AU2016315699B2 - AAV-EPO for treating companion animals - Google Patents

AAV-EPO for treating companion animals Download PDF

Info

Publication number
AU2016315699B2
AU2016315699B2 AU2016315699A AU2016315699A AU2016315699B2 AU 2016315699 B2 AU2016315699 B2 AU 2016315699B2 AU 2016315699 A AU2016315699 A AU 2016315699A AU 2016315699 A AU2016315699 A AU 2016315699A AU 2016315699 B2 AU2016315699 B2 AU 2016315699B2
Authority
AU
Australia
Prior art keywords
epo
raav
seq
promoter
nucleic acid
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
AU2016315699A
Other languages
English (en)
Other versions
AU2016315699A1 (en
Inventor
Christian HINDERER
James M. Wilson
Matthew Wilson
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Pennsylvania Penn
Original Assignee
University of Pennsylvania Penn
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 University of Pennsylvania Penn filed Critical University of Pennsylvania Penn
Publication of AU2016315699A1 publication Critical patent/AU2016315699A1/en
Application granted granted Critical
Publication of AU2016315699B2 publication Critical patent/AU2016315699B2/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475—Growth factors; Growth regulators
    • C07K14/505—Erythropoietin [EPO]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Dermatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Urology & Nephrology (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2016315699A 2015-08-31 2016-08-30 AAV-EPO for treating companion animals Active AU2016315699B2 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562212144P 2015-08-31 2015-08-31
US62/212,144 2015-08-31
US201662336211P 2016-05-13 2016-05-13
US62/336,211 2016-05-13
PCT/US2016/049487 WO2017040524A1 (en) 2015-08-31 2016-08-30 Aav-epo for treating companion animals

Publications (2)

Publication Number Publication Date
AU2016315699A1 AU2016315699A1 (en) 2018-03-08
AU2016315699B2 true AU2016315699B2 (en) 2021-09-09

Family

ID=58188403

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2016315699A Active AU2016315699B2 (en) 2015-08-31 2016-08-30 AAV-EPO for treating companion animals

Country Status (9)

Country Link
US (2) US11117942B2 (https=)
EP (1) EP3344650B1 (https=)
JP (1) JP6877408B2 (https=)
KR (1) KR20180057636A (https=)
CN (1) CN108137664B (https=)
AU (1) AU2016315699B2 (https=)
CA (1) CA2995733A1 (https=)
HK (1) HK1257024A1 (https=)
WO (1) WO2017040524A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3068328A1 (en) * 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
EP3829619A4 (en) * 2018-07-27 2022-05-18 REGENXBIO Inc. TREATMENT OF IVA MUCOPOLYSACCHARIDOSIS
AU2019397516A1 (en) * 2018-12-12 2021-06-17 Elanco Us Inc. Erythropoietin analogs for veterinary use
CN116710148A (zh) * 2020-09-28 2023-09-05 斯克奥特比奥股份有限公司 组合物及其用途
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
KR20240107201A (ko) 2021-08-31 2024-07-08 스카우트 바이오, 인코포레이티드 항원-결합 분자 및 이의 용도

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010129021A1 (en) * 2009-05-02 2010-11-11 Genzyme Corporation Gene therapy for neurodegenerative disorders

