WO1998051285A3 - Cationic amphiphile formulations - Google Patents

Cationic amphiphile formulations Download PDF

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Publication number
WO1998051285A3
WO1998051285A3 PCT/US1998/009974 US9809974W WO9851285A3 WO 1998051285 A3 WO1998051285 A3 WO 1998051285A3 US 9809974 W US9809974 W US 9809974W WO 9851285 A3 WO9851285 A3 WO 9851285A3
Authority
WO
WIPO (PCT)
Prior art keywords
cells
therapeutic
formulations
invention include
biologically active
Prior art date
Application number
PCT/US1998/009974
Other languages
French (fr)
Other versions
WO1998051285A2 (en
Inventor
Jennifer D Tousignant
John Marshall
Simon J Eastman
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
Priority to AU74900/98A priority Critical patent/AU7490098A/en
Publication of WO1998051285A2 publication Critical patent/WO1998051285A2/en
Publication of WO1998051285A3 publication Critical patent/WO1998051285A3/en

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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • 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
    • 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

Landscapes

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

Abstract

Novel formulations of cationic amphiphiles are provided that facilitate transport of biologically active (therapeutic) molecules into cells. The formulations comprise one or more cationic amphiphiles, a neutral co-lipid such as diphytanoylphosphatidylethanolamine and one or more derivatives of polyethylene glycol. Therapeutic molecules that can be delivered into cells according to the practice of the invention include DNA, RNA, and polypeptides. Representative uses of the therapeutic compositions of the invention include providing gene therapy, and delivery of antisense polynucleotides or biologically active polypeptides to cells.
PCT/US1998/009974 1997-05-15 1998-05-15 Cationic amphiphile formulations WO1998051285A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU74900/98A AU7490098A (en) 1997-05-15 1998-05-15 Cationic amphiphile formulations

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US4659997P 1997-05-15 1997-05-15
US60/046,599 1997-05-15

Publications (2)

Publication Number Publication Date
WO1998051285A2 WO1998051285A2 (en) 1998-11-19
WO1998051285A3 true WO1998051285A3 (en) 1999-02-25

Family

ID=21944334

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1998/009974 WO1998051285A2 (en) 1997-05-15 1998-05-15 Cationic amphiphile formulations

Country Status (2)

Country Link
AU (1) AU7490098A (en)
WO (1) WO1998051285A2 (en)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9018187B2 (en) 2009-07-01 2015-04-28 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents

Families Citing this family (33)

