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1. (WO1998051285) CATIONIC AMPHIPHILE FORMULATIONS
Latest bibliographic data on file with the International Bureau

Pub. No.: WO/1998/051285 International Application No.: PCT/US1998/009974
Publication Date: 19.11.1998 International Filing Date: 15.05.1998
Chapter 2 Demand Filed: 14.12.1998
IPC:
A61K 9/127 (2006.01) ,A61K 48/00 (2006.01) ,C12N 15/88 (2006.01)
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9
Medicinal preparations characterised by special physical form
10
Dispersions; Emulsions
127
Liposomes
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
87
Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
88
using micro-encapsulation, e.g. using liposome vesicle
Applicants:
GENZYME CORPORATION [US/US]; One Mountain Road Framingham, MA 01701-9322, US
Inventors:
TOUSIGNANT, Jennifer, D.; US
MARSHALL, John; US
EASTMAN, Simon, J.; US
Agent:
GARRETT, Arthur, S.; Finnegan, Henderson, Farabow, Garrett & Dunner, L.L.P. 1300 I Street, N.W. Washington, DC 20005-3315 , US
Priority Data:
60/046,59915.05.1997US
Title (EN) CATIONIC AMPHIPHILE FORMULATIONS
(FR) FORMULATIONS AMPHIPHILES CATIONIQUES
Abstract:
(EN) 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.
(FR) Cette invention se rapporte à de nouvelles formulations d'amphiphiles cationiques qui facilitent le transport de molécules biologiquement actives (thérapeutiques) à l'intérieur de cellules. Ces formulations comportent un ou plusieurs amphiphiles cationiques, un co-lipide neutre tel qu'une diphytanoylphosphatidyléthanolamine, et un ou plusieurs dérivés de polyéthylène glycol. Les molécules thérapeutiques que l'on peut transporter à l'intérieur de cellules, conformément à cette invention, contiennent de l'ADN, de l'ARN et des polypeptides. Les utilisations possibles des formulations thérapeutiques de cette invention incluent, entre autres, certaines thérapies géniques et l'administration à des cellules de polynucléotides antisens ou de polypeptides biologiquement actifs.
Designated States: AU, CA, JP
European Patent Office (AT, BE, CH, CY, DE, DK, ES, FI, FR, GB, GR, IE, IT, LU, MC, NL, PT, SE)
Publication Language: English (EN)
Filing Language: English (EN)
Also published as:
AU1998074900