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 PDFInfo
- 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
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- 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
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Classifications
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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
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- 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
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- 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
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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
- 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
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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
- 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)
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
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
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| 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 (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)
| 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 |
Family Cites Families (3)
| 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 |
| EP0933997A4 (en) * | 1996-09-06 | 2000-08-16 | Chiron Corp | METHODS AND COMPOSITIONS FOR LIVER-SPECIFIC ADMINISTRATION OF THERAPEUTIC MOLECULES USING RECOMBINANT AAV VECTORS |
-
1999
- 1999-12-02 JP JP2000584922A patent/JP2002531418A/ja active Pending
- 1999-12-02 AT AT99964038T patent/ATE226449T1/de not_active IP Right Cessation
- 1999-12-02 CA CA002353417A patent/CA2353417C/en not_active Expired - Lifetime
- 1999-12-02 WO PCT/US1999/028460 patent/WO2000032233A2/en not_active Ceased
- 1999-12-02 EP EP99964038A patent/EP1144009B1/en not_active Expired - Lifetime
- 1999-12-02 PT PT99964038T patent/PT1144009E/pt unknown
- 1999-12-02 AU AU20361/00A patent/AU773834B2/en not_active Expired
- 1999-12-02 DE DE69903666T patent/DE69903666T2/de not_active Expired - Lifetime
- 1999-12-02 ES ES99964038T patent/ES2185417T3/es not_active Expired - Lifetime
-
2004
- 2004-08-27 AU AU2004205308A patent/AU2004205308A1/en not_active Abandoned
-
2010
- 2010-12-08 JP JP2010274133A patent/JP5663285B2/ja not_active Expired - Lifetime
-
2014
- 2014-01-06 JP JP2014000095A patent/JP2014058578A/ja not_active Withdrawn
-
2015
- 2015-12-11 JP JP2015241786A patent/JP2016040330A/ja not_active Withdrawn
-
2017
- 2017-08-18 JP JP2017157878A patent/JP2017206556A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| PT1144009E (pt) | 2003-01-31 |
| AU2036100A (en) | 2000-06-19 |
| JP2002531418A (ja) | 2002-09-24 |
| 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 |
| AU773834B2 (en) | 2004-06-10 |
| 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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Effective date: 20191202 |