HUE065885T2 - Adeno-asszociált vírus (AAV) F klád vektor és alkalmazásai - Google Patents

Adeno-asszociált vírus (AAV) F klád vektor és alkalmazásai

Info

Publication number
HUE065885T2
HUE065885T2 HUE18711202A HUE18711202A HUE065885T2 HU E065885 T2 HUE065885 T2 HU E065885T2 HU E18711202 A HUE18711202 A HU E18711202A HU E18711202 A HUE18711202 A HU E18711202A HU E065885 T2 HUE065885 T2 HU E065885T2
Authority
HU
Hungary
Prior art keywords
clade
aav
adeno
vector
associated virus
Prior art date
Application number
HUE18711202A
Other languages
English (en)
Hungarian (hu)
Inventor
James Wilson
Qiang Wang
April Giles
Kevin Turner
Original Assignee
Univ Pennsylvania
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 Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of HUE065885T2 publication Critical patent/HUE065885T2/hu

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
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • A61K35/761Adenovirus
    • 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
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/32Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against translation products of oncogenes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • 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
    • C12N2750/14121Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • 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)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Virology (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Mycology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Oncology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
HUE18711202A 2017-02-28 2018-02-27 Adeno-asszociált vírus (AAV) F klád vektor és alkalmazásai HUE065885T2 (hu)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762464748P 2017-02-28 2017-02-28
US201762591002P 2017-11-27 2017-11-27
US201862614002P 2018-01-05 2018-01-05

Publications (1)

Publication Number Publication Date
HUE065885T2 true HUE065885T2 (hu) 2024-06-28

Family

ID=61628475

Family Applications (1)

Application Number Title Priority Date Filing Date
HUE18711202A HUE065885T2 (hu) 2017-02-28 2018-02-27 Adeno-asszociált vírus (AAV) F klád vektor és alkalmazásai

Country Status (29)

Country Link
US (2) US11827906B2 (OSRAM)
EP (2) EP3589730B1 (OSRAM)
JP (3) JP7455579B2 (OSRAM)
KR (1) KR102775350B1 (OSRAM)
CN (2) CN118956781A (OSRAM)
AU (2) AU2018227440B2 (OSRAM)
BR (1) BR112019017839A2 (OSRAM)
CA (1) CA3053399A1 (OSRAM)
CL (1) CL2019002474A1 (OSRAM)
CO (1) CO2019009525A2 (OSRAM)
DK (1) DK3589730T5 (OSRAM)
ES (1) ES2971872T3 (OSRAM)
FI (1) FI3589730T3 (OSRAM)
HR (1) HRP20240257T1 (OSRAM)
HU (1) HUE065885T2 (OSRAM)
IL (1) IL268644B2 (OSRAM)
LT (1) LT3589730T (OSRAM)
MD (1) MD3589730T2 (OSRAM)
MX (2) MX2019010275A (OSRAM)
PE (1) PE20191362A1 (OSRAM)
PL (1) PL3589730T3 (OSRAM)
PT (1) PT3589730T (OSRAM)
RS (1) RS65241B1 (OSRAM)
SA (1) SA519402565B1 (OSRAM)
SG (4) SG10201912969XA (OSRAM)
SI (1) SI3589730T1 (OSRAM)
SM (1) SMT202400080T1 (OSRAM)
WO (1) WO2018160582A1 (OSRAM)
ZA (1) ZA201905407B (OSRAM)

