CO2019009525A2 - 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

Info

Publication number
CO2019009525A2
CO2019009525A2 CONC2019/0009525A CO2019009525A CO2019009525A2 CO 2019009525 A2 CO2019009525 A2 CO 2019009525A2 CO 2019009525 A CO2019009525 A CO 2019009525A CO 2019009525 A2 CO2019009525 A2 CO 2019009525A2
Authority
CO
Colombia
Prior art keywords
associated virus
seq
adeno
aav
subtype
Prior art date
Application number
CONC2019/0009525A
Other languages
English (en)
Spanish (es)
Inventor
Qiang Wang
James M Wilson
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 CO2019009525A2 publication Critical patent/CO2019009525A2/es

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)
  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • General Engineering & Computer Science (AREA)
  • 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)
CONC2019/0009525A 2017-02-28 2019-08-30 Novedoso vector del subtipo f del virus adenoasociado (aav) y usos de este CO2019009525A2 (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
CO2019009525A2 true CO2019009525A2 (es) 2019-09-18

Family

ID=61628475

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2019/0009525A CO2019009525A2 (es) 2017-02-28 2019-08-30 Novedoso vector del subtipo f del virus adenoasociado (aav) y usos de este

Country Status (29)

Country Link
US (2) US11827906B2 (enExample)
EP (2) EP3589730B1 (enExample)
JP (3) JP7455579B2 (enExample)
KR (1) KR102775350B1 (enExample)
CN (2) CN110741082B (enExample)
AU (2) AU2018227440B2 (enExample)
BR (1) BR112019017839A2 (enExample)
CA (1) CA3053399A1 (enExample)
CL (1) CL2019002474A1 (enExample)
CO (1) CO2019009525A2 (enExample)
DK (1) DK3589730T5 (enExample)
ES (1) ES2971872T3 (enExample)
FI (1) FI3589730T3 (enExample)
HR (1) HRP20240257T1 (enExample)
HU (1) HUE065885T2 (enExample)
IL (1) IL268644B2 (enExample)
LT (1) LT3589730T (enExample)
MD (1) MD3589730T2 (enExample)
MX (2) MX2019010275A (enExample)
PE (1) PE20191362A1 (enExample)
PL (1) PL3589730T3 (enExample)
PT (1) PT3589730T (enExample)
RS (1) RS65241B1 (enExample)
SA (1) SA519402565B1 (enExample)
SG (4) SG10201912973XA (enExample)
SI (1) SI3589730T1 (enExample)
SM (1) SMT202400080T1 (enExample)
WO (1) WO2018160582A1 (enExample)
ZA (1) ZA201905407B (enExample)

