PL3356390T3 - Sposoby i kompozycje dla wektorów wirusowych unikających przeciwciał - Google Patents

Sposoby i kompozycje dla wektorów wirusowych unikających przeciwciał

Info

Publication number
PL3356390T3
PL3356390T3 PL16852471T PL16852471T PL3356390T3 PL 3356390 T3 PL3356390 T3 PL 3356390T3 PL 16852471 T PL16852471 T PL 16852471T PL 16852471 T PL16852471 T PL 16852471T PL 3356390 T3 PL3356390 T3 PL 3356390T3
Authority
PL
Poland
Prior art keywords
antibody
compositions
methods
virus vectors
evading
Prior art date
Application number
PL16852471T
Other languages
English (en)
Polish (pl)
Inventor
Aravind Asokan
Mavis Agbandje-Mckenna
Long Ping Victor TSE
Brittney GURDA
Original Assignee
The University Of North Carolina At Chapel Hill
University Of Florida Research Foundation, Inc.
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 The University Of North Carolina At Chapel Hill, University Of Florida Research Foundation, Inc. filed Critical The University Of North Carolina At Chapel Hill
Publication of PL3356390T3 publication Critical patent/PL3356390T3/pl

Links

Classifications

    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/08—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from viruses
    • C07K16/081—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from viruses from DNA 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
    • A61K2039/507—Comprising a combination of two or more separate antibodies
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/30—Immunoglobulins specific features characterized by aspects of specificity or valency
    • C07K2317/33—Crossreactivity, e.g. for species or epitope, or lack of said crossreactivity
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/30—Immunoglobulins specific features characterized by aspects of specificity or valency
    • C07K2317/34—Identification of a linear epitope shorter than 20 amino acid residues or of a conformational epitope defined by amino acid residues
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/76—Antagonist effect on antigen, e.g. neutralization or inhibition of binding
    • 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
    • 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6027—Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
PL16852471T 2015-09-28 2016-09-28 Sposoby i kompozycje dla wektorów wirusowych unikających przeciwciał PL3356390T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562234016P 2015-09-28 2015-09-28
EP16852471.8A EP3356390B1 (en) 2015-09-28 2016-09-28 Methods and compositions for antibody-evading virus vectors
PCT/US2016/054143 WO2017058892A2 (en) 2015-09-28 2016-09-28 Methods and compositions for antibody-evading virus vectors

Publications (1)

Publication Number Publication Date
PL3356390T3 true PL3356390T3 (pl) 2021-07-05

Family

ID=58424549

Family Applications (1)

Application Number Title Priority Date Filing Date
PL16852471T PL3356390T3 (pl) 2015-09-28 2016-09-28 Sposoby i kompozycje dla wektorów wirusowych unikających przeciwciał

Country Status (14)

Country Link
US (4) US10745447B2 (enExample)
EP (2) EP3831842A1 (enExample)
JP (3) JP7064214B2 (enExample)
CN (2) CN114606267A (enExample)
AU (2) AU2016332821B2 (enExample)
BR (1) BR112018003665A2 (enExample)
CA (1) CA2996420A1 (enExample)
DK (1) DK3356390T3 (enExample)
ES (1) ES2865487T3 (enExample)
IL (4) IL257628B2 (enExample)
PL (1) PL3356390T3 (enExample)
PT (1) PT3356390T (enExample)
SG (1) SG10202107733QA (enExample)
WO (1) WO2017058892A2 (enExample)

