JP7064214B2 - 抗体回避性ウイルスベクターのための方法および組成物 - Google Patents
抗体回避性ウイルスベクターのための方法および組成物 Download PDFInfo
- Publication number
- JP7064214B2 JP7064214B2 JP2018515949A JP2018515949A JP7064214B2 JP 7064214 B2 JP7064214 B2 JP 7064214B2 JP 2018515949 A JP2018515949 A JP 2018515949A JP 2018515949 A JP2018515949 A JP 2018515949A JP 7064214 B2 JP7064214 B2 JP 7064214B2
- Authority
- JP
- Japan
- Prior art keywords
- amino acid
- aav
- positions
- seq
- capsid protein
- 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.)
- Expired - Fee Related
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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/08—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from viruses
- C07K16/081—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from viruses from DNA 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
- A61K2039/507—Comprising a combination of two or more separate antibodies
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/30—Immunoglobulins specific features characterized by aspects of specificity or valency
- C07K2317/33—Crossreactivity, e.g. for species or epitope, or lack of said crossreactivity
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/30—Immunoglobulins specific features characterized by aspects of specificity or valency
- C07K2317/34—Identification of a linear epitope shorter than 20 amino acid residues or of a conformational epitope defined by amino acid residues
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
- C07K2317/76—Antagonist effect on antigen, e.g. neutralization or inhibition of binding
-
- 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
- C12N2810/00—Vectors comprising a targeting moiety
- C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
- C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
- C12N2810/6027—Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Immunology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Veterinary Medicine (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022011956A JP7390693B2 (ja) | 2015-09-28 | 2022-01-28 | 抗体回避性ウイルスベクターのための方法および組成物 |
| JP2023193120A JP2024026088A (ja) | 2015-09-28 | 2023-11-13 | 抗体回避性ウイルスベクターのための方法および組成物 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562234016P | 2015-09-28 | 2015-09-28 | |
| US62/234,016 | 2015-09-28 | ||
| PCT/US2016/054143 WO2017058892A2 (en) | 2015-09-28 | 2016-09-28 | Methods and compositions for antibody-evading virus vectors |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022011956A Division JP7390693B2 (ja) | 2015-09-28 | 2022-01-28 | 抗体回避性ウイルスベクターのための方法および組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018528253A JP2018528253A (ja) | 2018-09-27 |
| JP2018528253A5 JP2018528253A5 (enExample) | 2019-11-07 |
| JP7064214B2 true JP7064214B2 (ja) | 2022-05-10 |
Family
ID=58424549
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018515949A Expired - Fee Related JP7064214B2 (ja) | 2015-09-28 | 2016-09-28 | 抗体回避性ウイルスベクターのための方法および組成物 |
| JP2022011956A Active JP7390693B2 (ja) | 2015-09-28 | 2022-01-28 | 抗体回避性ウイルスベクターのための方法および組成物 |
| JP2023193120A Pending JP2024026088A (ja) | 2015-09-28 | 2023-11-13 | 抗体回避性ウイルスベクターのための方法および組成物 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022011956A Active JP7390693B2 (ja) | 2015-09-28 | 2022-01-28 | 抗体回避性ウイルスベクターのための方法および組成物 |
| JP2023193120A Pending JP2024026088A (ja) | 2015-09-28 | 2023-11-13 | 抗体回避性ウイルスベクターのための方法および組成物 |
Country Status (14)
| Country | Link |
|---|---|
| US (4) | US10745447B2 (enExample) |
| EP (2) | EP3831842A1 (enExample) |
| JP (3) | JP7064214B2 (enExample) |
| CN (2) | CN108137655B (enExample) |
| AU (2) | AU2016332821B2 (enExample) |
| BR (1) | BR112018003665A2 (enExample) |
| CA (1) | CA2996420A1 (enExample) |
| DK (1) | DK3356390T3 (enExample) |
| ES (1) | ES2865487T3 (enExample) |
| IL (4) | IL314171A (enExample) |
| PL (1) | PL3356390T3 (enExample) |
| PT (1) | PT3356390T (enExample) |
| SG (1) | SG10202107733QA (enExample) |
| WO (1) | WO2017058892A2 (enExample) |
Families Citing this family (91)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3049527A4 (en) | 2013-09-26 | 2017-08-16 | University of Florida Research Foundation, Inc. | Synthetic combinatorial aav capsid library for targeted gene therapy |
| GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| SG10202007103TA (en) | 2014-11-05 | 2020-09-29 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
| MX2017006216A (es) | 2014-11-14 | 2018-08-29 | Voyager Therapeutics Inc | Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela). |
| IL284949B2 (en) | 2014-11-14 | 2024-06-01 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| WO2016094783A1 (en) | 2014-12-12 | 2016-06-16 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| WO2017070476A2 (en) | 2015-10-22 | 2017-04-27 | University Of Florida Research Foundation, Inc. | Synthetic combinatorial aav3 capsid library |
| WO2017096162A1 (en) | 2015-12-02 | 2017-06-08 | Voyager Therapeutics, Inc. | Assays for the detection of aav neutralizing antibodies |
| AU2016371779C1 (en) * | 2015-12-14 | 2022-10-13 | The University Of North Carolina At Chapel Hill | Modified capsid proteins for enhanced delivery of parvovirus vectors |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| AU2017268382B2 (en) | 2016-05-18 | 2023-09-28 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's disease |
| EP3458588B1 (en) | 2016-05-18 | 2025-10-29 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| CN109890966A (zh) * | 2016-08-16 | 2019-06-14 | 北卡罗来纳-查佩尔山大学 | 用于靶向基因转移的方法和组合物 |
