JP2022547197A - アデノ随伴ウイルス(aav)の生体内分布の変更に向けた、aavとaav受容体(aavr)との間の相互作用を調節するための方法および組成物 - Google Patents
アデノ随伴ウイルス(aav)の生体内分布の変更に向けた、aavとaav受容体(aavr)との間の相互作用を調節するための方法および組成物 Download PDFInfo
- Publication number
- JP2022547197A JP2022547197A JP2022515606A JP2022515606A JP2022547197A JP 2022547197 A JP2022547197 A JP 2022547197A JP 2022515606 A JP2022515606 A JP 2022515606A JP 2022515606 A JP2022515606 A JP 2022515606A JP 2022547197 A JP2022547197 A JP 2022547197A
- Authority
- JP
- Japan
- Prior art keywords
- unmodified
- modified
- capsid protein
- raav
- aav
- 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
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
-
- 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
-
- 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
- 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
- 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
-
- 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
- C12N2750/14145—Special targeting system for viral vectors
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Virology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Mycology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (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)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962897973P | 2019-09-09 | 2019-09-09 | |
| US62/897,973 | 2019-09-09 | ||
| US201962934996P | 2019-11-13 | 2019-11-13 | |
| US62/934,996 | 2019-11-13 | ||
| PCT/US2020/050027 WO2021050614A2 (en) | 2019-09-09 | 2020-09-09 | Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022547197A true JP2022547197A (ja) | 2022-11-10 |
| JP2022547197A5 JP2022547197A5 (https=) | 2023-09-15 |
Family
ID=72826975
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022515606A Pending JP2022547197A (ja) | 2019-09-09 | 2020-09-09 | アデノ随伴ウイルス(aav)の生体内分布の変更に向けた、aavとaav受容体(aavr)との間の相互作用を調節するための方法および組成物 |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US12570998B2 (https=) |
| EP (1) | EP4028531A2 (https=) |
| JP (1) | JP2022547197A (https=) |
| KR (1) | KR20220054435A (https=) |
| CN (1) | CN114667349A (https=) |
| AU (1) | AU2020347165A1 (https=) |
| BR (1) | BR112022004183A2 (https=) |
| CA (1) | CA3153972A1 (https=) |
| IL (1) | IL291136A (https=) |
| WO (1) | WO2021050614A2 (https=) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3100006A1 (en) | 2018-05-11 | 2019-11-14 | Massachusetts Eye And Ear Infirmary | Altering tissue tropism of adeno-associated viruses |
| CA3153972A1 (en) | 2019-09-09 | 2021-03-18 | Massachusetts Eye And Ear Infirmary | Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav |
| WO2022150634A2 (en) * | 2021-01-08 | 2022-07-14 | Massachusetts Eye And Ear Infirmary | Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav |
| US20240352077A1 (en) * | 2021-08-25 | 2024-10-24 | Children's Medical Research Institute | Modified aav capsids and vectors |
| US20240425549A1 (en) * | 2021-08-25 | 2024-12-26 | Children's Medical Research Institute | Modified aav capsid polypeptides and vectors |
| KR20240095539A (ko) | 2021-10-08 | 2024-06-25 | 디노 테라퓨틱스, 인코포레이티드 | 캡시드 변이체 및 이의 사용 방법 |
| WO2023095034A1 (en) * | 2021-11-24 | 2023-06-01 | Novartis Ag | Modulators of adeno-associated virus transduction and uses thereof |
| WO2023106256A1 (ja) * | 2021-12-06 | 2023-06-15 | 学校法人順天堂 | 改変型アデノ随伴ウイルスベクター |
| WO2024238807A2 (en) | 2023-05-16 | 2024-11-21 | Affinia Therapeutics Inc. | Recombinant aavs with improved tropism and specificity |
| WO2025179124A1 (en) * | 2024-02-23 | 2025-08-28 | The Board Of Regents Of The University Of Texas System | Enhanced aav9 capsid protein |