Families Citing this family (78)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2915124C (en) 2001-11-13 2018-08-14 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
EP2298926A1 (en) 2003-09-30 2011-03-23 The Trustees of The University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
US10494645B2 (en) 2013-04-18 2019-12-03 Fondazione Telethon Effective delivery of large genes by dual AAV vectors
EP3831842A1 (en) 2015-09-28 2021-06-09 The University of North Carolina at Chapel Hill Methods and compositions for antibody-evading virus vectors
CA3011529A1 (en) * 2016-01-15 2017-07-20 American Gene Technologies International Inc. Methods and compositions 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
US11827906B2 (en) 2017-02-28 2023-11-28 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clade f vector and uses therefor
RU2019139555A (ru) 2017-05-11 2021-06-11 Зе Трастис Оф Зе Юниверсити Оф Пеннсильвания Генная терапия нейрональных цероидных липофусцинозов
CA3083761A1 (en) 2017-11-30 2019-06-06 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
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
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.
EP3773743A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
CN112543766A (zh) 2018-04-03 2021-03-23 斯特里迪比奥公司 抗体逃避性病毒载体
WO2019195449A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
KR20210071017A (ko) * 2018-10-01 2021-06-15 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Gm1 강글리오시드증 치료에 유용한 조성물
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
SG11202108614QA (en) 2019-02-15 2021-09-29 Sangamo Therapeutics Inc Compositions and methods for producing recombinant aav
EP3927381A1 (en) 2019-02-22 2021-12-29 The Trustees of The University of Pennsylvania Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration
MX2021010266A (es) * 2019-02-26 2021-09-23 Univ Pennsylvania Composiciones utiles en el tratamiento de la enfermedad de krabbe.
CN113727992A (zh) 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
US20220202960A1 (en) * 2019-04-24 2022-06-30 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
PH12021552608A1 (en) * 2019-04-29 2022-06-20 Univ Pennsylvania Novel aav capsids and compositions containing same
WO2020227166A1 (en) * 2019-05-03 2020-11-12 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
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
EP4072572A4 (en) * 2019-12-10 2024-01-03 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
CN115287350B (zh) * 2020-03-30 2025-07-04 中国医学科学院肿瘤医院 包括外泌体miR-106b-3p、miR-10a-3p等在肺癌诊断中的应用
IL297635A (en) * 2020-04-27 2022-12-01 Univ Pennsylvania Compositions useful in treatment of cdkl5 deficiency disorder (cdd)
AU2021270447A1 (en) 2020-05-12 2023-01-05 The Trustees Of The University Of Pennsylvania Compositions for DRG-specific reduction of transgene expression
TW202208622A (zh) * 2020-05-12 2022-03-01 賓州大學委員會 用於治療克拉培氏病之組成物
WO2021242909A1 (en) 2020-05-26 2021-12-02 Shape Therapeutics Inc. High throughput engineering of functional aav capsids
TW202216244A (zh) 2020-07-13 2022-05-01 賓州大學委員會 有用於治療夏馬杜三氏病之組成物
JP2023536148A (ja) 2020-07-30 2023-08-23 シェイプ セラピューティクス インク. Raavウイルス粒子の誘導産生のための安定な細胞株
TW202227632A (zh) 2020-08-19 2022-07-16 美商史崔德生物公司 用於治療雷特症候群之腺相關病毒載體
MX2023002293A (es) 2020-08-24 2023-05-19 Univ Pennsylvania Vectores virales que codifican fusiones de agonistas del receptor de glp-1 y usos de estos en el tratamiento de enfermedades metabólica.
MX2023002364A (es) 2020-08-26 2023-05-22 Univ Pennsylvania Virus adenoasociado recombinante para el tratamiento de la neurodegeneración iniciada en adultos asociada a grn.
KR20230118075A (ko) 2020-10-09 2023-08-10 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 파브리병 치료를 위한 조성물 및 방법
TW202229315A (zh) 2020-10-18 2022-08-01 賓州大學委員會 改良的腺相關病毒(aav)載體及其用途
CA3199234A1 (en) * 2020-10-22 2022-04-28 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
EP4236975A1 (en) * 2020-10-29 2023-09-06 The Trustees of The University of Pennsylvania Aav capsids and compositions containing same
EP4236974A2 (en) * 2020-10-29 2023-09-06 RegenxBio Inc. Vectorized factor xii antibodies and administration thereof
TW202233584A (zh) 2020-11-11 2022-09-01 歐洲分子生物學實驗室 用於基因治療之經修飾病毒顆粒
EP4256065A2 (en) 2020-12-01 2023-10-11 The Trustees of The University of Pennsylvania 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
EP4284335A1 (en) 2021-02-01 2023-12-06 RegenxBio Inc. Gene therapy for neuronal ceroid lipofuscinoses
US20240191258A1 (en) 2021-04-12 2024-06-13 The Trustees Of The University Of Pennsylvania Compositions useful for treating spinal and bulbar muscular atrophy (sbma)
EP4334334A1 (en) 2021-04-23 2024-03-13 The Trustees of The University of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
AU2022274162A1 (en) 2021-05-12 2023-11-30 Fondazione Telethon Vector system
IL308787A (en) * 2021-06-08 2024-01-01 Univ Pennsylvania Recombinant adeno-associated viruses for Leish-Nihan disorders and their uses
TW202325845A (zh) 2021-10-02 2023-07-01 賓州大學委員會 新穎aav衣殼及含其之組成物
US20250295807A1 (en) 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
US20250026795A1 (en) 2021-12-01 2025-01-23 Shape Therapeutics Inc. Functional aav capsids for intravitreal administration
