JP6985795B2 - 標的遺伝子治療のための合成コンビナトリアルaavカプシドライブラリー - Google Patents

標的遺伝子治療のための合成コンビナトリアルaavカプシドライブラリー Download PDF

Info

Publication number
JP6985795B2
JP6985795B2 JP2016517431A JP2016517431A JP6985795B2 JP 6985795 B2 JP6985795 B2 JP 6985795B2 JP 2016517431 A JP2016517431 A JP 2016517431A JP 2016517431 A JP2016517431 A JP 2016517431A JP 6985795 B2 JP6985795 B2 JP 6985795B2
Authority
JP
Japan
Prior art keywords
aav
sequence
capsid
library
gene
Prior art date
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.)
Active
Application number
JP2016517431A
Other languages
English (en)
Japanese (ja)
Other versions
JP2016533709A5 (enExample
JP2016533709A (ja
Inventor
マルシク,ダミアン
ゾロツキン,セルゲイ
アグバンジェ−マッケナ,メイビス
Original Assignee
ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド filed Critical ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド
Publication of JP2016533709A publication Critical patent/JP2016533709A/ja
Publication of JP2016533709A5 publication Critical patent/JP2016533709A5/ja
Application granted granted Critical
Publication of JP6985795B2 publication Critical patent/JP6985795B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145Special targeting system for viral vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)
JP2016517431A 2013-09-26 2014-09-26 標的遺伝子治療のための合成コンビナトリアルaavカプシドライブラリー Active JP6985795B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361883063P 2013-09-26 2013-09-26
US61/883,063 2013-09-26
PCT/US2014/057842 WO2015048534A1 (en) 2013-09-26 2014-09-26 Synthetic combinatorial aav capsid library for targeted gene therapy

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2019137247A Division JP7007337B2 (ja) 2013-09-26 2019-07-25 標的遺伝子治療のための合成コンビナトリアルaavカプシドライブラリー

Publications (3)

Publication Number Publication Date
JP2016533709A JP2016533709A (ja) 2016-11-04
JP2016533709A5 JP2016533709A5 (enExample) 2017-09-21
JP6985795B2 true JP6985795B2 (ja) 2021-12-22

Family

ID=52744537

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2016517431A Active JP6985795B2 (ja) 2013-09-26 2014-09-26 標的遺伝子治療のための合成コンビナトリアルaavカプシドライブラリー
JP2019137247A Active JP7007337B2 (ja) 2013-09-26 2019-07-25 標的遺伝子治療のための合成コンビナトリアルaavカプシドライブラリー

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2019137247A Active JP7007337B2 (ja) 2013-09-26 2019-07-25 標的遺伝子治療のための合成コンビナトリアルaavカプシドライブラリー

Country Status (5)

Country Link
US (2) US20160369298A1 (enExample)
EP (2) EP3049527A4 (enExample)
JP (2) JP6985795B2 (enExample)
AU (1) AU2014324717B2 (enExample)
WO (1) WO2015048534A1 (enExample)