Families Citing this family (78)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE317916T1 (de) 2001-11-13 2006-03-15 Univ Pennsylvania Verfahren zur identifizierung von adeno- assoziiertem virus (aav) sequenzen sowie kit zur ausführung der methode
ES2874298T3 (es) 2003-09-30 2021-11-04 Univ Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
WO2014170480A1 (en) 2013-04-18 2014-10-23 Fondazione Telethon Effective delivery of large genes by dual aav vectors
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
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
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
WO2018160582A1 (en) 2017-02-28 2018-09-07 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor
CA3061655A1 (en) 2017-05-11 2018-11-15 The Trustees Of The University Of Pennsylvania Gene therapy for neuronal ceroid lipofuscinoses
KR102823833B1 (ko) 2017-11-30 2025-06-23 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 뮤코다당류증 iiia형에 대한 유전자 요법
US11723989B2 (en) 2017-11-30 2023-08-15 The Trustees Of The University Of Pennsylvania Gene therapy for mucopolysaccharidosis IIIB
US12416016B2 (en) 2018-02-27 2025-09-16 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) vectors, AAV vectors having reduced capsid deamidation and uses therefor
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
SG11202009452WA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
JP2021519581A (ja) 2018-04-03 2021-08-12 ストライドバイオ,インコーポレイテッド 抗体を回避するウイルスベクター
AU2019247191A1 (en) 2018-04-03 2020-10-15 Ginkgo Bioworks, Inc. Virus vectors for targeting ophthalmic tissues
EP3860659A1 (en) * 2018-10-01 2021-08-11 The Trustees of the University of Pennsylvania Compositions useful for treating gm1 gangliosidosis
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
AU2020223149B2 (en) 2019-02-15 2025-05-22 Sangamo Therapeutics, Inc. Compositions and methods for producing recombinant AAV
SG11202108504YA (en) 2019-02-22 2021-09-29 Univ Pennsylvania Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration
EP3931337A1 (en) * 2019-02-26 2022-01-05 The Trustees of The University of Pennsylvania Compositions useful in treatment of krabbe disease
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
CA3133889A1 (en) * 2019-04-24 2020-10-29 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
SG11202111102SA (en) * 2019-04-29 2021-11-29 Univ Pennsylvania Novel aav capsids and compositions containing same
US12338451B2 (en) * 2019-05-03 2025-06-24 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
CA3157700A1 (en) 2019-10-17 2021-04-22 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
CA3161367A1 (en) * 2019-12-10 2021-06-17 Homology Medicines, Inc. Adeno-associated virus compositions and methods of use thereof
CN115867323A (zh) 2020-02-02 2023-03-28 宾夕法尼亚州大学信托人 可用于治疗gm1神经节苷脂症的组成物
CN113881770B (zh) * 2020-03-30 2022-04-12 中国医学科学院肿瘤医院 肺癌诊断的试剂盒和装置
BR112022021762A2 (pt) * 2020-04-27 2023-01-17 Univ Pennsylvania Composições úteis em tratamento de distúrbio de deficiência de cdkl5 (cdd)
BR112022021786A2 (pt) 2020-05-12 2023-01-17 Univ Pennsylvania Composições úteis em tratamento de doença de krabbe
BR112022022212A2 (pt) 2020-05-12 2022-12-13 Univ Pennsylvania Composições para redução específica de drg de expressão de transgene
US20230048732A1 (en) 2020-05-26 2023-02-16 Shape Therapeutics Inc. High throughput engineering of functional aav capsids
JP2023534037A (ja) 2020-07-13 2023-08-07 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 シャルコー・マリー・トゥース病の治療に有用な組成物
EP4189077A1 (en) 2020-07-30 2023-06-07 Shape Therapeutics Inc. Stable cell lines for inducible production of raav virions
CA3189878A1 (en) 2020-08-19 2022-02-24 Colin O'BANION Adeno-associated virus vectors for treatment of rett syndrome
EP4200429A1 (en) 2020-08-24 2023-06-28 The Trustees of The University of Pennsylvania Viral vectors encoding glp-1 receptor agonist fusions and uses thereof in treating metabolic diseases
EP4204014A1 (en) 2020-08-26 2023-07-05 The Trustees of The University of Pennsylvania Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration
KR20230118075A (ko) 2020-10-09 2023-08-10 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 파브리병 치료를 위한 조성물 및 방법
