Title:
THERAPEUTIC NANOSPHERES
Document Type and Number:
WIPO Patent Application WO1998056370
Kind Code:
A3
Abstract:
4-Phenylbutyrate exerts many beneficial biological effects. It appears to induce the transcription of certain promoters, as well as having a remedial effect on proteins which are aberrantly localized within the cell. In addition, it appears to cause cells to developmentally differentiate. The present invention provides nanosphere formulations of 4-phenylbutyrate and other drugs which remediate defective protein localization intracellularly. These formulations permit lower concentrations of drugs to be administered, providing both cost and safety benefits.
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Inventors:
WALSH SCOTT (US)
RUBENSTEIN RONALD (US)
ZEITLIN PAMELA (US)
LEONG KAM (US)
RUBENSTEIN RONALD (US)
ZEITLIN PAMELA (US)
LEONG KAM (US)
Application Number:
PCT/US1998/011880
Publication Date:
April 01, 1999
Filing Date:
June 11, 1998
Export Citation:
Assignee:
UNIV JOHNS HOPKINS MED (US)
WALSH SCOTT (US)
RUBENSTEIN RONALD (US)
ZEITLIN PAMELA (US)
LEONG KAM (US)
WALSH SCOTT (US)
RUBENSTEIN RONALD (US)
ZEITLIN PAMELA (US)
LEONG KAM (US)
International Classes:
A61K9/51; A61K31/19; A61K31/192; A61K31/352; A61K31/435; A61K31/52; A61K45/06; A61K47/30; A61K47/36; A61K47/42; A61K47/48; A61K48/00; A61P7/00; A61P11/00; A61P35/00; A61P43/00; (IPC1-7): A61K31/19; A61K9/51
Domestic Patent References:
| WO1997046588A1 | 1997-12-11 | |||
| WO1994004671A1 | 1994-03-03 | |||
| WO1996029998A1 | 1996-10-03 |
Other References:
WALSH S M ET AL: "COMBINATION OF DRUG AND GENE DELIVERY BY GELATIN NANOSPHERES FOR THE TREATMENT OF CYSTIC FIBROSIS", PROCEEDINGS OF THE 24TH. INTERNATIONAL SYMPOSIUM ON CONTROLLED RELEASE OF BIOACTIVE MATERIALS, STOCKHOLM, JUNE 15 - 19, 1997, no. SYMP. 24, 15 June 1997 (1997-06-15), CONTROLLED RELEASE SOCIETY, pages 75/76, XP002052415
RUBENSTEIN, R.C., ET AL.: "In vitro pharmacologic restoration of CFTR-mediated chloride transport with sodium 4-phenylbutyrate in cystic fibrosis epithelial cells containing delta.F508-CFTR", J. CLIN. INVEST., vol. 100, no. 10, 15 November 1997 (1997-11-15), pages 2457 - 2465, XP002076381
KO Y H ET AL: "THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR OVEREXPRESSION, PURIFICATION, AND CHARACTERIZATION OF WILD TYPE ANDDELTAF508 MUTANT FORMS OF THE FIRST NUCLEOTIDE BINDING FOLD IN FUSION WITH THE MALTOSE-BINDING PROTEIN", J. BIOL. CHEM., vol. 268, no. 32, 15 November 1993 (1993-11-15), pages 24330 - 24338, XP002055212
NEWMARK H L ET AL: "BUTYRATE AS A DIFFERENTIATING AGENT: PHARMACOKINETICS, ANALOGUES AND CURRENT STATUS", CANCER LETTERS, vol. 78, 1994, pages 1 - 5, XP000615955
MAO H -Q ET AL: "DNA-CHITOSAN NANOSPHERES FOR GENE DELIVERY", PROCEEDINGS OF THE INTERNATIONAL SYMPOSIUM ON CONTROLLED RELEASE BIOACTIVE MATERIALS, 7 July 1996 (1996-07-07), pages 401/402, XP002052414
