IL276859B2 - Novel adeno-associated virus (AAV) vectors, AAV vectors with reduced capsid deamidation and uses therefor - Google Patents
Novel adeno-associated virus (AAV) vectors, AAV vectors with reduced capsid deamidation and uses thereforInfo
- Publication number
- IL276859B2 IL276859B2 IL276859A IL27685920A IL276859B2 IL 276859 B2 IL276859 B2 IL 276859B2 IL 276859 A IL276859 A IL 276859A IL 27685920 A IL27685920 A IL 27685920A IL 276859 B2 IL276859 B2 IL 276859B2
- Authority
- IL
- Israel
- Prior art keywords
- aav
- vectors
- associated virus
- uses therefor
- novel adeno
- Prior art date
Links
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
- 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
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862635964P | 2018-02-27 | 2018-02-27 | |
| US201862667585P | 2018-05-29 | 2018-05-29 | |
| US201862677471P | 2018-05-29 | 2018-05-29 | |
| US201862703670P | 2018-07-26 | 2018-07-26 | |
| US201862722382P | 2018-08-24 | 2018-08-24 | |
| PCT/US2019/019804 WO2019168961A1 (en) | 2018-02-27 | 2019-02-27 | Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| IL276859A IL276859A (en) | 2020-10-29 |
| IL276859B1 IL276859B1 (en) | 2025-08-01 |
| IL276859B2 true IL276859B2 (en) | 2025-12-01 |
Family
ID=67805569
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL276859A IL276859B2 (en) | 2018-02-27 | 2020-08-23 | Novel adeno-associated virus (AAV) vectors, AAV vectors with reduced capsid deamidation and uses therefor |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20200407750A1 (enExample) |
| EP (1) | EP3758724A4 (enExample) |
| JP (3) | JP2021516046A (enExample) |
| KR (1) | KR20210010434A (enExample) |
| CN (2) | CN112352050A (enExample) |
| AU (1) | AU2019227726B2 (enExample) |
| BR (1) | BR112020017348A2 (enExample) |
| CA (1) | CA3091806A1 (enExample) |
| CL (2) | CL2020002200A1 (enExample) |
| IL (1) | IL276859B2 (enExample) |
| MX (1) | MX2020008933A (enExample) |
| SG (1) | SG11202008182TA (enExample) |
| WO (1) | WO2019168961A1 (enExample) |
Families Citing this family (39)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN114606267A (zh) | 2015-09-28 | 2022-06-10 | 北卡罗来纳-查佩尔山大学 | 逃避抗体的病毒载体的方法和组合物 |
| EA202092362A1 (ru) | 2018-04-03 | 2021-01-11 | Страйдбайо, Инк. | Вирусные векторы для нацеливания на ткани глаза |
| BR112020020266A2 (pt) | 2018-04-03 | 2021-01-19 | Stridebio, Inc. | Vetores de vírus com evasão de anticorpos |
| EP3774852A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Antibody-evading virus vectors |
| CA3113975A1 (en) | 2018-10-01 | 2020-04-09 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating propionic acidemia |
| BR112021018776A2 (pt) | 2019-03-21 | 2021-11-30 | Stridebio Inc | Vetores de vírus adenoassociado recombinante |
| JP2022550435A (ja) | 2019-10-04 | 2022-12-01 | ウルトラジェニックス ファーマシューティカル インコーポレイテッド | 組換えａａｖの改善された治療的使用のための方法 |
| CN114787180A (zh) | 2019-10-17 | 2022-07-22 | 斯特里迪比奥公司 | 用于治疗c型尼曼-匹克病的腺相关病毒载体 |
| TW202130653A (zh) * | 2019-10-21 | 2021-08-16 | 賓州大學委員會 | 具改進生產產率及肝臟趨性之ａａｖ３ｂ變異體 |
| CA3222852A1 (en) * | 2020-01-03 | 2021-07-08 | Sarepta Therapeutics, Inc. | Methods for analyzing aav capsid proteins |
| MX2022009982A (es) * | 2020-02-14 | 2022-09-12 | Ultragenyx Pharmaceutical Inc | Terapia genica para el tratamiento de transtorno por deficiencia de cdkl5. |
| EP4127189A1 (en) | 2020-03-31 | 2023-02-08 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating propionic acidemia |
| CN115885040A (zh) * | 2020-04-27 | 2023-03-31 | 宾夕法尼亚州大学信托人 | 可用于治疗cdkl5缺乏症（cdd）的组合物 |
| CA3183153A1 (en) | 2020-06-17 | 2021-12-23 | Christian HINDERER | Compositions and methods for treatment of gene therapy patients |
