Drug – bio-affecting and body treating compositions – Designated organic active ingredient containing – Carbohydrate doai
Patent
1996-07-12
1999-09-07
Campell, Bruce R.
Drug, bio-affecting and body treating compositions
Designated organic active ingredient containing
Carbohydrate doai
435455, 435458, 4353201, 435325, A61K 4800
Patent
active
059487674
ABSTRACT:
Novel cationic amphiphiles are provided that facilitate transport of biologically active (therapeutic) molecules into cells. The amphiphiles contain lipophilic groups derived from steroids, from mono or dialkylamines, or from alkyl or acyl groups; and cationic groups, protonatable at physiological pH, derived from amines, alkylamines or polyalkylamines. There are provided also therapeutic compositions prepared typically by contacting a dispersion of one or more cationic amphiphiles with the therapeutic molecules. 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. With respect to therapeutic compositions for gene therapy, the DNA is provided typically in the form of a plasmid for complexing with the cationic amphiphile.
Novel and highly effective plasmid constructs are also disclosed, including those that are particularly effective at providing gene therapy for clinical conditions complicated by inflammation. Additionally, targeting of organs for gene therapy by intravenous administration of therapeutic compositions is described.
REFERENCES:
patent: 4394448 (1983-07-01), Szoka et al.
patent: 4544545 (1985-10-01), Ryan et al.
patent: 4897355 (1990-01-01), Eppstein et al.
patent: 4958013 (1990-09-01), Letsinger
patent: 4971803 (1990-11-01), Li et al.
patent: 5004737 (1991-04-01), Kim et al.
patent: 5171678 (1992-12-01), Behr et al.
patent: 5264618 (1993-11-01), Felgner et al.
patent: 5283185 (1994-02-01), Epand et al.
patent: 5328470 (1994-07-01), Nabel et al.
patent: 5334761 (1994-08-01), Gebeyehu et al.
patent: 5356633 (1994-10-01), Woodle et al.
patent: 5416203 (1995-05-01), Letsinger
patent: 5650096 (1997-07-01), Harris et al.
patent: 5661025 (1997-08-01), Szoka et al.
H. Ueno, "Adenovirus-Mediated Gene Transfer into Injured Arteries: Quantitative Analysis of Repeated Administration", Gerontology, 42(suppl. 1), 1996, pp. 25-36.
Gunzburg et al. (Mol. Medicine Today, 1 (9), pp. 410-417, 1995).
Ledley (Human Gene Ther. (1995) 6:1129-1144).
Mastrangelo et al. (Seminars in Oncology, 1996, vol. 23, 1:4-21).
Bennett et al., "Cholestrol Enhances Cationic Liposome-Mediated DNA Transfection of Human Respiratory Epithelial Cells," Bioscience Reports, vol. 15, No.1 (1995).
Li et al., "DC-Chol Lipid System in Gene Transfer," Journal of Controlled Release, 39, 373-381 (1996).
Behr et al., "Efficient Gene Transfer into Mammalian Primary Endocrine Cells with Lipopoly-amine-coated DNA," Proc. Natl. Acad. Sci., vol. 86, pp. 6982-6986 (1989).
Egilmez et al., "Evaluation and Optimization of Different Cationic Liposome Formulations for in Vivo-Gene Transfer," Biochemical and Biophysical Research Communication, 221, 169-173 (1996).
Guo et al., "Cationic Liposome Containing Noncytotoxic Phospholipid and Cholestrol Derivatives," Journal of Liposomes Research, 3(1), pp. 51-70 (1993).
Moradpour et al., "Efficient Gene Transfer into Mammalian Cells with Cholesteryl-Spermidine," Biochemical and Piophysical Research Communications, 221, pp. 82-88 (1986).
Takeuchi et al., "Effect of Zeta Potential of Cationic Liposomes Containing Cationic Cholestrol Derivatives on Gene Transfection," FEBS Letters, 397, pp. 207-209 (1996).
Liu et al., "New Cationic Lipid Formulations for Gene Transfer," Pharmaceuticals Research, vol. 13, No. 12 (1996).
Lee et al., "Detailed Analysis of Structures and Formulations of Cationinc Lipids for Efficient Gene Transfer to the Lung," Human Gene Therapy, 7, pp. 1701-1717 (1996).
Vigneron et al., "Guanidinium-cholestrol Cationic Lipids: Efficient Vectors for the Transfection of Eukaryotic Cells," Proc. Natl. Acad. Sci., vol. 93, pp. 9682-9686 (1996).
Weignberg, Robert A., "The Retinoblastoma Protein and Cell Cycle Control," Cell, vol. 81, pp. 323-330 (1995).
Zalipsky et al., FEBS Letter 353, 71-74, 1994.
Wheeler et al. (Biochim. et Biophsica Acta 1280, 1996, 1-11).
Remy et al. (Bioconjugate Chem., 1994, 5, 647-654).
Felgner et al. (J. Biol. Chem. 1994, vol. 269, 4:2550-2561).
Crystal, Science, vol. 270, pp. 404-409, 1995.
K. Patel et al., "Modification of Vesicle Surfaces with Amphiphilic Sterols. Effect on Permeability and In Vivo Tissue Distribution", Biochimica et Biophysica Acta, 814, 1985, pp. 256-264.
