PL3589730T3 - Wektor z kladu f wirusa związanego z adenowirusami (aav) i zastosowania tego wektora - Google Patents

Wektor z kladu f wirusa związanego z adenowirusami (aav) i zastosowania tego wektora

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Publication number
PL3589730T3
PL3589730T3 PL18711202.4T PL18711202T PL3589730T3 PL 3589730 T3 PL3589730 T3 PL 3589730T3 PL 18711202 T PL18711202 T PL 18711202T PL 3589730 T3 PL3589730 T3 PL 3589730T3
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Poland
Prior art keywords
vector
aav
adeno
class
associated virus
Prior art date
Application number
PL18711202.4T
Other languages
English (en)
Inventor
James M. Wilson
Qiang Wang
April GILES
Kevin Turner
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The Trustees Of The University Of Pennsylvania
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Application filed by The Trustees Of The University Of Pennsylvania filed Critical The Trustees Of The University Of Pennsylvania
Publication of PL3589730T3 publication Critical patent/PL3589730T3/pl

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    • 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
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    • 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
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    • 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
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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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    • C12N2750/00011Details
    • 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
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    • 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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    • C12N2750/00011Details
    • 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)
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  • Virology (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
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  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Mycology (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Oncology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
PL18711202.4T 2017-02-28 2018-02-27 Wektor z kladu f wirusa związanego z adenowirusami (aav) i zastosowania tego wektora PL3589730T3 (pl)

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
PL3589730T3 true PL3589730T3 (pl) 2024-09-02

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PL18711202.4T PL3589730T3 (pl) 2017-02-28 2018-02-27 Wektor z kladu f wirusa związanego z adenowirusami (aav) i zastosowania tego wektora

Country Status (29)