CA3195553A1 (en) 2020-10-18 2022-04-21 Qiang Wang Improved adeno-associated virus (aav) vector and uses therefor
EP4232096A4 (en) * 2020-10-22 2024-10-09 Scout Bio, Inc. Method of suppressing immune response to vector-delivered therapeutic protein
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
TW202233844A (zh) * 2020-10-29 2022-09-01 賓州大學委員會 Aav衣殼及含有其之組成物
EP4236974A2 (en) * 2020-10-29 2023-09-06 RegenxBio Inc. Vectorized factor xii antibodies and administration thereof
EP4244209A1 (en) 2020-11-11 2023-09-20 European Molecular Biology Laboratory Modified viral particles for gene therapy
AU2021392642A1 (en) 2020-12-01 2023-06-22 The Trustees Of The University Of Pennsylvania Compositions and uses thereof for treatment of angelman syndrome
JP2023551903A (ja) 2020-12-01 2023-12-13 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 組織特異的標的化モチーフを有する新規組成物及びそれを含有する組成物
BR112023015303A2 (pt) 2021-02-01 2023-11-14 Regenxbio Inc Método para tratar doença cln2 devido a deficiência de tpp1 em um sujeito
KR20230170022A (ko) 2021-04-12 2023-12-18 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 척수 및 연수 근위축증(sbma) 치료에 유용한 조성물
MX2023012513A (es) 2021-04-23 2023-12-15 Univ Pennsylvania Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen.
KR20240005950A (ko) 2021-05-12 2024-01-12 폰다지오네 텔레톤 이티에스 벡터 시스템
IL308787A (en) * 2021-06-08 2024-01-01 Univ Pennsylvania Recombinant adeno-associated viruses for Leish-Nihan disorders and their uses
US20240384298A1 (en) 2021-10-02 2024-11-21 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
JP2024543994A (ja) 2021-12-01 2024-11-26 シェイプ セラピューティクス インコーポレイテッド 硝子体内投与用の機能性aavカプシド
US20250205363A1 (en) 2021-12-01 2025-06-26 Shape Therapeutics Inc. Functional aav capsids for systemic administration
WO2023102517A1 (en) 2021-12-02 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
US20250177495A1 (en) 2022-01-10 2025-06-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
WO2023133574A1 (en) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful for treatment of c9orf72-mediated disorders
US20250144243A1 (en) 2022-01-25 2025-05-08 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
WO2023196892A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
CN119317645A (zh) 2022-04-06 2025-01-14 宾夕法尼亚州大学信托人 用于治疗her2阳性转移性乳腺癌及其他癌症的组合物和方法
US20250353883A1 (en) 2022-05-06 2025-11-20 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
US20250295809A1 (en) * 2022-05-13 2025-09-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
WO2024130067A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
WO2024130070A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
AU2024221161A1 (en) 2023-02-17 2025-08-28 Biogen Ma Inc. Rgd-containing peptides for delivering payloads
WO2024258961A1 (en) 2023-06-12 2024-12-19 The Trustees Of The University Of Pennsylvania Aav gene therapy for mucopolysaccharidosis iiib
WO2025007046A1 (en) 2023-06-29 2025-01-02 The Trustees Of The University Of Pennsylvania Mutant aav with central nervous system targeting motifs and compositions containing same
WO2025035143A1 (en) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
WO2025076091A1 (en) * 2023-10-03 2025-04-10 Brave Bio, Inc. Cd20 antibody aav vectors and methods of use thereof
WO2025090858A1 (en) 2023-10-27 2025-05-01 Biogen Ma Inc. Methods for identifying aav capsid variants with desired characteristics
WO2025096967A1 (en) 2023-11-02 2025-05-08 Biogen Ma Inc. Redirection of aav capsids for central nervous system targeting
WO2025102034A1 (en) 2023-11-10 2025-05-15 The Trustees Of The University Of Pennsylvania Gene therapy for barth syndrome
WO2025106661A1 (en) 2023-11-14 2025-05-22 The Trustees Of The University Of Pennsylvania Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof
WO2025129157A1 (en) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease
WO2025188625A1 (en) 2024-03-03 2025-09-12 Passage Bio, Inc. Recombinant adeno-associated virus for treatment of neurodegenerative disorders
WO2025207948A1 (en) 2024-03-27 2025-10-02 Biogen Ma Inc. Aav capsids for targeting human transferrin receptor
WO2025207932A1 (en) 2024-03-27 2025-10-02 Biogen Ma Inc. Aav capsids comprising anti-transferrin antibody domains