| US11298041B2 (en) | 2016-08-30 | 2022-04-12 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| US11192925B2 (en) | 2016-10-19 | 2021-12-07 | Adverum Biotechnologies, Inc. | Modified AAV capsids and uses thereof |
| CA3054131C (en) | 2017-02-21 | 2021-05-25 | University Of Florida Research Foundation, Incorporated | Modified aav capsid proteins and uses thereof |
| EP3618839A4 (en) | 2017-05-05 | 2021-06-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| CN111108198A (zh) | 2017-05-05 | 2020-05-05 | 沃雅戈治疗公司 | 治疗亨廷顿病的组合物和方法 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| AU2018302016A1 (en) | 2017-07-17 | 2020-02-06 | The Regents Of The University Of California | Trajectory array guide system |
| TWI832036B (zh) | 2017-08-03 | 2024-02-11 | 美商航海家醫療公司 | 用於aav之遞送之組合物及方法 |
| EP3687582A4 (en) | 2017-09-29 | 2021-07-14 | Voyager Therapeutics, Inc. | RESCUE OF CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE OF FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| CN111479924B (zh) | 2017-10-16 | 2024-06-14 | 沃雅戈治疗公司 | 肌萎缩性侧索硬化症(als)的治疗 |
| SG11202003479TA (en) * | 2017-10-18 | 2020-05-28 | Regenxbio Inc | Fully-human post-translationally modified antibody therapeutics |
| CA3092107A1 (en) * | 2018-02-28 | 2019-09-06 | The University Of North Carolina At Chapel Hill | Methods and compositions for antibody-evading virus vectors |
| CN111819286B (zh) * | 2018-03-16 | 2024-05-24 | 国家儿童医院研究所 | 通过衣壳修饰增加组织特异性的基因递送 |
| JP2021519612A (ja) * | 2018-03-29 | 2021-08-12 | 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 | 中和を回避する肝臓向性組換えaav6ベクター |
| US20210017542A1 (en) * | 2018-03-29 | 2021-01-21 | University Of Florida Research Foundation, Incorporated | Aav6 variants |
| CN112272672A (zh) * | 2018-04-03 | 2021-01-26 | 斯特里迪比奥公司 | 抗体逃避性病毒载体 |
| WO2019195444A1 (en) * | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Antibody-evading virus vectors |
| JP7425738B2 (ja) | 2018-04-03 | 2024-01-31 | ギンコ バイオワークス インコーポレイテッド | 眼組織を標的とするウイルスベクター |
| WO2019213668A1 (en) * | 2018-05-04 | 2019-11-07 | Oregon Health & Science University | Human anti-aav2 capsid polyclonal antibody epitopes |
| TW202015742A (zh) | 2018-05-15 | 2020-05-01 | 美商航海家醫療公司 | 投遞腺相關病毒(aav)之組成物和方法 |
| MA52631A (fr) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics Inc | Compositions et méthodes pour le traitement de la maladie de parkinson |
| WO2019241486A1 (en) | 2018-06-13 | 2019-12-19 | Voyager Therapeutics, Inc. | Engineered 5' untranslated regions (5' utr) for aav production |
| JP7142815B2 (ja) * | 2018-06-21 | 2022-09-28 | 株式会社遺伝子治療研究所 | ヒト肝臓への遺伝子導入のためのアデノ随伴ウイルスビリオン |
| JP2021530548A (ja) | 2018-07-24 | 2021-11-11 | ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics, Inc. | 遺伝子治療製剤を生産するための系および方法 |
| AU2019346447A1 (en) * | 2018-09-26 | 2021-04-29 | California Institute Of Technology | Adeno-associated virus compositions for targeted gene therapy |
| JP7528066B2 (ja) | 2018-09-28 | 2024-08-05 | ボイジャー セラピューティクス インコーポレイテッド | 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法 |
| EP3861010A1 (en) | 2018-10-02 | 2021-08-11 | Voyager Therapeutics, Inc. | Redirection of tropism of aav capsids |
| EP3861113A1 (en) | 2018-10-04 | 2021-08-11 | Voyager Therapeutics, Inc. | Methods for measuring the titer and potency of viral vector particles |
| CA3115248A1 (en) | 2018-10-05 | 2020-04-09 | Voyager Therapeutics, Inc. | Engineered nucleic acid constructs encoding aav production proteins |
| WO2020077165A1 (en) | 2018-10-12 | 2020-04-16 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| TW202028468A (zh) | 2018-10-15 | 2020-08-01 | 美商航海家醫療公司 | 用於桿狀病毒/Sf9系統中rAAV之大規模生產的表現載體 |
| SG11202107645RA (en) | 2019-01-18 | 2021-08-30 | Voyager Therapeutics Inc | Methods and systems for producing aav particles |
| TW202045529A (zh) | 2019-02-15 | 2020-12-16 | 美商聖加莫治療股份有限公司 | 用於生產重組aav之組合物及方法 |
| CA3133453A1 (en) | 2019-03-21 | 2020-09-24 | Daniel Mccoy | Recombinant adeno-associated virus vectors |
| WO2020223274A1 (en) | 2019-04-29 | 2020-11-05 | Voyager Therapeutics, Inc. | SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS |
| US20240124889A1 (en) | 2019-05-07 | 2024-04-18 | Voyager Therapeutics, Inc. | Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation |
| AU2020299026A1 (en) * | 2019-07-04 | 2022-01-20 | Children's Medical Research Institute | Methods and AAV vectors for in vivo transduction |
| EP4006160A4 (en) * | 2019-07-12 | 2023-06-28 | Gene Therapy Research Institution Co., Ltd. | Adeno-associated virus virion for gene transfer to human liver |
| CN110295151A (zh) * | 2019-07-17 | 2019-10-01 | 博鑫仪器(天津)有限公司 | 一类腺依赖病毒病毒重组质粒、重组病毒及构建方法 |
| WO2021030125A1 (en) | 2019-08-09 | 2021-02-18 | Voyager Therapeutics, Inc. | Cell culture medium for use in producing gene therapy products in bioreactors |
| RU2751592C2 (ru) * | 2019-08-22 | 2021-07-15 | Общество С Ограниченной Ответственностью "Анабион" | Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе |
| WO2021041485A1 (en) | 2019-08-26 | 2021-03-04 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| WO2021046155A1 (en) | 2019-09-03 | 2021-03-11 | Voyager Therapeutics, Inc. | Vectorized editing of nucleic acids to correct overt mutations |
| AU2020367532A1 (en) | 2019-10-17 | 2022-05-12 | Ginkgo Bioworks, Inc. | Adeno-associated viral vectors for treatment of Niemann-Pick disease type C |
| US20230340526A1 (en) * | 2019-11-25 | 2023-10-26 | University Of Florida Research Foundation, Incorporated | Novel aav3b variants that target hepatocytes and evade the humoral immune response |
| JP2021097617A (ja) * | 2019-12-20 | 2021-07-01 | 学校法人自治医科大学 | オルニチントランスカルバミラーゼ欠損症の治療用アデノ随伴ウイルスビリオン |
| AR122013A1 (es) | 2020-05-05 | 2022-08-03 | Univ Duke | Composiciones de virus adenoasociados compatibles entre especies y métodos para su uso |
