ES2862912T3 - Composición y procedimientos para la transferencia genética de alta eficiencia utilizando variantes de cápside de VAA - Google Patents

Composición y procedimientos para la transferencia genética de alta eficiencia utilizando variantes de cápside de VAA Download PDF

Info

Publication number
ES2862912T3
ES2862912T3 ES13778449T ES13778449T ES2862912T3 ES 2862912 T3 ES2862912 T3 ES 2862912T3 ES 13778449 T ES13778449 T ES 13778449T ES 13778449 T ES13778449 T ES 13778449T ES 2862912 T3 ES2862912 T3 ES 2862912T3
Authority
ES
Spain
Prior art keywords
aav
capsid protein
vector
nucleic acid
lysine
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
ES13778449T
Other languages
English (en)
Spanish (es)
Inventor
Mustafa Yazicioglu
Federico Mingozzi
Xavier Anguela
Katherine High
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Childrens Hospital of Philadelphia CHOP
Original Assignee
Childrens Hospital of Philadelphia CHOP
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 Childrens Hospital of Philadelphia CHOP filed Critical Childrens Hospital of Philadelphia CHOP
Application granted granted Critical
Publication of ES2862912T3 publication Critical patent/ES2862912T3/es
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • 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
    • 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
    • C07K14/01DNA viruses
    • C07K14/015Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
    • 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
    • C12N15/864Parvoviral vectors, e.g. parvovirus, densovirus
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oncology (AREA)
  • Hematology (AREA)
  • Communicable Diseases (AREA)
  • Diabetes (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
ES13778449T 2012-04-18 2013-04-18 Composición y procedimientos para la transferencia genética de alta eficiencia utilizando variantes de cápside de VAA Active ES2862912T3 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261635273P 2012-04-18 2012-04-18
US201361794995P 2013-03-15 2013-03-15
PCT/US2013/037170 WO2013158879A1 (en) 2012-04-18 2013-04-18 Composition and methods for highly efficient gene transfer using aav capsid variants

Publications (1)

Publication Number Publication Date
ES2862912T3 true ES2862912T3 (es) 2021-10-08

Family

ID=49384070

Family Applications (1)

Application Number Title Priority Date Filing Date
ES13778449T Active ES2862912T3 (es) 2012-04-18 2013-04-18 Composición y procedimientos para la transferencia genética de alta eficiencia utilizando variantes de cápside de VAA

Country Status (26)

Country Link
US (2) US9909142B2 (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
EP (1) EP2839014B1 (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
JP (1) JP6342886B2 (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
KR (1) KR102063483B1 (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
CN (1) CN104487579B (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
AU (1) AU2013249202B2 (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
BR (2) BR122021020419B1 (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
CA (1) CA2870736C (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
CO (1) CO7200251A2 (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
DK (1) DK2839014T3 (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
ES (1) ES2862912T3 (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
HU (1) HUE054087T2 (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
IL (1) IL235102B (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
IN (1) IN2014DN08812A (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
MX (1) MX359518B (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
MY (1) MY172457A (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
NZ (1) NZ701693A (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
PE (1) PE20150163A1 (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
PH (1) PH12014502347B1 (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
PL (1) PL2839014T3 (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
PT (1) PT2839014T (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
RU (1) RU2683497C2 (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
SG (1) SG11201406776TA (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
SI (1) SI2839014T1 (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
WO (1) WO2013158879A1 (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)
ZA (1) ZA201407582B (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html)

