HK1202434A1 - Virus vectors for highly efficient transgene delivery - Google Patents

Virus vectors for highly efficient transgene delivery Download PDF

Info

Publication number
HK1202434A1
HK1202434A1 HK15103023.8A HK15103023A HK1202434A1 HK 1202434 A1 HK1202434 A1 HK 1202434A1 HK 15103023 A HK15103023 A HK 15103023A HK 1202434 A1 HK1202434 A1 HK 1202434A1
Authority
HK
Hong Kong
Prior art keywords
vector
highly efficient
viral
virus vectors
therapeutic
Prior art date
Application number
HK15103023.8A
Other languages
English (en)
Chinese (zh)
Inventor
Federico Mingozzi
Xavier ANGUELA
J. Fraser WRIGHT
Katherine A. High
Original Assignee
The Children's Hospital Of Philadelphia
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=48470322&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=HK1202434(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by The Children's Hospital Of Philadelphia filed Critical The Children's Hospital Of Philadelphia
Publication of HK1202434A1 publication Critical patent/HK1202434A1/xx

Links

Classifications

    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/644—Coagulation factor IXa (3.4.21.22)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2750/14123—Virus like particles [VLP]
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
HK15103023.8A 2011-11-22 2012-11-21 Virus vectors for highly efficient transgene delivery HK1202434A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201161562795P 2011-11-22 2011-11-22
US201161562795P 2011-11-22
US201261639025P 2012-04-26 2012-04-26
US201261639025P 2012-04-26
US201261682019P 2012-08-10 2012-08-10
US201261682019P 2012-08-10
PCT/US2012/066380 WO2013078400A1 (en) 2011-11-22 2012-11-21 Virus vectors for highly efficient transgene delivery

Publications (1)

Publication Number Publication Date
HK1202434A1 true HK1202434A1 (en) 2015-10-02

Family

ID=48470322

Family Applications (1)

Application Number Title Priority Date Filing Date
HK15103023.8A HK1202434A1 (en) 2011-11-22 2012-11-21 Virus vectors for highly efficient transgene delivery

Country Status (7)

Country Link
US (1) US10640785B2 (https=)
EP (2) EP3539568A1 (https=)
JP (1) JP6348064B2 (https=)
AU (1) AU2012340567B2 (https=)
CA (1) CA2856137A1 (https=)
HK (1) HK1202434A1 (https=)
WO (1) WO2013078400A1 (https=)