| US20230203102A1 (en) | 2020-05-13 | 2023-06-29 | Voyager Therapeutics, Inc. | Redirection of tropism of aav capsids |
| WO2021247995A2 (en) | 2020-06-04 | 2021-12-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating neuropathic pain |
| WO2022026410A2 (en) | 2020-07-27 | 2022-02-03 | Voyager Therapeutics, Inc | Compositions and methods for the treatment of niemann-pick type c1 disease |
| MX2023000815A (es) | 2020-07-27 | 2023-04-11 | Voyager Therapeutics Inc | Composiciones y metodos para el tratamiento de trastornos neurologicos relacionados con la deficiencia de glucosilceramidasa beta. |
| EP4192514A1 (en) | 2020-08-06 | 2023-06-14 | Voyager Therapeutics, Inc. | Cell culture medium for use in producing gene therapy products in bioreactors |
| KR20230068444A (ko) | 2020-08-19 | 2023-05-17 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 레트 증후군의 치료를 위한 아데노 관련 바이러스 벡터 |
| CN111925438B (zh) * | 2020-08-28 | 2021-03-09 | 和元生物技术(上海)股份有限公司 | 能够与aav1-13结合的抗体 |
| US20240141378A1 (en) | 2021-03-03 | 2024-05-02 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| EP4301768A2 (en) | 2021-03-03 | 2024-01-10 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| WO2022229703A2 (en) * | 2021-04-30 | 2022-11-03 | Takeda Pharmaceutical Company, Ltd. | New aav8 based immune escaping variants |
| AR126839A1 (es) * | 2021-08-20 | 2023-11-22 | Llc «Anabion» | Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 9 (aav9), cápside y vector basado en esta |
| CA3234664A1 (en) | 2021-10-08 | 2023-04-13 | Dyno Therapeutics, Inc. | Capsid variants and methods of using the same |
| MX2024004936A (es) | 2021-11-02 | 2024-05-08 | Voyager Therapeutics Inc | Variantes de capsides de aav y sus usos. |
| CN118525098A (zh) | 2021-11-29 | 2024-08-20 | 上海瑞宏迪医药有限公司 | Aadc、gdnf多核苷酸及其用于治疗帕金森病 |
| TW202342759A (zh) | 2022-02-04 | 2023-11-01 | 美商史崔德生物公司 | 重組腺相關病毒載體及其使用方法 |
| WO2023155918A1 (zh) | 2022-02-21 | 2023-08-24 | 上海瑞宏迪医药有限公司 | Vegf结合分子及其医药用途 |
| WO2023164448A1 (en) * | 2022-02-22 | 2023-08-31 | The University Of North Carolina At Chapel Hill | Chimeric neurotropic aav capsids |
| WO2024054983A1 (en) | 2022-09-08 | 2024-03-14 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| CN115850415B (zh) * | 2022-09-14 | 2024-05-28 | 青岛农业大学 | 一种PbCaM7基因、蛋白及其应用 |
| KR102855674B1 (ko) * | 2022-10-06 | 2025-09-08 | 주식회사 글루진테라퓨틱스 | 폐 기관지 특이적 유전자 전달이 가능한 아데노부속바이러스 벡터 |
| WO2024191778A1 (en) | 2023-03-10 | 2024-09-19 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2024191354A1 (en) * | 2023-03-13 | 2024-09-19 | Agency For Science, Technology And Research | Engineered adeno-associated viruses |
| AU2024290842A1 (en) | 2023-07-03 | 2026-01-15 | UCB Biopharma SRL | Engineered aav capsid proteins |
| WO2025049993A1 (en) | 2023-08-31 | 2025-03-06 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| KR102844372B1 (ko) * | 2023-12-06 | 2025-08-11 | 주식회사 글루진테라퓨틱스 | 뇌 성상교세포 특이적 유전자 전달이 가능한 아데노부속바이러스 변이체와 이의 응용 |
| WO2025171227A1 (en) | 2024-02-08 | 2025-08-14 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| CN120842371B (zh) * | 2025-09-25 | 2025-11-21 | 北京溯本源和生物科技有限公司 | 一种可广谱识别aav多种抗原的单克隆抗体5f11及应用 |
Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20050287122A1 (en) | 2001-01-05 | 2005-12-29 | Children's Hospital Inc. | AAV vectors and methods |
| WO2015121501A1 (en) | 2014-02-17 | 2015-08-20 | King's College London | Adeno-associated virus vector |
Family Cites Families (192)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US1453547A (en) | 1922-05-18 | 1923-05-01 | Doyle | Barrel-holding device |
| US1777296A (en) | 1928-08-22 | 1930-10-07 | Superior Locker Company | Indicating device for locker doors |
| US4501729A (en) | 1982-12-13 | 1985-02-26 | Research Corporation | Aerosolized amiloride treatment of retained pulmonary secretions |
| AU7966987A (en) | 1986-09-08 | 1988-04-07 | Applied Biotechnology, Inc. | Empty viral capsid vaccines |
| US4968603A (en) | 1986-12-31 | 1990-11-06 | The Regents Of The University Of California | Determination of status in neoplastic disease |
| JPH04501719A (ja) | 1988-11-10 | 1992-03-26 | インペリアル・キヤンサー・リサーチ・テクノロジー・リミテツド | ポリペプチド |
| US5916563A (en) | 1988-11-14 | 1999-06-29 | United States Of America | Parvovirus protein presenting capsids |
| US5399346A (en) | 1989-06-14 | 1995-03-21 | The United States Of America As Represented By The Department Of Health And Human Services | Gene therapy |
| ES2026826A6 (es) | 1991-03-26 | 1992-05-01 | Ercros Sa | Procedimiento para la produccion de una vacuna subunidad contra el parvovirus canino y otros virus relacionados. |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5869248A (en) | 1994-03-07 | 1999-02-09 | Yale University | Targeted cleavage of RNA using ribonuclease P targeting and cleavage sequences |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5599706A (en) | 1994-09-23 | 1997-02-04 | Stinchcomb; Dan T. | Ribozymes targeted to apo(a) mRNA |
| US6040183A (en) | 1995-06-07 | 2000-03-21 | University Of North Carloina At Chapel Hill | Helper virus-free AAV production |
| DE69638103D1 (de) | 1995-06-07 | 2010-02-04 | Univ North Carolina | Die transduktion von myoblasten mittels vektoren aus adenoassoziierten viren |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| US6083702A (en) | 1995-12-15 | 2000-07-04 | Intronn Holdings Llc | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
| EP0883344B2 (en) | 1995-12-15 | 2010-06-09 | VIRxSYS Corporation | Therapeutic molecules generated by trans-splicing |