Families Citing this family (128)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2359866B1 (en) * 2005-04-07 2013-07-17 The Trustees of The University of Pennsylvania Modified AAV rh48 capsids, compositions containing same and uses thereof
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
WO2008124724A1 (en) 2007-04-09 2008-10-16 University Of Florida Research Foundation, Inc. Raav vector compositions having tyrosine-modified capsid proteins and methods for use
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
MX2013012599A (es) 2011-04-29 2014-08-21 Selecta Biosciences Inc Liberación controlada de inmunosupresores a partir de nanoportadores sintéticos.
PE20150163A1 (es) 2012-04-18 2015-02-23 Philadelphia Children Hospital Composicion y metodos para una transferencia de genes altamente eficiente usando variantes capside aav
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
US12358954B2 (en) 2012-05-15 2025-07-15 University Of Florida Research Foundation, Incorporated Capsid-modified rAAV vector compositions and methods therefor
CA2907799A1 (en) * 2013-05-31 2014-12-04 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
MY175477A (en) * 2013-07-22 2020-06-30 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
US20160369298A1 (en) 2013-09-26 2016-12-22 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
WO2015134643A1 (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
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
CA2957737A1 (en) 2014-09-07 2016-03-10 Selecta Biosciences, Inc. Methods and compositions for attenuating gene expression modulating anti-viral transfer vector immune responses
EP3209311B1 (en) 2014-10-21 2024-03-06 University of Massachusetts Recombinant aav variants and uses thereof
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
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
KR20230169197A (ko) 2014-11-14 2023-12-15 보이저 테라퓨틱스, 인크. 근위축성 측삭 경화증(als)을 치료하는 조성물 및 방법
KR102584655B1 (ko) 2014-11-14 2023-10-06 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
EP3230441A4 (en) 2014-12-12 2018-10-03 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
WO2016126857A1 (en) 2015-02-03 2016-08-11 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
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
WO2016134337A1 (en) * 2015-02-19 2016-08-25 University Of Florida Research Foundation, Inc. Site-specific integrating recombinant aav vectors for gene therapy and improved production methods
JP6519080B2 (ja) * 2015-06-22 2019-05-29 ライオン株式会社 繊維製品用抗ウイルス組成物
EP3313991B1 (en) 2015-06-23 2024-07-17 The Children's Hospital of Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
CN114606267A (zh) 2015-09-28 2022-06-10 北卡罗来纳-查佩尔山大学 逃避抗体的病毒载体的方法和组合物
US12188037B2 (en) 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated Synthetic combinatorial AAV3 capsid library
AU2016344065A1 (en) 2015-10-28 2018-05-10 The Trustees Of The University Of Pennsylvania Intrathecal administration of adeno-associated-viral vectors for gene therapy
PE20231949A1 (es) 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
CA3006569A1 (en) 2015-12-02 2017-06-08 Voyager Therapeutics, Inc. Assays for the detection of aav neutralizing antibodies
WO2017181162A1 (en) * 2016-04-16 2017-10-19 University Of Florida Research Foundation, Incorporated Methods of enhancing biological potency of baculovirus system-produced recombinant adeno-associated virus