Families Citing this family (109)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100183558A1 (en) 2008-10-17 2010-07-22 Zhennan Lai Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
MX374963B (es) 2011-04-29 2025-03-06 Selecta Biosciences Inc Nanoportadores sintéticos tolerogénicos para reducir las respuestas de anticuerpos.
US12024568B2 (en) 2012-09-13 2024-07-02 Cornell University Treatment of brain cancers using central nervous system mediated gene transfer of monoclonal antibodies
WO2014093709A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
EP3031921B1 (en) 2012-12-12 2025-03-12 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
BR122020023215B1 (pt) 2013-05-03 2022-11-22 Selecta Biosciences, Inc Composição e kit de nanocarreadores sintéticos tolerogênicos para reduzir ou prevenir anafilaxia em resposta a um antígeno não alergênico
WO2014204724A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
CN105683379A (zh) * 2013-06-17 2016-06-15 布罗德研究所有限公司 用于对有丝分裂后细胞的疾病和障碍进行靶向和建模的系统、方法和组合物的递送、工程化和优化
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
MX374532B (es) 2013-06-17 2025-03-06 Broad Inst Inc Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos.
EP3725885A1 (en) 2013-06-17 2020-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
RU2716420C2 (ru) 2013-06-17 2020-03-11 Те Брод Инститьют Инк. Доставка и применение систем crispr-cas, векторов и композиций для целенаправленного воздействия и терапии в печени
HK1222673A1 (zh) * 2013-07-12 2017-07-07 The Children's Hospital Of Philadelphia Aav载体和用於抗aav(腺相关病毒)中和抗体的检测
IL297919A (en) * 2013-07-22 2023-01-01 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
WO2015035190A1 (en) * 2013-09-05 2015-03-12 Cornell University Gene therapy for alzheimer's and other neurodegenerative diseases and conditions
JP6793547B2 (ja) 2013-12-12 2020-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物
JP6712948B2 (ja) 2013-12-12 2020-06-24 ザ・ブロード・インスティテュート・インコーポレイテッド 組成物、及びヌクレオチドリピート障害におけるｃｒｉｓｐｒ−ｃａｓ系の使用方法
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
EP3080259B1 (en) 2013-12-12 2023-02-01 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
SG10201804975PA (en) 2013-12-12 2018-07-30 Broad Inst Inc Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for HBV and Viral Diseases and Disorders
JP7103750B2 (ja) 2013-12-12 2022-07-20 ザ・ブロード・インスティテュート・インコーポレイテッド ゲノム編集のためのＣＲＩＳＰＲ－Ｃａｓ系及び組成物の送達、使用及び治療適用
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
US10781459B2 (en) 2014-06-20 2020-09-22 University Of Florida Research Foundation, Incorporated Methods of packaging multiple adeno-associated virus vectors
JP7242180B2 (ja) 2014-09-07 2023-03-20 セレクタ バイオサイエンシーズ インコーポレーテッド 抗ウイルス導入ベクター免疫応答を減弱化するための方法および組成物
US10370432B2 (en) * 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
EP3204503A1 (en) * 2014-10-06 2017-08-16 Arthrogen BV Aav-based gene therapy
CN107073051B (zh) * 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
RU2716991C2 (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
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
EP3889260A1 (en) 2014-12-12 2021-10-06 The Broad Institute, Inc. Protected guide rnas (pgrnas)
CA2970370A1 (en) 2014-12-24 2016-06-30 Massachusetts Institute Of Technology Crispr having or associated with destabilization domains
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
CN107635584B (zh) 2015-04-09 2022-06-17 康奈尔大学 基因治疗以预防对过敏原的反应
US10214731B2 (en) * 2015-05-28 2019-02-26 Cornell University Adeno-associated virus mediated delivery of C1E1 as a therapy for angioedema
TWI906646B (zh) 2015-06-18 2025-12-01 美商博得學院股份有限公司 降低脫靶效應的ｃｒｉｓｐｒ酶突變
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
MX390848B (es) 2015-06-23 2025-03-21 Childrens Hospital Philadelphia Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.
EP3319631A4 (en) 2015-07-08 2019-01-09 American Gene Technologies International Inc. HIV PREMUNICATION AND IMMUNOTHERAPY
EP3384035A4 (en) 2015-12-02 2019-08-07 Voyager Therapeutics, Inc. ASSAYS FOR THE DETECTION OF AAV-NEUTRALIZING ANTIBODIES
CN116064678A (zh) 2016-01-15 2023-05-05 美国基因技术国际有限公司 用于活化γ-δT细胞的方法和组合物
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
WO2017139065A1 (en) 2016-02-08 2017-08-17 American Gene Technologies International Inc. Hiv vaccination and immunotherapy
WO2017156311A2 (en) 2016-03-09 2017-09-14 American Gene Technologies International Inc. Combination vectors and methods for treating cancer
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
WO2017197301A1 (en) * 2016-05-12 2017-11-16 Hanley Brian P Safe delivery of crispr and other gene therapies to large fractions of somatic cells in humans and animals
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
EP3468617A4 (en) * 2016-06-08 2020-01-22 American Gene Technologies International Inc. SYSTEM FOR THE NON-INTEGRAL VIRAL ISSUE AND PROCEDURE RELATING TO IT
AU2017292582C1 (en) 2016-07-08 2021-11-11 American Gene Technologies International Inc. HIV pre-immunization and immunotherapy
EP3487507A4 (en) 2016-07-21 2020-04-08 American Gene Technologies International, Inc. VIRAL VECTORS FOR THE TREATMENT OF PARKINSON'S DISEASE
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
AU2017341849B2 (en) 2016-10-13 2024-03-21 University Of Massachusetts AAV capsid designs
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
WO2018089527A1 (en) * 2016-11-09 2018-05-17 Intrexon Corporation Frataxin expression constructs
US11905312B2 (en) * 2017-02-15 2024-02-20 The University Of North Carolina At Chapel Hill Methods and compositions for gene transfer across the vasculature
ES3036482T3 (en) 2017-03-11 2025-09-19 Cartesian Therapeutics Inc Methods and compositions related to combined treatment with anti-inflammatories and synthetic nanocarriers comprising an immunosuppressant
US11820999B2 (en) 2017-04-03 2023-11-21 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
US10898585B2 (en) 2017-04-14 2021-01-26 Ptc Therapeutics .Inc. Gene therapy for AADC deficiency
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
CN110945127B (zh) 2017-05-22 2024-07-12 武田药品工业株式会社 用于血友病b的基因疗法的编码具有增加的表达的重组fix的病毒载体
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
JP2020524996A (ja) 2017-06-16 2020-08-27 アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド ヒトガンマデルタｔ細胞を介する腫瘍細胞傷害性の活性化のための方法および組成物
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
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
EP3697908A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
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
WO2019126222A1 (en) * 2017-12-18 2019-06-27 Spark Therapeutics, Inc. Adeno-associated virus (aav) vector lipid nanoparticle compositions and methods of use
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
US20190256867A1 (en) 2018-02-01 2019-08-22 Homology Medicines, Inc. Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
JP7244547B2 (ja) 2018-02-19 2023-03-22 ホモロジー・メディシンズ・インコーポレイテッド Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法
WO2019178288A2 (en) * 2018-03-13 2019-09-19 The Regents Of The University Of California Virus-like nanocapsid for oral delivery of insulin
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
AU2019247748A1 (en) 2018-04-03 2020-10-08 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
WO2019200016A1 (en) 2018-04-10 2019-10-17 President And Fellows Of Harvard College Aav vectors encoding clarin-1 or gjb2 and uses thereof
KR20210019996A (ko) 2018-05-15 2021-02-23 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 조성물 및 방법
US11466055B2 (en) * 2018-06-06 2022-10-11 The Regents Of The University Of California Virus-like nanoparticles for oral delivery
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
CA3119349A1 (en) * 2018-11-16 2020-05-22 Spark Therapeutics, Inc. In vitro assay for detecting enhancers and inhibitors of adeno associated virus (aav) vector transduction and/or detecting or quantitating anti-aav binding antibodies
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
KR20220019669A (ko) 2019-04-12 2022-02-17 프리라인 테라퓨틱스 리미티드 플라스미드 시스템
DK3722434T3 (da) 2019-04-12 2022-10-24 Freeline Therapeutics Ltd Plasmidsystem
AU2020287627A1 (en) 2019-06-04 2021-11-18 Selecta Biosciences, Inc. Formulations and doses of PEGylated uricase
US20200405824A1 (en) * 2019-06-26 2020-12-31 University Of Washington Use of ribonucleotide reductase alone or in combination with micro-dystrophin to treat duchenne muscular dystrophy striated muscle disease
KR20220053575A (ko) * 2019-08-01 2022-04-29 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 항체 결합 및 중화 항체 억제를 위한 조성물 및 방법
US11155835B2 (en) 2019-08-19 2021-10-26 The Catholic University Of America Prokaryotic-eukaryotic hybrid viral vector for delivery of large cargos of genes and proteins into human cells
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
IL293658A (en) * 2019-12-10 2022-08-01 Homology Medicines Inc Adeno-associated virus preparations and a method of using them
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
CA3179895A1 (en) * 2020-04-24 2021-10-28 Inserm (Institut National De La Sante Et De La Recherche Medicale) Methods for preventing induction of immune responses to the transduced cells expressing a transgene product after ocular gene therapy
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
AU2021328475A1 (en) 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
US12129287B2 (en) 2020-09-14 2024-10-29 President And Fellows Of Harvard College Recombinant adeno associated virus encoding clarin-1 and uses thereof
GB202016254D0 (en) 2020-10-13 2020-11-25 Freeline Therapeutics Ltd Plasmid system
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
EP4727963A2 (en) * 2023-06-14 2026-04-22 Fractyl Health, Inc. Gene therapies for metabolic disorders
EP4628576A1 (en) 2024-04-07 2025-10-08 Ascend Advanced Therapies GmbH Microrna molecules for use in methods for improving recombinant aav production
WO2025238067A1 (en) 2024-05-15 2025-11-20 Ascend Advanced Therapies Gmbh Novel vector system for aav production

