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WO2002000680A3 - Materiaux et procedes relatifs a l'administration de genes - Google Patents

Materiaux et procedes relatifs a l'administration de genes 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
Application number
PCT/GB2001/002859
Other languages
English (en)
Other versions
WO2002000680A2 (fr
Inventor
Kam Man Hui
Hui Gao
Original Assignee
Ncc Technology Pte Ltd
Stuart Ian
Kam Man Hui
Hui Gao
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, Kam Man Hui, Hui Gao filed Critical Ncc Technology Pte Ltd
Priority to AU2001266189A priority Critical patent/AU2001266189A1/en
Publication of WO2002000680A2 publication Critical patent/WO2002000680A2/fr
Publication of WO2002000680A3 publication Critical patent/WO2002000680A3/fr

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

Abstract

Nouveaux lipides pour former des liposomes destinés à être utilisés comme véhicules pour administrer des acides nucléiques, p.ex. en thérapie génique; ils possèdent une structure avec une domaine stéroïde (cholestéryle) lié à travers une liaison carbamoyle à un groupe de tête qui est positivement chargé pendant l'utilisation (en règle générale, par protonation). Le groupe de tête comprend de préférence un hétérocycle d'azote, p.ex. un système de noyau pipérazine ou morpholine.
PCT/GB2001/002859 2000-06-26 2001-06-26 Materiaux et procedes relatifs a l'administration de genes WO2002000680A2 (fr)

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 (fr) 2002-01-03
WO2002000680A3 true WO2002000680A3 (fr) 2002-05-16

Family

ID=9894419

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/GB2001/002859 WO2002000680A2 (fr) 2000-06-26 2001-06-26 Materiaux et procedes relatifs a l'administration de genes

Country Status (3)

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

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE10109898A1 (de) * 2001-02-21 2002-09-05 Novosom Gmbh Lipide mit veränderlicher Ladung
CN117003808A (zh) * 2022-04-28 2023-11-07 北京科兴中维生物技术有限公司 一种阳离子脂质化合物及其制备方法和用途
CN115487168A (zh) * 2022-09-27 2022-12-20 浙江大学 一种基于含氮杂环胆固醇衍生物的脂质纳米颗粒及其应用
WO2025140596A1 (fr) * 2023-12-29 2025-07-03 康希诺(上海)生物研发有限公司 Composé lipidique cationique ionisable pour l'administration et la composition d'acides nucléiques et son utilisation
WO2025140618A1 (fr) * 2023-12-29 2025-07-03 康希诺(上海)生物研发有限公司 Nanoparticule lipidique pour l'administration d'acide nucléique, son procédé de préparation et son utilisation

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1996018372A2 (fr) * 1994-12-09 1996-06-20 Genzyme Corporation Amphiphiles cationiques et plasmides destines a la liberation intracellulaire de molecules therapeutiques
WO1996040726A1 (fr) * 1995-06-07 1996-12-19 Genta Incorporated Nouveaux lipides cationiques a base de carbamate
WO1997004748A2 (fr) * 1995-08-01 1997-02-13 Advanced Therapies, Inc. Enveloppes virales artificielles renforcees pour l'apport de substances therapeutiques dans les cellules
WO1997039019A1 (fr) * 1996-04-12 1997-10-23 University Of Pittsburgh Nouveaux derives de cholesteryle cationiques contenant des groupes polaires cycliques
WO1998045317A1 (fr) * 1997-04-04 1998-10-15 Genzyme Corporation Amphiphiles cationiques contenant de l'imidazole, pour liberation intracellulaire de molecules therapeutiques

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1996018372A2 (fr) * 1994-12-09 1996-06-20 Genzyme Corporation Amphiphiles cationiques et plasmides destines a la liberation intracellulaire de molecules therapeutiques
WO1996040726A1 (fr) * 1995-06-07 1996-12-19 Genta Incorporated Nouveaux lipides cationiques a base de carbamate
WO1997004748A2 (fr) * 1995-08-01 1997-02-13 Advanced Therapies, Inc. Enveloppes virales artificielles renforcees pour l'apport de substances therapeutiques dans les cellules
WO1997039019A1 (fr) * 1996-04-12 1997-10-23 University Of Pittsburgh Nouveaux derives de cholesteryle cationiques contenant des groupes polaires cycliques
WO1998045317A1 (fr) * 1997-04-04 1998-10-15 Genzyme Corporation Amphiphiles cationiques contenant de l'imidazole, pour liberation intracellulaire de molecules therapeutiques

Also Published As

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

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