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

Families Citing this family (78)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ618298A (en) 2001-11-13 2015-04-24 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
EP2292780B1 (en) 2003-09-30 2017-08-23 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
DK2986635T3 (en) 2013-04-18 2019-01-28 Fond Telethon EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS
CN108137655B (zh) 2015-09-28 2022-04-22 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
IL279520B2 (en) * 2016-01-15 2023-03-01 American Gene Tech Int Inc Methods and preparations for the activation of gamma-delta t cells
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
US11591614B2 (en) 2017-05-11 2023-02-28 The Trustees Of The University Of Pennsylvania Gene therapy for ceroid lipofuscinoses
AU2018375163A1 (en) 2017-11-30 2020-06-11 The Trustees Of The University Of Pennsylvania Gene therapy for mucopolysaccharidosis IIIB
JP7389744B2 (ja) 2017-11-30 2023-11-30 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 ムコ多糖症iiia型のための遺伝子療法
CN112352050A (zh) * 2018-02-27 2021-02-09 宾夕法尼亚州大学信托人 新颖腺相关病毒(aav)载体、具有降低的衣壳脱酰胺化的aav载体及其用途
EP3768695A4 (en) * 2018-02-27 2022-04-06 The Trustees of the University of Pennsylvania NOVEL ADENO-ASSOCIATED VIRUS (AAV) VECTORS, AAV VECTORS WITH REDUCED CAPSIID DEAMIDATION AND USES THEREOF
SG11202009451VA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス 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
MX2021003869A (es) 2018-10-01 2021-09-08 Univ Pennsylvania Composiciones útiles para tratar la gangliosidosis gm1.
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
KR20210131370A (ko) 2019-02-22 2021-11-02 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Grn-연관 성인-발병 신경퇴화의 치료를 위한 재조합 아데노-연관 바이러스
TW202045728A (zh) * 2019-02-26 2020-12-16 賓州大學委員會 用於治療克拉培氏病之組成物
CA3133453A1 (en) 2019-03-21 2020-09-24 Daniel Mccoy Recombinant adeno-associated virus vectors
KR20220003553A (ko) * 2019-04-24 2022-01-10 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 레트 증후군의 치료에 유용한 조성물
BR112021021502A2 (pt) * 2019-04-29 2022-07-19 Univ Pennsylvania Capsídeos de aav e composições contendo os mesmos
MX2021013420A (es) * 2019-05-03 2022-02-03 Univ Pennsylvania Composiciones utiles en el tratamiento de leucodistrofia metacromatica.
TW202128736A (zh) 2019-10-17 2021-08-01 美商史崔德生物公司 用於治療c型尼曼—匹克病之腺相關病毒載體
AU2020398904A1 (en) * 2019-12-10 2022-06-30 Homology Medicines, Inc. Adeno-associated virus compositions and methods of use thereof
US20230190966A1 (en) 2020-02-02 2023-06-22 The Trustees Of The University Of Pennsylvania Compositions useful for treating gm1 gangliosidosis
CN113621708B (zh) * 2020-03-30 2022-02-15 中国医学科学院肿瘤医院 用于肺癌诊断的试剂盒、装置及方法
CN115885040A (zh) * 2020-04-27 2023-03-31 宾夕法尼亚州大学信托人 可用于治疗cdkl5缺乏症(cdd)的组合物
CN116057175A (zh) * 2020-05-12 2023-05-02 宾夕法尼亚州大学信托人 可用于治疗克拉伯病的组合物
KR20230010670A (ko) 2020-05-12 2023-01-19 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 이식유전자 발현의 drg-특이적 감소를 위한 조성물
AU2021282257A1 (en) 2020-05-26 2022-12-01 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
CN116323953A (zh) 2020-07-30 2023-06-23 沙普治疗股份有限公司 诱导产生raav病毒粒子的稳定细胞系
CN116745409A (zh) 2020-08-19 2023-09-12 萨雷普塔治疗公司 用于治疗雷特综合征的腺相关病毒载体
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
US20230364264A1 (en) 2020-08-26 2023-11-16 The Trustees Of The University Of Pennsylvania Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration
CA3193833A1 (en) 2020-10-09 2022-04-14 Juliette HORDEAUX Compositions and methods for treatment of fabry disease
CA3195553A1 (en) 2020-10-18 2022-04-21 Qiang Wang Improved adeno-associated virus (aav) vector and uses therefor
JP2023546689A (ja) * 2020-10-22 2023-11-07 スカウト バイオ, インコーポレイテッド ベクター送達される治療用タンパク質に対する免疫応答を抑制する方法
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
US20230390418A1 (en) * 2020-10-29 2023-12-07 Regenxbio Inc. Vectorized factor xii antibodies and administration thereof
US20230407333A1 (en) * 2020-10-29 2023-12-21 The Trustees Of The University Of Pennsylvania Aav capsids and compositions containing same
KR102862676B1 (ko) 2020-11-11 2025-09-23 유럽피안 몰레큘러 바이올로지 래보러토리 유전자 치료를 위한 변형된 바이러스 입자
US20240024507A1 (en) 2020-12-01 2024-01-25 The Trustees Of The University Of Pennsylvania Novel compositions with tissue-specific targeting motifs and compositions containing same
MX2023006445A (es) 2020-12-01 2023-08-10 Univ Pennsylvania Composiciones y usos de estas para el tratamiento del síndrome de angelman.
CA3209779A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
AU2022258312A1 (en) 2021-04-12 2023-10-26 The Trustees Of The University Of Pennsylvania Compositions useful for treating spinal and bulbar muscular atrophy (sbma)
AR125406A1 (es) 2021-04-23 2023-07-12 Univ Pennsylvania Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen
IL308356A (en) 2021-05-12 2024-01-01 Fond Telethon Vector system
CA3220037A1 (en) * 2021-06-08 2022-12-15 The Trustees Of The University Of Pennsylvania Recombinant adeno-associated viruses for lesch-nyhan disorders and uses thereof
US20240384298A1 (en) 2021-10-02 2024-11-21 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
US20250295807A1 (en) 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2023102078A2 (en) 2021-12-01 2023-06-08 Shape Therapeutics Inc. Functional aav capsids for intravitreal administration
JP2024543211A (ja) 2021-12-01 2024-11-19 シェイプ セラピューティクス インコーポレイテッド 全身投与用の機能的aavカプシド
WO2023102517A1 (en) 2021-12-02 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
AR128239A1 (es) 2022-01-10 2024-04-10 Univ Pennsylvania Composiciones y métodos útiles para el tratamiento de trastornos mediados por c9orf72