US11299751B2 (en) 2016-04-29 2022-04-12 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
BR112018072849B1 (pt) 2016-05-13 2020-12-22 4D Molecular Therapeutics Inc. proteína de capsídeo variante de vírus adeno-associado, ácido nucleico isolado, vírion de raav infeccioso e respectivo uso e composição farmacêutica
JP7066635B2 (ja) 2016-05-18 2022-05-13 ボイジャー セラピューティクス インコーポレイテッド 調節性ポリヌクレオチド
US11951121B2 (en) 2016-05-18 2024-04-09 Voyager Therapeutics, Inc. Compositions and methods for treating Huntington's disease
CR20190127A (es) * 2016-08-15 2019-06-25 Genzyme Corp Métodos para detectar aav
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
JP7577291B6 (ja) * 2016-10-13 2024-11-26 ユニバーシティ オブ マサチューセッツ Aavカプシド設計
WO2018129268A1 (en) 2017-01-07 2018-07-12 Selecta Biosciences, Inc. Patterned dosing of immunosuppressants coupled to synthetic nanocarriers
CN110462037A (zh) * 2017-01-30 2019-11-15 学校法人日本医科大学 腺相关病毒(aav)衣壳蛋白的突变体
CA3114549A1 (en) 2017-02-21 2018-08-30 University Of Florida Research Foundation, Incorporated Modified aav capsid proteins and uses thereof
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
CA3055936A1 (en) 2017-03-11 2018-09-20 Selecta Biosciences, Inc. Methods and compositions related to combined treatment with anti-inflammatories and synthetic nanocarriers comprising an immunosuppressant
WO2018170310A1 (en) 2017-03-15 2018-09-20 The University Of North Carolina At Chapel Hill Polyploid adeno-associated virus vectors and methods of making and using the same
US10550405B2 (en) 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
DK3601367T3 (da) 2017-03-30 2025-01-13 Univ Queensland Kimære molekyler og anvendelse deraf
WO2018204803A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
CA3061652A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
AU2018281306B2 (en) 2017-06-07 2025-04-17 Spark Therapeutics, Inc. Enhancing agents for improved cell transfection and/or rAAV vector production
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CN110461368A (zh) 2017-06-30 2019-11-15 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
AU2018302016A1 (en) 2017-07-17 2020-02-06 The Regents Of The University Of California Trajectory array guide system
US20210228738A1 (en) 2017-07-17 2021-07-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins
US12076369B2 (en) 2017-09-01 2024-09-03 The Frances Crick Institute Limited Immunoregulatory molecules and uses therefor
JP6825167B2 (ja) 2017-09-20 2021-02-03 4ディー モレキュラー セラピューティクス インコーポレイテッド アデノ随伴ウイルス変異キャプシドおよびその使用方法
US20200263199A1 (en) 2017-09-29 2020-08-20 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
AU2018347583A1 (en) 2017-10-13 2020-05-21 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector IgM responses
JP7502991B2 (ja) 2017-10-16 2024-06-19 ボイジャー セラピューティクス インコーポレイテッド 筋萎縮性側索硬化症(als)の治療
EP4454654A3 (en) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
JP7184894B2 (ja) 2017-11-27 2022-12-06 4ディー モレキュラー セラピューティクス インコーポレイテッド アデノ随伴ウイルス変異キャプシドおよび血管新生の阻害のための使用
US11905533B2 (en) 2017-12-19 2024-02-20 The University Of North Carolina At Chapel Hill Methods and compositions for delivery of viral vectors across the blood-brain barrier
WO2019152843A1 (en) 2018-02-01 2019-08-08 Homology Medicines, Inc. Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
