AU773834B2 - 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
AU773834B2
AU773834B2 AU20361/00A AU2036100A AU773834B2 AU 773834 B2 AU773834 B2 AU 773834B2 AU 20361/00 A AU20361/00 A AU 20361/00A AU 2036100 A AU2036100 A AU 2036100A AU 773834 B2 AU773834 B2 AU 773834B2
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AU
Australia
Prior art keywords
concentration
present
virion
sorbitol
tween
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
Application number
AU20361/00A
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English (en)
Other versions
AU2036100A (en
Inventor
Yero J. Espinoza
Hema S. Sista
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.)
Avigen Inc
Original Assignee
Avigen 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 Avigen Inc filed Critical Avigen Inc
Publication of AU2036100A publication Critical patent/AU2036100A/en
Application granted granted Critical
Publication of AU773834B2 publication Critical patent/AU773834B2/en
Anticipated expiration legal-status Critical
Expired legal-status Critical Current

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    • 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)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Dermatology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (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)
AU20361/00A 1998-12-03 1999-12-02 Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith Expired AU773834B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US11068998P 1998-12-03 1998-12-03
US60/110689 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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2004205308A Division AU2004205308A1 (en) 1998-12-03 2004-08-27 Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith

Publications (2)

Publication Number Publication Date
AU2036100A AU2036100A (en) 2000-06-19
AU773834B2 true AU773834B2 (en) 2004-06-10

Family

ID=22334360

Family Applications (2)

Application Number Title Priority Date Filing Date
AU20361/00A Expired AU773834B2 (en) 1998-12-03 1999-12-02 Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
AU2004205308A Abandoned AU2004205308A1 (en) 1998-12-03 2004-08-27 Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith

Family Applications After (1)

Application Number Title Priority Date Filing Date
AU2004205308A Abandoned AU2004205308A1 (en) 1998-12-03 2004-08-27 Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith

Country Status (9)

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

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2002229823B2 (en) * 2001-04-04 2007-02-15 Delsitech Oy Biodegradable carrier and method for preparation thereof
WO2003049763A1 (en) 2001-12-12 2003-06-19 Fh Faulding & Co Limited Composition for the preservation of 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
US8980610B2 (en) * 2008-11-20 2015-03-17 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
EP4008356A1 (en) 2016-03-31 2022-06-08 University of Cincinnati Methods and compositions for the treatment of als
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING NON-AGE-ASSOCIATED HEARING DEFICIENCY IN A HUMAN SUBJECT
WO2018212271A1 (ja) 2017-05-18 2018-11-22 国立大学法人京都大学 脊髄小脳変性症３６型の予防又は治療用組成物
CA3084802A1 (en) * 2017-12-05 2019-06-13 Applied Genetic Technologies Corporation Formulation optimization for viral particles
JP7630443B2 (ja) * 2019-04-19 2025-02-17 レジェンクスバイオ インコーポレーテッド アデノ随伴ウイルスベクター製剤及び方法
US20220325250A1 (en) 2019-08-14 2022-10-13 Leukocare Ag Method for obtaining efficient compositions comprising viral vectors for vaccination or gene therapy
WO2021168362A1 (en) 2020-02-21 2021-08-26 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject

Citations (2)

* 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
WO1998009524A1 (en) * 1996-09-06 1998-03-12 Chiron Corporation Methods and compositions for liver specific delivery of therapeutic molecules using recombinant aav vectors

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2751343B1 (fr) * 1996-07-16 1998-12-18 Transgene Sa Procede de conservation de virus recombinants infectieux, suspension aqueuse virale et utilisation comme medicament

Patent Citations (2)

* 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
WO1998009524A1 (en) * 1996-09-06 1998-03-12 Chiron Corporation Methods and compositions for liver specific delivery of therapeutic molecules using recombinant aav vectors

Also Published As

Publication number Publication date
PT1144009E (pt) 2003-01-31
AU2036100A (en) 2000-06-19
JP2002531418A (ja) 2002-09-24
CA2353417C (en) 2008-04-22
ES2185417T3 (es) 2003-04-16
DE69903666T2 (de) 2003-07-03
JP2011046748A (ja) 2011-03-10
JP2016040330A (ja) 2016-03-24
JP5663285B2 (ja) 2015-02-04
AU2004205308A1 (en) 2004-09-23
EP1144009B1 (en) 2002-10-23
ATE226449T1 (de) 2002-11-15
CA2353417A1 (en) 2000-06-08
EP1144009A3 (en) 2002-02-06
DE69903666D1 (de) 2002-11-28
EP1144009A2 (en) 2001-10-17
JP2014058578A (ja) 2014-04-03
WO2000032233A2 (en) 2000-06-08
WO2000032233A3 (en) 2001-10-18
JP2017206556A (ja) 2017-11-24

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MK14 Patent ceased section 143(a) (annual fees not paid) or expired