KR20180057636A - 반려 동물 치료용 aav-epo - Google Patents

반려 동물 치료용 aav-epo Download PDF

Info

Publication number
KR20180057636A
KR20180057636A KR1020187008958A KR20187008958A KR20180057636A KR 20180057636 A KR20180057636 A KR 20180057636A KR 1020187008958 A KR1020187008958 A KR 1020187008958A KR 20187008958 A KR20187008958 A KR 20187008958A KR 20180057636 A KR20180057636 A KR 20180057636A
Authority
KR
South Korea
Prior art keywords
epo
sequence
raav
aav
promoter
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.)
Withdrawn
Application number
KR1020187008958A
Other languages
English (en)
Korean (ko)
Inventor
크리스티안 힌더러
제임스 엠. 윌슨
매튜 윌슨
Original Assignee
더 트러스티스 오브 더 유니버시티 오브 펜실바니아
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 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 filed Critical 더 트러스티스 오브 더 유니버시티 오브 펜실바니아
Publication of KR20180057636A publication Critical patent/KR20180057636A/ko
Withdrawn legal-status Critical Current

Links

Images

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
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • 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
    • 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)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Dermatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Virology (AREA)
  • Microbiology (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020187008958A 2015-08-31 2016-08-30 반려 동물 치료용 aav-epo Withdrawn KR20180057636A (ko)

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 (1)

Publication Number Publication Date
KR20180057636A true KR20180057636A (ko) 2018-05-30

Family

ID=58188403

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020187008958A Withdrawn KR20180057636A (ko) 2015-08-31 2016-08-30 반려 동물 치료용 aav-epo

Country Status (9)

Country Link
US (2) US11117942B2 (enExample)
EP (1) EP3344650A4 (enExample)
JP (1) JP6877408B2 (enExample)
KR (1) KR20180057636A (enExample)
CN (1) CN108137664B (enExample)
AU (1) AU2016315699B2 (enExample)
CA (1) CA2995733A1 (enExample)
HK (1) HK1257024A1 (enExample)
WO (1) WO2017040524A1 (enExample)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020526514A (ja) * 2017-07-06 2020-08-31 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ｉ型を治療するための遺伝子治療
BR112021001498A2 (pt) * 2018-07-27 2021-04-27 Regenxbio Inc. vírus adeno-associado recombinante (raav), composição farmacêutica, polinucleotídeo, plasmídeo de raav, célula ex vivo, método de produção de um raav e métodos para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo iva (mps iva)
CN113423721A (zh) * 2018-12-12 2021-09-21 金德雷德生物科学股份有限公司 兽用促红细胞生成素类似物
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
EP4284335A1 (en) 2021-02-01 2023-12-06 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

Family Cites Families (36)

* 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
DE69824859T2 (de) 1997-04-14 2005-08-04 Cell Genesys, Inc., Foster City Methoden zur erhöhung der effizienz rekombinanter aav produkte
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
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
AU6814901A (en) 2000-06-01 2001-12-11 Univ North Carolina Methods and compounds for controlled release of recombinant parvovirus vectors
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
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
EP2357010B1 (en) 2005-04-07 2013-06-12 The Trustees of The University of Pennsylvania Method of increasing the function of an AAV vector
EP1736481A1 (en) 2005-05-13 2006-12-27 Charite Universitätsmedizin-Berlin Erythropoietin variants
US9988427B2 (en) * 2005-05-13 2018-06-05 Charite Universitaetsmedizen-Berlin Erythropoietin variants
US7456683B2 (en) 2005-06-09 2008-11-25 Panasonic Corporation Amplitude error compensating device and quadrature skew error compensating device
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University 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
BRPI0919403A2 (pt) * 2008-09-26 2017-09-26 Ambrx Inc polipeptídeos modificados de eritropoetina animal e seus usos
RS67077B1 (sr) * 2009-05-02 2025-08-29 Genzyme Corp Genska terapija za neurodegenerativne poremećaje
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
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
US20160207978A1 (en) * 2013-05-06 2016-07-21 Cell Machines Inc. Methods and compositions related to large scale production of proteins

Also Published As

Publication number Publication date
JP2018526994A (ja) 2018-09-20
AU2016315699B2 (en) 2021-09-09
CN108137664B (zh) 2021-11-26
AU2016315699A1 (en) 2018-03-08
US20180251508A1 (en) 2018-09-06
US20220056090A1 (en) 2022-02-24
WO2017040524A1 (en) 2017-03-09
JP6877408B2 (ja) 2021-05-26
CA2995733A1 (en) 2017-03-09
EP3344650A1 (en) 2018-07-11
EP3344650A4 (en) 2019-04-17
US11117942B2 (en) 2021-09-14
HK1257024A1 (zh) 2019-10-11
CN108137664A (zh) 2018-06-08

Similar Documents

Publication Publication Date Title
AU2016315699B2 (en) AAV-EPO for treating companion animals
AU2021261838A1 (en) Gene therapy for juvenile batten disease
CN111518175B (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
US11446395B2 (en) AAV vector for treatment of Friedreich's ataxia
US20250154511A1 (en) Products and Methods for Treatment of Amyotrophic Lateral Sclerosis
US11773392B2 (en) AAV treatment of Huntington's disease
CN111575248A (zh) 一种慢病毒、重组间充质干细胞及其构建方法和应用
CN115074369B (zh) 表达全长abca4基因的腺相关病毒载体及应用
AU2023241362A1 (en) Chimeric opsin GPCR proteins
CN113943749B (zh) 一种基于crispr基因编辑系统提高同源重组效率的方法
KR102570821B1 (ko) 재조합 바이러스 벡터 및 이를 이용한 약학 조성물
TW202227634A (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
US20220330533A1 (en) Systems and methods for batch cultivation of non-transgenic heterogametes
CN110423776B (zh) 表达重组凝血因子fix的载体及其构建方法和应用
TW202302858A (zh) 治療糖尿病之胰島素基因療法
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
KR20130059526A (ko) 인간 뼈 형성촉진효과를 위해 인간 ｂｍｐ2/7-ｐｔｄ 발현벡터를 통해 형질전환된 세포주 및 이 형질전환된 세포주를 이용한 3차원 세포 배양방법

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20180329

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
PC1203 Withdrawal of no request for examination