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US6583301B1 (en) 1998-04-08 2003-06-24 Celltech R & D Limited Lipids
US6852334B1 (en) 1999-04-20 2005-02-08 The University Of British Columbia Cationic peg-lipids and methods of use
CA2370690A1 (en) * 1999-04-20 2000-10-26 The University Of British Columbia Cationic peg-lipids and methods of use
AUPQ259399A0 (en) 1999-09-01 1999-09-23 Lustre Investments Pte Ltd Therapeutic agents
US8916539B2 (en) 2000-01-10 2014-12-23 Yissum Research Development Company Of The Hebrew University Of Jerusalem Use of lipid conjugates in the treatment of disease
US8076312B2 (en) 2000-01-10 2011-12-13 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd Use of lipid conjugates in the treatment of disease
US7811999B2 (en) 2000-01-10 2010-10-12 Yissum Research Development Company Of The Hebrew University Of Jerusalem, Ltd. Use of lipid conjugates in the treatment of diseases
US7608598B2 (en) 2000-01-10 2009-10-27 Yissum Research Development Company Of The Hebrew University Of Jerusalem Use of lipid conjugates in the treatment of conjunctivitis
US7772196B2 (en) 2000-01-10 2010-08-10 Yissum Research Development Company Of The Hebrew University Of Jerusalem Use of lipid conjugates in the treatment of diseases
US7893226B2 (en) 2004-09-29 2011-02-22 Yissum Research Development Company Of The Hebrew University Of Jerusalem, Ltd. Use of lipid conjugates in the treatment of diseases
US8304395B2 (en) 2000-01-10 2012-11-06 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Lipid conjugates in the treatment of disease
US9040078B2 (en) 2000-01-10 2015-05-26 Yissum Research Development Company Of The Hebrew University Of Jerusalem Use of lipid conjugates in the treatment of diseases of the nervous system
US7141552B2 (en) * 2000-01-10 2006-11-28 Yissum Research Development Company Of The Hebrew University Of Jerusalem Use of lipid conjugates in the treatment of diseases
US8501701B2 (en) 2000-01-10 2013-08-06 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Use of lipid conjugates in the treatment of disease
US7189705B2 (en) 2000-04-20 2007-03-13 The University Of British Columbia Methods of enhancing SPLP-mediated transfection using endosomal membrane destabilizers
AU2001254548B2 (en) * 2000-04-20 2006-09-28 The University Of British Columbia Enhanced stabilised plasmid-lipid particle-mediated transfection using endosomal membrane
US8883761B2 (en) 2001-01-10 2014-11-11 Yissum Research Development Company Of The Hebrew University Of Jerusalem Use of lipid conjugates in the treatment of diseases associated with vasculature
DE60334618D1 (en) 2002-06-28 2010-12-02 Protiva Biotherapeutics Inc METHOD AND DEVICE FOR PREPARING LIPOSOMES
SG190613A1 (en) 2003-07-16 2013-06-28 Protiva Biotherapeutics Inc Lipid encapsulated interfering rna
JP4842821B2 (en) 2003-09-15 2011-12-21 プロチバ バイオセラピューティクス インコーポレイティッド Polyethylene glycol modified lipid compounds and uses thereof
HUE036916T2 (en) 2004-05-05 2018-08-28 Silence Therapeutics Gmbh Lipids, lipid complexes and use thereof
EP1766035B1 (en) 2004-06-07 2011-12-07 Protiva Biotherapeutics Inc. Lipid encapsulated interfering rna
JP5292572B2 (en) 2004-12-27 2013-09-18 サイレンス・セラピューティクス・アーゲー Coated lipid complexes and their use
US8859524B2 (en) 2005-11-17 2014-10-14 Yissum Research Development Company Of The Hebrew University Of Jerusalem Lipid conjugates in the treatment of chronic rhinosinusitis
US8906882B2 (en) 2005-11-17 2014-12-09 Yissum Research Development Company Of The Hebrew University Of Jerusalem Lipid conjugates in the treatment of allergic rhinitis
JP2009544281A (en) 2006-07-21 2009-12-17 サイレンス・セラピューティクス・アーゲー Means for inhibiting the expression of protein kinase 3
CA2705785A1 (en) 2006-11-14 2008-05-22 Saul Yedgar Use of lipid conjugates in the treatment of diseases or disorders of the eye
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
JP2012526819A (en) 2009-05-11 2012-11-01 イッサム リサーチ ディベロプメント カンパニー オブ ザ ヘブリュー ユニバーシティ オブ エルサレム リミテッド Lipid-polymer complex, its preparation and its use
JP5766188B2 (en) 2009-07-01 2015-08-19 プロチバ バイオセラピューティクス インコーポレイティッド Lipid formulations for delivering therapeutic agents to solid tumors
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
US9006417B2 (en) 2010-06-30 2015-04-14 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
US11591544B2 (en) 2020-11-25 2023-02-28 Akagera Medicines, Inc. Ionizable cationic lipids

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1996018372A2 (en) * 1994-12-09 1996-06-20 Genzyme Corporation Cationic amphiphiles and plasmids for intracellular delivery of therapeutic molecules

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1996018372A2 (en) * 1994-12-09 1996-06-20 Genzyme Corporation Cationic amphiphiles and plasmids for intracellular delivery of therapeutic molecules

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
E.K. WASAN ET AL.: "plasmid dna is protected against ultrasonic cavitation-induced damage when complexed to cationic liposomes", JOURNAL OF PHARMACEUTICAL SCIENCES, vol. 85, no. 4, April 1996 (1996-04-01), washington (us), pages 427 - 433, XP000558752 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9018187B2 (en) 2009-07-01 2015-04-28 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents

Also Published As

Publication number Publication date
AU7490098A (en) 1998-12-08
WO1998051285A2 (en) 1998-11-19

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