WO2023133584A1 (en) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
MX2024009186A (es) 2022-01-25 2024-09-30 Univ Pennsylvania Cápsides de aav para mejorar la transducción cardiaca y desdireccionamiento del hígado.
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
EP4504258A1 (en) 2022-04-06 2025-02-12 The Trustees of the University of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
JP2025517628A (ja) 2022-05-06 2025-06-10 ノバルティス アーゲー 新規の組換えaav vp2融合ポリペプチド
US20250295809A1 (en) * 2022-05-13 2025-09-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
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
AU2023398931A1 (en) 2022-12-17 2025-07-10 The Trustees Of The University Of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
EP4665743A2 (en) 2023-02-17 2025-12-24 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
TW202516019A (zh) 2023-06-29 2025-04-16 賓州大學委員會 具中樞神經系統靶向模體的突變aav及含有其之組成物
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
TW202535908A (zh) 2023-11-02 2025-09-16 美商渤健麻塞諸塞州股份有限公司 中樞神經系統靶向之aav衣殼之重定向
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
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
NZ261277A (en) 1993-02-05 1997-04-24 Laporte Group Australia Ltd Slag defoaming composition comprising a carbon source; an exothermic material (eg aluminium) and a dense inert granular material (eg calcium silicate)
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
US5830462A (en) 1993-02-12 1998-11-03 President & Fellows Of Harvard College 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
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
WO1994018317A1 (en) 1993-02-12 1994-08-18 The Board Of Trustees Of The Leland Stanford Junior University 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
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
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
US6133456A (en) 1994-08-18 2000-10-17 Ariad Gene Therapeutics, Inc. Synthetic 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
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
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
US6506379B1 (en) 1995-06-07 2003-01-14 Ariad Gene Therapeutics, Inc. Intramuscular delivery of recombinant AAV
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
WO1996041865A1 (en) 1995-06-07 1996-12-27 Ariad Gene Therapeutics, Inc. Rapamcycin-based regulation of biological events
AU2192797A (en) 1996-02-28 1997-09-16 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
DE69837913T2 (de) 1997-04-01 2008-02-07 Solexa Ltd., Saffron Walden Verfahren zur vervielfältigung von nukleinsäure
DE69824859T2 (de) 1997-04-14 2005-08-04 Cell Genesys, Inc., Foster City Methoden zur erhöhung der effizienz rekombinanter aav produkte
US6015709A (en) 1997-08-26 2000-01-18 Ariad Pharmaceuticals, Inc. Transcriptional activators, and compositions and uses related thereto
EP1017829A2 (en) 1997-08-26 2000-07-12 Ariad Gene Therapeutics, Inc. Fusion proteins comprising a dimerization, trimerization or tetramerization domain and an additional heterologous transcription activation, transcription repression, dna binding or ligand binding domain
US6479653B1 (en) 1997-08-26 2002-11-12 Ariad Gene Therapeutics, Inc. Compositions and method for regulation of transcription
CA2303482A1 (en) 1997-08-27 1999-03-04 Ariad Gene Therapeutics, Inc. Chimeric transcriptional activators and compositions and uses related thereto
WO1999036553A2 (en) 1998-01-15 1999-07-22 Ariad Gene Therapeutics, Inc. Regulation of biological events using multimeric chimeric proteins
CA2319492A1 (en) 1998-02-13 1999-08-19 President And Fellows Of Harvard College Novel dimerizing agents, their production and use
US6984635B1 (en) 1998-02-13 2006-01-10 Board Of Trustees Of The Leland Stanford Jr. University 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
EP1080218A1 (en) 1998-05-27 2001-03-07 University of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient
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
DE69941905D1 (de) 1998-11-10 2010-02-25 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
ES2308989T3 (es) 1999-08-09 2008-12-16 Targeted Genetics Corporation Aumento de la expresion de una secuencia nucleotidica heterologa a partir de vectores viricos recombinantes que contienen una secuencia que forman pares de bases intracatenarios.
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
IL147803A0 (en) 1999-08-24 2002-08-14 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
CA2404253C (en) 2000-03-22 2014-05-13 Rohm And Haas Company Novel ecdysone receptor-based inducible gene expression system
US20040033600A1 (en) 2001-03-21 2004-02-19 Palli Subba Reddy Ecdysone receptor-based inducible gene expression system
EP1286703B1 (en) 2000-06-01 2009-08-05 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
ES2390070T3 (es) 2001-02-20 2012-11-06 Intrexon Corporation Nuevos receptores mutantes de sustitución y su uso en un sistema de expresión génica inducible basado en receptores nucleares
WO2002066614A2 (en) 2001-02-20 2002-08-29 Rheogene Holdings, Inc. Chimeric retinoid x receptors and their use in a novel ecdysone receptor-based inducible gene expression system
CA2447240C (en) 2001-05-18 2013-02-19 Wisconsin Alumni Research Foundation Method for the synthesis of dna sequences
NZ618298A (en) 2001-11-13 2015-04-24 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
PT2359869T (pt) 2001-12-17 2019-04-16 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
WO2003104413A2 (en) 2002-06-05 2003-12-18 University Of Florida Production of pseudotyped recombinant aav virions