Family Cites Families (141)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4816567A (en) 1983-04-08 1989-03-28 Genentech, Inc. Recombinant immunoglobin preparations
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
WO1994018347A1 (en) 1993-02-05 1994-08-18 Laporte Group Australia Limited A slag defoaming composite
US5830462A (en) 1993-02-12 1998-11-03 President & Fellows Of Harvard College Regulated transcription of targeted genes and other biological events
US5834266A (en) 1993-02-12 1998-11-10 President & Fellows Of Harvard College Regulated apoptosis
US5869337A (en) 1993-02-12 1999-02-09 President And Fellows Of Harvard College Regulated transcription of targeted genes and other biological events
US6972193B1 (en) 1993-02-12 2005-12-06 Board Of Trustees Of Leland Stanford Junior University Regulated transcription of targeted genes and other biological events
HUT73101A (en) 1993-02-12 1996-06-28 Harvard College Method for regulating transcription of targeted genes and other biological events
US6063625A (en) 1993-02-12 2000-05-16 Board Of Trustees Of Leland S, Stanford, Jr. University Regulated transcription of targeted genes and other biological events
US20020173474A1 (en) 1993-02-12 2002-11-21 President And Fellows Of Harvard College Methods & materials involving dimerization-mediated regulation of biological events
US6150137A (en) 1994-05-27 2000-11-21 Ariad Pharmaceuticals, Inc. Immunosuppressant target proteins
US6476200B1 (en) 1994-06-27 2002-11-05 The Johns Hopkins University Mammalian proteins that bind to FKBP12 in a rapamycin-dependent fashion
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6150527A (en) 1994-08-18 2000-11-21 Ariad Pharmaceuticals, Inc. Synthetic multimerizing agents
US6133456A (en) 1994-08-18 2000-10-17 Ariad Gene Therapeutics, Inc. Synthetic multimerizing agents
JP4470226B2 (ja) 1994-08-18 2010-06-02 アリアド・ファーマシューティカルズ・インコーポレイテッド 新規な多量体化剤
US5786464C1 (en) 1994-09-19 2012-04-24 Gen Hospital Corp Overexpression of mammalian and viral proteins
US6326166B1 (en) 1995-12-29 2001-12-04 Massachusetts Institute Of Technology Chimeric DNA-binding proteins
CA2209183A1 (en) 1994-12-29 1996-07-11 Joel L. Pomerantz Chimeric dna-binding proteins
US6166197A (en) 1995-03-06 2000-12-26 Isis Pharmaceuticals, Inc. Oligomeric compounds having pyrimidine nucleotide (S) with 2'and 5 substitutions
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US6506379B1 (en) 1995-06-07 2003-01-14 Ariad Gene Therapeutics, Inc. Intramuscular delivery of recombinant AAV
CA2219080A1 (en) 1995-06-07 1996-12-27 Ariad Gene Therapeutics, Inc. Rapamycin-based regulation of biological events
US6187757B1 (en) 1995-06-07 2001-02-13 Ariad Pharmaceuticals, Inc. Regulation of biological events using novel compounds
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US5811524A (en) 1995-06-07 1998-09-22 Idec Pharmaceuticals Corporation Neutralizing high affinity human monoclonal antibodies specific to RSV F-protein and methods for their manufacture and therapeutic use thereof
CA2244363C (en) 1996-02-28 2006-11-14 Ariad Gene Therapeutics, Inc. Synthetic derivatives of rapamycin as multimerizing agents for chimeric proteins with immunophilin-derived domains
US6723531B2 (en) 1996-04-05 2004-04-20 The Salk Institute For Biological Studies Method for modulating expression of exogenous genes in mammalian systems, and products related thereto
AU735648B2 (en) 1996-07-12 2001-07-12 Ariad Pharmaceuticals, Inc. Materials and method for treating or preventing pathogenic fungal infection
WO1998044151A1 (en) 1997-04-01 1998-10-08 Glaxo Group Limited Method of nucleic acid amplification
CA2287478C (en) 1997-04-14 2007-06-19 Richard J. Samulski Methods for increasing the efficiency of recombinant aav product
US6015709A (en) 1997-08-26 2000-01-18 Ariad Pharmaceuticals, Inc. Transcriptional activators, and compositions and uses related thereto
US6117680A (en) 1997-08-26 2000-09-12 Ariad Gene Therapeutics, Inc. Compositions and methods for regulation of transcription
US6479653B1 (en) 1997-08-26 2002-11-12 Ariad Gene Therapeutics, Inc. Compositions and method for regulation of transcription
