PE20191362A1 - Novedoso vector del subtipo f del virus adenoasociado (aav) y usos de este - Google Patents

Novedoso vector del subtipo f del virus adenoasociado (aav) y usos de este

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Publication number
PE20191362A1
PE20191362A1 PE2019001786A PE2019001786A PE20191362A1 PE 20191362 A1 PE20191362 A1 PE 20191362A1 PE 2019001786 A PE2019001786 A PE 2019001786A PE 2019001786 A PE2019001786 A PE 2019001786A PE 20191362 A1 PE20191362 A1 PE 20191362A1
Authority
PE
Peru
Prior art keywords
associated virus
seq
aavhu68
adeno
aav
Prior art date
Application number
PE2019001786A
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English (en)
Inventor
James M Wilson
Qiang Wang
April Giles
Kevin Turner
Original Assignee
Univ Pennsylvania
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Filing date
Publication date
Application filed by Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of PE20191362A1 publication Critical patent/PE20191362A1/es

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    • 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
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    • 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
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    • 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
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    • 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
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Oncology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)

Abstract

Un vector de virus adenoasociado recombinante (rAAV), que comprende una capside de AAVhu68 producida en un sistema de produccion que comprende una secuencia de nucleotidos de la SEQ ID NO: 1, o una secuencia al menos 75 % identica a esta, que codifica la SEQ ID N0:2. La capside de AAVhu68 comprende subpoblaciones de residuos de asparagina muy desamidados en los pares de asparagina-glicina en la secuencia de aminoacidos de la SEQ ID NO: 2. Tambien se proporcionan composiciones que contienen el rAAV y usos de estas. Ademas, se proporcionan rAAV que tienen una capside de AAV modificada, que comprende al menos una subpoblacion de proteinas vp1 o vp2 con una Val en la posicion de aminoacidos 157, con referencia a la numeracion de vp1 de AAVhu68
PE2019001786A 2017-02-28 2018-02-27 Novedoso vector del subtipo f del virus adenoasociado (aav) y usos de este PE20191362A1 (es)

Applications Claiming Priority (4)

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
PCT/US2018/019992 WO2018160582A1 (en) 2017-02-28 2018-02-27 Adeno-associated virus (aav) clade f vector and uses therefor

Publications (1)

Publication Number Publication Date
PE20191362A1 true PE20191362A1 (es) 2019-10-01

Family

ID=61628475

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2019001786A PE20191362A1 (es) 2017-02-28 2018-02-27 Novedoso vector del subtipo f del virus adenoasociado (aav) y usos de este

Country Status (23)

Country Link
US (2) US11827906B2 (es)
EP (2) EP4272765A3 (es)
JP (2) JP7455579B2 (es)
KR (1) KR20190132639A (es)
CN (1) CN110741082A (es)
BR (1) BR112019017839A2 (es)
CA (1) CA3053399A1 (es)
CL (1) CL2019002474A1 (es)
CO (1) CO2019009525A2 (es)
DK (1) DK3589730T3 (es)
FI (1) FI3589730T3 (es)
HR (1) HRP20240257T1 (es)
IL (1) IL268644A (es)
LT (1) LT3589730T (es)
MD (1) MD3589730T2 (es)
MX (1) MX2019010275A (es)
PE (1) PE20191362A1 (es)
PT (1) PT3589730T (es)
RS (1) RS65241B1 (es)
SG (4) SG10201912976WA (es)
SI (1) SI3589730T1 (es)
WO (1) WO2018160582A1 (es)
ZA (1) ZA201905407B (es)

