WO2005067632A3 - Compositions lipidiques et leurs utilisations - Google Patents

Compositions lipidiques et leurs utilisations Download PDF

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Publication number
WO2005067632A3
WO2005067632A3 PCT/US2005/000418 US2005000418W WO2005067632A3 WO 2005067632 A3 WO2005067632 A3 WO 2005067632A3 US 2005000418 W US2005000418 W US 2005000418W WO 2005067632 A3 WO2005067632 A3 WO 2005067632A3
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WO
WIPO (PCT)
Prior art keywords
cell
composition
transfection
agent
provides
Prior art date
Application number
PCT/US2005/000418
Other languages
English (en)
Other versions
WO2005067632A2 (fr
Inventor
Moghis U Ahmad
Saifuddin Sheikh
Shoukath Ali
Pei-Yu Chien
Haris Jamil
Jia-Ai Zhang
Sydney Ugwu
Zhi-Yi Zhang
Jinkang Wang
Shastri Bhamidipati
Zafeer Ahmad
Imran Ahmad
Original Assignee
Neopharm Inc
Moghis U Ahmad
Saifuddin Sheikh
Shoukath Ali
Pei-Yu Chien
Haris Jamil
Jia-Ai Zhang
Sydney Ugwu
Zhi-Yi Zhang
Jinkang Wang
Shastri Bhamidipati
Zafeer Ahmad
Imran Ahmad
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 Neopharm Inc, Moghis U Ahmad, Saifuddin Sheikh, Shoukath Ali, Pei-Yu Chien, Haris Jamil, Jia-Ai Zhang, Sydney Ugwu, Zhi-Yi Zhang, Jinkang Wang, Shastri Bhamidipati, Zafeer Ahmad, Imran Ahmad filed Critical Neopharm Inc
Priority to EP05705189A priority Critical patent/EP1742661A2/fr
Priority to CA002559352A priority patent/CA2559352A1/fr
Priority to JP2006549412A priority patent/JP2007524667A/ja
Priority to AU2005204689A priority patent/AU2005204689A1/en
Publication of WO2005067632A2 publication Critical patent/WO2005067632A2/fr
Publication of WO2005067632A3 publication Critical patent/WO2005067632A3/fr

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/13—Amines
    • A61K31/14—Quaternary ammonium compounds, e.g. edrophonium, choline
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/35—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom
    • A61K31/352—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom condensed with carbocyclic rings, e.g. methantheline 
    • A61K31/353—3,4-Dihydrobenzopyrans, e.g. chroman, catechin
    • A61K31/355—Tocopherols, e.g. vitamin E
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/575—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of three or more carbon atoms, e.g. cholane, cholestane, ergosterol, sitosterol
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/66—Phosphorus compounds
    • A61K31/683—Diesters of a phosphorus acid with two hydroxy compounds, e.g. phosphatidylinositols
    • A61K31/685—Diesters of a phosphorus acid with two hydroxy compounds, e.g. phosphatidylinositols one of the hydroxy compounds having nitrogen atoms, e.g. phosphatidylserine, lecithin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K48/0008—Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • 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
    • A61K48/0008—Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • 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
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1135—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against oncogenes or tumor suppressor genes
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Oncology (AREA)
  • Dispersion Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)

Abstract

La présente invention a trait à une composition apte à être utilisée comme agent de transfection, comportant un analogue de cardiolipine cationique et, une autre espèce de lipide. La composition de la présente invention peut faciliter la transfection d'une grande variété d'espèces de polynucléotides (par exemple, des oligodésoxyribonucléotides, des plasmides, des espèces d'ARNi et autres). En outre l'agent de transfection de la présente invention est efficace dans la promotion de transfection de cultures de cellules primaires ainsi que des cellules transformées. L'agent de transfection est également apte à une utilisation in vitro et in vivo. La composition de l'invention a également d'autres utilisations, telle que sous la forme de distributeur d'agents actifs, des utilisations dermatologiques et cosmétiques, et des applications dans l'agriculture. L'invention a trait en outre à un procédé d'introduction d'un agent actif dans une cellule par la mise en contact de la cellule avec la composition de l'invention. L'invention a également trait à un procédé d'inhibition de la croissance de cellules néoplasiques et à un procédé de traitement d'un patient souffrant d'une maladie néoplasique par l'utilisation de la composition de l'invention, dans lequel un agent actif est un agent antinéoplasique. L'invention a également trait à un procédé de validation d'une cible génétique comprenant (a) l'administration à une cellule d'une composition comportant un liposome cationique et un ARNsi, grâce auquel l'ARNsi pénètre dans la cellule pour l'inhibition de l'expression d'un gène à l'intérieur de la cellule et (b) le dosage pour l'inhibition du gène. Le procédé comprend également un analogue de cardiolipine cationique fluorescent/luminescent et des compositions comportant de tels analogues. Grâce à l'utilisation d'un analogue de cardiolipine cationique, l'invention fournit un procédé de repérage de la migration d'une substance lipidique chez un animal.
PCT/US2005/000418 2004-01-07 2005-01-07 Compositions lipidiques et leurs utilisations WO2005067632A2 (fr)