AU2022401548A1 (en) 2021-12-01 2024-07-04 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
WO2023133584A1 (en) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
TW202340467A (zh) 2022-01-10 2023-10-16 賓州大學委員會 有用於治療c9orf72介導之病症之組成物及方法
IL314434A (en) 2022-01-25 2024-09-01 Univ Pennsylvania AAV capsids for enhanced cardiac transduction and liver targeting neutralization
CA3247109A1 (en) 2022-04-06 2023-10-12 Univ Pennsylvania Compositions and treatment methods for metastatic breast cancer that is human epidermal growth factor receptor 2 positive and other cancers
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
US20250353883A1 (en) 2022-05-06 2025-11-20 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
EP4522757A2 (en) * 2022-05-13 2025-03-19 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
CN121127599A (zh) 2022-12-17 2025-12-12 宾夕法尼亚州大学信托人 具有心肌和骨骼肌特异性靶向基序的重组aav突变载体及含有其的组合物
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
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
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
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
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
PL310327A1 (en) 1993-02-12 1995-12-11 Univ Leland Stanford Junior Adjustable transcription of target genes and other biological processes
US5834266A (en) 1993-02-12 1998-11-10 President & Fellows Of Harvard College Regulated apoptosis
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
US20020173474A1 (en) 1993-02-12 2002-11-21 President And Fellows Of Harvard College Methods & materials involving dimerization-mediated regulation of biological events
US5830462A (en) 1993-02-12 1998-11-03 President & Fellows Of Harvard College Regulated transcription of targeted genes and other biological events
US5869337A (en) 1993-02-12 1999-02-09 President And 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
EP0776327B1 (en) 1994-08-18 2005-07-06 Ariad Gene Therapeutics, 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
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
CA2219080A1 (en) 1995-06-07 1996-12-27 Ariad Gene Therapeutics, Inc. Rapamycin-based regulation of biological events
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
US6506379B1 (en) 1995-06-07 2003-01-14 Ariad Gene Therapeutics, Inc. Intramuscular delivery of recombinant AAV
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
DE69739853D1 (de) 1996-02-28 2010-06-02 Ariad Pharma Inc Synthetische rapamycinderivate als multimerisierende wirkstoffe für chimere proteine mit von immunophilin abgeleiteten domänen
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
US6258823B1 (en) 1996-07-12 2001-07-10 Ariad Pharmaceuticals, Inc. Materials and method for treating or preventing pathogenic fungal infection
ATE364718T1 (de) 1997-04-01 2007-07-15 Solexa Ltd 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
JP2003524368A (ja) 1997-08-26 2003-08-19 アリアド ジーン セラピューティクス インコーポレイテッド ニ量化ドメイン、三量化ドメインまたは四量化ドメインおよび補足的非相同転写活性化ドメイン、転写抑制ドメイン、dna結合ドメインまたはリガンド結合ドメインを含む融合蛋白
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
AU752129B2 (en) 1997-08-27 2002-09-05 Ariad Gene Therapeutics, Inc. Chimeric transcriptional activators and compositions and uses related thereto
AU755784B2 (en) 1998-01-15 2002-12-19 Ariad Pharmaceuticals, 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
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
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus 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
AU780231B2 (en) 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
DE60039766D1 (de) 1999-08-09 2008-09-18 Targeted Genetics Corp Terologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen
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
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
CN1304578C (zh) 2000-03-22 2007-03-14 罗姆和哈斯公司 新的基于蜕皮激素受体的可诱导的基因表达系统
US20040033600A1 (en) 2001-03-21 2004-02-19 Palli Subba Reddy Ecdysone receptor-based inducible gene expression system
AU6814901A (en) 2000-06-01 2001-12-11 Univ North Carolina 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
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
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
EP1534738B1 (en) 2001-02-20 2012-06-20 Intrexon Corporation Novel substitution mutant receptors and their use in a nuclear receptor-based inducible gene expression system
AU2002247184B2 (en) 2001-02-20 2008-06-19 Intrexon Corporation Novel ecdysone receptor/invertebrate retinoid X receptor-based inducible gene expression system
EP1392868B2 (en) 2001-05-18 2013-09-04 Wisconsin Alumni Research Foundation Method for the synthesis of dna sequences using photo-labile linkers
CA2915124C (en) 2001-11-13 2018-08-14 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
ES2602352T3 (es) 2001-12-17 2017-02-20 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (VAA), vectores que las contienen y usos de las mismas
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
EP1660970A4 (en) 2003-08-01 2007-02-14 Dna Twopointo Inc SYSTEMS AND METHODS FOR BIOPOLYMER TECHNOLOGY
EP2298926A1 (en) 2003-09-30 2011-03-23 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