| CA2265460A1 (en) | 1996-09-11 | 1998-03-19 | The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services | Aav4 vector and uses thereof |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| ATE402254T1 (de) | 1998-05-28 | 2008-08-15 | Us Gov Health & Human Serv | Aav5 vektoren und deren verwendung |
| US6984517B1 (en) | 1998-05-28 | 2006-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | AAV5 vector and uses thereof |
| US6562958B1 (en) | 1998-06-09 | 2003-05-13 | Genome Therapeutics Corporation | Nucleic acid and amino acid sequences relating to Acinetobacter baumannii for diagnostics and therapeutics |
| US20100293663A2 (en) | 1998-06-16 | 2010-11-18 | Thomas La Rosa | Nucleic Acid Molecules and Other Molecules Associated with Plants and Uses Thereof for Plant Improvement |
| US8299321B2 (en) | 1998-06-16 | 2012-10-30 | Monsanto Technology Llc | Nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement |
| DE19830522A1 (de) | 1998-07-08 | 2000-01-13 | Pritt Produktionsgesellschaft | Vorrichtung zum Übertragen eines in Form eines Filmes auf ein Trägerband aufgebrachten Stoffes |
| WO2000017377A2 (en) | 1998-09-22 | 2000-03-30 | University Of Florida | Methods for large-scale production of recombinant aav vectors |
| GB9822763D0 (en) | 1998-10-20 | 1998-12-16 | Univ Sheffield | Immunoglobin variant |
| US6759237B1 (en) | 1998-11-05 | 2004-07-06 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| EP1127150B1 (en) | 1998-11-05 | 2007-05-16 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| US6822071B1 (en) | 1998-11-12 | 2004-11-23 | The Regents Of The University Of California | Polypeptides from Chlamydia pneumoniae and their use in the diagnosis, prevention and treatment of disease |
| EP1887081A2 (en) | 1999-02-25 | 2008-02-13 | Ceres Incorporated | DNA Sequences |
| EP1033405A3 (en) | 1999-02-25 | 2001-08-01 | Ceres Incorporated | Sequence-determined DNA fragments and corresponding polypeptides encoded thereby |
| US20090087878A9 (en) | 1999-05-06 | 2009-04-02 | La Rosa Thomas J | Nucleic acid molecules associated with plants |
| US20110093981A9 (en) | 1999-05-06 | 2011-04-21 | La Rosa Thomas J | Nucleic acid molecules and other molecules associated with transcription in plants and uses thereof for plant improvement |
| US20110214206A1 (en) | 1999-05-06 | 2011-09-01 | La Rosa Thomas J | Nucleic acid molecules and other molecules associated with plants |
| US20130326723A1 (en) | 1999-05-06 | 2013-12-05 | Thomas J. La Rosa | Soy nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement |
| US20080229439A1 (en) | 1999-05-06 | 2008-09-18 | La Rosa Thomas J | Nucleic acid molecules and other molecules associated with transcription in plants and uses thereof for plant improvement |
| US20040031072A1 (en) | 1999-05-06 | 2004-02-12 | La Rosa Thomas J. | Soy nucleic acid molecules and other molecules associated with transcription plants and uses thereof for plant improvement |
| US6498244B1 (en) * | 1999-05-28 | 2002-12-24 | Cell Genesys, Inc. | Adeno-associated virus capsid immunologic determinants |
| US7314912B1 (en) | 1999-06-21 | 2008-01-01 | Medigene Aktiengesellschaft | AAv scleroprotein, production and use thereof |
| DE19933288A1 (de) | 1999-07-15 | 2001-01-18 | Medigene Ag | Strukturprotein von Adeno-assoziiertem Virus mit veränderter Antigenität, seine Herstellung und Verwendung |
| ES2308989T3 (es) | 1999-08-09 | 2008-12-16 | Targeted Genetics Corporation | Aumento de la expresion de una secuencia nucleotidica heterologa a partir de vectores viricos recombinantes que contienen una secuencia que forman pares de bases intracatenarios. |
| US6468524B1 (en) | 2000-03-22 | 2002-10-22 | The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services | AAV4 vector and uses thereof |
| US20140130203A1 (en) | 2000-04-19 | 2014-05-08 | Thomas J. La Rosa | Rice nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement |
| US20110131679A2 (en) | 2000-04-19 | 2011-06-02 | Thomas La Rosa | Rice Nucleic Acid Molecules and Other Molecules Associated with Plants and Uses Thereof for Plant Improvement |
| WO2001081581A2 (en) | 2000-04-21 | 2001-11-01 | Corixa Corporation | Compositions and methods for the therapy and diagnosis of acne vulgaris |
| AU6814901A (en) | 2000-06-01 | 2001-12-11 | Univ North Carolina | Methods and compounds for controlled release of recombinant parvovirus vectors |
| ES2505700T3 (es) | 2000-06-01 | 2014-10-10 | University Of North Carolina At Chapel Hill | Vectores de parvovirus duplicados |
| JP2002130156A (ja) | 2000-10-20 | 2002-05-09 | Anest Iwata Corp | 多段式流体圧縮部を備えたスクロール流体機械 |
| US7214786B2 (en) | 2000-12-14 | 2007-05-08 | Kovalic David K | Nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement |
| AU2002248297A1 (en) | 2001-01-05 | 2002-07-16 | Children's Hospital, Inc. | Aav2 vectors and methods |
| EP1402042A2 (en) | 2001-06-22 | 2004-03-31 | Syngenta Participations AG | Abiotic stress responsive polynucleotides and polypeptides |
| AU2002345250A1 (en) | 2001-06-22 | 2003-01-08 | Syngenta Participations Ag | Plant disease resistance genes |
| US6623729B2 (en) | 2001-07-09 | 2003-09-23 | Korea Advanced Institute Of Science And Technology | Process for preparing sustained release micelle employing conjugate of anticancer drug and biodegradable polymer |
| WO2002010210A2 (en) | 2001-08-28 | 2002-02-07 | Bayer Cropscience Ag | Polypeptides for identifying herbicidally active compounds |
| WO2003033515A1 (en) | 2001-10-15 | 2003-04-24 | Corixa Corporation | Compositions and methods for the therapy and diagnosis of acne vulgaris |
| EP1572893B1 (en) | 2001-11-09 | 2009-01-07 | THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES | Production of adeno-associated virus in insect cells |
