Title:
TREATMENT OF PROTEIN AGGREGATION MYOPATHIC AND NEURODEGENERATIVE DISEASES BY PARENTERAL ADMINISTRATION OF TREHALOSE
Document Type and Number:
WIPO Patent Application WO/2014/181333
Kind Code:
A3
Abstract:
Disclosed is a method of treatment of a disease associated with abnormal protein aggregation comprising parenterally administering pharmaceutical formulations comprising trehalose. Also disclosed is an injectable aqueous pharmaceutical formulation comprising a therapeutically effective amount of trehalose.
Inventors:
MEGIDDO DALIA (IL)
Application Number:
PCT/IL2014/050411
Publication Date:
January 08, 2015
Filing Date:
May 07, 2014
Export Citation:
Assignee:
BIO BLAST PHARMA LTD (IL)
International Classes:
A61K31/7016; A61K9/00; A61P21/00; A61P25/14; A61P25/16; A61P25/28
Domestic Patent References:
| WO2010008860A1 | 2010-01-21 |
Foreign References:
| JP2001302517A | 2001-10-31 | |||
| US20100035837A1 | 2010-02-11 | |||
| JP2006342108A | 2006-12-21 | |||
| US20090110746A1 | 2009-04-30 |
Other References:
ANONYMOUS: "Trehalose in oculopharyngeal muscular dystrophy: The HOPEMD; NCT02015481 study", INTEGRITY, THOMSON-PHARMA, 20 December 2013 (2013-12-20), XP055137435, Retrieved from the Internet [retrieved on 20140901]
EMEA: "Avastin : EPAR - European Medicines Agency", 1 January 2005 (2005-01-01), pages 1 - 61, XP055138503, Retrieved from the Internet [retrieved on 20140905]
J. E. DAVIES: "Trehalose reduces aggregate formation and delays pathology in a transgenic mouse model of oculopharyngeal muscular dystrophy", HUMAN MOLECULAR GENETICS, vol. 15, no. 1, 30 November 2005 (2005-11-30), pages 23 - 31, XP055137287, ISSN: 0964-6906, DOI: 10.1093/hmg/ddi422
HERWEG W LANGE: "Aktueller Forschungsstand zur neuroprotektiven Therapie der Huntington-Krankheit", MATERIALIEN ZUR HUNTINGTON-KRANKHEIT, NR. 180, 1 June 2008 (2008-06-01), pages 1 - 12, XP055137420, Retrieved from the Internet [retrieved on 20140901]
MOTOMASA TANAKA ET AL: "Trehalose alleviates polyglutamine-mediated pathology in a mouse model of Huntington disease", NATURE MEDICINE, vol. 10, no. 2, 18 January 2004 (2004-01-18), pages 148 - 154, XP055137246, ISSN: 1078-8956, DOI: 10.1038/nm985
JOHN STOLZ ET AL: "Trehalose: an intriguing disaccharide with potential for medical application in ophthalmology", CLINICAL OPHTHALMOLOGY, 1 May 2011 (2011-05-01), pages 577, XP055137154, DOI: 10.2147/OPTH.S18827
DAVIES J E ET AL: "Oculopharyngeal muscular dystrophy: Potential therapies for an aggregate-associated disorder", INTERNATIONAL JOURNAL OF BIOCHEMISTRY AND CELL BIOLOGY, PERGAMON, GB, vol. 38, no. 9, 1 January 2006 (2006-01-01), pages 1457 - 1462, XP024987439, ISSN: 1357-2725, [retrieved on 20060101], DOI: 10.1016/J.BIOCEL.2006.01.016
EMEA: "Avastin : EPAR - European Medicines Agency", 1 January 2005 (2005-01-01), pages 1 - 61, XP055138503, Retrieved from the Internet
J. E. DAVIES: "Trehalose reduces aggregate formation and delays pathology in a transgenic mouse model of oculopharyngeal muscular dystrophy", HUMAN MOLECULAR GENETICS, vol. 15, no. 1, 30 November 2005 (2005-11-30), pages 23 - 31, XP055137287, ISSN: 0964-6906, DOI: 10.1093/hmg/ddi422
HERWEG W LANGE: "Aktueller Forschungsstand zur neuroprotektiven Therapie der Huntington-Krankheit", MATERIALIEN ZUR HUNTINGTON-KRANKHEIT, NR. 180, 1 June 2008 (2008-06-01), pages 1 - 12, XP055137420, Retrieved from the Internet
MOTOMASA TANAKA ET AL: "Trehalose alleviates polyglutamine-mediated pathology in a mouse model of Huntington disease", NATURE MEDICINE, vol. 10, no. 2, 18 January 2004 (2004-01-18), pages 148 - 154, XP055137246, ISSN: 1078-8956, DOI: 10.1038/nm985
JOHN STOLZ ET AL: "Trehalose: an intriguing disaccharide with potential for medical application in ophthalmology", CLINICAL OPHTHALMOLOGY, 1 May 2011 (2011-05-01), pages 577, XP055137154, DOI: 10.2147/OPTH.S18827
DAVIES J E ET AL: "Oculopharyngeal muscular dystrophy: Potential therapies for an aggregate-associated disorder", INTERNATIONAL JOURNAL OF BIOCHEMISTRY AND CELL BIOLOGY, PERGAMON, GB, vol. 38, no. 9, 1 January 2006 (2006-01-01), pages 1457 - 1462, XP024987439, ISSN: 1357-2725, [retrieved on 20060101], DOI: 10.1016/J.BIOCEL.2006.01.016
Attorney, Agent or Firm:
HACKMEY, Michal (P.O. Box 1323962 Tel-Aviv, Israel, IL)
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