EP2357010B1 (en) 2005-04-07 2013-06-12 The Trustees of The University of Pennsylvania Method of increasing the function of an AAV vector
US7456683B2 (en) 2005-06-09 2008-11-25 Panasonic Corporation Amplitude error compensating device and quadrature skew error compensating device
DK2548560T3 (en) 2005-06-23 2015-07-13 Cold Spring Harbor Lab Compositions and Methods for Modulating 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
EP2018421B1 (en) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
ES2596885T3 (es) 2006-06-07 2017-01-12 Genzyme Corporation Terapia génica para esclerosis lateral amiotrófica y otros trastornos de la medula espinal
JP2010520748A (ja) 2007-02-20 2010-06-17 アナプティスバイオ インコーポレイティッド 体細胞超変異系
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
CA2684534C (en) 2007-04-17 2017-04-04 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
CN102089324B (zh) 2007-11-12 2014-04-16 特罗科隆科学有限公司 用于对流行性感冒进行治疗以及诊断的组合物以及方法
RU2010127156A (ru) 2007-12-06 2012-01-20 Дана-Фарбер Кэнсер Инститьют, Инк. (Us) Антитела против вируса гриппа и их применение
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
CN102216327A (zh) 2008-07-25 2011-10-12 生物医学研究所 中和抗甲型流感病毒抗体及其用途
US8871207B2 (en) 2008-07-25 2014-10-28 Humabs, LLC Neutralizing anti-influenza A virus antibodies and uses thereof
MX353900B (es) 2008-11-07 2018-02-01 Massachusetts Inst Technology Lipidoides de aminoalcohol y usos de los mismos.
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
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts 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)
SI3449926T1 (sl) 2009-06-17 2020-04-30 Biogen Ma Inc. Sestave in metode za modulacijo združevanja SMN2 pri subjektu
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
SG10201502270TA (en) 2010-03-29 2015-05-28 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
JP6091435B2 (ja) 2011-02-22 2017-03-08 カリフォルニア インスティチュート オブ テクノロジー アデノ随伴ウイルス(aav)ベクターを用いたタンパク質の送達
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
US20140206753A1 (en) 2011-06-08 2014-07-24 Shire Human Genetic Therapies, 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
JP5683752B2 (ja) 2011-07-14 2015-03-11 クルセル ホランド ベー ヴェー 系統グループ1及び系統グループ2のインフルエンザa型ウイルス、並びにインフルエンザb型ウイルスを中和することが可能なヒト結合分子
JP6170507B2 (ja) 2012-01-31 2017-07-26 国立大学法人大阪大学 B型インフルエンザウイルスに対する広範な保護を与えるヒトモノクローナル抗体及びその使用方法
EA028433B1 (ru) 2012-03-08 2017-11-30 Янссен Вэксинс Энд Превеншн Б.В. Антитело, связывающееся с вирусами гриппа b и его применение
WO2013155222A2 (en) 2012-04-10 2013-10-17 The Regents Of The University Of California Brain-specific enhancers for cell-based therapy
WO2013182683A1 (en) 2012-06-08 2013-12-12 Ethris Gmbh Pulmonary delivery of messenger rna
CN104704123A (zh) 2012-06-21 2015-06-10 肌肉学研究协会 基因治疗载体的广泛的基因递送
ES2791778T3 (es) 2012-08-01 2020-11-05 Ikaika Therapeutics Llc Mitigación del daño tisular y la fibrosis a través de anticuerpos anti-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
AU2013388083B2 (en) 2013-05-01 2019-08-22 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
KR20170047192A (ko) 2014-02-19 2017-05-04 조디 베리 에볼라 단클론성 항체
WO2015164778A1 (en) 2014-04-25 2015-10-29 The Trustees Of The University Of Pennysylvania Ldlr variants and their use in compositions for reducing cholesterol levels
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
WO2015164757A1 (en) * 2014-04-25 2015-10-29 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
SG11201609207SA (en) 2014-05-13 2016-12-29 Univ Pennsylvania Compositions comprising aav expressing dual antibody constructs and uses thereof
EP3800260A1 (en) * 2014-09-24 2021-04-07 City of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
WO2016054598A2 (en) 2014-10-03 2016-04-07 Massachusetts Institute Of Technology Antibodies that bind ebola glycoprotein and uses thereof
JP6772157B2 (ja) 2015-02-05 2020-10-21 ヤンセン ファッシンズ アンド プリベンション ベーフェーJanssen Vaccines & Prevention B.V. インフルエンザヘマグルチニンに対する結合分子及びその使用
CN108135994B (zh) 2015-05-13 2022-01-25 宾夕法尼亚州大学信托人 Aav-介导的抗-流感抗体的表达及其使用方法
WO2017075119A1 (en) 2015-10-28 2017-05-04 The Trustees Of The Univeresity 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
ES2934848T3 (es) 2015-12-11 2023-02-27 Univ Pennsylvania Método de purificación escalable para AAV8
WO2017100704A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
WO2017160360A2 (en) 2015-12-11 2017-09-21 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
US20190002915A1 (en) 2015-12-14 2019-01-03 The Trustees Of The University Of Pennsylvania Compositions and methods for regulatable antibody expression
WO2017106326A1 (en) 2015-12-14 2017-06-22 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
US10973929B2 (en) 2016-02-03 2021-04-13 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type I
CN110168080B (zh) * 2016-08-15 2024-05-31 建新公司 检测aav的方法
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
SG11201907611WA (en) * 2017-02-28 2019-09-27 Univ Pennsylvania Influenza vaccines based on aav vectors
US11827906B2 (en) 2017-02-28 2023-11-28 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clade f vector and uses therefor
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي

Also Published As

Publication number Publication date
FI3589730T3 (fi) 2024-02-22
BR112019017839A2 (pt) 2020-04-14
JP2025081544A (ja) 2025-05-27
DK3589730T3 (da) 2024-03-04
IL268644B2 (en) 2025-06-01
ZA201905407B (en) 2020-11-25
SG10201912973XA (en) 2020-03-30
LT3589730T (lt) 2024-03-12
HUE065885T2 (hu) 2024-06-28
SG11201907714UA (en) 2019-09-27
JP2020510429A (ja) 2020-04-09
PE20191362A1 (es) 2019-10-01
SG10201912976WA (en) 2020-02-27
EP3589730A1 (en) 2020-01-08
JP7754872B2 (ja) 2025-10-15
WO2018160582A8 (en) 2019-09-12
EP4272765A2 (en) 2023-11-08
KR102775350B1 (ko) 2025-03-05
CL2019002474A1 (es) 2019-11-29
SA519402565B1 (ar) 2023-12-05
PT3589730T (pt) 2024-02-28
MD3589730T2 (ro) 2024-04-30
AU2018227440A1 (en) 2019-08-29
CN110741082B (zh) 2024-09-06
SI3589730T1 (sl) 2024-04-30
EP4272765A3 (en) 2024-02-21
ES2971872T3 (es) 2024-06-10
EP3589730B1 (en) 2023-12-27
JP2023089134A (ja) 2023-06-27
CN118956781A (zh) 2024-11-15
IL268644A (en) 2019-10-31
PL3589730T3 (pl) 2024-09-02
AU2018227440B2 (en) 2024-06-06
RS65241B1 (sr) 2024-03-29
WO2018160582A1 (en) 2018-09-07
IL268644B1 (en) 2025-02-01
JP7455579B2 (ja) 2024-03-26
CA3053399A1 (en) 2018-09-07
SG10201912969XA (en) 2020-02-27
US20200056159A1 (en) 2020-02-20
CN110741082A (zh) 2020-01-31
US20240117322A1 (en) 2024-04-11
AU2024219391A1 (en) 2024-10-03
DK3589730T5 (da) 2024-09-02
HRP20240257T1 (hr) 2024-05-24
MX2019010275A (es) 2019-10-15
US11827906B2 (en) 2023-11-28
KR20190132639A (ko) 2019-11-28
MX2024012403A (es) 2024-11-08
SMT202400080T1 (it) 2024-05-14