Families Citing this family (86)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
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
TWI698240B (zh) 2012-05-15 2020-07-11 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
EP3003391B1 (en) 2013-05-31 2021-09-22 The Regents of The University of California Adeno-associated virus variants and methods of use thereof
WO2015048534A1 (en) 2013-09-26 2015-04-02 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
EP3800191B1 (en) 2014-03-17 2025-08-20 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
RU2716991C2 (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
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
JP6805174B2 (ja) 2015-05-12 2020-12-23 アメリカ合衆国 神経成長因子シグナルペプチド及び副甲状腺ホルモンを含むaav分離株及び融合タンパク質
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
US12188037B2 (en) 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated Synthetic combinatorial AAV3 capsid library
JP7406783B2 (ja) * 2015-12-14 2023-12-28 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質
GB2545763A (en) 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
KR102427379B1 (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
WO2017212019A1 (en) * 2016-06-09 2017-12-14 Centre National De La Recherche Scientifique (Cnrs) Raav with chemically modified capsid
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
WO2018044933A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
EP3585883A4 (en) * 2017-02-21 2021-04-14 University of Florida Research Foundation, Incorporated MODIFIED AAV CAPSID PROTEINS AND USES THEREOF
JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. ハンチントン病治療組成物および方法
JP2020518258A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. 筋萎縮性側索硬化症(als)治療組成物および方法
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
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
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
EP3808849A1 (en) 2017-08-03 2021-04-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
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
AU2018338728B2 (en) 2017-09-29 2025-01-02 Centre National De La Recherche Scientifique (Cnrs) Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
EP3697908A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
SG11202009854XA (en) * 2018-02-15 2020-11-27 Bjoerklund Tomas Modified viral capsids
AU2019247748A1 (en) 2018-04-03 2020-10-08 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
CA3100006A1 (en) * 2018-05-11 2019-11-14 Massachusetts Eye And Ear Infirmary Altering tissue tropism of adeno-associated viruses
KR20210019996A (ko) 2018-05-15 2021-02-23 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 조성물 및 방법
WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
US11821009B2 (en) 2018-05-15 2023-11-21 Cornell University Genetic modification of the AAV capsid resulting in altered tropism and enhanced vector delivery
WO2019241486A1 (en) 2018-06-13 2019-12-19 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
CN112770812A (zh) 2018-07-24 2021-05-07 沃雅戈治疗公司 产生基因治疗制剂的系统和方法
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
WO2020072849A1 (en) 2018-10-04 2020-04-09 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
WO2020072844A1 (en) 2018-10-05 2020-04-09 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding aav production proteins
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
CN113166781A (zh) 2018-10-15 2021-07-23 沃雅戈治疗公司 在杆状病毒/Sf9系统中大规模生产rAAV的表达载体
SG11202107645RA (en) 2019-01-18 2021-08-30 Voyager Therapeutics Inc Methods and systems for producing aav particles
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
JP2022530457A (ja) * 2019-04-26 2022-06-29 サンガモ セラピューティクス, インコーポレイテッド 遺伝子操作aav
EP3962536A1 (en) 2019-04-29 2022-03-09 Voyager Therapeutics, Inc. Systems and methods for producing baculoviral infected insect cells (biics) in bioreactors
US20240124889A1 (en) 2019-05-07 2024-04-18 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
EP4010465A1 (en) 2019-08-09 2022-06-15 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
EP4022070A1 (en) 2019-08-26 2022-07-06 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
CA3153972A1 (en) 2019-09-09 2021-03-18 Massachusetts Eye And Ear Infirmary Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav
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
US12611436B2 (en) 2019-10-17 2026-04-28 Sarepta Therapeutics, Inc. AAV transfer cassette
KR20220094216A (ko) 2019-11-08 2022-07-05 코브 테라퓨틱스 변형된 아데노-관련 바이러스 벡터 및 이의 중추신경계로의 전달
CN116390934A (zh) * 2020-05-26 2023-07-04 塑造治疗公司 功能性aav衣壳的高通量工程化
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
WO2022026410A2 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
BR112023001456A2 (pt) 2020-07-27 2023-04-11 Voyager Therapeutics Inc Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase
WO2022032153A1 (en) 2020-08-06 2022-02-10 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
AU2021328475A1 (en) 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
CN116802279A (zh) 2020-11-06 2023-09-22 科亚韦治疗公司 内酰胺修饰的腺相关病毒载体
WO2022187548A1 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022187473A2 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
TW202325850A (zh) 2021-11-29 2023-07-01 大陸商上海瑞宏迪醫藥有限公司 Aadc、gdnf多核苷酸及其用於治療帕金森病
AU2023220237A1 (en) 2022-02-21 2024-08-15 Shanghai Regenelead Therapies Co., Ltd Vegf-binding molecule and pharmaceutical use thereof
EP4514984A4 (en) * 2022-04-25 2026-04-22 Beijing Hanmoluojie Tech Co Ltd 3D MOLECULAR SURFACE FEATURES MAPPING OF AAV CAPSIDS
US20260085328A1 (en) 2022-09-08 2026-03-26 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
TW202508610A (zh) 2023-08-23 2025-03-01 大陸商上海瑞宏迪醫藥有限公司 醫藥組成物及其用途
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
CN119751599B (zh) * 2024-07-16 2025-08-19 广州派真生物技术有限公司 腺相关病毒突变体及其应用
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

