AU2019227726B2 - 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

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Publication number
AU2019227726B2
AU2019227726B2 AU2019227726A AU2019227726A AU2019227726B2 AU 2019227726 B2 AU2019227726 B2 AU 2019227726B2 AU 2019227726 A AU2019227726 A AU 2019227726A AU 2019227726 A AU2019227726 A AU 2019227726A AU 2019227726 B2 AU2019227726 B2 AU 2019227726B2
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hsa
mir
deamidation
capsid
aav
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AU2019227726A
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AU2019227726A1 (en
Inventor
Joshua Joyner SIMS
April TEPE
Kevin Turner
James M. Wilson
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University of Pennsylvania Penn
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University of Pennsylvania Penn
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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)
AU2019227726A 2018-02-27 2019-02-27 Novel adeno-associated virus (AAV) vectors, aav vectors having reduced capsid deamidation and uses therefor Active AU2019227726B2 (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 (2)

Publication Number Publication Date
AU2019227726A1 AU2019227726A1 (en) 2020-09-10
AU2019227726B2 true AU2019227726B2 (en) 2025-09-04

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AU2019227726A Active AU2019227726B2 (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)

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US (1) US20200407750A1 (enExample)
EP (1) EP3758724A4 (enExample)
JP (3) JP2021516046A (enExample)
KR (1) KR20210010434A (enExample)
CN (2) CN119842635A (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)

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JP2021519581A (ja) 2018-04-03 2021-08-12 ストライドバイオ，インコーポレイテッド 抗体を回避するウイルスベクター
SG11202009452WA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
AU2019247191A1 (en) 2018-04-03 2020-10-15 Ginkgo Bioworks, Inc. Virus vectors for targeting ophthalmic tissues
WO2020072451A1 (en) 2018-10-01 2020-04-09 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
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
CA3157700A1 (en) 2019-10-17 2021-04-22 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
JP2022552892A (ja) * 2019-10-21 2022-12-20 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 改善された産生収率および肝臓向性を有するａａｖ３ｂ変異体
CA3165911A1 (en) * 2020-01-03 2021-07-08 Sarepta Therapeutics, Inc. Methods for analyzing aav capsid proteins
IL294781A (en) * 2020-02-14 2022-09-01 Ultragenyx Pharmaceutical Inc Gene therapy for treating cdkl5 deficiency disorder
JP2023520402A (ja) 2020-03-31 2023-05-17 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 プロピオン酸血症を処置するための遺伝子治療
BR112022021762A2 (pt) * 2020-04-27 2023-01-17 Univ Pennsylvania Composições úteis em tratamento de distúrbio de deficiência de cdkl5 (cdd)
US20230220069A1 (en) 2020-06-17 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of gene therapy patients
JP2023534037A (ja) 2020-07-13 2023-08-07 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 シャルコー・マリー・トゥース病の治療に有用な組成物
CA3189878A1 (en) 2020-08-19 2022-02-24 Colin O'BANION Adeno-associated virus vectors for treatment of rett syndrome
CA3195553A1 (en) * 2020-10-18 2022-04-21 Qiang Wang Improved adeno-associated virus (aav) vector and uses therefor
TW202233844A (zh) * 2020-10-29 2022-09-01 賓州大學委員會 Aav衣殼及含有其之組成物
JP2023551903A (ja) 2020-12-01 2023-12-13 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 組織特異的標的化モチーフを有する新規組成物及びそれを含有する組成物
BR112023015303A2 (pt) 2021-02-01 2023-11-14 Regenxbio Inc Método para tratar doença cln2 devido a deficiência de tpp1 em um sujeito
KR20230170022A (ko) 2021-04-12 2023-12-18 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 척수 및 연수 근위축증(sbma) 치료에 유용한 조성물
MX2023012513A (es) 2021-04-23 2023-12-15 Univ Pennsylvania Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen.
AU2022423978A1 (en) * 2021-12-28 2024-07-18 Chengdu Origen Biotechnology Co., Ltd. Modified aav capsid protein and use thereof
US20250177495A1 (en) 2022-01-10 2025-06-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
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
US20250144243A1 (en) 2022-01-25 2025-05-08 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
TW202342740A (zh) 2022-03-07 2023-11-01 美商奧崔基尼克斯製藥公司 改良的批量aav生產系統和方法
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
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
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
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
TW202432191A (zh) * 2022-12-23 2024-08-16 美商星火治療公司 腺相關病毒調配物
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
CN121127597A (zh) * 2023-05-03 2025-12-12 沃雅戈治疗公司 用于治疗与葡糖苷酰鞘氨醇酶β1缺陷相关的病症的组合物及方法
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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

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Also Published As

Publication number Publication date
US20200407750A1 (en) 2020-12-31
CL2020002200A1 (es) 2021-01-29
MX2020008933A (es) 2021-01-15
CN112352050A (zh) 2021-02-09
EP3758724A4 (en) 2022-07-06
BR112020017348A2 (pt) 2020-12-29
AU2019227726A1 (en) 2020-09-10
IL276859A (en) 2020-10-29
CL2022003757A1 (es) 2023-05-19
JP2023159235A (ja) 2023-10-31
IL276859B1 (en) 2025-08-01
JP2024041967A (ja) 2024-03-27
SG11202008182TA (en) 2020-09-29
KR20210010434A (ko) 2021-01-27
CN119842635A (zh) 2025-04-18
EP3758724A1 (en) 2021-01-06
IL276859B2 (en) 2025-12-01
WO2019168961A1 (en) 2019-09-06
JP2021516046A (ja) 2021-07-01
CA3091806A1 (en) 2019-09-06

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