AU2019228504B2 - Novel adeno-associated virus (AAV) vectors, AAV vectors having reduced capsid deamidation and uses therefor - Google Patents
Novel adeno-associated virus (AAV) vectors, AAV vectors having reduced capsid deamidation and uses therefor Download PDFInfo
- Publication number
- AU2019228504B2 AU2019228504B2 AU2019228504A AU2019228504A AU2019228504B2 AU 2019228504 B2 AU2019228504 B2 AU 2019228504B2 AU 2019228504 A AU2019228504 A AU 2019228504A AU 2019228504 A AU2019228504 A AU 2019228504A AU 2019228504 B2 AU2019228504 B2 AU 2019228504B2
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- Prior art keywords
- hsa
- mir
- capsid
- aav
- proteins
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- 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.)
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Classifications
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- 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
- 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
- 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)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Virology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (29)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862635964P | 2018-02-27 | 2018-02-27 | |
| US201862635968P | 2018-02-27 | 2018-02-27 | |
| US62/635,968 | 2018-02-27 | ||
| US62/635,964 | 2018-02-27 | ||
| US201862663788P | 2018-04-27 | 2018-04-27 | |
| US201862663797P | 2018-04-27 | 2018-04-27 | |
| US62/663,788 | 2018-04-27 | ||
| US62/663,797 | 2018-04-27 | ||
| US201862667587P | 2018-05-06 | 2018-05-06 | |
| US62/667,587 | 2018-05-06 | ||
| US201862667888P | 2018-05-07 | 2018-05-07 | |
| US201862667881P | 2018-05-07 | 2018-05-07 | |
| US62/667,881 | 2018-05-07 | ||
| US62/667,888 | 2018-05-07 | ||
| US201862677474P | 2018-05-29 | 2018-05-29 | |
| US201862667585P | 2018-05-29 | 2018-05-29 | |
| US201862677471P | 2018-05-29 | 2018-05-29 | |
| US62/677,474 | 2018-05-29 | ||
| US62/667,585 | 2018-05-29 | ||
| US62/677,471 | 2018-05-29 | ||
| US201862703670P | 2018-07-26 | 2018-07-26 | |
| US201862703673P | 2018-07-26 | 2018-07-26 | |
| US62/703,673 | 2018-07-26 | ||
| US62/703,670 | 2018-07-26 | ||
| US201862722382P | 2018-08-24 | 2018-08-24 | |
| US201862722388P | 2018-08-24 | 2018-08-24 | |
| US62/722,388 | 2018-08-24 | ||
| US62/722,382 | 2018-08-24 | ||
| PCT/US2019/019861 WO2019169004A1 (en) | 2018-02-27 | 2019-02-27 | Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| AU2019228504A1 AU2019228504A1 (en) | 2020-09-10 |
| AU2019228504B2 true AU2019228504B2 (en) | 2025-06-26 |
Family
ID=67809202
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2019228504A Active AU2019228504B2 (en) | 2018-02-27 | 2019-02-27 | Novel adeno-associated virus (AAV) vectors, AAV vectors having reduced capsid deamidation and uses therefor |
Country Status (14)
| Country | Link |
|---|---|
| US (2) | US12416016B2 (2) |
| EP (1) | EP3768695A4 (2) |
| JP (2) | JP7498665B2 (2) |
| KR (1) | KR102848518B1 (2) |
| CN (2) | CN112236443B (2) |
| AU (1) | AU2019228504B2 (2) |
| BR (1) | BR112020017278A2 (2) |
| CA (1) | CA3091795A1 (2) |
| CL (1) | CL2020002201A1 (2) |
| IL (1) | IL276860A (2) |
| MA (1) | MA52087A (2) |
| MX (1) | MX2020008932A (2) |
| SG (1) | SG11202007942YA (2) |
| WO (1) | WO2019169004A1 (2) |
Families Citing this family (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2865487T3 (es) | 2015-09-28 | 2021-10-15 | Univ North Carolina Chapel Hill | Métodos y composiciones para vectores virales que evaden los anticuerpos |
| EP3768695A4 (en) * | 2018-02-27 | 2022-04-06 | The Trustees of the University of Pennsylvania | NOVEL ADENO-ASSOCIATED VIRUS (AAV) VECTORS, AAV VECTORS WITH REDUCED CAPSIID DEAMIDATION AND USES THEREOF |
