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Withdrawn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Withdrawn legal-status Critical Current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)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Dermatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Urology & Nephrology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Immunology (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)
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 (10)

Country Link
US (2) US11117942B2 (enExample)
EP (1) EP3344650B1 (enExample)
JP (1) JP6877408B2 (enExample)
KR (1) KR20180057636A (enExample)
CN (1) CN108137664B (enExample)
AU (1) AU2016315699B2 (enExample)
CA (1) CA2995733A1 (enExample)
ES (1) ES3069921T3 (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
CA3068328A1 (en) * 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
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)
JP2022511882A (ja) * 2018-12-12 2022-02-01 キンドレッド バイオサイエンシズ インコーポレイテッド 獣医学的使用のためのエリスロポエチンアナログ
CA3200863A1 (en) * 2020-09-28 2022-03-31 Scout Bio, Inc. Composition and uses thereof
MX2023008826A (es) 2021-02-01 2023-09-15 Regenxbio Inc Terapia génica para lipofuscinosis neuronal ceroidea.
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
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
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
US5846528A (en) * 1996-01-18 1998-12-08 Avigen, Inc. Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
EP1007637B1 (en) 1997-04-14 2004-06-30 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
WO2000015772A2 (en) * 1998-09-16 2000-03-23 Nexia Biotechnologies, Inc. Recombinant protein production in urine
EP1135468B1 (en) 1998-11-10 2010-01-06 University Of North Carolina At Chapel Hill 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
US6596535B1 (en) 1999-08-09 2003-07-22 Targeted Genetics Corporation Metabolically activated recombinant viral vectors and methods for the preparation and use
AU6814901A (en) 2000-06-01 2001-12-11 Univ North Carolina Methods and compounds for controlled release of recombinant parvovirus vectors
CN103589692B (zh) 2001-11-13 2018-07-24 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
EP1668143B1 (en) 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
DE23193606T1 (de) 2005-04-07 2024-02-15 The Trustees Of The University Of Pennsylvania Verfahren zur erhöhung der funktion eines aav-vektors
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
US8278418B2 (en) * 2008-09-26 2012-10-02 Ambrx, Inc. Modified animal erythropoietin polypeptides and their uses
SI3988660T1 (sl) * 2009-05-02 2025-09-30 Genzyme Corporation Genska terapija za nevrodegenerativne motnje
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
WO2014052693A2 (en) * 2012-09-29 2014-04-03 The Trustees Of The University Of Pennsylvania Veterinary composition and methods for non-surgical neutering and castration
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
EP2994483A4 (en) * 2013-05-06 2017-02-08 Cell Machines, Inc. Methods and compositions related to large scale production of proteins

Also Published As

Publication number Publication date
EP3344650A4 (en) 2019-04-17
WO2017040524A1 (en) 2017-03-09
US20220056090A1 (en) 2022-02-24
US11117942B2 (en) 2021-09-14
EP3344650C0 (en) 2026-03-04
JP2018526994A (ja) 2018-09-20
US12624076B2 (en) 2026-05-12
ES3069921T3 (en) 2026-06-11
AU2016315699B2 (en) 2021-09-09
HK1257024A1 (zh) 2019-10-11
CA2995733A1 (en) 2017-03-09
US20180251508A1 (en) 2018-09-06
CN108137664B (zh) 2021-11-26
EP3344650B1 (en) 2026-03-04
EP3344650A1 (en) 2018-07-11
CN108137664A (zh) 2018-06-08
JP6877408B2 (ja) 2021-05-26
AU2016315699A1 (en) 2018-03-08

Similar Documents

Publication Publication Date Title
AU2016315699B2 (en) AAV-EPO for treating companion animals
AU2021261838A1 (en) Gene therapy for juvenile batten disease
KR102745604B1 (ko) Glp-1 및 대사성 질환 치료용 조성물에서 이의 용도
US20260053951A1 (en) Aav vector for treatment of friedreich's ataxia
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
US12522824B2 (en) AAV treatment of Huntington's disease
US20250154511A1 (en) Products and Methods for Treatment of Amyotrophic Lateral Sclerosis
CN115074369B (zh) 表达全长abca4基因的腺相关病毒载体及应用
AU2020392702B2 (en) Chimeric opsin GPCR proteins
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
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
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
WITN Application deemed withdrawn, e.g. because no request for examination was filed or no examination fee was paid