Family Cites Families (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
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
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US5846528A (en) * 1996-01-18 1998-12-08 Avigen, Inc. Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
US6548286B1 (en) 1997-04-14 2003-04-15 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant AAV product
US6696411B1 (en) * 1998-04-22 2004-02-24 Cornell Research Foundation, Inc. Canine erythropoietin gene and recombinant protein
US6146874A (en) 1998-05-27 2000-11-14 University Of Florida Method of preparing recombinant adeno-associated virus compositions
JP2002525047A (ja) * 1998-09-16 2002-08-13 ネクシア バイオテクノロジーズ， インコーポレイテッド 尿における組換え蛋白質の産生
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
EP1520589A1 (en) 1998-12-17 2005-04-06 Pfizer Products Inc. Erythropoietin in the treatment of domestic and livestock animals for anaemia
CA2288341C (en) * 1998-12-17 2005-01-04 Pfizer Products Inc. Erythropoietin in the treatment of domestic and livestock animals for anemia
CA2379166C (en) 1999-08-09 2013-03-26 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs
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
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
US9988427B2 (en) * 2005-05-13 2018-06-05 Charite Universitaetsmedizen-Berlin Erythropoietin variants
EP1736481A1 (en) * 2005-05-13 2006-12-27 Charite Universitätsmedizin-Berlin Erythropoietin variants
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
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
WO2010011642A2 (en) 2008-07-21 2010-01-28 The Trustees Of The University Of Pennsylvania Methods and compositions using splicing regulatory proteins involved in tumor suppression
BR122012024318A2 (pt) * 2008-09-26 2019-07-30 Ambrx, Inc. Polipeptídeos modificados de eritropoetina animal e seus usos
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
CA2886161A1 (en) * 2012-09-29 2014-04-03 The Trustees Of The University Of Pennsylvania Veterinary composition and methods for non-surgical neutering and castration
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
WO2014182684A2 (en) * 2013-05-06 2014-11-13 Cell Machines, Inc. Methods and compositions related to large scale production of proteins

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010129021A1 (en) * 2009-05-02 2010-11-11 Genzyme Corporation Gene therapy for neurodegenerative disorders

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
BEALL et al., "Transfer of the feline erythropoietin gene to cats using a recombinant adeno-associated virus vector.", Gene Ther., (2000), vol. 7, no. 6, pages 534 - 9 *
WALKER MARK C ET AL, "Expression of erythropoietin in cats treated with a recombinant adeno-associated viral vector.", AMERICAN JOURNAL OF VETERINARY RESEARCH, (2005-03), vol. 66, no. 3, ISSN 0002-9645, pages 450 - 456 *

Also Published As

Publication number Publication date
CN108137664B (zh) 2021-11-26
AU2016315699A1 (en) 2018-03-08
CA2995733A1 (en) 2017-03-09
CN108137664A (zh) 2018-06-08
EP3344650A1 (en) 2018-07-11
US11117942B2 (en) 2021-09-14
HK1257024A1 (zh) 2019-10-11
EP3344650A4 (en) 2019-04-17
WO2017040524A1 (en) 2017-03-09
US20180251508A1 (en) 2018-09-06
EP3344650B1 (en) 2026-03-04
JP2018526994A (ja) 2018-09-20
JP6877408B2 (ja) 2021-05-26
KR20180057636A (ko) 2018-05-30
US20220056090A1 (en) 2022-02-24

Similar Documents

Publication Publication Date Title
AU2016315699B2 (en) AAV-EPO for treating companion animals
AU2021261838A1 (en) Gene therapy for juvenile batten disease
US20260053951A1 (en) Aav vector for treatment of friedreich's ataxia
CN112695057B (zh) Sars-cov-2抗原多肽及其重组腺相关病毒和在制备疫苗中的应用
KR102745604B1 (ko) Glp-1 및 대사성 질환 치료용 조성물에서 이의 용도
US20250154511A1 (en) Products and Methods for Treatment of Amyotrophic Lateral Sclerosis
US12522824B2 (en) AAV treatment of Huntington's disease
KR20220111294A (ko) 키메라 옵신 gpcr 단백질
CN115074369B (zh) 表达全长abca4基因的腺相关病毒载体及应用
CN111575248A (zh) 一种慢病毒、重组间充质干细胞及其构建方法和应用
CN113943749B (zh) 一种基于crispr基因编辑系统提高同源重组效率的方法
TW202541851A (zh) 表現γ-肌聚醣之腺相關病毒載體之全身性遞送及肌肉失養症之治療
TW202302858A (zh) 治療糖尿病之胰島素基因療法
CN110423776B (zh) 表达重组凝血因子fix的载体及其构建方法和应用

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)