| WO2025194042A1 (en) | 2024-03-14 | 2025-09-18 | Affinia Therapeutics Inc. | Plasmid system for production of recombinant adenoassociated virus |
| WO2025235491A1 (en) | 2024-05-07 | 2025-11-13 | Affinia Therapeutics Inc. | Recombinant aav for treatment of cardiac diseases |
Family Cites Families (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| JP5136766B2 (ja) | 2004-12-15 | 2013-02-06 | ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | キメラベクター |
| WO2010093784A2 (en) | 2009-02-11 | 2010-08-19 | The University Of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
| CN105755044A (zh) | 2011-04-22 | 2016-07-13 | 加利福尼亚大学董事会 | 具有变异衣壳的腺相关病毒病毒体及其使用方法 |
| JP5915467B2 (ja) | 2012-08-30 | 2016-05-11 | 株式会社島津製作所 | 液体クロマトグラフ検出器用送液管及び液体クロマトグラフ |
| ES2926774T3 (es) | 2013-03-15 | 2022-10-28 | Univ North Carolina Chapel Hill | Métodos y composiciones para vectores AAV de doble unión de glicano |
| EP3003391B1 (en) | 2013-05-31 | 2021-09-22 | The Regents of The University of California | Adeno-associated virus variants and methods of use thereof |
| WO2015048534A1 (en) | 2013-09-26 | 2015-04-02 | University Of Florida Research Foundation, Inc. | Synthetic combinatorial aav capsid library for targeted gene therapy |
| CN106232618A (zh) | 2013-10-11 | 2016-12-14 | 马萨诸塞眼科耳科诊所 | 预测祖先病毒序列的方法及其用途 |
| US9474181B2 (en) | 2013-12-23 | 2016-10-18 | Dell Products, L.P. | Combined power and management module for use in a rack-configured information handling system |
| WO2015164757A1 (en) * | 2014-04-25 | 2015-10-29 | Oregon Health & Science University | Methods of viral neutralizing antibody epitope mapping |
| JP6475998B2 (ja) | 2015-02-10 | 2019-02-27 | デクセリアルズ株式会社 | タッチパッド用アンテナ装置、及び電子機器 |
| CN108603235A (zh) | 2015-07-30 | 2018-09-28 | 马萨诸塞眼科耳科诊所 | 祖先病毒序列及其用途 |
| EP3384034B1 (en) | 2015-12-02 | 2020-07-08 | The Board of Trustees of the Leland Stanford Junior University | Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism |
| SG10201913266UA (en) | 2015-12-11 | 2020-02-27 | Massachusetts Eye & Ear Infirmary | Materials and methods for delivering nucleic acids to cochlear and vestibular cells |
| JP6947739B2 (ja) | 2016-02-16 | 2021-10-13 | ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー | 既存のヒト中和抗体に対して耐性である新規組換えアデノ随伴ウイルスカプシド |
| JP6936788B2 (ja) | 2016-04-01 | 2021-09-22 | 株式会社カネカ | 軽量化及びリブ設計が可能な樹脂組成物による表面性が良好な射出発泡成形体 |
| IL305149A (en) | 2016-07-26 | 2023-10-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| AU2017341849B2 (en) | 2016-10-13 | 2024-03-21 | University Of Massachusetts | AAV capsid designs |
| US11905312B2 (en) * | 2017-02-15 | 2024-02-20 | The University Of North Carolina At Chapel Hill | Methods and compositions for gene transfer across the vasculature |
| CA3061968A1 (en) | 2017-05-10 | 2018-11-15 | Massachusetts Eye And Ear Infirmary | Methods and compositions for modifying assembly-activating protein (aap)-dependence of viruses |
| EP3808849A1 (en) * | 2017-08-03 | 2021-04-21 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| US11247659B2 (en) | 2017-08-08 | 2022-02-15 | Aisin Seiki Kabushiki Kaisha | Vehicle control apparatus |
| CN111770999A (zh) * | 2017-11-27 | 2020-10-13 | 4D分子治疗有限公司 | 腺相关病毒变体衣壳和用于抑制血管生成的应用 |
| CA3100006A1 (en) | 2018-05-11 | 2019-11-14 | Massachusetts Eye And Ear Infirmary | Altering tissue tropism of adeno-associated viruses |
| WO2020041498A1 (en) | 2018-08-21 | 2020-02-27 | Massachusetts Eye And Ear Infirmary | Compositions and methods for modulating transduction efficiency of adeno-associated viruses |
| CA3153972A1 (en) | 2019-09-09 | 2021-03-18 | Massachusetts Eye And Ear Infirmary | Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav |
| WO2022150634A2 (en) | 2021-01-08 | 2022-07-14 | Massachusetts Eye And Ear Infirmary | Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav |
-
2020
- 2020-09-09 CA CA3153972A patent/CA3153972A1/en active Pending
- 2020-09-09 KR KR1020227011374A patent/KR20220054435A/ko active Pending
- 2020-09-09 JP JP2022515606A patent/JP2022547197A/ja active Pending
- 2020-09-09 CN CN202080077968.8A patent/CN114667349A/zh active Pending
- 2020-09-09 WO PCT/US2020/050027 patent/WO2021050614A2/en not_active Ceased
- 2020-09-09 AU AU2020347165A patent/AU2020347165A1/en not_active Abandoned
- 2020-09-09 BR BR112022004183A patent/BR112022004183A2/pt unknown
- 2020-09-09 US US17/641,535 patent/US12570998B2/en active Active
- 2020-09-09 EP EP20789739.8A patent/EP4028531A2/en active Pending
-
2022
- 2022-03-06 IL IL291136A patent/IL291136A/en unknown
Non-Patent Citations (2)
| Title |
|---|
| DR. BONOの生命科学データ解析, vol. 第1版第3刷, JPN6024036501, 2018, pages 7 - 10, ISSN: 0005412547 * |
| JOURNAL OF VIROLOGY, vol. 80, no. 2, JPN6024036500, 2006, pages 821 - 834, ISSN: 0005412546 * |
Also Published As
| Publication number | Publication date |
|---|---|
| CA3153972A1 (en) | 2021-03-18 |
| IL291136A (en) | 2022-05-01 |
| US12570998B2 (en) | 2026-03-10 |
| AU2020347165A1 (en) | 2022-03-31 |
| WO2021050614A3 (en) | 2021-05-20 |
| EP4028531A2 (en) | 2022-07-20 |
| KR20220054435A (ko) | 2022-05-02 |
| CN114667349A (zh) | 2022-06-24 |
| WO2021050614A2 (en) | 2021-03-18 |
| US20220411820A1 (en) | 2022-12-29 |
| BR112022004183A2 (pt) | 2022-05-31 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US12570998B2 (en) | Methods and compositions for modulating the interaction between adeno-associated virus (AAV) and the AAV receptor (AAVR) for altered bio-distribution of AAV | |
| US20240344085A1 (en) | Altering tissue tropism of adeno-associated viruses | |
| JP6619454B2 (ja) | キャプシド | |
| US20240060087A1 (en) | Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav | |
| CN113874385A (zh) | 突变的腺相关病毒衣壳蛋白、包含该蛋白的aav颗粒和肝定向aav载体基因治疗 | |
| US20110171262A1 (en) | Parvoviral capsid with incorporated gly-ala repeat region | |
| JP2019525758A (ja) | 新規アデノ随伴ウイルスキャプシドタンパク質 | |
| JP2011067212A (ja) | ハイブリッドウイルス粒子とその製造方法 | |
| CN116209768A (zh) | 用于通过高变区交换工程化新杂合aav衣壳的方法 | |
| KR20230029616A (ko) | 종간 호환가능한 아데노-관련 바이러스 조성물 및 이의 사용 방법 | |
| JP2001517071A (ja) | 組換えaavビリオン産生における使用のための補助機能 | |
| CN116249771A (zh) | 改进的腺相关病毒基因治疗载体 | |
| JP2025004107A (ja) | ガラクトース血症の処置のための遺伝子治療 | |
| WO2021113634A1 (en) | Transgene cassettes designed to express a human mecp2 gene | |
| JP7788594B2 (ja) | 乳児バッテン病の治療のための髄腔内および静脈内組合せ遺伝子療法 | |
| US20250297278A1 (en) | Insect cell-produced high potency AAV vectors with CNS-tropism | |
| WO2024163335A2 (en) | Pten gene therapy vectors and uses thereof | |
| HK40044351B (en) | Liver-specific tropism of adeno-associated viruses | |
| HK40044351A (en) | Liver-specific tropism of adeno-associated viruses |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20230907 |
|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20230907 |
|
| A977 | Report on retrieval |
Free format text: JAPANESE INTERMEDIATE CODE: A971007 Effective date: 20240823 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20240910 |
|
| A601 | Written request for extension of time |
Free format text: JAPANESE INTERMEDIATE CODE: A601 Effective date: 20241203 |
|
| A601 | Written request for extension of time |
Free format text: JAPANESE INTERMEDIATE CODE: A601 Effective date: 20250204 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20250310 |
|
| A02 | Decision of refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A02 Effective date: 20250610 |