EP1003886A1 (en) 1997-08-27 2000-05-31 Ariad Gene Therapeutics, Inc. Chimeric transcriptional activators and compositions and uses related thereto
CA2318402A1 (en) 1998-01-15 1999-07-22 Ariad Gene Therapeutics, Inc. Regulation of biological events using multimeric chimeric proteins
US6984635B1 (en) 1998-02-13 2006-01-10 Board Of Trustees Of The Leland Stanford Jr. University Dimerizing agents, their production and use
JP2002503667A (ja) 1998-02-13 2002-02-05 プレジデント・アンド・フェローズ・オブ・ハーバード・カレッジ 新規な二量体化剤、その製造および使用
US6333318B1 (en) 1998-05-14 2001-12-25 The Salk Institute For Biological Studies Formulations useful for modulating expression of exogenous genes in mammalian systems, and products related thereto
US6146874A (en) 1998-05-27 2000-11-14 University Of Florida Method of preparing recombinant adeno-associated virus compositions
US6258603B1 (en) 1998-06-17 2001-07-10 Rohm And Haas Company Ligands for modulating the expression of exogenous genes via an ecdysone receptor complex
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US7109317B1 (en) 1998-11-06 2006-09-19 President And Fellows Of Harvard College FK506-based regulation of biological events
US6491907B1 (en) 1998-11-10 2002-12-10 The University Of North Carolina At Chapel Hill Recombinant parvovirus vectors and method of making
WO2001011034A2 (en) 1999-08-09 2001-02-15 Targeted Genetics Corporation 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
US7067526B1 (en) 1999-08-24 2006-06-27 Ariad Gene Therapeutics, Inc. 28-epirapalogs
AU783158B2 (en) 1999-08-24 2005-09-29 Ariad Pharmaceuticals, Inc. 28-epirapalogs
US6780639B1 (en) 1999-08-24 2004-08-24 Universite Libre De Bruxelles Antibiotic inducible/repressible genetic construct for gene therapy or gene immunization
JP3961737B2 (ja) 2000-02-29 2007-08-22 株式会社小糸製作所 車両用灯具およびその製造方法
US20040033600A1 (en) 2001-03-21 2004-02-19 Palli Subba Reddy Ecdysone receptor-based inducible gene expression system
JP5031967B2 (ja) 2000-03-22 2012-09-26 イントレキソン コーポレーション 新規エクジソン受容体ベースの誘導性遺伝子発現系
AU2001268149B2 (en) 2000-06-01 2005-08-18 University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
US8105825B2 (en) 2000-10-03 2012-01-31 Intrexon Corporation Multiple inducible gene regulation system
CA2438119C (en) 2001-02-20 2014-12-16 Rheogene Holdings, Inc. Chimeric retinoid x receptors and their use in a novel ecdysone receptor-based inducible gene expression system
AU2002248500B2 (en) 2001-02-20 2007-12-13 Intrexon Corporation Novel substitution mutant receptors and their use in a nuclear receptor-based inducible gene expression system
ES2385598T3 (es) 2001-02-20 2012-07-27 Intrexon Corporation Sistema novedoso de expresión génica inducible basado en el receptor de ecdisona/receptor X retinoide de invertebrado
ES2422303T3 (es) 2001-02-20 2013-09-10 Intrexon Corp Receptores mutantes por sustitución novedosos y su uso en un sistema de expresión génica inducible basado en receptores nucleares
WO2002095073A1 (en) 2001-05-18 2002-11-28 Wisconsin Alumni Research Foundation Method for the synthesis of dna sequences
ATE317916T1 (de) 2001-11-13 2006-03-15 Univ Pennsylvania Verfahren zur identifizierung von adeno- assoziiertem virus (aav) sequenzen sowie kit zur ausführung der methode
EP1453547B1 (en) 2001-12-17 2016-09-21 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor
US20070015238A1 (en) 2002-06-05 2007-01-18 Snyder Richard O Production of pseudotyped recombinant AAV virions
US8005620B2 (en) 2003-08-01 2011-08-23 Dna Twopointo Inc. Systems and methods for biopolymer engineering
EP1660970A4 (en) 2003-08-01 2007-02-14 Dna Twopointo Inc SYSTEMS AND METHODS FOR BIOPOLYMER TECHNOLOGY
ES2874298T3 (es) * 2003-09-30 2021-11-04 Univ Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
US20060246079A1 (en) 2003-11-14 2006-11-02 Morrow Phillip R Neutralizing human antibodies to anthrax toxin
US7935510B2 (en) 2004-04-30 2011-05-03 Intrexon Corporation Mutant receptors and their use in a nuclear receptor-based inducible gene expression system
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
ES2428218T3 (es) 2005-04-07 2013-11-06 The Trustees Of The University Of Pennsylvania Cápsides de AAV rh48 modificadas, composiciones que las contienen y usos de las mismas
