ATE237312T1 - In liposomen verkapselte nukleinsäurekomplexe - Google Patents

In liposomen verkapselte nukleinsäurekomplexe

Info

Publication number
ATE237312T1
ATE237312T1 AT99934114T AT99934114T ATE237312T1 AT E237312 T1 ATE237312 T1 AT E237312T1 AT 99934114 T AT99934114 T AT 99934114T AT 99934114 T AT99934114 T AT 99934114T AT E237312 T1 ATE237312 T1 AT E237312T1
Authority
AT
Austria
Prior art keywords
liposomes
nucleic acid
acid complexes
complexes encapsulated
encapsulated
Prior art date
Application number
AT99934114T
Other languages
English (en)
Inventor
Anthony Boey
Tao Chen
Esther Leng
Paul G Tard
Jeffrey J Wheeler
Peter Scherrer
Roger Graham
Original Assignee
Protiva Biotherapeutics 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 Protiva Biotherapeutics Inc filed Critical Protiva Biotherapeutics Inc
Application granted granted Critical
Publication of ATE237312T1 publication Critical patent/ATE237312T1/de

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    • 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
    • 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
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Dispersion Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AT99934114T 1998-07-20 1999-07-16 In liposomen verkapselte nukleinsäurekomplexe ATE237312T1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US9348198P 1998-07-20 1998-07-20
PCT/US1999/016166 WO2000003683A2 (en) 1998-07-20 1999-07-16 Liposomal encapsulated nucleic acid-complexes

Publications (1)

Publication Number Publication Date
ATE237312T1 true ATE237312T1 (de) 2003-05-15

Family

ID=22239187

Family Applications (1)

Application Number Title Priority Date Filing Date
AT99934114T ATE237312T1 (de) 1998-07-20 1999-07-16 In liposomen verkapselte nukleinsäurekomplexe

Country Status (7)

Country Link
EP (1) EP1096921B1 (de)
JP (1) JP2002520038A (de)
AT (1) ATE237312T1 (de)
AU (1) AU747597B2 (de)
CA (1) CA2335393C (de)
DE (1) DE69906977T2 (de)
WO (1) WO2000003683A2 (de)

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* Cited by examiner, † Cited by third party
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JP2001510457A (ja) * 1996-11-12 2001-07-31 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア インビボ送達に有効な、脂質―核酸複合体の安定な製剤の調製
US6210707B1 (en) * 1996-11-12 2001-04-03 The Regents Of The University Of California Methods of forming protein-linked lipidic microparticles, and compositions thereof
FR2759298B1 (fr) * 1997-02-10 1999-04-09 Rhone Poulenc Rorer Sa Formulation de particules agent(s) de transfection cationique(s)/acides nucleiques stabilisees

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CA2335393C (en) 2008-09-23
AU5001999A (en) 2000-02-07
DE69906977T2 (de) 2004-05-19
JP2002520038A (ja) 2002-07-09
EP1096921A1 (de) 2001-05-09
WO2000003683A2 (en) 2000-01-27
WO2000003683A3 (en) 2000-04-27
DE69906977D1 (de) 2003-05-22
EP1096921B1 (de) 2003-04-16
CA2335393A1 (en) 2000-01-27

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