Similar Documents

Publication Publication Date Title
CO2019009525A2 (es) Novedoso vector del subtipo f del virus adenoasociado (aav) y usos de este
CO2020004738A2 (es) Variantes de cápsides de virus adenoasociados y métodos de uso de estas
CL2019002280A1 (es) Vectores virales, adenovirales y composiciones a base de una secuencia de ornitina transcarbamilasa humana (hotch) modificada genéticamente y su uso en el tratamiento de deficiencia de la otc. (divisional solicitud 201602235)
CL2020002201A1 (es) Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidación reducida de la cápside y usos de estos.
CL2022003757A1 (es) Vectores de aav que tienen una desamidación reducida de la cápside y sus usos (divisional solic 202002200).
CL2020001360A1 (es) Cápsides variantes de virus adenoasociados y su uso para inhibir la angiogénesis.
UA124343C2 (uk) Капсиди аденоасоційованого вірусу і спосіб його використання
ZA202004980B (en) A modified raav capsid protein for gene therapy
MX2021012311A (es) Variantes de capsides de aav para liberacion intravitrea.
MX388331B (es) Variantes de virus adenoasociado y métodos de uso de estas
ES2629380T3 (es) Viriones de AAV con inmunorreactividad reducida y usos de los mismos
UY37620A (es) Vacunas contra la gripe mediadas por aav novedosas
RU2017142006A (ru) Векторы на основе аденоассоциированных вирусов для лечения мукополисахаридозов
AR110502A1 (es) Polinucleótidos y polipéptidos de adenovirus
EA201791936A1 (ru) Рекомбинантные конструкции glut1 аденоассоциированного вирусного вектора и способы восстановления экспрессии glut1 на их основе
CO2020002283A2 (es) Métodos de terapia génica del factor viii (fviii)
PH12022550109A1 (en) Modified aav capsid proteins for treatment of arthritic disease
PE20221254A1 (es) Proteina vp1 modificada aislada de la capside del virus adeno-asociado de serotipo 5 (aav5), capside y vector basado en la misma
CO2024006328A2 (es) Composiciones útiles en el tratamiento del trastorno por deficiencia de cdkl5 (cdd)
MX2022000038A (es) Virus del dengue atenuados.
MX2021008832A (es) Composiciones y metodos para la administracion de polipeptidos cftr.
CO2022016956A2 (es) Composiciones útiles en el tratamiento del trastorno de deficiencia de cdkl5 (cdd)
EA201992023A1 (ru) Вектор на основе аденоассоциированного вируса (aav) клады f и его применения
PE20241345A1 (es) Proteina vp1 modificada y aislada de la capside del virus adenoasociado del serotipo 5 (aav5), capside y vector basados en esta
WO2023250447A3 (en) Methods of selection and use of engineered cyclic peptides for enhancing viral production