CA2353417C - Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith - Google Patents

Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith Download PDF

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Publication number
CA2353417C
CA2353417C CA002353417A CA2353417A CA2353417C CA 2353417 C CA2353417 C CA 2353417C CA 002353417 A CA002353417 A CA 002353417A CA 2353417 A CA2353417 A CA 2353417A CA 2353417 C CA2353417 C CA 2353417C
Authority
CA
Canada
Prior art keywords
recombinant aav
virion
aav virion
concentration
present
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.)
Expired - Lifetime
Application number
CA002353417A
Other languages
English (en)
French (fr)
Other versions
CA2353417A1 (en
Inventor
Hema S. Sista
Yero J. Espinoza
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.)
Individual
Original Assignee
Genzyme Corp
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 Genzyme Corp filed Critical Genzyme Corp
Publication of CA2353417A1 publication Critical patent/CA2353417A1/en
Application granted granted Critical
Publication of CA2353417C publication Critical patent/CA2353417C/en
Anticipated expiration legal-status Critical
Expired - Lifetime legal-status Critical Current

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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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482—Serine endopeptidases (3.4.21)
    • A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • 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
    • 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
    • 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
    • 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
    • C12N2750/14151—Methods of production or purification of viral material

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Dermatology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
CA002353417A 1998-12-03 1999-12-02 Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith Expired - Lifetime CA2353417C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US11068998P 1998-12-03 1998-12-03
US60/110,689 1998-12-03
PCT/US1999/028460 WO2000032233A2 (en) 1998-12-03 1999-12-02 Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith

Publications (2)

Publication Number Publication Date
CA2353417A1 CA2353417A1 (en) 2000-06-08
CA2353417C true CA2353417C (en) 2008-04-22

Family

ID=22334360

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002353417A Expired - Lifetime CA2353417C (en) 1998-12-03 1999-12-02 Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith

Country Status (9)

Country Link
EP (1) EP1144009B1 (https=)
JP (5) JP2002531418A (https=)
AT (1) ATE226449T1 (https=)
AU (2) AU773834B2 (https=)
CA (1) CA2353417C (https=)
DE (1) DE69903666T2 (https=)
ES (1) ES2185417T3 (https=)
PT (1) PT1144009E (https=)
WO (1) WO2000032233A2 (https=)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002080977A1 (en) * 2001-04-04 2002-10-17 Delsitech Oy Biodegradable carrier and method for preparation thereof
EP1453536A4 (en) 2001-12-12 2009-08-26 Mayne Pharma Int Pty Ltd COMPOSITION FOR PRESERVING VIRUSES
US7261882B2 (en) 2003-06-23 2007-08-28 Reagents Of The University Of Colorado Methods for treating neuropathic pain by administering IL-10 polypeptides
LT2816118T (lt) 2005-05-31 2018-12-10 The Regents Of The University Of Colorado, A Body Corporate Genų pristatymo būdai
CA2742817A1 (en) * 2008-11-20 2010-05-27 Biogen Idec Ma Inc. Arginine inactivation of viruses
JP6348064B2 (ja) 2011-11-22 2018-06-27 ザ チルドレンズ ホスピタル オブ フィラデルフィア 効率の高いトランスジーン送達のためのウイルスベクター
EP3436078B1 (en) 2016-03-31 2021-10-27 University of Cincinnati Methods and compositions for the treatment of als
JP2019530737A (ja) 2016-08-23 2019-10-24 アコーオス インコーポレイテッド ヒト対象において非加齢性聴力障害を治療するための組成物および方法
EP3626821A4 (en) 2017-05-18 2021-03-03 Kyoto University COMPOSITION FOR THE PREVENTION OR TREATMENT OF SPINOCEREBEL ATAXIA TYPE 36
EP3720500A4 (en) * 2017-12-05 2021-08-25 Applied Genetic Technologies Corporation FORMULATION OPTIMIZATION FOR VIRAL PARTICLES
US20220143115A1 (en) * 2019-04-19 2022-05-12 Regenxbio Inc. Adeno-Associated Virus Vector Formulations and Methods
US20220325250A1 (en) 2019-08-14 2022-10-13 Leukocare Ag Method for obtaining efficient compositions comprising viral vectors for vaccination or gene therapy
JP7807380B2 (ja) 2020-02-21 2026-01-27 アコーオス インコーポレイテッド ヒト対象において非加齢関連聴力障害を処置するための組成物および方法
US20240066146A1 (en) * 2020-12-18 2024-02-29 Sangamo Therapeutics, Inc. Improved pharmaceutical compositions containing adeno-associated viral vector

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5616487A (en) * 1994-09-15 1997-04-01 Aastrom Biosciences, Inc. Stabilized retrovirus compositions
FR2751343B1 (fr) * 1996-07-16 1998-12-18 Transgene Sa Procede de conservation de virus recombinants infectieux, suspension aqueuse virale et utilisation comme medicament
US6521225B1 (en) * 1996-09-06 2003-02-18 Chiron Corporation AAV vectors

Also Published As

Publication number Publication date
JP2016040330A (ja) 2016-03-24
AU2004205308A1 (en) 2004-09-23
AU2036100A (en) 2000-06-19
JP2002531418A (ja) 2002-09-24
DE69903666D1 (de) 2002-11-28
JP2014058578A (ja) 2014-04-03
WO2000032233A2 (en) 2000-06-08
EP1144009B1 (en) 2002-10-23
DE69903666T2 (de) 2003-07-03
JP5663285B2 (ja) 2015-02-04
AU773834B2 (en) 2004-06-10
EP1144009A2 (en) 2001-10-17
ATE226449T1 (de) 2002-11-15
PT1144009E (pt) 2003-01-31
JP2011046748A (ja) 2011-03-10
JP2017206556A (ja) 2017-11-24
ES2185417T3 (es) 2003-04-16
WO2000032233A3 (en) 2001-10-18
CA2353417A1 (en) 2000-06-08
EP1144009A3 (en) 2002-02-06

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Effective date: 20191202