JP2023528801A - バリアントキャプシドを有するアデノ随伴ウイルスビリオン及びその使用方法 - Google Patents

バリアントキャプシドを有するアデノ随伴ウイルスビリオン及びその使用方法 Download PDF

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JP2023528801A
JP2023528801A JP2022573159A JP2022573159A JP2023528801A JP 2023528801 A JP2023528801 A JP 2023528801A JP 2022573159 A JP2022573159 A JP 2022573159A JP 2022573159 A JP2022573159 A JP 2022573159A JP 2023528801 A JP2023528801 A JP 2023528801A
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amino acids
aav
cells
polypeptide
raav
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JP2023528801A5 (https=
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ジョン ジー． フラナリー
スコット エフ． ゲラー
カレン アイ． ゲーリン
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University of California San Diego UCSD
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University of California San Diego UCSD
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    • 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
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    • 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
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    • 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
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    • 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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    • 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
    • C12N2750/14145—Special targeting system for viral vectors

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  • Ophthalmology & Optometry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Epidemiology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2022573159A 2020-05-29 2021-05-27 バリアントキャプシドを有するアデノ随伴ウイルスビリオン及びその使用方法 Pending JP2023528801A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063032206P 2020-05-29 2020-05-29
US63/032,206 2020-05-29
US202163187154P 2021-05-11 2021-05-11
US63/187,154 2021-05-11
PCT/US2021/034624 WO2021243085A2 (en) 2020-05-29 2021-05-27 Adeno-associated virus virions with variant capsids and methods of use thereof

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JP2023528801A true JP2023528801A (ja) 2023-07-06
JP2023528801A5 JP2023528801A5 (https=) 2024-05-29

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US (1) US12031147B2 (https=)
EP (1) EP4157299A4 (https=)
JP (1) JP2023528801A (https=)
KR (1) KR20230043794A (https=)
CN (1) CN116249772A (https=)
AU (1) AU2021280317A1 (https=)
CA (1) CA3179597A1 (https=)
MX (1) MX2022015048A (https=)
WO (1) WO2021243085A2 (https=)

Families Citing this family (8)

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Publication number Priority date Publication date Assignee Title
WO2022150776A1 (en) 2021-01-11 2022-07-14 Vedere Bio Ii, Inc. OPTOGENETIC COMPOSITIONS COMPRISING A CBh PROMOTER SEQUENCE AND METHODS FOR USE
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
CN118791624B (zh) * 2023-04-11 2026-04-03 苏州星明优健生物技术有限公司 用于治疗视网膜神经退行性疾病的新型gpcr嵌合视蛋白
AU2024332181A1 (en) 2023-08-31 2026-02-12 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
TW202545971A (zh) 2024-02-08 2025-12-01 美商戴諾治療公司 殼體多肽及其使用方法
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
CN118955654B (zh) * 2024-10-15 2025-05-16 朗信启昇(苏州)生物制药有限公司 具有变异衣壳的腺相关病毒病毒体及其用途

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019518458A (ja) * 2016-05-13 2019-07-04 ４ディー モレキュラー セラピューティクス インコーポレイテッド アデノ関連ウイルス変異キャプシドおよびその使用方法

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8663624B2 (en) * 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
CN105755044A (zh) 2011-04-22 2016-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
US11021519B2 (en) * 2015-03-02 2021-06-01 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
JP6836999B2 (ja) 2015-03-24 2021-03-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアＴｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ アデノ随伴ウイルス変異体及びその使用方法
AU2017302013B2 (en) 2016-07-29 2022-05-26 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
CA3040179A1 (en) * 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
WO2018134168A1 (en) * 2017-01-17 2018-07-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods of expressing a polynucleotide of interest in the cone photoreceptors
CA3053154A1 (en) * 2017-06-30 2019-01-03 The Regents Of The University Of California Adeno-associated virus virions with variant capsids and methods of use thereof
JP2020534788A (ja) 2017-08-28 2020-12-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア アデノ随伴ウイルスカプシド変異体及びその使用方法
US20230049217A1 (en) * 2021-05-21 2023-02-16 Novartis Ag Compositions and methods for enhancing visual function

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019518458A (ja) * 2016-05-13 2019-07-04 ４ディー モレキュラー セラピューティクス インコーポレイテッド アデノ関連ウイルス変異キャプシドおよびその使用方法

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
"Expanding the Potentialof Adeno-Associated Virus for the Treatment of Intractable Inherited Retinal", PEER REVIEWED|THESIS/DISSERTATION, JPN6025019337, 2017, pages 1 - 82, ISSN: 0005726623 *

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WO2021243085A3 (en) 2022-01-06
EP4157299A2 (en) 2023-04-05
EP4157299A4 (en) 2024-10-02
US20210371879A1 (en) 2021-12-02
KR20230043794A (ko) 2023-03-31
US12031147B2 (en) 2024-07-09
AU2021280317A1 (en) 2023-01-05
MX2022015048A (es) 2023-03-08
WO2021243085A2 (en) 2021-12-02
CN116249772A (zh) 2023-06-09
CA3179597A1 (en) 2021-12-02

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