| EP3774852A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Antibody-evading virus vectors |
| AU2019247748A1 (en) | 2018-04-03 | 2020-10-08 | Ginkgo Bioworks, Inc. | Antibody-evading virus vectors |
| WO2020132385A1 (en) | 2018-12-21 | 2020-06-25 | The University Of North Carolina At Chapel Hill | Optimized galc genes and expression cassettes and their use |
| AR118465A1 (es) | 2019-03-21 | 2021-10-06 | Stridebio Inc | Vectores de virus adenoasociados recombinantes |
| SG11202111375RA (en) | 2019-04-29 | 2021-11-29 | Univ Pennsylvania | Novel aav capsids and compositions containing same |
| US12611436B2 (en) | 2019-10-17 | 2026-04-28 | Sarepta Therapeutics, Inc. | AAV transfer cassette |
| JP2022551739A (ja) | 2019-10-17 | 2022-12-13 | ストライドバイオ，インコーポレイテッド | ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター |
| EP4048681A4 (en) * | 2019-10-21 | 2024-03-20 | The Trustees of The University of Pennsylvania | AAV3B VARIANTS WITH IMPROVED PRODUCTION YIELD AND LIVER TROPISM |
| CN114902051B (zh) * | 2020-01-03 | 2026-04-17 | 萨勒普塔医疗公司 | 用于分析aav衣壳蛋白的方法 |
| US12497612B2 (en) | 2020-03-09 | 2025-12-16 | University Of Massachusetts | Gene replacement therapy for FOXG1 syndrome |
| US12552838B2 (en) | 2020-04-20 | 2026-02-17 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
| US20230175014A1 (en) * | 2020-04-27 | 2023-06-08 | The Trustees Of The University Of Pennsylvania | Compositions and methods for reducing nuclease expression and off-target activity using a promoter with low transcriptional activity |
| AU2021292200A1 (en) | 2020-06-17 | 2023-02-02 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of gene therapy patients |
| IL299762A (en) | 2020-07-13 | 2023-03-01 | Univ Pennsylvania | Useful preparations for the treatment of Charcot-Marietot disease |
| AU2021328475A1 (en) | 2020-08-19 | 2023-03-16 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for treatment of Rett syndrome |
| WO2022082109A1 (en) * | 2020-10-18 | 2022-04-21 | The Trustees Of The University Of Pennsylvania | Improved adeno-associated virus (aav) vector and uses therefor |
| WO2022165313A1 (en) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| IL307633A (en) | 2021-04-12 | 2023-12-01 | Univ Pennsylvania | Useful preparations in the treatment of spinal muscular atrophy (SBMA) |
| WO2023081807A1 (en) * | 2021-11-04 | 2023-05-11 | The Trustees of the University of Pennsylvania Penn Center for Innovation | Compositions and methods for reducing pcsk9 levels in a subject |
| WO2023133574A1 (en) | 2022-01-10 | 2023-07-13 | The Trustees Of The University Of Pennsylvania | Compositions and methods useful for treatment of c9orf72-mediated disorders |
| WO2023133584A1 (en) | 2022-01-10 | 2023-07-13 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of metachromatic leukodystrophy |
| WO2023196892A1 (en) | 2022-04-06 | 2023-10-12 | The Trustees Of The University Of Pennsylvania | Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors |
| WO2024206226A1 (en) * | 2023-03-25 | 2024-10-03 | Duke University | Compositions comprising kidney-tropic aavs and methods of use thereof |
| WO2025250683A1 (en) * | 2024-05-29 | 2025-12-04 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
Citations (3)
| 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 |
| 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 |
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| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
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| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| US20070172460A1 (en) * | 2003-03-19 | 2007-07-26 | Jurgen Kleinschmidt | Random peptide library displayed on aav vectors |
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| US20060246079A1 (en) | 2003-11-14 | 2006-11-02 | Morrow Phillip R | Neutralizing human antibodies to anthrax toxin |