| IL161827A0 (en) | 2001-11-13 | 2005-11-20 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novelsequences identified thereby |
| EP1463805B1 (en) | 2001-12-17 | 2014-10-22 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor |
| CA2469785C (en) | 2001-12-17 | 2014-04-22 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor |
| US7071172B2 (en) | 2002-04-30 | 2006-07-04 | The University Of North Carolina At Chapel Hill | Secretion signal vectors |
| ATE405295T1 (de) | 2002-05-01 | 2008-09-15 | Univ Florida | Verbesserte raav-expressionssysteme für die genetische modifikation spezifischer capsidproteine |
| ITRM20020253A1 (it) | 2002-05-08 | 2003-11-10 | Univ Roma | Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche. |
| JP2005185101A (ja) | 2002-05-30 | 2005-07-14 | National Institute Of Agrobiological Sciences | 植物の全長cDNAおよびその利用 |
| CN101014364B (zh) | 2002-12-24 | 2012-01-18 | 里纳特神经系统学公司 | 抗ngf抗体及其使用方法 |
| AU2004248136B2 (en) | 2003-06-02 | 2011-09-15 | University Of Massachusetts | Methods and compositions for controlling efficacy of RNA silencing |
| DK2657247T3 (en) | 2003-06-19 | 2017-07-10 | Genzyme Corp | AAV virions with reduced immunoreactivity and applications thereof |
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| WO2009105612A2 (en) | 2008-02-20 | 2009-08-27 | Ceres, Inc. | Nucleotide sequences and corresponding polypeptides conferring improved nitrogen use efficiency characteristics in plants |
| US20060107345A1 (en) | 2003-09-30 | 2006-05-18 | Nickolai Alexandrov | Sequence-determined DNA fragments and corresponding polypeptides encoded thereby |
| EP3211085B1 (en) | 2003-09-30 | 2021-03-31 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| ES2338344T3 (es) | 2004-04-07 | 2010-05-06 | Rinat Neuroscience Corporation | Procedimiento de tratamiento del dolor de cancer de hueso mediante la administracion de una antagonista del factor de crecimiento neuronal. |
| FR2874384B1 (fr) | 2004-08-17 | 2010-07-30 | Genethon | Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables |
| JP4346526B2 (ja) | 2004-08-31 | 2009-10-21 | 株式会社東芝 | 半導体集積回路装置 |
| US7366250B2 (en) | 2004-09-09 | 2008-04-29 | Agere Systems Inc. | Method and apparatus for improved efficiency in an extended multiple antenna communication system |
| EP1791432A4 (en) | 2004-09-09 | 2010-07-07 | Gen Hospital Corp | MODULATION OF PHOSPHATASE ACTIVITY IN HERZCELLS |
| AU2005316476A1 (en) * | 2004-12-15 | 2006-06-22 | University Of Florida Research Foundation, Inc. | Chimeric vectors |
| EP1839669A4 (en) | 2005-01-05 | 2012-12-12 | Shionogi & Co | NEW ANGIOGENESEHEMMER |
| EP2573188A2 (en) | 2005-03-02 | 2013-03-27 | Metanomics GmbH | Process for the production of fine chemicals |
| WO2006092449A2 (en) | 2005-03-02 | 2006-09-08 | Metanomics Gmbh | Process for the production of fine chemicals |
| CA2606362A1 (en) | 2005-04-29 | 2006-11-09 | The University Of North Carolina At Chapel Hill | Methods and compositions for regulated expression of nucleic acid at post-transcriptional level |
| EP2478760A1 (en) | 2005-05-10 | 2012-07-25 | Monsanto Technology LLC | Genes and uses for plant improvement |
| EP1981548A4 (en) | 2006-01-20 | 2010-03-24 | Univ North Carolina | REINFORCED PRODUCTION OF INFECTIOUS PARVOVIRUS VECTORS IN INSECT CELLS |
| US7867484B2 (en) * | 2006-01-27 | 2011-01-11 | University Of North Carolina At Chapel Hill | Heparin and heparan sulfate binding chimeric vectors |
| CN101437833A (zh) | 2006-02-10 | 2009-05-20 | 辛辛那提大学 | 作为心脏功能调节剂的磷酸酶抑制剂蛋白-1 |
| WO2007120542A2 (en) | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid library and aav capsid proteins |
| WO2008027084A2 (en) * | 2006-04-28 | 2008-03-06 | The Trustees Of The University Of Pennsylvania | Modified aav vectors having reduced capsid immunogenicity and use thereof |
| WO2007127264A2 (en) | 2006-04-28 | 2007-11-08 | The Trustees Of The University Of Pennsylvania | Scalable production method for aav |
| WO2007148971A2 (en) | 2006-06-21 | 2007-12-27 | Amsterdam Molecular Therapeutics B.V. | Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells |
| JP5575486B2 (ja) | 2007-01-18 | 2014-08-20 | ユニヴァーシティ オブ ミズーリー−コロンビア | 筋鞘にnNOSを回復させる合成ミニ/ミクロジストロフィン遺伝子 |
| US9611302B2 (en) * | 2007-04-09 | 2017-04-04 | University Of Florida Research Foundation, Inc. | High-transduction-efficiency RAAV vectors, compositions, and methods of use |
| US9725485B2 (en) | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
| DK2191001T3 (en) * | 2007-04-09 | 2016-09-19 | Univ Florida | RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF |
| CN103695459A (zh) | 2007-09-18 | 2014-04-02 | 巴斯夫植物科学有限公司 | 产量提高的植物 |
| US20090215879A1 (en) | 2008-02-26 | 2009-08-27 | University Of North Carolina At Chapel Hill | Methods and compositions for adeno-associated virus (aav) with hi loop mutations |
| CN102159713A (zh) | 2008-05-20 | 2011-08-17 | Eos神经科学公司 | 用于递送光敏蛋白的载体和使用方法 |
| JP2010002479A (ja) | 2008-06-18 | 2010-01-07 | Crossfor:Kk | メガネレンズ用装飾体およびメガネ用装飾体装着具 |
| 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 |
| US8679837B2 (en) | 2009-04-02 | 2014-03-25 | University Of Florida Research Foundation, Inc. | Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors |
| SG175409A1 (en) | 2009-05-02 | 2011-12-29 | Genzyme Corp | Gene therapy for neurodegenerative disorders |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| EP2292781A1 (en) | 2009-08-17 | 2011-03-09 | Genethon | Baculovirus-based production of biopharmaceuticals free of contaminating baculoviral virions |
| WO2011122950A1 (en) | 2010-04-01 | 2011-10-06 | Amsterdam Molecular Therapeutics (Amt) Ip B.V. | Monomeric duplex aav vectors |
