JP2021519612A - 中和を回避する肝臓向性組換えａａｖ６ベクター - Google Patents

中和を回避する肝臓向性組換えａａｖ６ベクター Download PDF

Info

Publication number
JP2021519612A
JP2021519612A JP2021502719A JP2021502719A JP2021519612A JP 2021519612 A JP2021519612 A JP 2021519612A JP 2021502719 A JP2021502719 A JP 2021502719A JP 2021502719 A JP2021502719 A JP 2021502719A JP 2021519612 A JP2021519612 A JP 2021519612A
Authority
JP
Japan
Prior art keywords
aav6
modified
vector
raav6
neutralization
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
JP2021502719A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2019191418A5 (https=
JP2021519612A5 (https=
Inventor
メイヴィス アグバンジェ−マッケンナ，
メイヴィス アグバンジェ−マッケンナ，
アントネット ベネット，
アントネット ベネット，
アール． ジュード サムルスキ，
アール． ジュード サムルスキ，
Original Assignee
アスクレピオス バイオファーマシューティカル， インコーポレイテッド
アスクレピオス バイオファーマシューティカル， インコーポレイテッド
ユニバーシティ オブ フロリダ リサーチ ファウンデーション，インコーポレイティド
ユニバーシティ オブ フロリダ リサーチ ファウンデーション，インコーポレイティド
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 アスクレピオス バイオファーマシューティカル， インコーポレイテッド, アスクレピオス バイオファーマシューティカル， インコーポレイテッド, ユニバーシティ オブ フロリダ リサーチ ファウンデーション，インコーポレイティド, ユニバーシティ オブ フロリダ リサーチ ファウンデーション，インコーポレイティド filed Critical アスクレピオス バイオファーマシューティカル， インコーポレイテッド
Publication of JP2021519612A publication Critical patent/JP2021519612A/ja
Publication of JPWO2019191418A5 publication Critical patent/JPWO2019191418A5/ja
Publication of JP2021519612A5 publication Critical patent/JP2021519612A5/ja
Pending legal-status Critical Current

Links

Images

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
    • C12N15/63—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
    • 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
    • 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

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2021502719A 2018-03-29 2019-03-28 中和を回避する肝臓向性組換えａａｖ６ベクター Pending JP2021519612A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862649691P 2018-03-29 2018-03-29
US62/649,691 2018-03-29
PCT/US2019/024567 WO2019191418A1 (en) 2018-03-29 2019-03-28 Liver tropic recombinant aav6 vectors that evade neutralization

Publications (3)

Publication Number Publication Date
JP2021519612A true JP2021519612A (ja) 2021-08-12
JPWO2019191418A5 JPWO2019191418A5 (https=) 2022-01-26
JP2021519612A5 JP2021519612A5 (https=) 2022-01-26

Family

ID=68060427

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021502719A Pending JP2021519612A (ja) 2018-03-29 2019-03-28 中和を回避する肝臓向性組換えａａｖ６ベクター

Country Status (6)

Country Link
US (1) US20210032660A1 (https=)
EP (1) EP3781187A4 (https=)
JP (1) JP2021519612A (https=)
CN (1) CN112218880A (https=)
CA (1) CA3095127A1 (https=)
WO (1) WO2019191418A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20210017542A1 (en) * 2018-03-29 2021-01-21 University Of Florida Research Foundation, Incorporated Aav6 variants
CN117801116A (zh) * 2022-09-30 2024-04-02 上海玮美基因科技有限责任公司 一种融合型新型腺相关病毒及其应用

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017058892A2 (en) * 2015-09-28 2017-04-06 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006110689A2 (en) * 2005-04-07 2006-10-19 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
US9533189B2 (en) * 2012-05-30 2017-01-03 Jaime Cameron Push-up exercise apparatus
KR102234672B1 (ko) * 2013-05-21 2021-04-06 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
JP2017048681A (ja) * 2015-08-31 2017-03-09 株式会社マーレ フィルターシステムズ ポンプ
EP4659816A3 (en) * 2015-12-14 2025-12-31 The University of North Carolina at Chapel Hill MODIFIED CAPSID PROTEINS FOR IMPROVED PARVOVIRUS VECTOR DELIVERY

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017058892A2 (en) * 2015-09-28 2017-04-06 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
BENNETT AD. ET AL.: "AAV6 K531 Serves a Dual Function in Selective Receptor and Antibody ADK6 Recognition", 2017, MOLECULAR THERAPY, VOL. 25, NO. 5S1, P. 44, [91], JPN6022053948, ISSN: 0005228659 *
HUANG LY. ET AL., CHARACTERIZATION OF THE ADENO-ASSOCIATED VIRUS 1 AND 6 SIALIC ACID BINDING SITE, J VIROL, 2016, VOL., JPN6022053946, ISSN: 0005228661 *
WU Z. ET AL.: "Single Amino Acid Changes Can Influence Titer, Heparin Binding, and Tissue Tropism in Different Aden", J VIROL, 2006, VOL. 80, NO. 22, P. 11393-11397, JPN6022053947, ISSN: 0005228660 *

Also Published As

Publication number Publication date
CA3095127A1 (en) 2019-10-03
US20210032660A1 (en) 2021-02-04
EP3781187A1 (en) 2021-02-24
WO2019191418A1 (en) 2019-10-03
CN112218880A (zh) 2021-01-12
EP3781187A4 (en) 2021-12-22

Similar Documents

Publication Publication Date Title
US12435342B2 (en) Methods of packaging multiple adeno-associated virus vectors
CN110770346B (zh) 多倍体腺相关病毒载体及其制备和使用方法
TWI791433B (zh) 治療ａ型血友病之基因治療
JP5136766B2 (ja) キメラベクター
JP2022065003A (ja) パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質
JP2021516044A (ja) 抗体回避ウイルスベクターのための方法および組成物
JP2016525356A (ja) 変異ａａｖ、及び、細胞、臓器並びに組織への遺伝子導入のための組成物、方法並びに使用法
CN116670152A (zh) 具有组织特异性靶向基序的新型组合物和含有其的组合物
KR20230034211A (ko) 변형된 아데노-관련 바이러스 5 캡시드 및 이의 용도
CN115960921B (zh) 一组肝靶向新型腺相关病毒的获得及其应用
Kochergin-Nikitsky et al. Tissue and cell-type-specific transduction using rAAV vectors in lung diseases
JP2021519612A (ja) 中和を回避する肝臓向性組換えａａｖ６ベクター
EP3773746A1 (en) Aav6 variants
HK40044939A (en) Liver tropic recombinant aav6 vectors that evade neutralization
HK40104382A (zh) 多倍体腺相关病毒载体及其制备和使用方法
HK40118611A (zh) 合成嗜肝性腺相关病毒衣壳及其用途
WO2025259575A1 (en) Methods and compositions for treatment of duchenne muscular dystrophy
HK40038424A (en) Methods and compositions for delivery of viral vectors across the blood-brain barrier
HK40048077B (zh) 合成嗜肝性腺相关病毒衣壳及其用途
HK40022719A (en) Polyploid adeno-associated virus vectors and methods of making and using the same
HK40022719B (zh) 多倍体腺相关病毒载体及其制备和使用方法
Drouin Characterization of AAV2 genome packaging

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220114

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20220114

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20221220

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20221223

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20230320

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230619

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20230703

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20231106

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20231122

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

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20231212

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

Free format text: JAPANESE INTERMEDIATE CODE: A912

Effective date: 20231228