CA3091806A1 - 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 PDF

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Publication number
CA3091806A1
CA3091806A1 CA3091806A CA3091806A CA3091806A1 CA 3091806 A1 CA3091806 A1 CA 3091806A1 CA 3091806 A CA3091806 A CA 3091806A CA 3091806 A CA3091806 A CA 3091806A CA 3091806 A1 CA3091806 A1 CA 3091806A1
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Canada
Prior art keywords
hsa
mir
capsid
deamidation
seq
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Pending
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CA3091806A
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English (en)
French (fr)
Inventor
James M. Wilson
April TEPE
Kevin Turner
Joshua Joyner SIMS
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University of Pennsylvania Penn
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University of Pennsylvania Penn
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Application filed by University of Pennsylvania Penn filed Critical University of Pennsylvania Penn
Publication of CA3091806A1 publication Critical patent/CA3091806A1/en
Pending legal-status Critical Current

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    • 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

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  • 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)
CA3091806A 2018-02-27 2019-02-27 Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor Pending CA3091806A1 (en)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201862635964P 2018-02-27 2018-02-27
US62/635,964 2018-02-27
US201862677471P 2018-05-29 2018-05-29
US201862667585P 2018-05-29 2018-05-29
US62/667,585 2018-05-29
US62/677,471 2018-05-29
US201862703670P 2018-07-26 2018-07-26
US62/703,670 2018-07-26
US201862722382P 2018-08-24 2018-08-24
US62/722,382 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 (1)

Publication Number Publication Date
CA3091806A1 true CA3091806A1 (en) 2019-09-06

Family

ID=67805569

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3091806A Pending CA3091806A1 (en) 2018-02-27 2019-02-27 Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor

Country Status (13)

Country Link
US (1) US20200407750A1 (https=)
EP (1) EP3758724A4 (https=)
JP (4) JP2021516046A (https=)
KR (1) KR20210010434A (https=)
CN (2) CN119842635A (https=)
AU (1) AU2019227726B2 (https=)
BR (1) BR112020017348A2 (https=)
CA (1) CA3091806A1 (https=)
CL (2) CL2020002200A1 (https=)
IL (1) IL276859B2 (https=)
MX (1) MX2020008933A (https=)
SG (1) SG11202008182TA (https=)
WO (1) WO2019168961A1 (https=)

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MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
BR112021006052A2 (pt) 2018-10-01 2021-09-08 Ultragenyx Pharmaceutical Inc. Terapia gênica para tratar acidemia propiônica
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
WO2021067598A1 (en) 2019-10-04 2021-04-08 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
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
WO2023172491A1 (en) 2022-03-07 2023-09-14 Ultragenyx Pharmaceutical Inc. Modified batch aav production systems and methods
JP2023514204A (ja) * 2020-02-14 2023-04-05 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 Ｃｄｋｌ５欠損障害を処置するための遺伝子治療
WO2021202532A1 (en) 2020-03-31 2021-10-07 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
KR20230003569A (ko) * 2020-04-27 2023-01-06 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Cdkl5 결핍 장애 (cdd)의 치료에 유용한 조성물
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
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
TW202237850A (zh) 2020-12-01 2022-10-01 賓州大學委員會 具有組織特異性靶向基序的新穎構成物及含有其之組成物
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)
CA3216004A1 (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
AU2022423978A1 (en) * 2021-12-28 2024-07-18 Chengdu Origen Biotechnology Co., Ltd. Modified aav capsid protein and use thereof
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
CN119317718A (zh) 2022-01-25 2025-01-14 宾夕法尼亚州大学信托人 用于改善心脏转导和肝脏去靶向的aav衣壳
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
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
US20260021205A1 (en) * 2022-07-15 2026-01-22 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
CN116350801A (zh) * 2022-11-22 2023-06-30 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
EP4634394A2 (en) 2022-12-17 2025-10-22 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
WO2024138129A2 (en) * 2022-12-23 2024-06-27 Spark Therapeutics, Inc. Adeno-associated virus formulations
KR20260021096A (ko) * 2023-05-03 2026-02-12 보이저 테라퓨틱스, 인크. Cdkl5 결핍과 관련된 장애의 치료를 위한 조성물 및 방법
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
TW202516019A (zh) 2023-06-29 2025-04-16 賓州大學委員會 具中樞神經系統靶向模體的突變aav及含有其之組成物
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
WO2026074494A1 (en) 2024-10-03 2026-04-09 Sarepta Therapeutics, Inc. Modified viral capsids and uses thereof

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ES2525067T3 (es) * 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
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WO2017100674A1 (en) * 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
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CA3033856A1 (en) * 2016-08-15 2018-02-22 Genzyme Corporation Methods for detecting aav
PT3589730T (pt) * 2017-02-28 2024-02-28 Univ Pennsylvania Vetor de vírus adenoassociado (aav) de clado f e suas utilizações

Also Published As

Publication number Publication date
AU2019227726A1 (en) 2020-09-10
EP3758724A4 (en) 2022-07-06
JP2026027394A (ja) 2026-02-18
MX2020008933A (es) 2021-01-15
CN112352050A (zh) 2021-02-09
JP2021516046A (ja) 2021-07-01
SG11202008182TA (en) 2020-09-29
CL2020002200A1 (es) 2021-01-29
EP3758724A1 (en) 2021-01-06
KR20210010434A (ko) 2021-01-27
IL276859B1 (en) 2025-08-01
CN119842635A (zh) 2025-04-18
IL276859A (en) 2020-10-29
CL2022003757A1 (es) 2023-05-19
AU2019227726B2 (en) 2025-09-04
WO2019168961A1 (en) 2019-09-06
JP2024041967A (ja) 2024-03-27
US20200407750A1 (en) 2020-12-31
IL276859B2 (en) 2025-12-01
JP2023159235A (ja) 2023-10-31
BR112020017348A2 (pt) 2020-12-29

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