| JP4495210B2 (ja) | 2005-06-09 | 2010-06-30 | パナソニック株式会社 | 振幅誤差補償装置及び直交度誤差補償装置 |
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| 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 |
| AU2015320694B2 (en) * | 2014-09-24 | 2021-11-11 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| CA2969145A1 (en) * | 2014-11-26 | 2016-06-02 | The Regents Of The University Of California | Therapeutic compositions comprising transcription factors and methods of making and using the same |
| CN108779167A (zh) | 2015-10-28 | 2018-11-09 | 宾夕法尼亚州大学信托人 | 鞘内施用腺伴随病毒载体用于基因治疗 |
| ES2934848T3 (es) | 2015-12-11 | 2023-02-27 | Univ Pennsylvania | Método de purificación escalable para AAV8 |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| SG10201912761UA (en) | 2016-04-15 | 2020-02-27 | The Trustees Of The Univ Of Pennsyvania | Gene therapy for treating mucopolysaccharidosis type ii |
| IL305149A (en) * | 2016-07-26 | 2023-10-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| PT3589730T (pt) | 2017-02-28 | 2024-02-28 | Univ Pennsylvania | Vetor de vírus adenoassociado (aav) de clado f e suas utilizações |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| EP3768695A4 (en) * | 2018-02-27 | 2022-04-06 | The Trustees of the University of Pennsylvania | NOVEL ADENO-ASSOCIATED VIRUS (AAV) VECTORS, AAV VECTORS WITH REDUCED CAPSIID DEAMIDATION AND USES THEREOF |
-
2019
- 2019-02-27 EP EP19760152.9A patent/EP3768695A4/en active Pending
- 2019-02-27 AU AU2019228504A patent/AU2019228504B2/en active Active
- 2019-02-27 MA MA052087A patent/MA52087A/fr unknown
- 2019-02-27 CN CN201980029022.1A patent/CN112236443B/zh active Active
- 2019-02-27 SG SG11202007942YA patent/SG11202007942YA/en unknown
- 2019-02-27 WO PCT/US2019/019861 patent/WO2019169004A1/en not_active Ceased
- 2019-02-27 US US16/975,545 patent/US12416016B2/en active Active
- 2019-02-27 JP JP2020545159A patent/JP7498665B2/ja active Active
- 2019-02-27 CN CN202510015875.7A patent/CN120519403A/zh active Pending
- 2019-02-27 MX MX2020008932A patent/MX2020008932A/es unknown
- 2019-02-27 KR KR1020207027462A patent/KR102848518B1/ko active Active
- 2019-02-27 BR BR112020017278-2A patent/BR112020017278A2/pt unknown
- 2019-02-27 CA CA3091795A patent/CA3091795A1/en active Pending
-
2020
- 2020-08-23 IL IL276860A patent/IL276860A/en unknown
- 2020-08-26 CL CL2020002201A patent/CL2020002201A1/es unknown
-
2024
- 2024-05-31 JP JP2024088641A patent/JP2024115558A/ja active Pending
-
2025
- 2025-08-12 US US19/297,827 patent/US20260049334A1/en active Pending
Patent Citations (3)
| 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 |
| 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 |
Also Published As
| Publication number | Publication date |
|---|---|
| CN112236443B (zh) | 2025-02-07 |
| CN112236443A (zh) | 2021-01-15 |
| JP7498665B2 (ja) | 2024-06-12 |
| BR112020017278A2 (pt) | 2020-12-22 |
| MA52087A (fr) | 2021-06-02 |
| EP3768695A1 (en) | 2021-01-27 |
| WO2019169004A1 (en) | 2019-09-06 |
| JP2021515548A (ja) | 2021-06-24 |
| EP3768695A4 (en) | 2022-04-06 |
| AU2019228504A1 (en) | 2020-09-10 |
| KR20210006327A (ko) | 2021-01-18 |
| WO2019169004A8 (en) | 2019-12-05 |
| JP2024115558A (ja) | 2024-08-26 |
| CA3091795A1 (en) | 2019-09-06 |
| US20210123073A1 (en) | 2021-04-29 |
| IL276860A (en) | 2020-10-29 |
| SG11202007942YA (en) | 2020-09-29 |
| US12416016B2 (en) | 2025-09-16 |
| MX2020008932A (es) | 2020-10-01 |
| KR102848518B1 (ko) | 2025-08-22 |
| CN120519403A (zh) | 2025-08-22 |
| CL2020002201A1 (es) | 2020-12-18 |
| US20260049334A1 (en) | 2026-02-19 |
| WO2019169004A9 (en) | 2019-10-31 |
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| FGA | Letters patent sealed or granted (standard patent) |