Families Citing this family (91)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6985795B2 (ja) 2013-09-26 2021-12-22 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 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
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
CN107106689A (zh) 2014-11-05 2017-08-29 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
KR102584655B1 (ko) 2014-11-14 2023-10-06 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
MX2017006216A (es) 2014-11-14 2018-08-29 Voyager Therapeutics Inc Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela).
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
US12188037B2 (en) 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated Synthetic combinatorial AAV3 capsid library
US10983110B2 (en) 2015-12-02 2021-04-20 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
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
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
AU2017268382B2 (en) 2016-05-18 2023-09-28 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
RU2758488C2 (ru) 2016-05-18 2021-10-28 Вояджер Терапьютикс, Инк. Модулирующие полинуклеотиды
BR112019002904A2 (pt) * 2016-08-16 2019-06-25 The University Of North Carolina At Chapel Hill métodos e composições para transferência gênica direcionada
JP2019531787A (ja) 2016-08-30 2019-11-07 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 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム
US11192925B2 (en) 2016-10-19 2021-12-07 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
CA3114549A1 (en) 2017-02-21 2018-08-30 University Of Florida Research Foundation, Incorporated Modified aav capsid proteins and uses thereof
AU2018261790B2 (en) 2017-05-05 2024-10-03 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
WO2018204803A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CN111132626B (zh) 2017-07-17 2024-01-30 沃雅戈治疗公司 轨迹阵列引导系统
EP3662060A2 (en) 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
EP3687582A4 (en) 2017-09-29 2021-07-14 Voyager Therapeutics, Inc. RESCUE OF CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE OF FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION
EP4454654A3 (en) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
US11434502B2 (en) 2017-10-16 2022-09-06 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)
AU2018350992A1 (en) * 2017-10-18 2020-05-21 Regenxbio Inc. Fully-human post-translationally modified antibody therapeutics
CN112041442A (zh) * 2018-02-28 2020-12-04 北卡罗来纳大学查佩尔希尔分校 用于逃避抗体的病毒载体的方法和组合物
EP3765624A4 (en) * 2018-03-16 2022-05-25 Research Institute at Nationwide Children's Hospital Increasing tissue specific gene delivery by capsid modification
EP3773746A4 (en) * 2018-03-29 2022-01-19 University of Florida Research Foundation, Incorporated AAV6 VARIANTS
CN112218880A (zh) * 2018-03-29 2021-01-12 阿斯克肋匹奥生物制药公司 逃避中和的肝趋向性重组aav6载体
EP3773743A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
WO2019195449A1 (en) * 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
CN112543766A (zh) 2018-04-03 2021-03-23 斯特里迪比奥公司 抗体逃避性病毒载体
JP2021523702A (ja) * 2018-05-04 2021-09-09 オレゴン ヘルス アンド サイエンス ユニバーシティ ヒト抗ａａｖ２カプシドポリクローナル抗体エピトープ
MX2020012077A (es) 2018-05-15 2021-03-09 Voyager Therapeutics Inc Composiciones y metodos para el tratamiento de la enfermedad de parkinson.
TW202015742A (zh) 2018-05-15 2020-05-01 美商航海家醫療公司 投遞腺相關病毒（ａａｖ）之組成物和方法
WO2019241486A1 (en) 2018-06-13 2019-12-19 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
JP7142815B2 (ja) * 2018-06-21 2022-09-28 株式会社遺伝子治療研究所 ヒト肝臓への遺伝子導入のためのアデノ随伴ウイルスビリオン
WO2020023612A1 (en) 2018-07-24 2020-01-30 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
KR20210084459A (ko) * 2018-09-26 2021-07-07 캘리포니아 인스티튜트 오브 테크놀로지 표적화된 유전자 요법을 위한 아데노-연관 바이러스 조성물
SG11202103151RA (en) 2018-09-28 2021-04-29 Voyager Therapeutics Inc Frataxin expression constructs having engineered promoters and methods of use thereof
AU2019354995B2 (en) 2018-10-02 2024-12-12 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