Families Citing this family (55)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2406745C (en) 2001-11-13 2006-01-10 The Trustees Of The University Of Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
EP3211085B1 (en) 2003-09-30 2021-03-31 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
JP7064214B2 (ja) 2015-09-28 2022-05-10 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 抗体回避性ウイルスベクターのための方法および組成物
IL310925A (en) * 2016-01-15 2024-04-01 American Gene Tech Int Inc Methods and preparations for activating GAMMA-DELTA T cells
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
JP7455579B2 (ja) 2017-02-28 2024-03-26 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 新規アデノ随伴ウイルス(aav)クレードfベクター及びその用途
RU2019139555A (ru) 2017-05-11 2021-06-11 Зе Трастис Оф Зе Юниверсити Оф Пеннсильвания Генная терапия нейрональных цероидных липофусцинозов
AU2018375164A1 (en) 2017-11-30 2020-06-04 The Trustees Of The University Of Pennsylvania Gene therapy for Mucopolysaccharidosis IIIA
WO2019108856A1 (en) 2017-11-30 2019-06-06 The Trustees Of The University Of Pennsylvania Gene therapy for mucopolysaccharidosis iiib
MX2020008932A (es) * 2018-02-27 2020-10-01 Univ Pennsylvania Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos.
CA3091806A1 (en) * 2018-02-27 2019-09-06 The Trustees Of The University Of Pennsylvania Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor
EP3774854A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
CN113438954A (zh) * 2018-10-01 2021-09-24 宾夕法尼亚州大学信托人 可用于治疗gm1神经节苷脂病的组合物
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
EP3924364A1 (en) 2019-02-15 2021-12-22 Sangamo Therapeutics, Inc. Compositions and methods for producing recombinant aav
TW202045730A (zh) 2019-02-22 2020-12-16 賓州大學委員會 用於治療grn相關的成年發病型神經系統退化症之重組腺相關病毒
MX2021010266A (es) 2019-02-26 2021-09-23 Univ Pennsylvania Composiciones utiles en el tratamiento de la enfermedad de krabbe.
EA202192501A1 (ru) 2019-03-21 2021-12-16 Страйдбайо, Инк. Рекомбинантные аденоассоциированные вирусные векторы
WO2020219766A1 (en) * 2019-04-24 2020-10-29 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
CA3135539A1 (en) * 2019-05-03 2020-11-12 Juliette HORDEAUX Compositions useful in treatment of metachromatic leukodystrophy
WO2021076925A1 (en) 2019-10-17 2021-04-22 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
AU2020398904A1 (en) * 2019-12-10 2022-06-30 Homology Medicines, Inc. Adeno-associated virus compositions and methods of use thereof
WO2021155337A1 (en) 2020-02-02 2021-08-05 The Trustees Of The University Of Pennsylvania Compositions useful for treating gm1 gangliosidosis
CA3177407A1 (en) 2020-05-12 2021-11-18 James M. Wilson Compositions for drg-specific reduction of transgene expression
KR20230023637A (ko) 2020-05-12 2023-02-17 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 크라베병의 치료에 유용한 조성물
EP4158037A1 (en) 2020-05-26 2023-04-05 Shape Therapeutics Inc. High throughput engineering of functional aav capsids
CN116438311A (zh) 2020-07-13 2023-07-14 宾夕法尼亚州大学信托人 可用于治疗夏科-马里-图思病的组合物
GB2599212A (en) 2020-07-30 2022-03-30 Shape Therapeutics Inc Stable cell lines for inducible production of rAAV virions
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
CN116887867A (zh) 2020-08-26 2023-10-13 宾夕法尼亚州大学信托人 用于治疗grn相关成人发作性神经退行性病变的重组腺相关病毒
EP4225906A1 (en) 2020-10-09 2023-08-16 The Trustees of The University of Pennsylvania Compositions and methods for treatment of fabry disease
AR123838A1 (es) 2020-10-18 2023-01-18 Univ Pennsylvania Vector mejorado de virus adenoasociado (aav) y usos de este
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
WO2022094255A2 (en) * 2020-10-29 2022-05-05 Regenxbio Inc. Vectorized factor xii antibodies and administration thereof
JP2023550581A (ja) * 2020-10-29 2023-12-04 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 Aavカプシド及びそれを含有する組成物
JP2023548432A (ja) 2020-11-11 2023-11-16 ヨーロピアン モレキュラー バイオロジー ラボラトリー 遺伝子療法のための改変ウイルス粒子
CA3200014A1 (en) 2020-12-01 2022-06-09 James M. Wilson Novel compositions with tissue-specific targeting motifs and compositions containing same
CA3200192A1 (en) 2020-12-01 2022-06-09 Justin PERCIVAL Compositions and uses thereof for treatment of angelman syndrome
JP2024505257A (ja) 2021-02-01 2024-02-05 レジェンクスバイオ インコーポレーテッド 神経セロイドリポフスチン症の遺伝子治療
EP4323520A1 (en) 2021-04-12 2024-02-21 The Trustees of The University of Pennsylvania Compositions useful for treating spinal and bulbar muscular atrophy (sbma)
BR112023021971A2 (pt) 2021-04-23 2024-02-20 Univ Pennsylvania Composições com motivos de direcionamento específicos do cérebro e composições contendo os mesmos
BR112023023599A2 (pt) 2021-05-12 2024-03-12 Fond Telethon Sistema de vetor
IL308787A (en) * 2021-06-08 2024-01-01 Univ Pennsylvania Recombinant adeno-associated viruses for Leish-Nihan disorders and their uses
WO2023056399A1 (en) 2021-10-02 2023-04-06 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
WO2023102079A1 (en) 2021-12-01 2023-06-08 Shape Therapeutics Inc. Functional aav capsids for systemic administration
WO2023102078A2 (en) 2021-12-01 2023-06-08 Shape Therapeutics Inc. Functional aav capsids for intravitreal administration
WO2023102517A1 (en) 2021-12-02 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
WO2023133584A1 (en) 2022-01-10 2023-07-13 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
WO2023147304A1 (en) 2022-01-25 2023-08-03 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
WO2023196893A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treating her2 positive metastatic breast cancer and other cancers
WO2023214346A1 (en) 2022-05-06 2023-11-09 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
WO2023220695A2 (en) * 2022-05-13 2023-11-16 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer

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
JPH08506144A (ja) 1993-02-05 1996-07-02 ラポート グループ オーストラリア リミティド スラグ脱泡複合材料
US5834266A (en) 1993-02-12 1998-11-10 President & Fellows Of Harvard College Regulated apoptosis
US20020173474A1 (en) 1993-02-12 2002-11-21 President And Fellows Of Harvard College Methods & materials involving dimerization-mediated regulation of biological events
US6140120A (en) 1993-02-12 2000-10-31 Board Of Trustees Of Leland Stanford Jr. University Regulated transcription of targeted genes and other biological events
JP3824633B2 (ja) 1993-02-12 2006-09-20 ザ・ボード・オブ・トラスティーズ・オブ・ザ・リランド・スタンフォード・ジュニアー・ユニバーシティ 標的遺伝子の調節的転写および他の生物学的結果
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
US5830462A (en) 1993-02-12 1998-11-03 President & Fellows Of Harvard College Regulated transcription of targeted genes and other 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
US6133456A (en) 1994-08-18 2000-10-17 Ariad Gene Therapeutics, Inc. Synthetic multimerizing agents
US6150527A (en) 1994-08-18 2000-11-21 Ariad Pharmaceuticals, Inc. Synthetic multimerizing agents
AU3367995A (en) 1994-08-18 1996-03-14 Ariad Pharmaceuticals, Inc. New multimerizing agents
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
AU719001B2 (en) 1994-12-29 2000-05-04 Massachusetts Institute Of Technology 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
CA2219080A1 (en) 1995-06-07 1996-12-27 Ariad Gene Therapeutics, Inc. Rapamycin-based regulation of biological events
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
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
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US6187757B1 (en) 1995-06-07 2001-02-13 Ariad Pharmaceuticals, Inc. Regulation of biological events using novel compounds
WO1997031899A1 (en) 1996-02-28 1997-09-04 Ariad Gene Therapeutics, Inc. Synthetic derivatives of rapamycin as multimerising 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
ATE545710T1 (de) 1997-04-01 2012-03-15 Illumina Cambridge Ltd Verfahren zur vervielfältigung von nukleinsäuren
US6548286B1 (en) 1997-04-14 2003-04-15 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant AAV product
US6479653B1 (en) 1997-08-26 2002-11-12 Ariad Gene Therapeutics, Inc. Compositions and method for regulation of transcription
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
AU752129B2 (en) 1997-08-27 2002-09-05 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
WO1999041258A1 (en) 1998-02-13 1999-08-19 President And Fellows Of Harvard College Novel dimerizing agents, their production and use
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
DK1916258T3 (da) 1999-08-09 2014-07-28 Genzyme Corp Forøgelse af ekspression af en enkeltstrenget, heterolog nukleotidsekvens fra rekombinante, virale vektorer ved en sådan udformning af sekvensen at den danner intrastrengbasepar
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
US7067526B1 (en) 1999-08-24 2006-06-27 Ariad Gene Therapeutics, Inc. 28-epirapalogs
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
AU2001249315A1 (en) 2000-03-22 2001-10-03 Rheogene, Inc. Ecdysone receptor-based inducible gene expression system
US7201898B2 (en) 2000-06-01 2007-04-10 The 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
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
JP4328094B2 (ja) 2001-02-20 2009-09-09 イントレクソン・コーポレイション 新規置換突然変異体受容体および核受容体−ベースの誘導性遺伝子発現システムにおけるその使用
JP4955905B2 (ja) 2001-02-20 2012-06-20 イントレキソン コーポレーション キメラレチノイドx受容体および新規エクジソン受容体−ベースの誘導性遺伝子発現システムにおけるそれらの使用
