WO2012112677A3 - Methods for treating lysosomal acid lipase deficiency - Google Patents

Methods for treating lysosomal acid lipase deficiency Download PDF

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Publication number
WO2012112677A3
WO2012112677A3 PCT/US2012/025233 US2012025233W WO2012112677A3 WO 2012112677 A3 WO2012112677 A3 WO 2012112677A3 US 2012025233 W US2012025233 W US 2012025233W WO 2012112677 A3 WO2012112677 A3 WO 2012112677A3
Authority
WO
WIPO (PCT)
Prior art keywords
disease
acid lipase
lysosomal acid
methods
lipase deficiency
Prior art date
Application number
PCT/US2012/025233
Other languages
French (fr)
Other versions
WO2012112677A2 (en
Inventor
Gregory Grabowski
Hong Du
Michael Heartlein
Michael Concino
Paolo Martini
Muthuraman Meiyappan
Alla Romashko
Brian PESCATORE
Lawrence Charnas
Original Assignee
Children's Hospital Medical Center
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 Children's Hospital Medical Center filed Critical Children's Hospital Medical Center
Publication of WO2012112677A2 publication Critical patent/WO2012112677A2/en
Priority to US13/967,446 priority Critical patent/US20130330317A1/en
Publication of WO2012112677A3 publication Critical patent/WO2012112677A3/en

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases

Abstract

The present invention provides compositions and methods for effective treatment of a lysosomal acid lipase deficiency (LALD) disease, in particular, Wolman's disease and Cholesteryl Ester Storage Disease (CESD). Among other things, the present invention provides a method of treating a lysosomal acid lipase deficiency (LALD) disease, including administering to an individual suffering from or susceptible to the LALD disease a therapeutic effective amount of a lysosomal acid lipase periodically at an administration interval such that lipid level in liver, spleen and/or small intestine is reduced by at least 20% as compared to an untreated control.
PCT/US2012/025233 2011-02-15 2012-02-15 Methods for treating lysosomal acid lipase deficiency WO2012112677A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
US13/967,446 US20130330317A1 (en) 2012-02-15 2013-08-15 Methods for Treating Lysosomal Acid Lipase Deficiency

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201161443079P 2011-02-15 2011-02-15
US61/443,079 2011-02-15

Related Child Applications (1)

Application Number Title Priority Date Filing Date
US13/967,446 Continuation US20130330317A1 (en) 2012-02-15 2013-08-15 Methods for Treating Lysosomal Acid Lipase Deficiency

Publications (2)

Publication Number Publication Date
WO2012112677A2 WO2012112677A2 (en) 2012-08-23
WO2012112677A3 true WO2012112677A3 (en) 2014-05-01

Family

ID=46673151

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2012/025233 WO2012112677A2 (en) 2011-02-15 2012-02-15 Methods for treating lysosomal acid lipase deficiency

Country Status (1)

Country Link
WO (1) WO2012112677A2 (en)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2398995C (en) 2000-02-04 2014-09-23 Children's Hospital Research Foundation Lipid hydrolysis therapy for atherosclerosis and related diseases
MX338426B (en) 2010-04-23 2016-04-15 Synageva Biopharma Corp Lysosomal storage disease enzyme.
ES2769836T3 (en) 2010-09-09 2020-06-29 Alexion Pharma Inc Isolated Human Recombinant N-glycosylated Lysosomal Acid Lipase
EP2675472A4 (en) 2011-02-15 2014-09-17 Synageva Biopharma Corp Methods for treating lysosomal acid lipase deficiency
KR20140130443A (en) * 2012-03-02 2014-11-10 시나게바 바이오파르마, 코포레이션 Truncated lysosomal acid lipase
WO2017218926A1 (en) 2016-06-17 2017-12-21 Alexion Pharmaceuticals, Inc. Lysosomal acid lipase deficiency compositions and methods

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040038365A1 (en) * 2000-10-31 2004-02-26 Yonghong Xiao Regulation of human lysosomal acid lipase
US20070280925A1 (en) * 2000-06-19 2007-12-06 Genzyme Corporation Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US20090297496A1 (en) * 2005-09-08 2009-12-03 Childrens Hospital Medical Center Lysosomal Acid Lipase Therapy for NAFLD and Related Diseases

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070280925A1 (en) * 2000-06-19 2007-12-06 Genzyme Corporation Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US20040038365A1 (en) * 2000-10-31 2004-02-26 Yonghong Xiao Regulation of human lysosomal acid lipase
US20090297496A1 (en) * 2005-09-08 2009-12-03 Childrens Hospital Medical Center Lysosomal Acid Lipase Therapy for NAFLD and Related Diseases

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
DU ET AL.: "Wolman disease/cholesteryl ester storage disease: efficacy of plant produced human lysosomal acid lipase in mice.", JOURNAL OF LIPID RESEARCH, vol. 49, 2008, Retrieved from the Internet <URL:http://www.jlr.org/contenU49/8/1646.full.pdf+html> [retrieved on 20120805] *

Also Published As

Publication number Publication date
WO2012112677A2 (en) 2012-08-23

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