EP3755795A4 (en) 2018-02-19 2022-07-20 Homology Medicines, Inc. Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof
US12178908B2 (en) 2018-02-26 2024-12-31 AnTolRx, Inc. Tolerogenic liposomes and methods of use thereof
IL276859B1 (en) * 2018-02-27 2025-08-01 Univ Pennsylvania Novel adeno-associated virus (AAV) vectors, AAV vectors with reduced capsid deamidation and uses therefor
CN111819286B (zh) * 2018-03-16 2024-05-24 国家儿童医院研究所 通过衣壳修饰增加组织特异性的基因递送
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
KR20210006358A (ko) 2018-04-03 2021-01-18 스트라이드바이오 인코포레이티드 안 조직을 표적으로 하기 위한 바이러스 벡터
AU2019249890B2 (en) 2018-04-05 2024-10-10 Association Institut De Myologie Hybrid recombinant adeno-associated virus serotype between AAV9 and AAVrh74 with reduced liver tropism
EP3784697A4 (en) * 2018-04-27 2022-07-06 Spark Therapeutics, Inc. Engineered aav capsids with increased tropism and aav vectors comprising the engineered capsids and methods of making and using same
CA3098198A1 (en) 2018-04-27 2019-10-31 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
BR112020023082A2 (pt) 2018-05-15 2021-02-09 Voyager Therapeutics, Inc. composições e métodos para o tratamento de doença de parkinson
KR20210021310A (ko) * 2018-05-16 2021-02-25 스파크 테라퓨틱스, 인코포레이티드 코돈-최적화된 산 알파-글루코시다제 발현 카세트 및 이를 사용하는 방법
KR20210034015A (ko) 2018-07-16 2021-03-29 셀렉타 바이오사이언시즈, 인크. Mma 구축물 및 벡터의 방법 및 조성물
CN112771070A (zh) 2018-07-16 2021-05-07 西莱克塔生物科技公司 Otc构建体和载体的方法和组合物
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
US11702673B2 (en) 2018-10-18 2023-07-18 University Of Florida Research Foundation, Incorporated Methods of enhancing biological potency of baculovirus system-produced recombinant adeno-associated virus
TW202027799A (zh) 2018-10-18 2020-08-01 美商英特利亞醫療公司 用於表現因子ix的組成物及方法
WO2020163743A1 (en) 2019-02-08 2020-08-13 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
JP7562544B2 (ja) 2019-02-08 2024-10-07 デシベル セラピューティクス インコーポレイテッド ミオシン15プロモーター及びその使用
CN113677801A (zh) 2019-02-25 2021-11-19 诺华股份有限公司 治疗bietti晶体营养不良的组合物和方法
EP3931335A2 (en) 2019-02-25 2022-01-05 Novartis AG Compositions and methods to treat bietti crystalline dystrophy
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
BR112021021566A2 (pt) 2019-04-28 2022-04-19 Selecta Biosciences Inc Métodos para tratamento de indivíduos com imunidade preexistente a vetores de transferência viral
WO2020243261A1 (en) 2019-05-28 2020-12-03 Selecta Biosciences, Inc. Methods and compositions for attenuated anti-viral transfer vector immune response
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
EP4038194A1 (en) 2019-10-04 2022-08-10 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
WO2021076925A1 (en) 2019-10-17 2021-04-22 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
IL294230A (en) 2020-01-03 2022-08-01 Sarepta Therapeutics Inc Methods for analyzing aav capsid proteins
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
CA3164714A1 (en) 2020-02-14 2021-08-19 Ultragenyx Pharmaceutical Inc. Gene therapy for treating cdkl5 deficiency disorder
EP4121544A1 (en) 2020-03-19 2023-01-25 Ultragenyx Pharmaceutical Inc. Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav
US20230129893A1 (en) 2020-03-31 2023-04-27 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