K. Moore et al., "Squalamine: An Aminosterol Antiobiotic from the Shark", Proceedings of the National Academy of Sciences USA, 90, 1993, pp. 1354-1358.
D. Litzinger et al., "Fate of Cationic Liposomes and their Complex with Oligonucleotide In Vivo", Biochimica et Biophysics Acta, 1281, 1996, pp. 139-149.
N. Egilmez et al., "Evaluation and Optimization of Different Cationic Liposome Formulations for In Vivo Gene Transfer", Biochemical and Biophysical Research Communications,221, 1996, pp. 169-173.
V. Dzau et al., "Fusigenic Viral Liposome for Gene Therapy in Cardiovascular Diseases", Proceedings of the National Academy of Sciences, USA, 93, 1996, pp. 11421-11425.
M. Tsukamoto et al., "Gene Transfer and Expression in Progeny after Intravenous DNA Injection into Pregnant Mice", Nature Genetics, 9, 1995, pp. 243-248.
A. Thierry et al., "Systemic Gene Therapy: Biodistribution and Long-term Expression of a Transgenic in Mice", Proceedings of the National Academy of Sciences, USA, 92, 1995, pp. 9742-9746.
R. Mahato et al., "In Vivo Disposition Characteristics of Plasmid DNA Complexed with Cationic Liposomes", Journal of Drug Targeting, 3, 1995, pp. 149-157.
D. Lew et al., "Cancer Gene Therapy Using Plasmid DNA: Pharmacokinetic Study of DNA Following Injection in Mice", Human Gene Therapy, 6, 1995, pp. 553-564.
N. Zhu et al., "Systemic Gene Expression After Intravenous DNA Delivery into Adult Mice", Science, 261, 1993, pp. 209-211.
E. Nabel et al., "Recombinant Fibroblast Growth Factor-1 Promotes Intimal Hyperplasia and Angiogenesis in Arteries In Vivo", Nature, 362, 1993, pp. 844-846.
E. Nabel et al., "Direct Transfer of Transforming growth Factor .beta.1 Gene into Arteries Stimulates Fibrocellular Hyperplasia", Proceedings of the National Academy of Sciences, USA, 90, 1993, pp. 10759-10763.
K. Brigham et al., "Expression of Human Growth Hormone Fusion Genes in Cultured Lung Endothelial Cells and in the Lungs of Mice", American Journal of Respiratory Cell and Molecular Biology, 8, 1993, pp. 209-213.
M. Stewart et al., "Gene Transfer In Vivo with DNA-Liposome Complexes: Safety and Acute Toxicity in Mice", Human Gene Therapy, 3, 1992, pp. 267-275.
E. Nabel et al., "Gene Transfer In Vivo with DNA-Liposome Complexes: Lack of Autoimmunity and Gonadal Localization", Human Gene Therapy, 3, 1992, pp. 649-656.
C. Lim et al., "Direct In Vivo Gene Transfer Into the Coronary and Peripheral Vasculatures of the Intact Dog", Circulation, 83, 1991, pp. 2007-2011.
E. Nabel et al., "Site-Specific Gene Expression in Vivo by Direct Gene Transfer into the Arterial Wall," Science, 249, 1990, pp. 1285-1288.
K. Brigham et al., "Rapid Communication: In vivo Transfection of Murine Lungs with a Functioning Prokaryotic Gene Using a Liposome Vehicle", The American Journal of the Medical Sciences, 298, 1989, pp. 278-281.
L. Feldman et al., "Perspectives Of Arterial Gene Therapy For the Prevention of Restenosis", Cardiovascular Research, 32, 1996, pp. 194-207.
D. Stephan, et al., "A New Cationic Liposome DNA Complex Enhances the Efficiency of Arterial Gene Transfer In Vivo", Human Gene Therapy, 7, 1996, pp. 1803-1812.
J. Zhu et al., "A Continuous Intracerebral Gene Delivery System for In Vivo Liposome-Mediated Gene Therapy", Gene Therapy, 3, 1996, pp. 472-476.
A. Canonico et al., "Aerosol and Intravenous Transfection of Human .alpha.1-Antitrypsin Gene to Lungs of Rabbits", American Journal of Respiratory Cell and Molecular Biology, 10, 1994, pp. 24-29.
Y. Liu et al., "Cationic Liposome-mediated Intravenous Gene Delivery", The Journal of Biological Chemistry, 270, 1995, pp. 24864-24870.
G. McLachlan et al., "Evaluation In Vitro and In Vivo of Cationic
Bagley Rebecca G.
Chang Chau-Dung
Cheng Seng H.
Eastman Simon J.
Harris David J.
Campell Bruce R.
Genzyme Corporation
Nguyen Dave Trong
LandOfFree
Cationic amphiphile/DNA complexes does not yet have a rating. At this time, there are no reviews or comments for this patent.
If you have personal experience with Cationic amphiphile/DNA complexes, we encourage you to share that experience with our LandOfFree.com community. Your opinion is very important and Cationic amphiphile/DNA complexes will most certainly appreciate the feedback.
Profile ID: LFUS-PAI-O-1804625