TRUONG V L ET AL: "A TARGET-SPECIFIC MICROSPHERES DRUG DELIVERY SYSTEM MADE OF ENZYMATICALLY DEGRADEABLE GELATIN AND CHONDROITIN SULFATE COACERVATES", PROCEEDINGS OF THE INTERNATIONAL SYMPOSIUM ON CONTROLLED RELEASE BIOACTIVE MATERIALS, vol. 20, 25 July 1993 (1993-07-25), pages 474/475, XP002016479
GALLET X ET AL: "PREDICTION OF THE ANTIGENIC SITES OF THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR PROTEIN BY MOLECULAR MODELLING", PROTEIN ENGINEERING, vol. 8, no. 8, 1 August 1995 (1995-08-01), pages 829 - 834, XP000565688
ZABNER J ET AL: "REPEAT ADMINISTRATION OF AN DENOVIRUS VECTOR ENCODING CYSTIC FIBROSIS TRANSMEMBRANCE CONDUCTANCE REGULATOR TO THE NASAL EPITHELIUM OF PATIENTS WITH CYSTIC FIBROSIS", JOURNAL OF CLINICAL INVESTIGATION, vol. 97, no. 6, 15 March 1996 (1996-03-15), pages 1504 - 1511, XP000575871
RUBENSTEIN, R.C., ET AL.: "In vitro pharmacologic restoration of CFTR-mediated chloride transport with sodium 4-phenylbutyrate in cystic fibrosis epithelial cells containing delta.F508-CFTR", J. CLIN. INVEST., vol. 100, no. 10, 15 November 1997 (1997-11-15), pages 2457 - 2465, XP002076381
KO Y H ET AL: "THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR OVEREXPRESSION, PURIFICATION, AND CHARACTERIZATION OF WILD TYPE ANDDELTAF508 MUTANT FORMS OF THE FIRST NUCLEOTIDE BINDING FOLD IN FUSION WITH THE MALTOSE-BINDING PROTEIN", J. BIOL. CHEM., vol. 268, no. 32, 15 November 1993 (1993-11-15), pages 24330 - 24338, XP002055212
NEWMARK H L ET AL: "BUTYRATE AS A DIFFERENTIATING AGENT: PHARMACOKINETICS, ANALOGUES AND CURRENT STATUS", CANCER LETTERS, vol. 78, 1994, pages 1 - 5, XP000615955
MAO H -Q ET AL: "DNA-CHITOSAN NANOSPHERES FOR GENE DELIVERY", PROCEEDINGS OF THE INTERNATIONAL SYMPOSIUM ON CONTROLLED RELEASE BIOACTIVE MATERIALS, 7 July 1996 (1996-07-07), pages 401/402, XP002052414
TRUONG V L ET AL: "A TARGET-SPECIFIC MICROSPHERES DRUG DELIVERY SYSTEM MADE OF ENZYMATICALLY DEGRADEABLE GELATIN AND CHONDROITIN SULFATE COACERVATES", PROCEEDINGS OF THE INTERNATIONAL SYMPOSIUM ON CONTROLLED RELEASE BIOACTIVE MATERIALS, vol. 20, 25 July 1993 (1993-07-25), pages 474/475, XP002016479
GALLET X ET AL: "PREDICTION OF THE ANTIGENIC SITES OF THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR PROTEIN BY MOLECULAR MODELLING", PROTEIN ENGINEERING, vol. 8, no. 8, 1 August 1995 (1995-08-01), pages 829 - 834, XP000565688
ZABNER J ET AL: "REPEAT ADMINISTRATION OF AN DENOVIRUS VECTOR ENCODING CYSTIC FIBROSIS TRANSMEMBRANCE CONDUCTANCE REGULATOR TO THE NASAL EPITHELIUM OF PATIENTS WITH CYSTIC FIBROSIS", JOURNAL OF CLINICAL INVESTIGATION, vol. 97, no. 6, 15 March 1996 (1996-03-15), pages 1504 - 1511, XP000575871
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