EP1777906A1 (en) 2005-06-09 2007-04-25 Matsushita Electric Industrial Co., Ltd. Amplitude error compensating apparatus and orthogonality error compensating apparatus
DK1910395T3 (da) 2005-06-23 2013-02-04 Isis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulation af SMN2-splejsning
US8877187B2 (en) 2005-07-25 2014-11-04 Avianax, Llc Therapeutic antibodies for treatment and prophylaxis of transmittable viral diseases
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
EP2018421B1 (en) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
CA2654292C (en) 2006-06-07 2022-01-11 Genzyme Corporation Gene therapy for motor neuron disorders
EP2126087A4 (en) 2007-02-20 2010-10-27 Anaptysbio Inc METHODS FOR PRODUCING PHARMACOTHEQUES, AND USES THEREOF
EP2125886A2 (en) 2007-03-13 2009-12-02 Humabs LLC Antibodies against h5n1 strains of influenza a virus
CA2684534C (en) 2007-04-17 2017-04-04 National Research Council Of Canada Constructs for enhancement of gene expression in muscle
WO2008156763A2 (en) 2007-06-15 2008-12-24 The Board Of Trustees Of The Leland Stanford Junior University Human neutralizing monoclonal antibodies to h5n1 influenza a virus
US7863425B2 (en) 2007-09-26 2011-01-04 Cornell University Compositions and methods for inhibiting Yersinia pestis infection
EP2220116B1 (en) 2007-11-12 2012-09-05 Theraclone Sciences, Inc. Compositions and methods for the therapy and diagnosis of influenza
WO2009079259A2 (en) 2007-12-06 2009-06-25 Dana-Farber Cancer Institute, Inc. Antibodies against influenza virus and methods of use thereof
ITTO20080204A1 (it) 2008-03-17 2009-09-18 Pomona Biotechnologies Llc Anticorpi monoclonali atti a reagire con una pluralita di sottotipi del virus influenzale a
AU2009228058A1 (en) 2008-03-28 2009-10-01 Sea Lane Biotechnologies, Llc Neutralizing molecules to viral antigens
ITTO20080398A1 (it) 2008-05-27 2009-11-28 Pomona Biotechnologies Llc Anticorpi monoclonali aventi proprieta' di cross-neutralizzazione omosubtipica per virus influenzali di tipo a sottotipo h1
EP2296700A2 (en) 2008-06-03 2011-03-23 Vaxin, Inc. Intranasal administration of receptor-binding ligands or genes encoding such ligands as a therapeutic regimen for mitigating infections caused by respiratory pathogens
US8871207B2 (en) 2008-07-25 2014-10-28 Humabs, LLC Neutralizing anti-influenza A virus antibodies and uses thereof
KR20110049802A (ko) 2008-07-25 2011-05-12 인스티튜트 포 리서치 인 바이오메드슨 중화 항-인플루엔자 a 바이러스 항체 및 이의 용도
US8969353B2 (en) 2008-11-07 2015-03-03 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
US8211631B2 (en) 2008-12-18 2012-07-03 Wisconsin Alumni Research Foundation In vitro model of spinal muscular atrophy
WO2010071832A1 (en) 2008-12-19 2010-06-24 Nationwide Children's Hospital Delivery of polynucleotides across the blood brain barrier using recombinant aav9
US8470327B2 (en) 2009-05-11 2013-06-25 Crucell Holland B.V. Human binding molecules capable of neutralizing influenza virus H3N2 and uses thereof
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
IT1395961B1 (it) 2009-06-01 2012-11-02 Pomona Biotechnologies Llc Anticorpi monoclonali come medicamento per il trattamento terapeutico e/o profilattico delle infezioni da virus influenzale a (h1n1) di origine suina (s-oiv)
SI3305302T1 (sl) 2009-06-17 2018-12-31 Biogen Ma Inc. Sestave in metode za modulacijo združevanja smn2 pri subjektu
RU2560420C2 (ru) 2009-06-25 2015-08-20 МЕДИММЬЮН, ЭлЭлСи Варианты гемагглютининов вируса свиного гриппа
US8598331B2 (en) 2009-09-28 2013-12-03 The University Of British Columbia CLDN5 mini-promoters
US20130023033A1 (en) 2010-03-29 2013-01-24 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
EP2678433B1 (en) 2011-02-22 2017-05-03 California Institute of Technology Delivery of proteins using adeno-associated virus (aav) vectors
WO2012145572A1 (en) 2011-04-20 2012-10-26 The Trustees Of The University Of Pennsylvania Regimens and compositions for aav-mediated passive immunization of airborne pathogens
WO2012145759A2 (en) 2011-04-21 2012-10-26 Arizona Board Of Regents, A Body Corporate Of The State Of Arizona Acting For And On Behalf Of Arizona State University Methods of protein production and compositions thereof