| US20230270884A1 (en) | 2020-07-13 | 2023-08-31 | The Trustees Of The University Of Pennsylvania | Compositions useful for treatment of charcot-marie-tooth disease |
| US12104163B2 (en) | 2020-08-19 | 2024-10-01 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for treatment of Rett syndrome |
| AU2021359874A1 (en) * | 2020-10-18 | 2023-05-25 | The Trustees Of The University Of Pennsylvania | Improved adeno-associated virus (aav) vector and uses therefor |
| JP2023550581A (ja) * | 2020-10-29 | 2023-12-04 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Ａａｖカプシド及びそれを含有する組成物 |
| MX2023006444A (es) | 2020-12-01 | 2023-08-10 | Univ Pennsylvania | Composiciones novedosas con motivos dirigidos a tejido específico y composiciones que los contienen. |
| JP2024505257A (ja) | 2021-02-01 | 2024-02-05 | レジェンクスバイオ インコーポレーテッド | 神経セロイドリポフスチン症の遺伝子治療 |
| MX2023012052A (es) | 2021-04-12 | 2024-03-15 | Univ Pennsylvania | Composiciones útiles para tratar la atrofia muscular espinal y bulbar (sbma). |
| WO2022226263A1 (en) | 2021-04-23 | 2022-10-27 | The Trustees Of The University Of Pennsylvania | Novel compositions with brain-specific targeting motifs and compositions containing same |
| US20250101386A1 (en) * | 2021-12-28 | 2025-03-27 | Chengdu Origen Biotechnology Co., Ltd. | Modified aav capsid protein and use thereof |
| WO2023133584A1 (en) | 2022-01-10 | 2023-07-13 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of metachromatic leukodystrophy |
| AR128239A1 (es) | 2022-01-10 | 2024-04-10 | Univ Pennsylvania | Composiciones y métodos útiles para el tratamiento de trastornos mediados por c9orf72 |
| AU2023211652A1 (en) | 2022-01-25 | 2024-08-08 | The Trustees Of The University Of Pennsylvania | Aav capsids for improved heart transduction and detargeting of liver |
| CA3245327A1 (en) | 2022-03-07 | 2023-09-14 | Ultragenyx Pharmaceutical Inc. | MODIFIED BATCH-BASED AAV PRODUCTION SYSTEMS AND PROCESSES |
| EP4504258A1 (en) | 2022-04-06 | 2025-02-12 | The Trustees of the University of Pennsylvania | Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors |
| CN115011601B (zh) * | 2022-06-27 | 2023-07-21 | 山东大学齐鲁医院 | 一种干扰JUND表达的shRNA、重组腺相关病毒载体及其应用 |
| WO2024015966A2 (en) * | 2022-07-15 | 2024-01-18 | The Trustees Of The University Of Pennsylvania | Recombinant aav having aav clade d and clade e capsids and compositions containing same |
| CN115554418B (zh) | 2022-11-22 | 2023-04-14 | 四川至善唯新生物科技有限公司 | 一种重组腺相关病毒载体的药物组合物及其用途 |
| WO2024130067A2 (en) | 2022-12-17 | 2024-06-20 | The Trustees Of The University Of Pennsylvania | Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same |
| WO2024130070A2 (en) | 2022-12-17 | 2024-06-20 | The Trustees Of The University Of Pennsylvania | Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof |
| AU2023412879A1 (en) * | 2022-12-23 | 2025-07-10 | Spark Therapeutics, Inc. | Adeno-associated virus formulations |
| CN121079310A (zh) * | 2023-05-03 | 2025-12-05 | 沃雅戈治疗公司 | 用于治疗与cdkl5缺乏相关的病症的组合物及方法 |
| WO2024229161A1 (en) * | 2023-05-03 | 2024-11-07 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency |
| AR133098A1 (es) | 2023-06-29 | 2025-08-27 | Univ Pennsylvania | Aav mutante con motivos dirigidos al sistema nervioso central y composiciones que lo contienen |
| WO2025102034A1 (en) | 2023-11-10 | 2025-05-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for barth syndrome |
| WO2025106661A1 (en) | 2023-11-14 | 2025-05-22 | The Trustees Of The University Of Pennsylvania | Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2017100674A1 (en) * | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| WO2017180854A1 (en) * | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsylvania | Novel aav8 mutant capsids and compositions containing same |