Family Cites Families (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6759237B1 (en) * 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
AU780231B2 (en) 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
JP4353799B2 (ja) 2001-10-09 2009-10-28 ザ・ジョーンズ・ホプキンス・ユニバーシティ 転移に関連するホスファターゼ
US20030225260A1 (en) * 2002-04-30 2003-12-04 Snyder Richard O. Production of recombinant AAV virions
AU2003265855A1 (en) 2002-08-28 2004-03-19 University Of Florida Modified aav
SI1633772T1 (sl) 2003-06-19 2016-06-30 Genzyme Corporation AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
JP5136766B2 (ja) 2004-12-15 2013-02-06 ユニバーシティ オブ ノース カロライナ アット チャペル ヒル キメラベクター
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
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
ES2714007T3 (es) 2007-04-09 2019-05-24 Univ Florida Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso
WO2010011404A2 (en) 2008-05-20 2010-01-28 Eos Neuroscience, Inc. Vectors for delivery of light-sensitive proteins and methods of use
EP2524037B1 (en) 2010-01-12 2018-05-16 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
WO2012057363A1 (ja) 2010-10-27 2012-05-03 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
WO2012109570A1 (en) 2011-02-10 2012-08-16 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
NZ701693A (en) 2012-04-18 2017-02-24 The Children’S Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using aav capsid variants
WO2013170078A1 (en) * 2012-05-09 2013-11-14 Oregon Health & Science University Adeno associated virus plasmids and vectors
ES2926774T3 (es) 2013-03-15 2022-10-28 Univ North Carolina Chapel Hill Métodos y composiciones para vectores AAV de doble unión de glicano
EP3492597A3 (en) 2013-05-21 2019-08-28 University of Florida Research Foundation, Inc. Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer
WO2015048534A1 (en) 2013-09-26 2015-04-02 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
US20160272687A1 (en) 2013-11-08 2016-09-22 The Board Of Trustees Of The University Of Arkansas Adeno-associated virus "x" oncogene
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
EP3113787B1 (en) 2014-03-04 2019-12-04 University of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
WO2016133917A1 (en) 2015-02-16 2016-08-25 University Of Florida Research Foundation Raav vector compositions, methods for targeting vascular endothelial cells and use in treatment of type i diabetes
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
US10081659B2 (en) 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
WO2017004514A1 (en) 2015-07-02 2017-01-05 University Of Florida Research Foundation, Inc. Recombinant adeno-associated virus vectors to target medullary thyroid carcinoma
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
US12188037B2 (en) 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated Synthetic combinatorial AAV3 capsid library
EP3585883A4 (en) 2017-02-21 2021-04-14 University of Florida Research Foundation, Incorporated MODIFIED AAV CAPSID PROTEINS AND USES THEREOF

Also Published As

Publication number Publication date
CA2930549A1 (en) 2015-04-02
US11091777B2 (en) 2021-08-17
WO2015048534A1 (en) 2015-04-02
US20190249195A1 (en) 2019-08-15
JP2016533709A (ja) 2016-11-04
JP2019195338A (ja) 2019-11-14
JP7007337B2 (ja) 2022-01-24
EP3633041A2 (en) 2020-04-08
AU2014324717B2 (en) 2020-10-08
EP3049527A1 (en) 2016-08-03
EP3049527A4 (en) 2017-08-16
US20160369298A1 (en) 2016-12-22
EP3633041A3 (en) 2020-07-29