EP3861113A1 (en) 2018-10-04 2021-08-11 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
EP3861107A1 (en) 2018-10-05 2021-08-11 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding aav production proteins
WO2020077165A1 (en) 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
JP2022505106A (ja) 2018-10-15 2022-01-14 ボイジャー セラピューティクス インコーポレイテッド バキュロウイルス／Ｓｆ９システムにおけるｒＡＡＶの大規模産生のための発現ベクター
CN113631225A (zh) 2019-01-18 2021-11-09 沃雅戈治疗公司 用于生产aav颗粒的方法和系统
SG11202108614QA (en) 2019-02-15 2021-09-29 Sangamo Therapeutics Inc Compositions and methods for producing recombinant aav
CN113727992A (zh) 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
US20220243225A1 (en) 2019-04-29 2022-08-04 Voyager Therapeutics, Inc. SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS
EP3966227A1 (en) 2019-05-07 2022-03-16 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
EP3999120A4 (en) * 2019-07-04 2024-02-21 Children's Medical Research Institute Methods and aav vectors for in vivo transduction
JP7371954B2 (ja) * 2019-07-12 2023-10-31 株式会社遺伝子治療研究所 ヒト肝臓への遺伝子導入のためのアデノ随伴ウイルスビリオン
CN110295151A (zh) * 2019-07-17 2019-10-01 博鑫仪器(天津)有限公司 一类腺依赖病毒病毒重组质粒、重组病毒及构建方法
WO2021030125A1 (en) 2019-08-09 2021-02-18 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
RU2751592C2 (ru) * 2019-08-22 2021-07-15 Общество С Ограниченной Ответственностью "Анабион" Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе
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
TW202128736A (zh) * 2019-10-17 2021-08-01 美商史崔德生物公司 用於治療ｃ型尼曼—匹克病之腺相關病毒載體
US20230340526A1 (en) * 2019-11-25 2023-10-26 University Of Florida Research Foundation, Incorporated Novel aav3b variants that target hepatocytes and evade the humoral immune response
JP2021097617A (ja) * 2019-12-20 2021-07-01 学校法人自治医科大学 オルニチントランスカルバミラーゼ欠損症の治療用アデノ随伴ウイルスビリオン
MX2022013963A (es) 2020-05-05 2023-02-22 Univ Duke Composiciones de virus adenoasociados compatibles entre especies y metodos de uso de las mismas.
US20230203102A1 (en) 2020-05-13 2023-06-29 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
US20230285596A1 (en) 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
CA3190309A1 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
US20230295656A1 (en) 2020-08-06 2023-09-21 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
TW202227632A (zh) 2020-08-19 2022-07-16 美商史崔德生物公司 用於治療雷特症候群之腺相關病毒載體
CN111925438B (zh) * 2020-08-28 2021-03-09 和元生物技术（上海）股份有限公司 能够与aav1-13结合的抗体
WO2022187473A2 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022229703A2 (en) * 2021-04-30 2022-11-03 Takeda Pharmaceutical Company, Ltd. New aav8 based immune escaping variants
AR126839A1 (es) * 2021-08-20 2023-11-22 Llc «Anabion» Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 9 (aav9), cápside y vector basado en esta
MX2024004217A (es) 2021-10-08 2024-06-26 Dyno Therapeutics Inc Variantes de cápside y métodos de uso de estas.
KR20240113624A (ko) 2021-11-02 2024-07-22 보이저 테라퓨틱스, 인크. Aav 캡시드 변이체 및 이의 용도
WO2023093905A1 (zh) 2021-11-29 2023-06-01 上海瑞宏迪医药有限公司 Aadc、gdnf多核苷酸及其用于治疗帕金森病
WO2023150687A1 (en) 2022-02-04 2023-08-10 Ginkgo Bioworks, Inc. Recombinant adeno-associated virus vectors, and methods of use thereof
WO2023155918A1 (zh) 2022-02-21 2023-08-24 上海瑞宏迪医药有限公司 Vegf结合分子及其医药用途
CN119095866A (zh) * 2022-02-22 2024-12-06 北卡罗来纳大学查佩尔希尔分校 嵌合神经向性aav衣壳
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
CN115850415B (zh) * 2022-09-14 2024-05-28 青岛农业大学 一种PbCaM7基因、蛋白及其应用
KR102855674B1 (ko) * 2022-10-06 2025-09-08 주식회사 글루진테라퓨틱스 폐 기관지 특이적 유전자 전달이 가능한 아데노부속바이러스 벡터
TW202502803A (zh) 2023-03-10 2025-01-16 美商戴諾治療公司 衣殼多肽及其使用方法
CN121039150A (zh) * 2023-03-13 2025-11-28 新加坡科技研究局 工程改造的腺相关病毒
AR133153A1 (es) 2023-07-03 2025-09-03 UCB Biopharma SRL Proteínas de cápside de aav manipuladas por ingeniería
TW202525832A (zh) 2023-08-31 2025-07-01 美商戴諾治療公司 衣殼多肽及其使用方法(二)
KR102844372B1 (ko) * 2023-12-06 2025-08-11 주식회사 글루진테라퓨틱스 뇌 성상교세포 특이적 유전자 전달이 가능한 아데노부속바이러스 변이체와 이의 응용
WO2025171227A1 (en) 2024-02-08 2025-08-14 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
CN120842371B (zh) * 2025-09-25 2025-11-21 北京溯本源和生物科技有限公司 一种可广谱识别aav多种抗原的单克隆抗体5f11及应用

