WO2012112677A3 - Methods for treating lysosomal acid lipase deficiency - Google Patents
Methods for treating lysosomal acid lipase deficiency Download PDFInfo
- 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
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.
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)
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)
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 |
-
2012
- 2012-02-15 WO PCT/US2012/025233 patent/WO2012112677A2/en active Application Filing
Patent Citations (3)
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)
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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