AR118928A1 - Métodos de redosificación de vectores de terapia génica - Google Patents

Métodos de redosificación de vectores de terapia génica

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Publication number
AR118928A1
AR118928A1 ARP200101369A ARP200101369A AR118928A1 AR 118928 A1 AR118928 A1 AR 118928A1 AR P200101369 A ARP200101369 A AR P200101369A AR P200101369 A ARP200101369 A AR P200101369A AR 118928 A1 AR118928 A1 AR 118928A1
Authority
AR
Argentina
Prior art keywords
gene therapy
redosing
methods
therapy vectors
vector
Prior art date
Application number
ARP200101369A
Other languages
English (en)
Inventor
Peter Colosi
Justin Ishida
Silvia Ramirez
Original Assignee
Biomarin Pharm Inc
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 Biomarin Pharm Inc filed Critical Biomarin Pharm Inc
Publication of AR118928A1 publication Critical patent/AR118928A1/es

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    • 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
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0083Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the administration regime
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS 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
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14142Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/48Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/80Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85Vector systems having a special element relevant for transcription from vertebrates mammalian

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

La presente descripción se refiere, en general, a métodos para readministrar, o redosificar, a un sujeto que se ha sometido a un primer régimen de terapia génica con una segunda o posterior administración de un régimen de terapia génica, en el que el primer vector de terapia génica y el segundo vector de terapia génica comprenden diferentes cápsides de AAV pero portan un transgen o polinucleótido útil para tratar la misma enfermedad o trastorno.
ARP200101369A 2019-05-14 2020-05-13 Métodos de redosificación de vectores de terapia génica AR118928A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201962847908P 2019-05-14 2019-05-14

Publications (1)

Publication Number Publication Date
AR118928A1 true AR118928A1 (es) 2021-11-10

Family

ID=70919191

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP200101369A AR118928A1 (es) 2019-05-14 2020-05-13 Métodos de redosificación de vectores de terapia génica

Country Status (5)

Country Link
US (2) US20200362368A1 (es)
EP (1) EP3969061A1 (es)
AR (1) AR118928A1 (es)
TW (1) TW202104592A (es)
WO (1) WO2020232044A1 (es)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2019000962A (es) 2016-07-26 2019-08-01 Biomarin Pharm Inc Novedosas proteinas de la capside del virus adenoasociado.
AU2019265560A1 (en) 2018-05-09 2020-11-26 Biomarin Pharmaceutical Inc. Methods of treating phenylketonuria
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
EP4237545A1 (en) 2020-11-02 2023-09-06 BioMarin Pharmaceutical Inc. Process for enriching adeno-associated virus
WO2023034980A1 (en) 2021-09-03 2023-03-09 Bomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023034994A1 (en) 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023034990A1 (en) 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
EP4396199A1 (en) 2021-09-03 2024-07-10 BioMarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
EP4396202A1 (en) 2021-09-03 2024-07-10 BioMarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023034989A1 (en) 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2024064863A2 (en) 2022-09-22 2024-03-28 Biomarin Pharmaceutical Inc. Treatment of arrhythmogenic cardiomyopathy with aav gene therapy vectors
WO2024064856A1 (en) 2022-09-22 2024-03-28 Biomarin Pharmaceutical Inc. Treatment of cardiomyopathy with aav gene therapy vectors

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE3485810T2 (de) 1983-05-27 1992-12-10 Texas A & M University Syst Verfahren zur herstellung eines rekombinanten baculovirus-expressionsvektors.
US4745051A (en) 1983-05-27 1988-05-17 The Texas A&M University System Method for producing a recombinant baculovirus expression vector
DK518384A (da) 1984-01-31 1985-07-01 Idaho Res Found Vektor til fremstilling af et gen-produkt i insektceller, fremgangsmaade til dens fremstilling samt dens anvendelse
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
WO1998010088A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
JP4060531B2 (ja) * 1998-05-28 2008-03-12 アメリカ合衆国 Aav5ベクターおよびその使用
US6221349B1 (en) * 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
AU2001255575B2 (en) 2000-04-28 2006-08-31 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
WO2003074714A1 (en) 2002-03-05 2003-09-12 Stichting Voor De Technische Wetenschappen Baculovirus expression system
DK3211085T3 (da) 2003-09-30 2021-06-21 Univ Pennsylvania Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf
WO2006073496A2 (en) * 2004-07-30 2006-07-13 Targeted Genetics Corporation Recombinant aav based vaccine methods
CN101124328A (zh) * 2004-12-15 2008-02-13 北卡罗来纳查佩尔山大学 嵌合载体
EP2007795B1 (en) * 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
US7943379B2 (en) 2008-04-30 2011-05-17 Nationwide Children's Hospital, Inc. Production of rAAV in vero cells using particular adenovirus helpers
IL298604A (en) * 2016-04-15 2023-01-01 Univ Pennsylvania Novel aav8 mutant capsids and preparations containing them
MX2019000962A (es) 2016-07-26 2019-08-01 Biomarin Pharm Inc Novedosas proteinas de la capside del virus adenoasociado.
US20210228738A1 (en) * 2017-07-17 2021-07-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體

Also Published As

Publication number Publication date
TW202104592A (zh) 2021-02-01
US20200362368A1 (en) 2020-11-19
WO2020232044A1 (en) 2020-11-19
EP3969061A1 (en) 2022-03-23
US20240200096A1 (en) 2024-06-20

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