US8005620B2 (en) 2003-08-01 2011-08-23 Dna Twopointo Inc. Systems and methods for biopolymer engineering
US20080050357A1 (en) 2003-08-01 2008-02-28 Claes Gustafsson Systems and Methods for Antibody Engineering
EP2292780B1 (en) * 2003-09-30 2017-08-23 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
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
EP2359865B1 (en) 2005-04-07 2013-10-02 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
WO2007002390A2 (en) 2005-06-23 2007-01-04 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing
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
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
PL2489733T3 (pl) 2006-06-07 2019-08-30 Genzyme Corporation Terapia genowa w stwardnieniu zanikowym bocznym i innych zaburzeniach rdzenia kręgowego
BRPI0807952A2 (pt) 2007-02-20 2014-06-10 Anaptysbio Inc Sistemas de hipermutação somática
EP2125886A2 (en) 2007-03-13 2009-12-02 Humabs LLC Antibodies against h5n1 strains of influenza a virus
WO2008124934A1 (en) 2007-04-17 2008-10-23 National Research Council Of Canada Constructs for enhancement of gene expression in muscle
US7879326B2 (en) 2007-06-15 2011-02-01 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
MX2010005244A (es) 2007-11-12 2010-10-25 Theraclone Sciences Inc Composiciones y métodos para la terapia y el diagnóstico de influenza.
EP3524619A1 (en) 2007-12-06 2019-08-14 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
EP2698380A1 (en) 2008-03-28 2014-02-19 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
RU2553325C2 (ru) 2008-07-25 2015-06-10 Институт Фо Ресёч Ин Биомедицин Нейтрализующие антитела против вируса гриппа а и их использование
JP6087504B2 (ja) 2008-11-07 2017-03-01 マサチューセッツ インスティテュート オブ テクノロジー アミノアルコールリピドイドおよびその使用
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
WO2010130636A1 (en) 2009-05-11 2010-11-18 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)
KR20200047790A (ko) 2009-06-17 2020-05-07 바이오젠 엠에이 인코포레이티드 대상에게서 smn2 스플라이싱을 조정하기 위한 조성물 및 방법
WO2010151673A1 (en) 2009-06-25 2010-12-29 Medimmune, Llc Swine influenza hemagglutinin variants
US8598331B2 (en) 2009-09-28 2013-12-03 The University Of British Columbia CLDN5 mini-promoters
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
SG183929A1 (en) 2010-03-29 2012-10-30 Univ Pennsylvania Pharmacologically induced transgene ablation system
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
WO2012115980A1 (en) 2011-02-22 2012-08-30 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
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
CA3107288A1 (en) 2011-06-08 2012-12-13 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
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
US9181328B2 (en) 2012-01-31 2015-11-10 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
BR112014029807A2 (pt) 2012-06-08 2017-06-27 Ethris Gmbh administração pulmonar de rna mensageiro
EP3536795A3 (en) 2012-06-21 2019-12-11 Association Institut de Myologie Widespread gene expression
ES2791778T3 (es) 2012-08-01 2020-11-05 Ikaika Therapeutics Llc Mitigación del daño tisular y la fibrosis a través de anticuerpos anti-LTBP4
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
US10746742B2 (en) * 2014-04-25 2020-08-18 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
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
JP6983511B2 (ja) 2014-04-25 2021-12-17 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 脳中の転移乳癌および他の癌を処置するための方法および組成物
PT3142750T (pt) 2014-05-13 2020-09-22 Univ Pennsylvania Composições que compreendem aav expressando construções de anticorpos duplos e suas utilizações
KR20230067694A (ko) * 2014-09-24 2023-05-16 시티 오브 호프 고효율 게놈 편집을 위한 아데노-관련 바이러스 벡터 변이체 및 이의 방법
CA2963470A1 (en) 2014-10-03 2016-04-07 Massachusetts Institute Of Technology Antibodies that bind ebola glycoprotein and uses thereof
EA038407B1 (ru) 2015-02-05 2021-08-24 Янссен Вэксинс Энд Превеншн Б.В. Связывающие молекулы, направленные против гемагглютинина вируса гриппа, и пути их применения
MA48852A (fr) 2015-05-13 2020-04-01 Univ Pennsylvania Expression médiée par aav d'anticorps anti-grippaux et leurs procédés d'utilisation
US11135313B2 (en) 2015-10-28 2021-10-05 The Trustees Of The University Of Pennsylvania Intrathecal administration of adeno-associated-viral vectors for gene therapy
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
EP3387117B1 (en) 2015-12-11 2022-11-23 The Trustees Of The University Of Pennsylvania Scalable purification method for aav8
ES2918998T3 (es) 2015-12-11 2022-07-21 Univ Pennsylvania Método de purificación escalable para AAVrh10
US11098286B2 (en) 2015-12-11 2021-08-24 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV9
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
US20180363000A1 (en) 2015-12-14 2018-12-20 The Trustees Of The University Of Pennsylvania Aav-anti pcsk9 antibody constructs and uses thereof
US20190002915A1 (en) 2015-12-14 2019-01-03 The Trustees Of The University Of Pennsylvania Compositions and methods for regulatable antibody expression
MA43968A (fr) 2016-02-03 2018-12-12 Univ Pennsylvania Thérapie génique pour traiter la mucopolysaccharidose de type i
CA3033856A1 (en) 2016-08-15 2018-02-22 Genzyme Corporation Methods for detecting aav
US20190240328A1 (en) 2016-09-24 2019-08-08 The Trustees Of The University Of Pennsylvania Novel humanized anti-ebola antibodies useful in preventing ebola infections
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
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

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