Family Cites Families (194)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US1453547A (en) 1922-05-18 1923-05-01 Doyle Barrel-holding device
US1777296A (en) 1928-08-22 1930-10-07 Superior Locker Company Indicating device for locker doors
US4501729A (en) 1982-12-13 1985-02-26 Research Corporation Aerosolized amiloride treatment of retained pulmonary secretions
JPH01503675A (ja) 1986-09-08 1989-12-14 アプライド・バイオテクノロジー・インコーポレーテツド 中空ウイルスキヤプシドのワクチン
US4968603A (en) 1986-12-31 1990-11-06 The Regents Of The University Of California Determination of status in neoplastic disease
EP0442926A1 (en) 1988-11-10 1991-08-28 Imperial Cancer Research Technology Limited Polypeptides
US5916563A (en) 1988-11-14 1999-06-29 United States Of America Parvovirus protein presenting capsids
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
ES2026826A6 (es) 1991-03-26 1992-05-01 Ercros Sa Procedimiento para la produccion de una vacuna subunidad contra el parvovirus canino y otros virus relacionados.
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5869248A (en) 1994-03-07 1999-02-09 Yale University Targeted cleavage of RNA using ribonuclease P targeting and cleavage sequences
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5599706A (en) 1994-09-23 1997-02-04 Stinchcomb; Dan T. Ribozymes targeted to apo(a) mRNA
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
JP4002605B2 (ja) 1995-06-07 2007-11-07 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 筋組織のａａｖ形質導入
US6040183A (en) 1995-06-07 2000-03-21 University Of North Carloina At Chapel Hill Helper virus-free AAV production
US6083702A (en) 1995-12-15 2000-07-04 Intronn Holdings Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
EP0883344B2 (en) 1995-12-15 2010-06-09 VIRxSYS Corporation Therapeutic molecules generated by trans-splicing
EP0932694A2 (en) 1996-09-11 1999-08-04 THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6984517B1 (en) 1998-05-28 2006-01-10 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector and uses thereof
ES2313784T3 (es) 1998-05-28 2009-03-01 The Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services Vector aav5 y usos del mismo.
US6562958B1 (en) 1998-06-09 2003-05-13 Genome Therapeutics Corporation Nucleic acid and amino acid sequences relating to Acinetobacter baumannii for diagnostics and therapeutics
US20100293663A2 (en) 1998-06-16 2010-11-18 Thomas La Rosa Nucleic Acid Molecules and Other Molecules Associated with Plants and Uses Thereof for Plant Improvement
US8299321B2 (en) 1998-06-16 2012-10-30 Monsanto Technology Llc Nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement
DE19830522A1 (de) 1998-07-08 2000-01-13 Pritt Produktionsgesellschaft Vorrichtung zum Übertragen eines in Form eines Filmes auf ein Trägerband aufgebrachten Stoffes
NZ511171A (en) 1998-09-22 2004-02-27 Univ Florida Methods for large-scale production of recombinant AAV vectors
GB9822763D0 (en) 1998-10-20 1998-12-16 Univ Sheffield Immunoglobin variant
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
ATE362542T1 (de) 1998-11-05 2007-06-15 Univ Pennsylvania Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
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
US6822071B1 (en) 1998-11-12 2004-11-23 The Regents Of The University Of California Polypeptides from Chlamydia pneumoniae and their use in the diagnosis, prevention and treatment of disease
EP1887081A2 (en) 1999-02-25 2008-02-13 Ceres Incorporated DNA Sequences
EP1033405A3 (en) 1999-02-25 2001-08-01 Ceres Incorporated Sequence-determined DNA fragments and corresponding polypeptides encoded thereby
US20040031072A1 (en) 1999-05-06 2004-02-12 La Rosa Thomas J. Soy nucleic acid molecules and other molecules associated with transcription plants and uses thereof for plant improvement
US20080229439A1 (en) 1999-05-06 2008-09-18 La Rosa Thomas J Nucleic acid molecules and other molecules associated with transcription in plants and uses thereof for plant improvement
US20130326723A1 (en) 1999-05-06 2013-12-05 Thomas J. La Rosa Soy nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement
US20110214206A1 (en) 1999-05-06 2011-09-01 La Rosa Thomas J Nucleic acid molecules and other molecules associated with plants
US20110093981A9 (en) 1999-05-06 2011-04-21 La Rosa Thomas J Nucleic acid molecules and other molecules associated with transcription in plants and uses thereof for plant improvement
US20090087878A9 (en) 1999-05-06 2009-04-02 La Rosa Thomas J Nucleic acid molecules associated with plants
US6498244B1 (en) 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
US7314912B1 (en) 1999-06-21 2008-01-01 Medigene Aktiengesellschaft AAv scleroprotein, production and use thereof
DE19933288A1 (de) 1999-07-15 2001-01-18 Medigene Ag Strukturprotein von Adeno-assoziiertem Virus mit veränderter Antigenität, seine Herstellung und Verwendung
DE60039766D1 (de) 1999-08-09 2008-09-18 Targeted Genetics Corp Terologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen
US6468524B1 (en) 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US20110131679A2 (en) 2000-04-19 2011-06-02 Thomas La Rosa Rice Nucleic Acid Molecules and Other Molecules Associated with Plants and Uses Thereof for Plant Improvement
US20140130203A1 (en) 2000-04-19 2014-05-08 Thomas J. La Rosa Rice nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement
AU2001255524A1 (en) 2000-04-21 2001-11-07 Corixa Corporation Compositions and methods for the therapy and diagnosis of acne vulgaris
ES2256265T3 (es) 2000-06-01 2006-07-16 University Of North Carolina At Chapel Hill Vectores de parvovirus duplicados.