JP6184945B2 (ja) 2011-06-08 2017-08-23 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド mRNA送達のための脂質ナノ粒子組成物および方法
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
US8961978B2 (en) 2011-07-14 2015-02-24 Crucell Holland B.V. Human binding molecules capable of neutralizing influenza A viruses of phylogenetic group 1 and phylogenetic group 2 and influenza B viruses
WO2013114885A1 (en) 2012-01-31 2013-08-08 Osaka University Human monoclonal antibodies broadly protective against influenza b virus and methods of using the same
ES2664625T3 (es) 2012-03-08 2018-04-20 Janssen Vaccines & Prevention B.V. Moléculas de unión humanas que pueden unirse a los virus de la influenza b y neutralizarlos, y sus usos
WO2013155222A2 (en) 2012-04-10 2013-10-17 The Regents Of The University Of California Brain-specific enhancers for cell-based therapy
DK2858677T3 (da) 2012-06-08 2020-08-31 Ethris Gmbh Pulmonær levering af messenger rna
US20150182637A1 (en) 2012-06-21 2015-07-02 Association Institut De Myologie Widespread gene delivery of gene therapy vectors
CA2880649C (en) 2012-08-01 2023-03-14 Elizabeth MCNALLY Mitigating tissue damage and fibrosis via latent transforming growth factor beta binding protein (ltbp4)
WO2015012924A2 (en) 2013-04-29 2015-01-29 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof
KR102116378B1 (ko) 2013-05-01 2020-06-01 젠자임 코포레이션 척수성 근위축증을 치료하기 위한 조성물 및 방법
AU2015218905A1 (en) 2014-02-19 2016-08-04 Emergent Biosolutions Canada Inc. Ebola monoclonal antibodies
JP6741590B2 (ja) 2014-04-25 2020-08-19 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 コレステロールレベルを低下させるためのldlr変異体および組成物中でのそれらの使用
US10780182B2 (en) 2014-04-25 2020-09-22 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
US10746742B2 (en) * 2014-04-25 2020-08-18 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
JP6974943B2 (ja) 2014-05-13 2021-12-01 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 2種の抗体構築物を発現するaavを含む組成物およびこの使用
PL3198018T3 (pl) * 2014-09-24 2021-07-19 City Of Hope Warianty wektora wirusa związanego z adenowirusami dla wysokiej skuteczności edycji genomu i jego sposoby
CN106999566B (zh) 2014-10-03 2022-01-28 麻省理工学院 结合埃博拉病毒糖蛋白的抗体及其用途
CA2975655C (en) 2015-02-05 2023-09-19 Janssen Vaccines & Prevention B.V. Binding molecules directed against influenza hemagglutinin and uses thereof
US11535665B2 (en) 2015-05-13 2022-12-27 The Trustees Of The University Of Pennsylvania AAV-mediated expression of anti-influenza antibodies and methods of use thereof
JP2019501115A (ja) 2015-10-28 2019-01-17 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 遺伝子治療のためのアデノ随伴ウイルスベクターのくも膜下腔内投与
EP3992283A1 (en) 2015-12-11 2022-05-04 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
EP3387117B1 (en) 2015-12-11 2022-11-23 The Trustees Of The University Of Pennsylvania Scalable purification method for aav8
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
US11098286B2 (en) 2015-12-11 2021-08-24 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV9
US20180363000A1 (en) 2015-12-14 2018-12-20 The Trustees Of The University Of Pennsylvania Aav-anti pcsk9 antibody constructs and uses thereof
EP3394270A1 (en) 2015-12-14 2018-10-31 The Trustees Of The University Of Pennsylvania Adeno-associated viral vectors useful in treatment of spinal muscular atropy
US20190002915A1 (en) 2015-12-14 2019-01-03 The Trustees Of The University Of Pennsylvania Compositions and methods for regulatable antibody expression
KR20180121899A (ko) 2016-02-03 2018-11-09 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 I형 점액다당류증을 치료하기 위한 유전자 요법
SG10201913002QA (en) * 2016-08-15 2020-03-30 Genzyme Corp Methods for detecting aav
WO2018057916A1 (en) 2016-09-24 2018-03-29 The Trustees Of The University Of Pennsylvania Novel humanized anti-ebola antibodies useful in preventing ebola infections
WO2018160573A1 (en) 2017-02-28 2018-09-07 The Trustees Of The University Of Pennsylvania Influenza vaccines based on aav vectors
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
WO2018160582A1 (en) 2017-02-28 2018-09-07 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor

Also Published As

Publication number Publication date
AU2018227440A1 (en) 2019-08-29
JP2025081544A (ja) 2025-05-27
WO2018160582A8 (en) 2019-09-12
US11827906B2 (en) 2023-11-28
SG10201912973XA (en) 2020-03-30
IL268644B2 (en) 2025-06-01
SMT202400080T1 (it) 2024-05-14
CA3053399A1 (en) 2018-09-07
KR102775350B1 (ko) 2025-03-05
SA519402565B1 (ar) 2023-12-05
MD3589730T2 (ro) 2024-04-30
FI3589730T3 (fi) 2024-02-22
KR20190132639A (ko) 2019-11-28
MX2024012403A (es) 2024-11-08
AU2024219391A1 (en) 2024-10-03
ES2971872T3 (es) 2024-06-10
JP7754872B2 (ja) 2025-10-15
EP4272765A3 (en) 2024-02-21
EP3589730A1 (en) 2020-01-08
CN110741082A (zh) 2020-01-31
ZA201905407B (en) 2020-11-25
SI3589730T1 (sl) 2024-04-30
BR112019017839A2 (pt) 2020-04-14
IL268644A (en) 2019-10-31
DK3589730T3 (da) 2024-03-04
WO2018160582A1 (en) 2018-09-07
CN110741082B (zh) 2024-09-06
LT3589730T (lt) 2024-03-12
US20240117322A1 (en) 2024-04-11
AU2018227440B2 (en) 2024-06-06
CN118956781A (zh) 2024-11-15
PT3589730T (pt) 2024-02-28
HRP20240257T1 (hr) 2024-05-24
IL268644B1 (en) 2025-02-01
CL2019002474A1 (es) 2019-11-29
JP2023089134A (ja) 2023-06-27
RS65241B1 (sr) 2024-03-29
SG10201912976WA (en) 2020-02-27
PL3589730T3 (pl) 2024-09-02
DK3589730T5 (da) 2024-09-02
SG11201907714UA (en) 2019-09-27
CO2019009525A2 (es) 2019-09-18
PE20191362A1 (es) 2019-10-01
EP4272765A2 (en) 2023-11-08
SG10201912969XA (en) 2020-02-27
US20200056159A1 (en) 2020-02-20
EP3589730B1 (en) 2023-12-27
MX2019010275A (es) 2019-10-15
JP7455579B2 (ja) 2024-03-26
JP2020510429A (ja) 2020-04-09

Similar Documents

Publication Publication Date Title
ZA201905407B (en) Adeno-associated virus (aav) clade f vector and uses therefor
IL282053A (en) Constructions of glut1-linked recombinant aav vectors, preparations and kits containing them and their use
IL279154B (en) Adenovirus-related vector
IL282885A (en) AAV viral vectors and their uses
IL286911A (en) Recombinant adeno-associated viruses and their uses
IL263801B1 (en) Novel adeno-associated virus capsid proteins
SG11202003398SA (en) Adenovirus vectors and uses thereof
ZA201801657B (en) Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same
IL287214A (en) Adeno-associated virus vector formulations and methods
EP3221456A4 (en) Genome-modified recombinant adeno-associated virus vectors
EP3589277A4 (en) MODIFIED AVA CAPSIDES AND USE OF THESE LATEST
EP3528785A4 (en) MODIFIED AAV CASPIDS AND USES THEREOF
IL272972A (en) ADENO-ASSOCIATED VIRUS (AAV with a modified phospholipase region
IL253103A0 (en) Adeno-associated viral vectors encoding adapted g6pc and uses thereof
SG11201704919SA (en) Methods of purifying adeno-associated virus (aav) and/or recombinant adeno-associated virus (raav)
MA47687A (fr) Vecteur de clade f de virus adéno-associé (aav) et ses utilisations
EP3371312A4 (en) VIRUS VECTOR TEST AND VECTOR
HK40061100A (en) Aav viral vectors and uses thereof
GB201707261D0 (en) Adeno-associated viral vector system
HK1259319A1 (en) Regulatable expression using adeno-associated virus (aav)
HK40011852A (en) Modified aav capsids and uses thereof
GB201610448D0 (en) Adeno-associated viral vector system
HK40012959A (en) Modified aav capsids and uses thereof