JP4994563B2 (ja) 2001-02-20 2012-08-08 イントレキソン コーポレーション 新規置換突然変異体受容体および核受容体−ベースの誘導性遺伝子発現システムにおけるその使用
IL158487A0 (en) 2001-05-18 2004-05-12 Wisconsin Alumni Res Found Method for the synthesis of dna sequences
CA2406745C (en) 2001-11-13 2006-01-10 The Trustees Of The University Of Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
FI3517134T3 (fi) 2001-12-17 2024-04-03 Univ Pennsylvania Adeno-assosioituneen viruksen (aav) serotyypin 8 sekvenssejä, niitä sisältäviä vektoreita ja niiden käyttöjä
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
EP2434420A3 (en) 2003-08-01 2012-07-25 Dna Twopointo Inc. Systems and methods for biopolymer engineering
EP3211085B1 (en) 2003-09-30 2021-03-31 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
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
EP2357010B1 (en) 2005-04-07 2013-06-12 The Trustees of The University of Pennsylvania Method of increasing the function of an AAV vector
JP4495210B2 (ja) 2005-06-09 2010-06-30 パナソニック株式会社 振幅誤差補償装置及び直交度誤差補償装置
PL2548560T3 (pl) 2005-06-23 2015-11-30 Biogen Ma Inc Kompozycje i sposoby modulowania splicingu SMN2
US8877187B2 (en) 2005-07-25 2014-11-04 Avianax, Llc Therapeutic antibodies for treatment and prophylaxis of transmittable viral diseases
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
ES2400235T3 (es) * 2006-04-28 2013-04-08 The Trustees Of The University Of Pennsylvania Método de producción escalable de AAV
LT2489733T (lt) 2006-06-07 2019-05-27 Genzyme Corporation Šoninės amiotrofinės sklerozės ir kitų nugaros smegenų sutrikimų genų terapija
US8603950B2 (en) 2007-02-20 2013-12-10 Anaptysbio, Inc. Methods of generating libraries and uses thereof
JP5346820B2 (ja) 2007-03-13 2013-11-20 エイチユーエムエイビーエス・リミテッド・ライアビリティ・カンパニー H5n1亜型a型インフルエンザウィルスに対する抗体
WO2008124934A1 (en) 2007-04-17 2008-10-23 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
NZ585500A (en) 2007-11-12 2012-06-29 Theraclone Sciences Inc Antibodies which bind to an epitope in the extracellular domain of the matrix 2 ectodomain polypeptide of an influenza virus
EP3333187B1 (en) 2007-12-06 2022-06-22 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
JP2011516423A (ja) 2008-03-28 2011-05-26 シー レーン バイオテクノロジーズ, エルエルシー ウイルス抗原に対する中和分子
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
CA2731686C (en) 2008-07-25 2020-04-07 Institute For Research In Biomedicine Neutralizing anti-influenza a virus antibodies and uses thereof
US8871207B2 (en) 2008-07-25 2014-10-28 Humabs, LLC Neutralizing anti-influenza A virus antibodies and uses thereof
CN102245559B (zh) 2008-11-07 2015-05-27 麻省理工学院 氨基醇类脂质和其用途
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
AU2010247530B2 (en) 2009-05-11 2016-10-13 Janssen Vaccines & Prevention 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)
EP3305302B1 (en) 2009-06-17 2018-09-19 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing in a subject
CN102802666B (zh) 2009-06-25 2015-06-03 米迪缪尼有限公司 猪流感血凝素变异体
US8598331B2 (en) 2009-09-28 2013-12-03 The University Of British Columbia CLDN5 mini-promoters
BR112012024934A2 (pt) 2010-03-29 2016-12-06 Univ Pennsylvania sistemas de ablação de transgene induzida farmacologicamente
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
CN103492574B (zh) 2011-02-22 2015-12-09 加州理工学院 使用腺相关病毒(aav)载体递送蛋白
EP3699286A1 (en) 2011-04-20 2020-08-26 The Trustees of the University of Pennsylvania Regimens and compositions for aav-mediated passive immunization of airborne pathogens
US20140127749A1 (en) 2011-04-21 2014-05-08 Arizona Borad Of Regents, A Body Corporate Of The State Of Arizona Acting For And On Behalf Of Arizo Methods of protein production and compositions thereof