AU6814901A (en) 2000-06-01 2001-12-11 Univ North Carolina Methods and compounds for controlled release of recombinant parvovirus vectors
JP2002130156A (ja) 2000-10-20 2002-05-09 Anest Iwata Corp 多段式流体圧縮部を備えたスクロール流体機械
US7214786B2 (en) 2000-12-14 2007-05-08 Kovalic David K Nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement
AU2002248297A1 (en) 2001-01-05 2002-07-16 Children's Hospital, Inc. Aav2 vectors and methods
US7749492B2 (en) * 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
EP1402042A2 (en) 2001-06-22 2004-03-31 Syngenta Participations AG Abiotic stress responsive polynucleotides and polypeptides
WO2003000906A2 (en) 2001-06-22 2003-01-03 Syngenta Participations Ag Plant disease resistance genes
US6623729B2 (en) 2001-07-09 2003-09-23 Korea Advanced Institute Of Science And Technology Process for preparing sustained release micelle employing conjugate of anticancer drug and biodegradable polymer
WO2002010210A2 (en) 2001-08-28 2002-02-07 Bayer Cropscience Ag Polypeptides for identifying herbicidally active compounds
WO2003033515A1 (en) 2001-10-15 2003-04-24 Corixa Corporation Compositions and methods for the therapy and diagnosis of acne vulgaris
AU2002360355B2 (en) 2001-11-09 2005-07-07 Government Of The United States Of America, Department Of Health And Human Services Production of adeno-associated virus in insect cells
CA2915124C (en) * 2001-11-13 2018-08-14 The Trustees Of The University Of Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
ES2602352T3 (es) 2001-12-17 2017-02-20 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (VAA), vectores que las contienen y usos de las mismas
JP4769417B2 (ja) 2001-12-17 2011-09-07 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 アデノ随伴ウイルス（ａａｖ）血清型９の配列、それを含むベクターおよびその使用
AU2003223766A1 (en) 2002-04-30 2003-11-17 University Of North Carolina At Chapel Hill Secretion signal vectors
DK1496944T3 (da) 2002-05-01 2008-12-01 Univ Florida Forbedrede rAAV-ekspressionssystemer til genetisk modificering af specifikke sapsid-proteiner
ITRM20020253A1 (it) 2002-05-08 2003-11-10 Univ Roma Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche.
JP2005185101A (ja) 2002-05-30 2005-07-14 National Institute Of Agrobiological Sciences 植物の全長ｃＤＮＡおよびその利用
CN101014364B (zh) 2002-12-24 2012-01-18 里纳特神经系统学公司 抗ngf抗体及其使用方法
AU2004248136B2 (en) 2003-06-02 2011-09-15 University Of Massachusetts Methods and compositions for controlling efficacy of RNA silencing
PL3235827T3 (pl) 2003-06-19 2021-07-05 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowania
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
EP2298926A1 (en) 2003-09-30 2011-03-23 The Trustees of The University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
US20060107345A1 (en) 2003-09-30 2006-05-18 Nickolai Alexandrov Sequence-determined DNA fragments and corresponding polypeptides encoded thereby
EP3372614B1 (en) 2004-04-07 2022-06-08 Rinat Neuroscience Corp. Methods for treating bone cancer pain by administering a nerve growth factor antagonist
FR2874384B1 (fr) 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
JP4346526B2 (ja) 2004-08-31 2009-10-21 株式会社東芝 半導体集積回路装置
US7366250B2 (en) 2004-09-09 2008-04-29 Agere Systems Inc. Method and apparatus for improved efficiency in an extended multiple antenna communication system
WO2006029319A2 (en) 2004-09-09 2006-03-16 The General Hospital Corporation Modulating phosphatase activity in cardiac cells
CA2591544A1 (en) * 2004-12-15 2006-06-22 The University Of North Carolina At Chapel Hill Chimeric vectors
US20080213759A1 (en) 2005-01-05 2008-09-04 Shionogi & Co., Ltd. Novel Angiogenesis Inhibitor
US20140199313A1 (en) 2005-03-02 2014-07-17 Metanomics Gmbh Process for the Production of Fine Chemicals
AU2006219823A1 (en) 2005-03-02 2006-09-08 Metanomics Gmbh Process for the production of fine chemicals
CN101213203A (zh) 2005-04-29 2008-07-02 教堂山北卡罗莱纳州大学 在转录后水平调节核酸表达的方法和组合物
EP2478760A1 (en) 2005-05-10 2012-07-25 Monsanto Technology LLC Genes and uses for plant improvement
US20090191597A1 (en) 2006-01-20 2009-07-30 Asklepios Biopharmaceutical, Inc. Enhanced production of infectious parvovirus vectors in insect cells
US7867484B2 (en) * 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
EP1994043A4 (en) 2006-02-10 2009-05-20 Univ Cincinnati PHOSPHATASE INHIBITOR PROTEIN 1 AS A REGULATOR OF HEART FUNCTION
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
EP2018421B1 (en) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
US20090317417A1 (en) * 2006-04-28 2009-12-24 The Trustees Of The University Of Pennsylvania Modified AAV Vectors Having Reduced Capsid Immunogenicity and Use Thereof
US8512981B2 (en) * 2006-06-21 2013-08-20 Amsterdam Molecular Therapeutics B.V. Vectors with modified initiation codon for the translation of AAV-Rep78 useful for production of AAV
JP5575486B2 (ja) 2007-01-18 2014-08-20 ユニヴァーシティ オブ ミズーリー−コロンビア 筋鞘にｎＮＯＳを回復させる合成ミニ／ミクロジストロフィン遺伝子
US9611302B2 (en) * 2007-04-09 2017-04-04 University Of Florida Research Foundation, Inc. High-transduction-efficiency RAAV vectors, compositions, and methods of use
HUE030719T2 (en) * 2007-04-09 2017-05-29 Univ Florida RAAV vector formulations and methods comprising tyrosine-modified capsid proteins for their use
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
CN103695459A (zh) 2007-09-18 2014-04-02 巴斯夫植物科学有限公司 产量提高的植物
EP2250268A4 (en) 2008-02-20 2011-08-24 Ceres Inc IMPROVED NITROGEN USE OF EFFICIENCY PROPERTIES IN PLANT NUCLEOTIDE SEQUENCES AND CORRESPONDING POLYPEPTIDES
WO2009108274A2 (en) 2008-02-26 2009-09-03 The University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