| WO2018035059A1 (en) * | 2016-08-15 | 2018-02-22 | Genzyme Corporation | Methods for detecting aav |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN102199626B (zh) * | 2003-09-30 | 2015-06-24 | 宾夕法尼亚大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| EP2383346B1 (en) * | 2005-04-07 | 2014-10-08 | The Trustees of the University of Pennsylvania | Modified AAVrh.64 capsids, compositions containing same and uses thereof |
| AU2013249202B2 (en) * | 2012-04-18 | 2018-08-09 | The Children's Hospital Of Philadelphia | Composition and methods for highly efficient gene transfer using AAV capsid variants |
| KR102380265B1 (ko) * | 2013-07-22 | 2022-03-29 | 더 칠드런스 호스피탈 오브 필라델피아 | 변종 ａａｖ 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도 |
| CN118956781A (zh) * | 2017-02-28 | 2024-11-15 | 宾夕法尼亚州大学信托人 | 腺相关病毒(aav)进化枝f载体及其用途 |
-
2019
- 2019-02-27 EP EP19760264.2A patent/EP3758724A4/en active Pending
- 2019-02-27 KR KR1020207027457A patent/KR20210010434A/ko not_active Ceased
- 2019-02-27 CA CA3091806A patent/CA3091806A1/en active Pending
- 2019-02-27 SG SG11202008182TA patent/SG11202008182TA/en unknown
- 2019-02-27 CN CN201980028806.2A patent/CN112352050A/zh active Pending
- 2019-02-27 WO PCT/US2019/019804 patent/WO2019168961A1/en not_active Ceased
- 2019-02-27 CN CN202411591073.2A patent/CN119842635A/zh active Pending
- 2019-02-27 AU AU2019227726A patent/AU2019227726B2/en active Active
- 2019-02-27 MX MX2020008933A patent/MX2020008933A/es unknown
- 2019-02-27 US US16/975,541 patent/US20200407750A1/en active Pending
- 2019-02-27 BR BR112020017348-7A patent/BR112020017348A2/pt unknown
- 2019-02-27 JP JP2020545160A patent/JP2021516046A/ja active Pending
-
2020
- 2020-08-23 IL IL276859A patent/IL276859B2/en unknown
- 2020-08-26 CL CL2020002200A patent/CL2020002200A1/es unknown
-
2022
- 2022-12-26 CL CL2022003757A patent/CL2022003757A1/es unknown
-
2023
- 2023-08-14 JP JP2023131761A patent/JP2023159235A/ja active Pending
-
2024
- 2024-01-18 JP JP2024005924A patent/JP2024041967A/ja active Pending
Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2017100674A1 (en) * | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| WO2017180854A1 (en) * | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsylvania | Novel aav8 mutant capsids and compositions containing same |
| WO2018035059A1 (en) * | 2016-08-15 | 2018-02-22 | Genzyme Corporation | Methods for detecting aav |
Non-Patent Citations (1)
| Title |
|---|
| GILES, APRIL R., ET AL., THE BIOCHEMICAL AND FUNCTIONAL EFFECTS OF SPONTANEOUS DEAMIDATION OF THE AAV CAPSID ON GENE THERAPY VECTORS., 1 May 2014 (2014-05-01) * |
Also Published As
| Publication number | Publication date |
|---|---|
| BR112020017348A2 (pt) | 2020-12-29 |
| CL2020002200A1 (es) | 2021-01-29 |
| JP2024041967A (ja) | 2024-03-27 |
| CN112352050A (zh) | 2021-02-09 |
| US20200407750A1 (en) | 2020-12-31 |
| SG11202008182TA (en) | 2020-09-29 |
| AU2019227726B2 (en) | 2025-09-04 |
| WO2019168961A1 (en) | 2019-09-06 |
| CN119842635A (zh) | 2025-04-18 |
| CA3091806A1 (en) | 2019-09-06 |
| JP2021516046A (ja) | 2021-07-01 |
| JP2023159235A (ja) | 2023-10-31 |
| EP3758724A1 (en) | 2021-01-06 |
| IL276859B1 (en) | 2025-08-01 |
| AU2019227726A1 (en) | 2020-09-10 |
| EP3758724A4 (en) | 2022-07-06 |
| CL2022003757A1 (es) | 2023-05-19 |
| MX2020008933A (es) | 2021-01-15 |
| KR20210010434A (ko) | 2021-01-27 |
| IL276859A (en) | 2020-10-29 |
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