WO2002000680A3 - Cationic steroid derivatives for gene delivery - Google Patents

Cationic steroid derivatives for gene delivery Download PDF

Info

Publication number
WO2002000680A3
WO2002000680A3 PCT/GB2001/002859 GB0102859W WO0200680A3 WO 2002000680 A3 WO2002000680 A3 WO 2002000680A3 GB 0102859 W GB0102859 W GB 0102859W WO 0200680 A3 WO0200680 A3 WO 0200680A3
Authority
WO
WIPO (PCT)
Prior art keywords
gene delivery
steroid derivatives
cationic steroid
head group
cationic
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.)
Ceased
Application number
PCT/GB2001/002859
Other languages
French (fr)
Other versions
WO2002000680A2 (en
Inventor
Kam Man Hui
Hui Gao
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.)
NCC TECHNOLOGY Pte Ltd
STUART IAN
Original Assignee
NCC TECHNOLOGY Pte Ltd
STUART IAN
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 NCC TECHNOLOGY Pte Ltd, STUART IAN filed Critical NCC TECHNOLOGY Pte Ltd
Priority to AU2001266189A priority Critical patent/AU2001266189A1/en
Publication of WO2002000680A2 publication Critical patent/WO2002000680A2/en
Publication of WO2002000680A3 publication Critical patent/WO2002000680A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07J—STEROIDS
    • C07J41/00—Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring
    • C07J41/0033—Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005
    • C07J41/0055—Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005 the 17-beta position being substituted by an uninterrupted chain of at least three carbon atoms which may or may not be branched, e.g. cholane or cholestane derivatives, optionally cyclised, e.g. 17-beta-phenyl or 17-beta-furyl derivatives
    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/554—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being a steroid plant sterol, glycyrrhetic acid, enoxolone or bile acid
    • 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—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272—Non-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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07J—STEROIDS
    • C07J43/00—Normal steroids having a nitrogen-containing hetero ring spiro-condensed or not condensed with the cyclopenta(a)hydrophenanthrene skeleton
    • C07J43/003—Normal steroids having a nitrogen-containing hetero ring spiro-condensed or not condensed with the cyclopenta(a)hydrophenanthrene skeleton not condensed
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Dispersion Chemistry (AREA)
  • Botany (AREA)
  • Medicinal Preparation (AREA)
  • Steroid Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Novel lipids for forming liposomes for use as nucleic acid delivery vehicles, e.g. for gene therapy, have structure with a steroid (cholesteryl) domain linked via a carbamoyl linkage to a head group which is positively charged in use (generally through protonation). The head group preferably comprises a nitrogen heterocycle, e.g. a piperazine or morpholine ring system.
PCT/GB2001/002859 2000-06-26 2001-06-26 Cationic steroid derivatives for gene delivery Ceased WO2002000680A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2001266189A AU2001266189A1 (en) 2000-06-26 2001-06-26 Materials and methods relating to gene delivery

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GBGB0015624.0A GB0015624D0 (en) 2000-06-26 2000-06-26 Materials and methods relating to improved gene delivery
GB0015624.0 2000-06-26

Publications (2)

Publication Number Publication Date
WO2002000680A2 WO2002000680A2 (en) 2002-01-03
WO2002000680A3 true WO2002000680A3 (en) 2002-05-16

Family

ID=9894419

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/GB2001/002859 Ceased WO2002000680A2 (en) 2000-06-26 2001-06-26 Cationic steroid derivatives for gene delivery

Country Status (3)

Country Link
AU (1) AU2001266189A1 (en)
GB (1) GB0015624D0 (en)
WO (1) WO2002000680A2 (en)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE10109898A1 (en) * 2001-02-21 2002-09-05 Novosom Gmbh Variable charge lipids
CN117003808A (en) * 2022-04-28 2023-11-07 北京科兴中维生物技术有限公司 A cationic lipid compound and its preparation method and use
CN115487168A (en) * 2022-09-27 2022-12-20 浙江大学 A kind of lipid nanoparticle based on nitrogen-containing heterocyclic cholesterol derivative and its application
CN120227354A (en) * 2023-12-29 2025-07-01 康希诺(上海)生物研发有限公司 Lipid nanoparticles for delivering nucleic acids and preparation method and use thereof
WO2025140596A1 (en) * 2023-12-29 2025-07-03 康希诺(上海)生物研发有限公司 Ionizable cationic lipid compound for nucleic acid delivery and composition, and use thereof

Citations (5)

* 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
WO1996040726A1 (en) * 1995-06-07 1996-12-19 Genta Incorporated Novel carbamate-based cationic lipids
WO1997004748A2 (en) * 1995-08-01 1997-02-13 Advanced Therapies, Inc. Enhanced artificial viral envelopes for cellular delivery of therapeutic substances
WO1997039019A1 (en) * 1996-04-12 1997-10-23 University Of Pittsburgh Novel cationic cholesteryl derivatives containing cyclic polar groups
WO1998045317A1 (en) * 1997-04-04 1998-10-15 Genzyme Corporation Imidazole-containing cationic amphiphiles for intracellular delivery of therapeutic molecules

Patent Citations (5)

* 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
WO1996040726A1 (en) * 1995-06-07 1996-12-19 Genta Incorporated Novel carbamate-based cationic lipids
WO1997004748A2 (en) * 1995-08-01 1997-02-13 Advanced Therapies, Inc. Enhanced artificial viral envelopes for cellular delivery of therapeutic substances
WO1997039019A1 (en) * 1996-04-12 1997-10-23 University Of Pittsburgh Novel cationic cholesteryl derivatives containing cyclic polar groups
WO1998045317A1 (en) * 1997-04-04 1998-10-15 Genzyme Corporation Imidazole-containing cationic amphiphiles for intracellular delivery of therapeutic molecules

Also Published As

Publication number Publication date
WO2002000680A2 (en) 2002-01-03
AU2001266189A1 (en) 2002-01-08
GB0015624D0 (en) 2000-08-16

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