| ES2605305T3 (es) | 2010-04-23 | 2017-03-13 | University Of Massachusetts | Vectores de AAV que se dirigen al SNC y métodos de uso de los mismos |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| US9839696B2 (en) | 2010-04-30 | 2017-12-12 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| US8628966B2 (en) | 2010-04-30 | 2014-01-14 | City Of Hope | CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer |
| WO2012057363A1 (ja) | 2010-10-27 | 2012-05-03 | 学校法人自治医科大学 | 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン |
| CA2826273C (en) * | 2011-02-10 | 2021-11-02 | The University Of North Carolina At Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
| WO2012112578A2 (en) | 2011-02-14 | 2012-08-23 | The Children's Hospital Of Philadelphia | Improved aav8 vector with enhanced functional activity and methods of use thereof |
| US9976157B2 (en) | 2011-08-23 | 2018-05-22 | Evogene Ltd. | Isolated polynucleotides and polypeptides, and methods of using same for increasing plant yield and/or agricultural characteristics |
| FI124176B (en) | 2011-12-01 | 2014-04-15 | Helsingin Yliopisto | Polypeptide, polynucleotide, expression cassette, vector, host cell, plant and uses |
| SG11201406776TA (en) | 2012-04-18 | 2015-03-30 | Philadelphia Children Hospital | Composition and methods for highly efficient gene transfer using aav capsid variants |
| EP2660325A3 (en) | 2012-05-02 | 2014-02-12 | Christian Medical College | AAV vectors and corresponding nucleotide sequences and methods |
| EP2847337A4 (en) | 2012-05-09 | 2016-04-27 | Univ Oregon Health & Science | ADENO ASSOCIATED VIRUS PLASMIDS AND VECTORS |
| US10294281B2 (en) * | 2012-05-15 | 2019-05-21 | University Of Florida Research Foundation, Incorporated | High-transduction-efficiency rAAV vectors, compositions, and methods of use |
| EP2492347A1 (en) | 2012-05-22 | 2012-08-29 | Laboratorios Del. Dr. Esteve, S.A. | Methods for the production of vectors |
| EP3536795A3 (en) | 2012-06-21 | 2019-12-11 | Association Institut de Myologie | Widespread gene expression |
| US9567376B2 (en) | 2013-02-08 | 2017-02-14 | The Trustees Of The University Of Pennsylvania | Enhanced AAV-mediated gene transfer for retinal therapies |
| US10266845B2 (en) | 2013-02-08 | 2019-04-23 | The Trustees Of The University Of Pennsylvania | Enhanced AAV-mediated gene transfer for retinal therapies |
| CN110885854A (zh) | 2013-03-15 | 2020-03-17 | 北卡罗来纳-查佩尔山大学 | 双重聚糖结合aav载体的方法和组合物 |
| CA2917018A1 (en) | 2013-05-21 | 2014-12-04 | University Of Florida Research Foundation, Inc. | Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer |
| CN105247044B (zh) | 2013-05-31 | 2021-05-07 | 加利福尼亚大学董事会 | 腺相关病毒变体及其使用方法 |
| HUE047996T2 (hu) | 2013-07-22 | 2020-05-28 | Childrens Hospital Philadelphia | AAV variáns és készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez |
| WO2015038958A1 (en) | 2013-09-13 | 2015-03-19 | California Institute Of Technology | Selective recovery |
| EP3049527A4 (en) | 2013-09-26 | 2017-08-16 | University of Florida Research Foundation, Inc. | Synthetic combinatorial aav capsid library for targeted gene therapy |
| EP3459965B1 (en) | 2013-10-11 | 2020-12-02 | Massachusetts Eye & Ear Infirmary | Methods of predicting ancestral virus sequences and uses thereof |
| CN104592364B (zh) * | 2013-10-30 | 2018-05-01 | 北京大学 | 定点突变和定点修饰的腺相关病毒、其制备方法及应用 |
| US10072251B2 (en) | 2014-02-19 | 2018-09-11 | University Of Massachusetts | Recombinant AAVS having useful transcytosis properties |
| WO2015126972A1 (en) | 2014-02-21 | 2015-08-27 | University Of Florida Research Foundation, Inc. | Methods and compositions for gene delivery to on bipolar cells |
| EP3113787B1 (en) | 2014-03-04 | 2019-12-04 | University of Florida Research Foundation, Inc. | Improved raav vectors and methods for transduction of photoreceptors and rpe cells |
| US10746742B2 (en) | 2014-04-25 | 2020-08-18 | Oregon Health & Science University | Methods of viral neutralizing antibody epitope mapping |
| EA034355B1 (ru) | 2014-05-02 | 2020-01-30 | Джензим Корпорейшн | Векторы на основе aav для генной терапии в сетчатке и cns |
| MX384480B (es) | 2014-05-28 | 2025-03-14 | Evogene Ltd | Polinucleotidos aislados, polipeptidos y metodos para utilizarlos para aumentar la tolerancia al stress abiotico, la biomasa y el rendimiento de plantas. |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| HUE060907T2 (hu) | 2014-06-25 | 2023-04-28 | Acuitas Therapeutics Inc | Új lipidek és lipid nanorészecske formulációk nukleinsavak bevitelére |
| AU2015320694B2 (en) | 2014-09-24 | 2021-11-11 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| US10711270B2 (en) | 2014-10-03 | 2020-07-14 | University Of Massachusetts | High efficiency library-identified AAV vectors |
| CN107073051B (zh) | 2014-10-21 | 2021-08-24 | 马萨诸塞大学 | 重组aav变体及其用途 |
| EP4344741A3 (en) | 2014-11-21 | 2024-08-28 | The University of North Carolina at Chapel Hill | Aav vectors targeted to the central nervous system |
| EP3245220B1 (en) | 2015-01-14 | 2023-09-20 | The University of North Carolina at Chapel Hill | Methods and compositions for targeted gene transfer |
| WO2016126857A1 (en) * | 2015-02-03 | 2016-08-11 | University Of Florida Research Foundation, Inc. | Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof |
| US20180135074A1 (en) * | 2015-02-19 | 2018-05-17 | University Of Florida Research Foundation, Incorporated | Recombinant aav vectors for gene therapy of human hematopoietic disorders |
| US11021519B2 (en) | 2015-03-02 | 2021-06-01 | Adverum Biotechnologies, Inc. | Compositions and methods for intravitreal delivery of polynucleotides to retinal cones |