AU2012267531B2 (en) 2011-06-08 2017-06-22 Translate Bio, Inc. Lipid nanoparticle compositions and methods for mRNA delivery
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
MX346206B (es) 2011-07-14 2017-03-09 Crucell Holland Bv Moleculas de enlace humanas capaces de neutralizar los virus de la influenza a del grupo filogenetico 1 y grupo filogenetico 2 y virus de la influenza b.
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
US8834881B2 (en) 2012-03-08 2014-09-16 Crucell Holland B.V. Human binding molecules capable of binding to and neutralizing influenza B viruses and uses thereof
WO2013155222A2 (en) 2012-04-10 2013-10-17 The Regents Of The University Of California Brain-specific enhancers for cell-based therapy
JP6208750B2 (ja) 2012-06-08 2017-10-04 エスリス ゲーエムベーハーethris GmbH メッセンジャーrnaの肺送達
CA2877428A1 (en) 2012-06-21 2013-12-27 Association Institut De Myologie Widespread gene delivery of gene therapy vectors
DK2880053T3 (da) 2012-08-01 2020-05-11 Ikaika Therapeutics Llc Lindring af vævsskade og fibrose via anti-LTBP4-antistof
US9719106B2 (en) 2013-04-29 2017-08-01 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof
KR102116378B1 (ko) 2013-05-01 2020-06-01 젠자임 코포레이션 척수성 근위축증을 치료하기 위한 조성물 및 방법
EP3107582A4 (en) 2014-02-19 2018-02-14 Emergent BioSolutions Canada Inc. Ebola monoclonal antibodies
WO2015164757A1 (en) * 2014-04-25 2015-10-29 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
EP3134115A4 (en) 2014-04-25 2017-10-25 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
CA2946392A1 (en) 2014-04-25 2015-10-29 James M. Wilson Ldlr variants and their use in compositions for reducing cholesterol levels
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
CN113667696A (zh) * 2014-09-24 2021-11-19 希望之城 用于高效基因组编辑的腺相关病毒载体变异体和其方法
US10894818B2 (en) 2014-10-03 2021-01-19 Massachusetts Institute Of Technology Antibodies that bind Ebola glycoprotein and uses thereof
BR112017016417A2 (pt) 2015-02-05 2018-03-27 Janssen Vaccines & Prevention B.V. moléculas de ligação dirigidas contra hemaglutinina da gripe e seus usos
EP3307310A2 (en) 2015-05-13 2018-04-18 The Trustees Of The University Of Pennsylvania Aav-mediated expression of anti-influenza antibodies and methods of use thereof
KR20180100304A (ko) 2015-10-28 2018-09-10 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 유전자 요법을 위한 아데노-관련-바이러스 벡터의 척추강 내 투여
US11015173B2 (en) 2015-12-11 2021-05-25 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
ES2918998T3 (es) 2015-12-11 2022-07-21 Univ Pennsylvania Método de purificación escalable para AAVrh10
US11015174B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV8
US20180363000A1 (en) 2015-12-14 2018-12-20 The Trustees Of The University Of Pennsylvania Aav-anti pcsk9 antibody constructs and uses thereof
JP7082050B2 (ja) 2015-12-14 2022-06-07 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 脊髄性筋萎縮症の処置において有用なアデノ-関連ウイルスベクター
WO2017106244A1 (en) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Compositions and methods for regulatable antibody expression
WO2017136500A1 (en) 2016-02-03 2017-08-10 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
SG11201901221YA (en) * 2016-08-15 2019-03-28 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
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
CN110573523A (zh) 2017-02-28 2019-12-13 宾夕法尼亚州大学信托人 基于aav载体的流感疫苗
JP7455579B2 (ja) 2017-02-28 2024-03-26 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 新規アデノ随伴ウイルス(aav)クレードfベクター及びその用途

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