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
MX2023002016A (es) 2020-08-19 2023-06-26 Sarepta Therapeutics Inc Vectores de virus adenoasociados para el tratamiento del sindrome de rett.
US12312606B2 (en) 2020-10-15 2025-05-27 Hoffmann-La Roche Inc. Nucleic acid constructs for VA RNA transcription
US20220154223A1 (en) 2020-10-15 2022-05-19 Hoffmann-La Roche Inc. Nucleic acid constructs for simultaneous gene activation
EP4274571A1 (en) 2021-01-05 2023-11-15 Selecta Biosciences, Inc. Viral vector dosing protocols
US20220347317A1 (en) * 2021-04-23 2022-11-03 University Of Florida Research Foundation, Incorporated Aavrh74 vectors for gene therapy of muscular dystrophies
JP2024531138A (ja) 2021-08-11 2024-08-29 ウルトラジェニックス ファーマシューティカル インコーポレイテッド 筋ジストロフィーを処置するための組成物および方法
CN118139974A (zh) 2021-08-20 2024-06-04 拜奥卡德联合股份公司 获得修饰的腺相关病毒衣壳的方法
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
AR126840A1 (es) 2021-08-20 2023-11-22 Llc «Anabion» Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 5 (aav5), cápside y vector basado en esta
US20230086710A1 (en) * 2021-09-14 2023-03-23 California Institute Of Technology Aav capsid variants
US20230141563A1 (en) 2021-10-12 2023-05-11 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
EP4415760A1 (en) 2021-10-12 2024-08-21 Cartesian Therapeutics, Inc. Viral vector dosing protocols
CA3237037A1 (en) 2021-11-14 2023-05-19 Cartesian Therapeutics, Inc. Multiple dosing with viral vectors
CN116554278A (zh) * 2022-01-30 2023-08-08 上海玮美基因科技有限责任公司 变异型腺相关病毒及其在疾病治疗中的应用
EP4490278A1 (en) 2022-03-07 2025-01-15 Ultragenyx Pharmaceutical Inc. Modified batch aav production systems and methods
US20230357437A1 (en) 2022-03-09 2023-11-09 Selecta Biosciences, Inc. Immunosuppressants in combination with anti-igm agents and related dosing
CN118974274A (zh) 2022-04-13 2024-11-15 豪夫迈·罗氏有限公司 用于确定aav基因组的方法
CN119654334A (zh) * 2022-05-11 2025-03-18 费城儿童医院 用于靶向深层脑结构的腺相关病毒载体
CN119256220A (zh) 2022-05-23 2025-01-03 豪夫迈·罗氏有限公司 用于区分aav颗粒血清型和aav颗粒荷载状态的基于拉曼的方法
JP2025519228A (ja) 2022-06-03 2025-06-24 エフ. ホフマン-ラ ロシュ アーゲー 組換えaav粒子を生成するための方法
KR20250036743A (ko) 2022-07-14 2025-03-14 에프. 호프만-라 로슈 아게 재조합 aav 입자를 생산하는 방법
AU2023321906A1 (en) 2022-08-11 2025-03-27 Cartesian Therapeutics, Inc. Compositions and methods related to immunoglobulin proteases and fusions thereof
WO2024038002A1 (en) 2022-08-15 2024-02-22 F. Hoffmann-La Roche Ag Prevention or mitigation of adverse effects related to recombinant viral vectors
AU2023342254A1 (en) 2022-09-12 2025-01-23 F. Hoffmann-La Roche Ag Method for separating full and empty aav particles
AU2024216670A1 (en) 2023-02-07 2025-07-31 F. Hoffmann-La Roche Ag Method for the detection of anti-aav particle antibodies
WO2024194280A1 (en) 2023-03-21 2024-09-26 F. Hoffmann-La Roche Ag Method for the production of recombinant aav particle preparations
WO2024213596A1 (en) 2023-04-13 2024-10-17 F. Hoffmann-La Roche Ag Improved recombinant polyadenylation signal sequences and use thereof
WO2025057182A1 (en) * 2023-09-15 2025-03-20 Indian Institute Of Technology Kanpur Modified aav vectors for enhanced gene therapy in hemophilia b
WO2025082618A1 (en) 2023-10-20 2025-04-24 Fuse Vectors Aps In vitro parvovirus capsid manufacturing
WO2025168663A1 (en) 2024-02-09 2025-08-14 F. Hoffmann-La Roche Ag Method for producing recombinant adeno-associated viral particles