Priority Applications (4)

Application Number Priority Date Filing Date Title
EP05705189A EP1742661A2 (fr) 2004-01-07 2005-01-07 Compositions lipidiques et leurs utilisations
CA002559352A CA2559352A1 (fr) 2004-01-07 2005-01-07 Compositions lipidiques et leurs utilisations
JP2006549412A JP2007524667A (ja) 2004-01-07 2005-01-07 脂質組成物及びその使用
AU2005204689A AU2005204689A1 (en) 2004-01-07 2005-01-07 Lipid compositions and use thereof

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US53504204P 2004-01-07 2004-01-07
US60/535,042 2004-01-07
US55684304P 2004-03-27 2004-03-27
US60/556,843 2004-03-27
US55723204P 2004-03-29 2004-03-29
US60/557,232 2004-03-29

Publications (2)

Publication Number Publication Date
WO2005067632A2 WO2005067632A2 (fr) 2005-07-28
WO2005067632A3 true WO2005067632A3 (fr) 2009-04-02

Family

ID=34799610

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2005/000418 WO2005067632A2 (fr) 2004-01-07 2005-01-07 Compositions lipidiques et leurs utilisations

Country Status (5)

Country Link
EP (1) EP1742661A2 (fr)
JP (1) JP2007524667A (fr)
AU (1) AU2005204689A1 (fr)
CA (1) CA2559352A1 (fr)
WO (1) WO2005067632A2 (fr)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8815599B2 (en) 2004-06-01 2014-08-26 Pronai Therapeutics, Inc. Methods and compositions for the inhibition of gene expression
WO2007065017A2 (fr) * 2005-12-01 2007-06-07 Pronai Therapeutics, Inc. Systeme d'administration de liposomes cationiques a oligonucleotides
EP1957044B1 (fr) 2005-12-01 2013-03-13 Pronai Therapeutics, Inc. Formulation de liposomes amphoteres
EP2069500B1 (fr) * 2006-10-04 2014-09-24 Centre National De La Recherche Scientifique (Cnrs) COMPOSITIONS INCLUANT UN siARN ET DES DÉRIVÉS LIPIDIQUES D'AMINOGLYCOSIDE À NOYAU 2-DÉSOXYSTREPTAMINE 4,5-DISUBSTITUÉE ET LEURS UTILISATIONS
WO2008109365A1 (fr) * 2007-03-02 2008-09-12 Mdrna, Inc. Composés d'acide nucléique permettant d'inhiber l'expression de gène raf1 et utilisations de ceux-ci
PT2992874T (pt) 2013-04-30 2018-11-09 Delta Fly Pharma Inc Lipossoma para administração tópica e aplicação do mesmo
EP3213755B1 (fr) * 2014-10-30 2020-06-24 Delta-Fly Pharma, Inc. Nouveau procédé de fabrication de lipoplexe pour une administration par voie locale, et médicament antitumoral utilisant le lipoplexe
KR101846090B1 (ko) * 2016-09-19 2018-04-06 동국제약 주식회사 독소루비신 함유 페길화된 리포좀 제제의 제조방법

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030108923A1 (en) * 2000-03-30 2003-06-12 Whitehead Institute For Biomedical Research RNA sequence-specific mediators of RNA interference

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030108923A1 (en) * 2000-03-30 2003-06-12 Whitehead Institute For Biomedical Research RNA sequence-specific mediators of RNA interference

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
MARCUSSON ET AL., NUCL. ACIDS RES., vol. 26, no. 8, pages 2016 - 2023, XP008112625 *

Also Published As

Publication number Publication date
EP1742661A2 (fr) 2007-01-17
WO2005067632A2 (fr) 2005-07-28
JP2007524667A (ja) 2007-08-30
AU2005204689A2 (en) 2005-07-28
CA2559352A1 (fr) 2005-07-28
AU2005204689A1 (en) 2005-07-28

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