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA1202962A (en) 1982-10-19 1986-04-08 David P. Clifford Substituted n-phenyl-n'-benzoyl ureas and their use as insecticides and acaricides
US4965188A (en) 1986-08-22 1990-10-23 Cetus Corporation Process for amplifying, detecting, and/or cloning nucleic acid sequences using a thermostable enzyme
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
ATE226449T1 (de) 1998-12-03 2002-11-15 Avigen Inc Hilfsstoffe zur verwendung in adeno-assoziiertes virus enthaltenden pharmazeutischen zubereitungen und damit hergestellte pharmazeutische zubereitungen
US20080220015A1 (en) 2001-08-03 2008-09-11 Nokad, S.A. Method of modulating neutralizing antibodies formation in mammals, and uses thereof in gene therapy, animal trangenesis and in functional inactivation of endogenous proteins
EP1496979A4 (en) 2002-03-22 2007-10-31 Univ Texas PROTAMINE-ADENOVIRAL VECTOR COMPLEXES AND APPLICATION METHODS
SI1496944T1 (sl) 2002-05-01 2009-02-28 Univ Florida Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinov
JP4566129B2 (ja) 2003-02-17 2010-10-20 扶桑薬品工業株式会社 新規ウイルスベクター
BRPI0511764B8 (pt) 2004-06-01 2021-05-25 Avigen Inc método de prevenção de agregação de vírions de vírus adeno-associado recombinante (raav) em uma preparação purificada de virions raav
CA2580189C (en) 2004-09-29 2013-05-21 Children's Memorial Hospital Sirna-mediated gene silencing of alpha synuclein
WO2008027084A2 (en) * 2006-04-28 2008-03-06 The Trustees Of The University Of Pennsylvania Modified aav vectors having reduced capsid immunogenicity and use thereof
MX367100B (es) * 2012-02-17 2019-08-05 The Children´S Hospital Of Philadelphia Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos.