MX2010012592A (es) 2008-05-20 2011-05-05 Eos Neuroscience Inc Vectores para la administracion de proteinas sensibles a la luz y metodos de uso de las mismas.
JP2010002479A (ja) 2008-06-18 2010-01-07 Crossfor:Kk メガネレンズ用装飾体およびメガネ用装飾体装着具
ES2634118T3 (es) * 2009-02-11 2017-09-26 The University Of North Carolina At Chapel Hill Vectores de virus modificados y métodos para fabricar y utilizar los mismos
WO2010114948A2 (en) 2009-04-02 2010-10-07 University Of Florida Research Foundation, Inc. An inducible system for highly efficient production of recombinant adeno-associated virus (raav) vectors
RS67077B1 (sr) 2009-05-02 2025-08-29 Genzyme Corp Genska terapija za neurodegenerativne poremećaje
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
EP2292781A1 (en) 2009-08-17 2011-03-09 Genethon Baculovirus-based production of biopharmaceuticals free of contaminating baculoviral virions
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
EP3514232A1 (en) 2010-04-23 2019-07-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
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
US9839696B2 (en) 2010-04-30 2017-12-12 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
CN107828820B (zh) 2010-10-27 2022-06-07 学校法人自治医科大学 用于向神经系统细胞导入基因的腺相关病毒粒子
EP4234571A3 (en) * 2011-02-10 2023-09-27 The University of North Carolina at Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
CA2827375C (en) 2011-02-14 2022-07-19 The Children's Hospital Of Philadelphia Improved aav8 vector with enhanced functional activity and methods of use thereof
CA3177736A1 (en) 2011-08-23 2013-02-28 Evogene Ltd. Isolated polynucleotides and polypeptides, and methods of using same for increasing plant yield and/or agricultural characteristics
FI124176B (en) 2011-12-01 2014-04-15 Helsingin Yliopisto Polypeptide, polynucleotide, expression cassette, vector, host cell, plant and uses
SI2839014T1 (sl) 2012-04-18 2021-05-31 The Children's Hospital Of Philadelphia Sestavek in postopki za zelo učinkovit prenos genov z uporabo variant kapside AAV-JA
US20140162319A2 (en) 2012-05-02 2014-06-12 Sangeetha Hareendran Nucleotide sequences, methods, kit and a recombinant cell thereof
EP2847337A4 (en) 2012-05-09 2016-04-27 Univ Oregon Health & Science ADENO ASSOCIATED VIRUS PLASMIDS AND VECTORS
US10294281B2 (en) * 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
EP2492347A1 (en) 2012-05-22 2012-08-29 Laboratorios Del. Dr. Esteve, S.A. Methods for the production of vectors
CN104704123A (zh) 2012-06-21 2015-06-10 肌肉学研究协会 基因治疗载体的广泛的基因递送
US10266845B2 (en) 2013-02-08 2019-04-23 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
US9567376B2 (en) 2013-02-08 2017-02-14 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
CA2904396A1 (en) 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav vectors
AU2014274457B2 (en) 2013-05-21 2019-10-24 University Of Florida Research Foundation, Inc. Capsid-modified, rAAV3 vector compositions and uses in gene therapy of human liver cancer
ES2897508T3 (es) 2013-05-31 2022-03-01 Univ California Variantes de virus adenoasociados y métodos de uso de las mismas
HUE047996T2 (hu) 2013-07-22 2020-05-28 Childrens Hospital Philadelphia AAV variáns és készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez
US9585971B2 (en) 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
JP6985795B2 (ja) 2013-09-26 2021-12-22 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 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
JP6552511B2 (ja) 2013-10-11 2019-07-31 マサチューセッツ アイ アンド イヤー インファーマリー 祖先ウイルス配列を予測する方法およびその使用
CN104592364B (zh) * 2013-10-30 2018-05-01 北京大学 定点突变和定点修饰的腺相关病毒、其制备方法及应用
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
WO2015127128A2 (en) 2014-02-19 2015-08-27 University Of Massachusetts Recombinant aavs having useful transcytosis properties
WO2015126972A1 (en) 2014-02-21 2015-08-27 University Of Florida Research Foundation, Inc. Methods and compositions for gene delivery to on bipolar cells
CA2941640A1 (en) 2014-03-04 2015-09-11 University Of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
WO2015164757A1 (en) 2014-04-25 2015-10-29 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
EP4600255A3 (en) 2014-05-02 2025-10-22 Genzyme Corporation Aav vectors for retinal and cns gene therapy
WO2015181823A1 (en) 2014-05-28 2015-12-03 Evogene Ltd. Isolated polynucleotides, polypeptides and methods of using same for increasing abiotic stress tolerance, biomass and yield of plants
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
HRP20221536T1 (hr) 2014-06-25 2023-02-17 Acuitas Therapeutics Inc. Novi lipidi i formulacije lipidnih nanočestica za isporuku nukleinskih kiselina
EP3800260A1 (en) 2014-09-24 2021-04-07 City of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
US10711270B2 (en) 2014-10-03 2020-07-14 University Of Massachusetts High efficiency library-identified AAV vectors
RU2020140209A (ru) 2014-10-21 2021-01-25 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
RU2727015C2 (ru) 2014-11-21 2020-07-17 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл Векторы aav, нацеленные на центральную нервную систему
WO2016115382A1 (en) 2015-01-14 2016-07-21 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
US20180030096A1 (en) * 2015-02-03 2018-02-01 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
WO2016134338A1 (en) 2015-02-19 2016-08-25 University Of Florida Research Foundation, Inc. Recombinant aav vectors for gene therapy of human hematopoietic disorders
KR20170137730A (ko) 2015-03-02 2017-12-13 애드베룸 바이오테크놀로지스, 인코포레이티드 망막 추상체에 폴리뉴클레오타이드의 유리체 내 전달을 위한 조성물 및 방법