Similar Documents

Publication Publication Date Title
JP7007337B2 (ja) 標的遺伝子治療のための合成コンビナトリアルaavカプシドライブラリー
AU2014324717A1 (en) Synthetic combinatorial AAV capsid library for targeted gene therapy
JP7727029B2 (ja) アデノ随伴ウイルスの肝臓特異的向性
JP7416443B2 (ja) 高形質導入効率rAAVベクター、組成物、および使用方法
US12188037B2 (en) Synthetic combinatorial AAV3 capsid library
US12570699B2 (en) Capsid-modified, rAAV3 vector compositions and methods of use in gene therapy of human liver cancer
JP6921788B2 (ja) アデノ随伴ウイルスプラスミド及びベクター
AU2014274457B2 (en) Capsid-modified, rAAV3 vector compositions and uses in gene therapy of human liver cancer
US20230049066A1 (en) Novel aav3b variants that target human hepatocytes in the liver of humanized mice
CN111718418B (zh) 一种增强基因编辑的融合蛋白及其应用
CN111718420B (zh) 一种用于基因治疗的融合蛋白及其应用
CA3271456A1 (en) AAVS RECOMMENDATIONS WITH IMPROVED TROPISM AND SPECIFICITY
WO2013159036A1 (en) Adeno associated virus plasmids and vectors
US20260041792A1 (en) Novel aav3b capsid variants with enhanced hepatocyte tropism
CA2930549C (en) Synthetic combinatorial aav capsid library for targeted gene therapy
US20230340526A1 (en) Novel aav3b variants that target hepatocytes and evade the humoral immune response
US20240425879A1 (en) Compositions and methods for adeno-associated (aav) virus dnase expression
CN121203037A (zh) 基于整合素改造的特异性靶向心脏及肌肉的aav9优势突变体
WO2023133593A2 (en) Aav5 capsid variants
CN121752725A (zh) 经修饰的腺相关病毒载体及其在治疗中枢神经系统疾病中的用途
CN121974988A (en) Adeno-associated virus capsid protein VP1 mutant and application thereof

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20170810

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20170810

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20180813

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20181112

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20190325

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20190725

C60 Trial request (containing other claim documents, opposition documents)

Free format text: JAPANESE INTERMEDIATE CODE: C60

Effective date: 20190725

C11 Written invitation by the commissioner to file amendments

Free format text: JAPANESE INTERMEDIATE CODE: C11

Effective date: 20190806

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20190725

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20190913

C21 Notice of transfer of a case for reconsideration by examiners before appeal proceedings

Free format text: JAPANESE INTERMEDIATE CODE: C21

Effective date: 20190917

A912 Re-examination (zenchi) completed and case transferred to appeal board

Free format text: JAPANESE INTERMEDIATE CODE: A912

Effective date: 20191011

C211 Notice of termination of reconsideration by examiners before appeal proceedings

Free format text: JAPANESE INTERMEDIATE CODE: C211

Effective date: 20191016

C22 Notice of designation (change) of administrative judge

Free format text: JAPANESE INTERMEDIATE CODE: C22

Effective date: 20200408

C13 Notice of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: C13

Effective date: 20210107

C28A Non-patent document cited

Free format text: JAPANESE INTERMEDIATE CODE: C2838

Effective date: 20210107

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20210407

C22 Notice of designation (change) of administrative judge

Free format text: JAPANESE INTERMEDIATE CODE: C22

Effective date: 20210408

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210507

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20210507

C23 Notice of termination of proceedings

Free format text: JAPANESE INTERMEDIATE CODE: C23

Effective date: 20210916

C22 Notice of designation (change) of administrative judge

Free format text: JAPANESE INTERMEDIATE CODE: C22

Effective date: 20211007

C03 Trial/appeal decision taken

Free format text: JAPANESE INTERMEDIATE CODE: C03

Effective date: 20211028

C30A Notification sent

Free format text: JAPANESE INTERMEDIATE CODE: C3012

Effective date: 20211028

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20211126

R150 Certificate of patent or registration of utility model

Ref document number: 6985795

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250