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
US4501729A (en) 1982-12-13 1985-02-26 Research Corporation Aerosolized amiloride treatment of retained pulmonary secretions
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
US6127525A (en) * 1995-02-21 2000-10-03 Cornell Research Foundation, Inc. Chimeric adenoviral coat protein and methods of using same
CA2264482A1 (en) 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
JP4888876B2 (ja) * 2003-06-13 2012-02-29 田平 武 アルツハイマー病の治療のための組換えアデノ随伴ウィルスベクター
EP2359866B1 (en) * 2005-04-07 2013-07-17 The Trustees of The University of Pennsylvania Modified AAV rh48 capsids, compositions containing same and uses thereof
CN101528916B (zh) 2006-04-28 2013-09-04 宾夕法尼亚大学托管会 规模可调的aav生产方法
SG10201908848RA (en) * 2010-03-29 2019-10-30 Univ Pennsylvania Pharmacologically induced transgene ablation system
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
PE20150163A1 (es) 2012-04-18 2015-02-23 Philadelphia Children Hospital Composicion y metodos para una transferencia de genes altamente eficiente usando variantes capside aav

Also Published As

Publication number Publication date
CN104487579B (zh) 2022-01-18
CA2870736C (en) 2021-11-02
JP2015514427A (ja) 2015-05-21
CA2870736A1 (en) 2013-10-24
AU2013249202A1 (en) 2014-11-06
PH12014502347A1 (en) 2014-12-22
MY172457A (en) 2019-11-26
PE20150163A1 (es) 2015-02-23
RU2683497C2 (ru) 2019-03-28
US9909142B2 (en) 2018-03-06
HUE054087T2 (hu) 2021-09-28
NZ701693A (en) 2017-02-24
CN104487579A (zh) 2015-04-01
US11279950B2 (en) 2022-03-22
IL235102B (en) 2019-08-29
PL2839014T3 (pl) 2021-08-23
AU2013249202B2 (en) 2018-08-09
EP2839014B1 (en) 2020-12-16
JP6342886B2 (ja) 2018-06-13
SI2839014T1 (sl) 2021-05-31
EP2839014A1 (en) 2015-02-25
PH12014502347B1 (en) 2014-12-22
MX359518B (es) 2018-10-01
KR20150004859A (ko) 2015-01-13
IN2014DN08812A (c_deeref_Disk_and_Scratch_Disk_Pools_and_Their_Defaults.html) 2015-05-22
BR122021020419B1 (pt) 2023-01-17
ZA201407582B (en) 2021-05-26
WO2013158879A1 (en) 2013-10-24
EP2839014A4 (en) 2015-12-30
US20180245098A1 (en) 2018-08-30
HK1207663A1 (en) 2016-02-05
MX2014012680A (es) 2015-05-11
PT2839014T (pt) 2021-03-19
BR112014025985A2 (pt) 2017-07-11
SG11201406776TA (en) 2015-03-30
CO7200251A2 (es) 2015-02-27
DK2839014T3 (da) 2021-03-08
US20150065562A1 (en) 2015-03-05
KR102063483B1 (ko) 2020-01-08
IL235102A0 (en) 2014-12-31
RU2014146159A (ru) 2016-06-10

Similar Documents

Publication Publication Date Title
ES2862912T3 (es) Composición y procedimientos para la transferencia genética de alta eficiencia utilizando variantes de cápside de VAA
EP2675484B1 (en) Improved aav8 vector with enhanced functional activity and methods of use thereof
EP3294894B1 (en) Aav isolate and fusion protein comprising nerve growth factor signal peptide and parathyroid hormone
JP6516713B2 (ja) アデノ随伴ウイルス(aav)の同源系統群(クレイド)、配列、それらを含有するベクターおよびそれらの用途
KR102516647B1 (ko) 고 형질도입 효율 raav 벡터, 조성물 및 사용 방법
AU6626500A (en) Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs
CN111065741A (zh) 脊髓性肌萎缩症的治疗
CN116685329B (zh) 核酸构建体及其用于治疗脊髓性肌肉萎缩症的用途
WO2021165544A1 (en) Viral vector particle based on aav2 for gene therapy
US20230321220A1 (en) Aav5-based vaccine against sars-cov-2
WO2022139631A1 (en) Aav5-based vaccine for induction of specific immunity and/or prevention of sars-cov-2-related infection
CA3088079A1 (en) Compositions and methods for treating retinal disorders
HK1207663B (en) Composition and methods for highly efficient gene transfer using aav capsid variants
EA050137B1 (ru) ВАКЦИНА НА ОСНОВЕ AAV5 ПРОТИВ SARS-CoV-2
WO2024238448A2 (en) Chimeric capsid recombinant adeno-associated virus with bbb penetration and microglia targeting (aav-bm)
WO2023022633A1 (ru) Выделенный модифицированный белок vp1 капсида aav5