| EP3277819B1 (en) | 2015-03-24 | 2021-03-03 | The Regents of The University of California | Adeno-associated virus variants and methods of use thereof |
| US10081659B2 (en) | 2015-04-06 | 2018-09-25 | The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services | Adeno-associated vectors for enhanced transduction and reduced immunogenicity |
| WO2016164642A1 (en) | 2015-04-08 | 2016-10-13 | The United States Of America, As Represented By The Secretary Of Health And Human Services | Viral gene therapy as treatment for cholesterol storage disease or disorder |
| US11046955B2 (en) | 2015-04-24 | 2021-06-29 | University Of Massachusetts | Modified AAV constructs and uses thereof |
| WO2016179644A1 (en) | 2015-05-08 | 2016-11-17 | Children's Medical Research Institute | Promoters for expression of heterologous genes |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| EP3294894B8 (en) | 2015-05-12 | 2019-09-25 | The U.S.A. as represented by the Secretary, Department of Health and Human Services | Aav isolate and fusion protein comprising nerve growth factor signal peptide and parathyroid hormone |
| US20180216133A1 (en) | 2015-07-17 | 2018-08-02 | The Trustees Of The University Of Pennsylvania | Compositions and methods for achieving high levels of transduction in human liver cells |
| WO2017019876A1 (en) | 2015-07-28 | 2017-02-02 | University Of Massachusetts | Transgenic expression of dnase i in vivo delivered by an adeno-associated virus vector |
| WO2017019994A2 (en) | 2015-07-30 | 2017-02-02 | Massachusetts Eye And Ear Infirmary | Ancestral virus sequences and uses thereof |
| WO2017066764A2 (en) | 2015-10-16 | 2017-04-20 | William Marsh Rice University | Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses |
| WO2017070516A1 (en) | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
| IL259595B2 (en) | 2015-12-01 | 2024-01-01 | Spark Therapeutics Inc | Gradual methods for the production of a recombinant adenovirus (AAV) vector in a serum-free suspension cell culture system suitable for clinical use |
| US10406244B2 (en) | 2015-12-02 | 2019-09-10 | The Board Of Trustees Of The Leland Stanford Junior University | AAV vectors with expanded packaging capacity |
| 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 |
| AU2016371779C1 (en) | 2015-12-14 | 2022-10-13 | The University Of North Carolina At Chapel Hill | Modified capsid proteins for enhanced delivery of parvovirus vectors |
| WO2017139643A1 (en) | 2016-02-12 | 2017-08-17 | University Of Massachusetts | Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization |
| WO2017143100A1 (en) | 2016-02-16 | 2017-08-24 | The Board Of Trustees Of The Leland Stanford Junior University | Novel recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies |
| CN108697774A (zh) | 2016-02-22 | 2018-10-23 | 北卡罗来纳大学教堂山分校 | 治疗mps i-相关失明的aav-idua载体 |
| SG11201808812RA (en) | 2016-04-15 | 2018-11-29 | Univ Pennsylvania | Novel aav8 mutant capsids and compositions containing same |
| US11866462B2 (en) | 2016-05-04 | 2024-01-09 | Oregon Health & Science University | Recombinant adeno-associated viral vectors |
| RU2725286C2 (ru) | 2016-05-13 | 2020-06-30 | 4Д Молекьюлар Терапьютикс Инк. | Варианты капсидов аденоассоциированного вируса и способы их применения |
| EP3458588B1 (en) | 2016-05-18 | 2025-10-29 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| CA3031095A1 (en) | 2016-07-21 | 2018-01-25 | Spark Therapeutics, Inc. | Scalable high recovery methods for producing high yield recombinant adeno-associated viral (raav) vector and recombinant adeno-associated viral (raav) vectors produced thereby |
| IL305149A (en) | 2016-07-26 | 2023-10-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| CN109890966A (zh) | 2016-08-16 | 2019-06-14 | 北卡罗来纳-查佩尔山大学 | 用于靶向基因转移的方法和组合物 |
| US11078238B2 (en) | 2016-09-29 | 2021-08-03 | University Of Florida Research Foundation, Incorporated | AAVrh.10 variants with host antibody escape capabilities and altered tissue targeting properties |
| US11192925B2 (en) | 2016-10-19 | 2021-12-07 | Adverum Biotechnologies, Inc. | Modified AAV capsids and uses thereof |
| WO2018119330A2 (en) | 2016-12-22 | 2018-06-28 | Oregon Health & Science University | Adeno associated viral vectors |
| CA3052829A1 (en) | 2017-02-15 | 2018-08-23 | The University Of North Carolina At Chapel Hill | Methods and compositions for gene transfer across the vasculature |
| EP3589730B1 (en) | 2017-02-28 | 2023-12-27 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clade f vector and uses therefor |
| EP3610023B1 (en) | 2017-03-15 | 2023-10-04 | The University of North Carolina at Chapel Hill | Polyploid adeno-associated virus vectors and methods of making and using the same |
| JP7330899B2 (ja) | 2017-05-10 | 2023-08-22 | マサチューセッツ アイ アンド イヤー インファーマリー | ウイルスのアセンブリ活性化タンパク質(aap)依存性を改変する方法および組成物 |
| CA3068010A1 (en) | 2017-06-20 | 2018-12-27 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Codon-optimized human npc1 genes for the treatment of niemann-pick type c1 deficiency and related conditions |
| JP2020530307A (ja) | 2017-06-30 | 2020-10-22 | インティマ・バイオサイエンス,インコーポレーテッド | 遺伝子治療のためのアデノ随伴ウイルスベクター |
| JP7389040B2 (ja) | 2018-01-17 | 2023-11-29 | メイラジーティーエックス・ユーケー・ザ・セカンド・リミテッド | 遺伝子治療のための修飾rAAVキャプシドタンパク質 |
| CN120519403A (zh) | 2018-02-27 | 2025-08-22 | 宾夕法尼亚州大学信托人 | 新颖腺相关病毒(aav)载体、具有降低的衣壳脱酰胺化的aav载体及其用途 |
| IL276859B2 (en) | 2018-02-27 | 2025-12-01 | Univ Pennsylvania | Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor |
| CA3092107A1 (en) | 2018-02-28 | 2019-09-06 | The University Of North Carolina At Chapel Hill | Methods and compositions for antibody-evading virus vectors |