Also Published As

Publication number Publication date
EP2782596A1 (en) 2014-10-01
CA2856137A1 (en) 2013-05-30
WO2013078400A1 (en) 2013-05-30
EP2782596A4 (en) 2015-07-29
JP2014534245A (ja) 2014-12-18
JP6348064B2 (ja) 2018-06-27
US10640785B2 (en) 2020-05-05
EP3539568A1 (en) 2019-09-18
US20140336245A1 (en) 2014-11-13
AU2012340567B2 (en) 2017-11-23
AU2012340567A1 (en) 2014-06-05

Similar Documents

Publication Publication Date Title
HK1202434A1 (en) Virus vectors for highly efficient transgene delivery
Crisci et al. Virus-like particles: the new frontier of vaccines for animal viral infections
Nayak et al. Progress and prospects: immune responses to viral vectors
Lasaro et al. New insights on adenovirus as vaccine vectors
PH12020550094A1 (en) Adeno-associated virus variant capsids and methods of use thereof
WO2013096866A3 (en) Processes using vlps with capsids resistant to hydrolases
WO2008122811A3 (en) Adenoviral vectors encoding a pathogen or tumour antigen
EP3795581A3 (en) New avv capsid proteins for nucleic acid transfer
WO2012106377A3 (en) Nucleic acid molecules encoding novel herpes antigens, vaccine comprising the same, and methods of use thereof
WO2015158749A3 (de) Viraler vektor für den zielgerichteten gentransfer in gehirn und rückenmark
WO2008145401A3 (en) Mutated parvovirus structural proteins as vaccines
EP4219695A3 (en) Adeno-associated virus variant capsids and use for inhibiting angiogenesis
EA201991911A1 (ru) Способы и композиции для переноса генов по сосудистой сети
WO2012142434A8 (en) Aav mediated ctla-4 gene transfer to treat sjogren's syndrome
PE20150163A1 (es) Composicion y metodos para una transferencia de genes altamente eficiente usando variantes capside aav
WO2010138263A3 (en) Novel aav 's and uses thereof
SG195181A1 (en) Simian adenovirus and hybrid adenoviral vectors
RU2016123086A (ru) Способы и композиции для лечения болезни хантингтона
BR112015008930A2 (pt) vacinas baseadas em partícula recombinante contra a infecção por citomegalovírus humano
WO2012159006A8 (en) Polypeptides and vectors for targeting her2/neu expressing cells and uses thereof
WO2013052832A3 (en) Simian (gorilla) adenovirus or adenoviral vectors and methods of use
CA2860388C (en) Parainfluenza virus 5 based vaccines
WO2013110818A3 (en) Immunogens for hiv vaccination
WO2013052859A3 (en) Adenoviral vector-based respiratory syncytial virus (rsv) vaccine
PH12013501116A1 (en) Raccoon poxvirus expressing rabies glycoproteins