EP3277819B1 (en) 2015-03-24 2021-03-03 The Regents of The University of California Adeno-associated virus variants and methods of use thereof
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
US20180104289A1 (en) 2015-04-08 2018-04-19 The United States Of America, As Represented By The Secretary, Department Of Health And Human Viral gene therapy as treatment for cholesterol storage disease or disorder
WO2016172008A1 (en) 2015-04-24 2016-10-27 University Of Massachusetts Modified aav constructions and uses thereof
WO2016179644A1 (en) 2015-05-08 2016-11-17 Children's Medical Research Institute Promoters for expression of heterologous genes
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
CA2985786A1 (en) 2015-05-12 2016-11-17 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav isolate and fusion protein comprising nerve growth factor signal peptide and parathyroid hormone
US20180216133A1 (en) 2015-07-17 2018-08-02 The Trustees Of The University Of Pennsylvania Compositions and methods for achieving high levels of transduction in human liver cells
US20180214576A1 (en) 2015-07-28 2018-08-02 University Of Massachusetts Transgenic expression of dnasei in vivo delivered by an adeno-associated virus vector
LT3872085T (lt) 2015-07-30 2023-05-10 Massachusetts Eye & Ear Infirmary Viruso prototipo sekos ir jų panaudojimas
WO2017066764A2 (en) 2015-10-16 2017-04-20 William Marsh Rice University Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses
US11426469B2 (en) 2015-10-22 2022-08-30 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
KR20180091863A (ko) 2015-12-01 2018-08-16 스파크 테라퓨틱스, 인코포레이티드 임상적 사용에 적합한 무혈청 현탁 세포 배양 시스템에서 재조합 아데노-관련 바이러스(aav) 벡터를 생성하기 위한 확장가능한 방법
WO2017096164A1 (en) 2015-12-02 2017-06-08 The Board Of Trustees Of The Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
US10406244B2 (en) 2015-12-02 2019-09-10 The Board Of Trustees Of The Leland Stanford Junior University AAV vectors with expanded packaging capacity
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
CA3012344A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
AU2017219865B2 (en) 2016-02-16 2023-04-13 The Board Of Trustees Of The Leland Stanford Junior University Novel recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies
MX2018009426A (es) 2016-02-22 2018-12-19 Univ North Carolina Chapel Hill Vector aav-idua para el tratamiento de ceguera asociada con mucopolisacaridosis i (mps i).
CN109661470A (zh) 2016-04-15 2019-04-19 宾夕法尼亚州大学信托人 新型aav8突变衣壳和含有其的组合物
WO2017192750A1 (en) 2016-05-04 2017-11-09 Oregon Health & Science University Recombinant adeno-associated viral vectors
CN109476707B (zh) 2016-05-13 2022-12-02 4D分子治疗有限公司 腺相关病毒变体衣壳和其使用方法
RU2758488C2 (ru) 2016-05-18 2021-10-28 Вояджер Терапьютикс, Инк. Модулирующие полинуклеотиды
DK3488007T3 (da) 2016-07-21 2024-02-19 Spark Therapeutics Inc Skalerbare fremgangsmåder med høj indvinding til fremstilling af højt udbytte rekombinant adeno-associeret viral (raav) vektor og rekombinante adeno-associeret virale (raav) vektorer produceret derved
IL263801B2 (en) 2016-07-26 2024-01-01 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
BR112019002904A2 (pt) 2016-08-16 2019-06-25 The University Of North Carolina At Chapel Hill métodos e composições para transferência gênica direcionada
US11078238B2 (en) 2016-09-29 2021-08-03 University Of Florida Research Foundation, Incorporated AAVrh.10 variants with host antibody escape capabilities and altered tissue targeting properties
US11192925B2 (en) 2016-10-19 2021-12-07 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
US20180182497A1 (en) 2016-12-22 2018-06-28 DxRx, Inc. Creating engagement with an inner circle social network in substance abuse treatment
JP7291397B2 (ja) 2017-02-15 2023-06-15 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 血管系を通過する遺伝子移入の方法および組成物
US11827906B2 (en) 2017-02-28 2023-11-28 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clade f vector and uses therefor
EP4303225B1 (en) 2017-03-15 2025-11-26 The University of North Carolina at Chapel Hill Polyploid adeno-associated virus vectors and methods of making and using the same
JP7330899B2 (ja) 2017-05-10 2023-08-22 マサチューセッツ アイ アンド イヤー インファーマリー ウイルスのアセンブリ活性化タンパク質（ａａｐ）依存性を改変する方法および組成物
WO2018237066A1 (en) 2017-06-20 2018-12-27 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services CODON OPTIMIZED HUMAN NPC1 GENES FOR THE TREATMENT OF C1-TYPE NIEMANN-PICK DISEASE AND ASSOCIATED STATES
WO2019006418A2 (en) 2017-06-30 2019-01-03 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
US12480138B2 (en) 2018-01-17 2025-11-25 MeiraGTx Gene Regulation Limited Modified rAAV capsid protein for gene therapy
MX2020008932A (es) 2018-02-27 2020-10-01 Univ Pennsylvania Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos.
EP3758724A4 (en) 2018-02-27 2022-07-06 The Trustees of The University of Pennsylvania NOVEL VECTORS OF ADENO-ASSOCIATED VIRUS (AAV), AAV VECTORS WITH REDUCED CAPSIID DEAMIDATION AND USES THEREOF
CN112041442A (zh) 2018-02-28 2020-12-04 北卡罗来纳大学查佩尔希尔分校 用于逃避抗体的病毒载体的方法和组合物
WO2019173538A1 (en) 2018-03-06 2019-09-12 University Of Florida Research Foundation, Incorporated Aav chimeras
US20210010028A1 (en) 2018-03-06 2021-01-14 Voyager Therapeutics, Inc. Insect cell manufactured partial self-complementary aav genomes
EP3765624A4 (en) 2018-03-16 2022-05-25 Research Institute at Nationwide Children's Hospital Increasing tissue specific gene delivery by capsid modification
WO2019195449A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
CN112543766A (zh) 2018-04-03 2021-03-23 斯特里迪比奥公司 抗体逃避性病毒载体
EP3773743A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues

Also Published As

Publication number Publication date
SG10202107733QA (en) 2021-09-29
IL257628A (en) 2018-04-30
CN108137655A (zh) 2018-06-08
JP7064214B2 (ja) 2022-05-10
JP2022068187A (ja) 2022-05-09
US20190048041A1 (en) 2019-02-14
AU2022203144A1 (en) 2022-06-02
ES2865487T3 (es) 2021-10-15
EP3356390A4 (en) 2019-08-28
JP2018528253A (ja) 2018-09-27
CN114606267A (zh) 2022-06-10
JP7390693B2 (ja) 2023-12-04
CN108137655B (zh) 2022-04-22
AU2016332821A1 (en) 2018-04-26
JP2024026088A (ja) 2024-02-28
CA2996420A1 (en) 2017-04-06
US11840555B2 (en) 2023-12-12
IL314171A (en) 2024-09-01
IL292830B2 (en) 2023-06-01
WO2017058892A3 (en) 2017-05-11
EP3356390B1 (en) 2021-01-20
IL292830A (en) 2022-07-01
EP3356390A2 (en) 2018-08-08
IL257628B (en) 2022-12-01
US20220089651A1 (en) 2022-03-24
US20240158450A1 (en) 2024-05-16
DK3356390T3 (da) 2021-04-12
IL299901B2 (en) 2024-12-01
US10745447B2 (en) 2020-08-18
IL257628B2 (en) 2023-04-01
US11208438B2 (en) 2021-12-28
AU2016332821B2 (en) 2022-02-17
IL299901A (en) 2023-03-01
WO2017058892A2 (en) 2017-04-06
US20200399321A1 (en) 2020-12-24
HK1257410A1 (en) 2019-10-18
IL299901B1 (en) 2024-08-01
BR112018003665A2 (en) 2018-09-25
EP3831842A1 (en) 2021-06-09
PT3356390T (pt) 2021-04-21

Similar Documents

Publication Publication Date Title
IL257628A (en) Methods and compositions for stealth virus antibody vectors
IL304398A (en) Preparations and methods for genome modification
IL259576A (en) Preparations and methods for immuno-oncology
GB2546350B (en) Compositions and methods
IL256295A (en) Anti-ntb-a antibodies and related compositions and methods
SG10201609929QA (en) Etching compositions and methods for using same
IL260124A (en) Compounds and methods for reducing tau expression
GB201718876D0 (en) Antithrombin-heparin compositions and methods
IL258292A (en) Methods and compositions for reducing metastases
IL246879B (en) Preparations of Epilimod and methods of using them
PT3250550T (pt) Composições e métodos para sensibilização viral
PL3220908T3 (pl) Kompozycje i sposoby leczenia endometriozy
GB2550632B (en) Compositions and uses and methods relating thereto
GB201715820D0 (en) Compositions and methods
GB201705626D0 (en) Compositions and methods
ZA201807665B (en) Methods and compositions
GB201512996D0 (en) Compositions and methods
PT3160263T (pt) Composições para prevenção de infeções
GB201514413D0 (en) Compositions and methods
GB201522814D0 (en) Compositions and uses thereof
ZA201807782B (en) Methods and compositions
IL256261B (en) Hide1 compositions and methods
GB201522554D0 (en) Methods and compositions
GB201518167D0 (en) Methods and compositions
GB201514415D0 (en) Compositions and methods