| EP3762500A1 (en) | 2018-03-06 | 2021-01-13 | Voyager Therapeutics, Inc. | Insect cell manufactured partial self-complementary aav genomes |
| KR20200130337A (ko) | 2018-03-06 | 2020-11-18 | 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 | Aav 키메라 |
| CN111819286B (zh) | 2018-03-16 | 2024-05-24 | 国家儿童医院研究所 | 通过衣壳修饰增加组织特异性的基因递送 |
| WO2019195444A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Antibody-evading virus vectors |
| JP7425738B2 (ja) | 2018-04-03 | 2024-01-31 | ギンコ バイオワークス インコーポレイテッド | 眼組織を標的とするウイルスベクター |
| CN112272672A (zh) | 2018-04-03 | 2021-01-26 | 斯特里迪比奥公司 | 抗体逃避性病毒载体 |
-
2016
- 2016-09-28 AU AU2016332821A patent/AU2016332821B2/en not_active Ceased
- 2016-09-28 EP EP20212583.7A patent/EP3831842A1/en not_active Withdrawn
- 2016-09-28 SG SG10202107733QA patent/SG10202107733QA/en unknown
- 2016-09-28 IL IL314171A patent/IL314171A/en unknown
- 2016-09-28 US US15/763,706 patent/US10745447B2/en active Active
- 2016-09-28 CN CN201680056241.5A patent/CN108137655B/zh not_active Expired - Fee Related
- 2016-09-28 WO PCT/US2016/054143 patent/WO2017058892A2/en not_active Ceased
- 2016-09-28 EP EP16852471.8A patent/EP3356390B1/en active Active
- 2016-09-28 CN CN202210436783.2A patent/CN114606267A/zh active Pending
- 2016-09-28 JP JP2018515949A patent/JP7064214B2/ja not_active Expired - Fee Related
- 2016-09-28 CA CA2996420A patent/CA2996420A1/en active Pending
- 2016-09-28 IL IL299901A patent/IL299901B2/en unknown
- 2016-09-28 BR BR112018003665-0A patent/BR112018003665A2/en not_active IP Right Cessation
- 2016-09-28 IL IL292830A patent/IL292830B2/en unknown
- 2016-09-28 ES ES16852471T patent/ES2865487T3/es active Active
- 2016-09-28 DK DK16852471.8T patent/DK3356390T3/da active
- 2016-09-28 PL PL16852471T patent/PL3356390T3/pl unknown
- 2016-09-28 IL IL257628A patent/IL257628B2/en unknown
- 2016-09-28 PT PT168524718T patent/PT3356390T/pt unknown
-
2020
- 2020-07-06 US US16/921,239 patent/US11208438B2/en active Active
-
2021
- 2021-11-15 US US17/526,510 patent/US11840555B2/en active Active
-
2022
- 2022-01-28 JP JP2022011956A patent/JP7390693B2/ja active Active
- 2022-05-11 AU AU2022203144A patent/AU2022203144A1/en not_active Abandoned
-
2023
- 2023-10-17 US US18/488,604 patent/US20240158450A1/en not_active Abandoned
- 2023-11-13 JP JP2023193120A patent/JP2024026088A/ja active Pending
Patent Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20050287122A1 (en) | 2001-01-05 | 2005-12-29 | Children's Hospital Inc. | AAV vectors and methods |
| WO2015121501A1 (en) | 2014-02-17 | 2015-08-20 | King's College London | Adeno-associated virus vector |
Non-Patent Citations (4)
| Title |
|---|
| Journal of Virology,2012年,Vol.86, No.15,p.7739-7751 |
| Molecular Therapy,2012年,Vol.20, No.2,p.329-338 |
| Nature Communications,2014年,Vol.5, article No.3075,p.1-14 |
| Selection of neutralizing antibody-resistant AAV8 variants with structure-guided site-specific saturated mutagenesis,Molecular Therapy,2011年,Vol.19 Suppl.1,S129 |
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7064214B2 (ja) | 抗体回避性ウイルスベクターのための方法および組成物 | |
| JP6042825B2 (ja) | 改変された形質導入プロファイルを有するウイルスベクターならびにその製造および使用の方法 | |
| 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 | |
| JP7291397B2 (ja) | 血管系を通過する遺伝子移入の方法および組成物 | |
| JP6396988B2 (ja) | 二重グリカン結合性aavベクターのための方法および組成物 | |
| JP5136766B2 (ja) | キメラベクター | |
| 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 | |
| JP2021519581A (ja) | 抗体を回避するウイルスベクター | |
| JP2022525955A (ja) | 組換えアデノ随伴ウイルスベクター | |
| JP2021519099A (ja) | 抗体を回避するウイルスベクター | |
| JP2018503376A (ja) | 標的化遺伝子移入のための方法および組成物 | |
| JP2021522775A (ja) | 合理的なポリプロイド性アデノ随伴ウイルスベクターならびにその製造法および使用法 | |
| HK40053197A (en) | Methods and compositions for antibody-evading virus vectors | |
| HK1257410B (en) | Methods and compositions for antibody-evading virus vectors |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20190930 |
|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20190930 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20201006 |
|
| A601 | Written request for extension of time |
Free format text: JAPANESE INTERMEDIATE CODE: A601 Effective date: 20201221 |
|
| A601 | Written request for extension of time |
Free format text: JAPANESE INTERMEDIATE CODE: A601 Effective date: 20210226 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20210406 |
|
| A02 | Decision of refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A02 Effective date: 20211001 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20220128 |
|
| C60 | Trial request (containing other claim documents, opposition documents) |
Free format text: JAPANESE INTERMEDIATE CODE: C60 Effective date: 20220128 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A821 Effective date: 20220131 |
|
| A911 | Transfer to examiner for re-examination before appeal (zenchi) |
Free format text: JAPANESE INTERMEDIATE CODE: A911 Effective date: 20220218 |
|
| C21 | Notice of transfer of a case for reconsideration by examiners before appeal proceedings |
Free format text: JAPANESE INTERMEDIATE CODE: C21 Effective date: 20220222 |
|
| TRDD | Decision of grant or rejection written | ||
| A01 | Written decision to grant a patent or to grant a registration (utility model) |
Free format text: JAPANESE INTERMEDIATE CODE: A01 Effective date: 20220318 |
|
| A61 | First payment of annual fees (during grant procedure) |
Free format text: JAPANESE INTERMEDIATE CODE: A61 Effective date: 20220412 |
|
| R150 | Certificate of patent or registration of utility model |
Ref document number: 7064214 Country of ref document: JP Free format text: JAPANESE INTERMEDIATE CODE: R150 |
|
| LAPS | Cancellation because of no payment of annual fees |