1, 288, JPN5021006656, 2014, pages 111, ISSN: 0004983417 *
"MAPPING OF CONFORMATIONAL EPITOPES OF MONOCLONAL AND POLYCLONAL ANTIBODIES AGAINST AAV CAPSIDS BY A", MOLECULAR THERAPY, vol. Vol.25, No.5S1, 309, JPN5021006657, 2017, pages 145 - 146, ISSN: 0004983418 *
"NOVEL BARCODE-BASED IN VIVO SCREENING METHOD FOR GENERATING DE NOVO AAV SEROTYPES FOR CNS-DIRECTED G", MOLECULAR THERAPY, vol. Vol.24, Suppl.1, 542, JPN5021006655, 2016, pages 216 - 217, ISSN: 0004983416 *
"USING NOVEL ENGINEERED AAV CAPSIDS WITH HIGH EFFICIENCY RETROGRADE TRANSPORT TO MAP AND MODULATE THE", MOLECULAR THERAPY, vol. Vol.25, No.5S1, 729, JPN5021006658, 2017, pages 335 - 336, ISSN: 0004983419 *

Also Published As

Publication number Publication date
BR112020016666A2 (pt) 2020-12-15
US20210107947A1 (en) 2021-04-15
CA3095660A1 (en) 2019-08-22
SG11202009854XA (en) 2020-11-27
WO2019158619A1 (en) 2019-08-22
AU2019220368A1 (en) 2020-10-08
AU2019220368B2 (en) 2024-06-13
AU2024204557A1 (en) 2024-07-25
EP3752521A1 (en) 2020-12-23
CN112105630A (zh) 2020-12-18
US20250051396A1 (en) 2025-02-13
JP2024050766A (ja) 2024-04-10

Similar Documents

Publication Publication Date Title
US20250051396A1 (en) Modified viral capsids
US11932668B2 (en) Selective recovery
CN112004820B (zh) 具有增强的人胰腺向性的新型重组腺相关病毒衣壳
KR102423442B1 (ko) 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
JP2021522827A (ja) アデノ随伴ウイルスの肝臓特異的向性
JP2024515807A (ja) Ａａｖカプシドおよびその使用
KR102917057B1 (ko) 뇌에 지향성을 가지는 aav 돌연변이체
US20220372512A1 (en) Methods and aav vectors for in vivo transduction
CN111718418B (zh) 一种增强基因编辑的融合蛋白及其应用
CN111718420A (zh) 一种用于基因治疗的融合蛋白及其应用
WO2023184107A1 (en) Crispr-cas13 system for treating mecp2-associated diseases
Davidsson et al. Barcoded Rational AAV Vector Evolution enables systematic in vivo mapping of peptide binding motifs
AU2024330245A1 (en) Gene therapy vector for treating parkinson's disease and use thereof
JP2024517957A (ja) ベクター系
EP4121443A1 (en) Method of engineering and isolating adeno-associated virus
EP4516805A1 (en) Viral vector for fibroblast transfection
Weinmann Massively parallel in vivo characterization of novel adeno-associated viral (AAV) capsids using DNA/RNA barcoding and next generation sequencing
Goertsen Expanding adeno-associated viral capsid engineering to multiple variable regions for diversified tropism
Zdechlik Engineering Adeno-Associated Virus for Receptor-Mediated Gene Delivery
CN117836420A (zh) 重组tert编码病毒基因组和运载体
EP4658800A1 (en) An aav9 capsid variant for targeted gene transfer
HK40109651A (zh) 重组tert编码病毒基因组和运载体
Herrmann Dissection and optimization of Adeno-associated virus (AAV) DNA family shuffling technology: The journey is the reward
WO2020187272A1 (zh) 一种用于基因治疗的融合蛋白及其应用

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20201015

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220214

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20220214

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230207

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20230426

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230630

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20230703

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20231003

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240202

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

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20240327

A912 Re-examination (zenchi) completed and case transferred to appeal board

Free format text: JAPANESE INTERMEDIATE CODE: A912

Effective date: 20240510

A711 Notification of change in applicant

Free format text: JAPANESE INTERMEDIATE CODE: A711

Effective date: 20241225

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20241225