JP2014518222A5 - - Google Patents
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- Publication number
- JP2014518222A5 JP2014518222A5 JP2014517125A JP2014517125A JP2014518222A5 JP 2014518222 A5 JP2014518222 A5 JP 2014518222A5 JP 2014517125 A JP2014517125 A JP 2014517125A JP 2014517125 A JP2014517125 A JP 2014517125A JP 2014518222 A5 JP2014518222 A5 JP 2014518222A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- subject
- composition according
- lysosomal storage
- therapy
- Prior art date
- Legal status (The legal status 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 status listed.)
- Granted
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- 208000015439 Lysosomal storage disease Diseases 0.000 claims description 34
- 208000010346 Sphingolipidoses Diseases 0.000 claims description 14
- 201000001307 Sphingolipidosis Diseases 0.000 claims description 14
- 208000011580 syndromic disease Diseases 0.000 claims description 14
- 239000003814 drug Substances 0.000 claims description 12
- 238000011374 additional therapy Methods 0.000 claims description 10
- 229940124597 therapeutic agent Drugs 0.000 claims description 10
- 208000002678 Mucopolysaccharidoses Diseases 0.000 claims description 9
- 206010028093 mucopolysaccharidosis Diseases 0.000 claims description 9
- 238000002560 therapeutic procedure Methods 0.000 claims description 9
- 238000002641 enzyme replacement therapy Methods 0.000 claims description 8
- 230000003902 lesion Effects 0.000 claims description 8
- 102100033342 Lysosomal acid glucosylceramidase Human genes 0.000 claims description 6
- 108010006519 Molecular Chaperones Proteins 0.000 claims description 6
- 238000010322 bone marrow transplantation Methods 0.000 claims description 6
- 238000001415 gene therapy Methods 0.000 claims description 6
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 claims description 5
- 206010053185 Glycogen storage disease type II Diseases 0.000 claims description 5
- 229920002683 Glycosaminoglycan Polymers 0.000 claims description 5
- 208000008955 Mucolipidoses Diseases 0.000 claims description 5
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 claims description 5
- 206010028095 Mucopolysaccharidosis IV Diseases 0.000 claims description 5
- 206010056893 Mucopolysaccharidosis VII Diseases 0.000 claims description 5
- 208000014060 Niemann-Pick disease Diseases 0.000 claims description 5
- 201000010099 disease Diseases 0.000 claims description 5
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 5
- 201000004502 glycogen storage disease II Diseases 0.000 claims description 5
- 238000002347 injection Methods 0.000 claims description 5
- 239000007924 injection Substances 0.000 claims description 5
- 238000001361 intraarterial administration Methods 0.000 claims description 5
- 238000010255 intramuscular injection Methods 0.000 claims description 5
- 239000007927 intramuscular injection Substances 0.000 claims description 5
- 238000010253 intravenous injection Methods 0.000 claims description 5
- 108010045758 lysosomal proteins Proteins 0.000 claims description 5
- 201000007769 mucolipidosis Diseases 0.000 claims description 5
- 201000002273 mucopolysaccharidosis II Diseases 0.000 claims description 5
- 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 claims description 5
- 208000010978 mucopolysaccharidosis type 4 Diseases 0.000 claims description 5
- 208000025919 mucopolysaccharidosis type 7 Diseases 0.000 claims description 5
- 210000004877 mucosa Anatomy 0.000 claims description 5
- 230000001225 therapeutic effect Effects 0.000 claims description 5
- 238000011282 treatment Methods 0.000 claims description 5
- 108010008165 Etanercept Proteins 0.000 claims description 4
- 229960002964 adalimumab Drugs 0.000 claims description 4
- 229960000403 etanercept Drugs 0.000 claims description 4
- 229960001743 golimumab Drugs 0.000 claims description 4
- 229960000598 infliximab Drugs 0.000 claims description 4
- 229960005027 natalizumab Drugs 0.000 claims description 4
- 102000004127 Cytokines Human genes 0.000 claims description 3
- 108090000695 Cytokines Proteins 0.000 claims description 3
- 102100029199 Iduronate 2-sulfatase Human genes 0.000 claims description 3
- 102100031688 N-acetylgalactosamine-6-sulfatase Human genes 0.000 claims description 3
- 101710099863 N-acetylgalactosamine-6-sulfatase Proteins 0.000 claims description 3
- 229960003122 alglucerase Drugs 0.000 claims description 3
- 108010060162 alglucerase Proteins 0.000 claims description 3
- -1 alglucosidase alpha Proteins 0.000 claims description 3
- 229960005390 galsulfase Drugs 0.000 claims description 3
- 108010089296 galsulfase Proteins 0.000 claims description 3
- 229960002396 idursulfase Drugs 0.000 claims description 3
- 108010072166 idursulfase Proteins 0.000 claims description 3
- 229960002127 imiglucerase Drugs 0.000 claims description 3
- 108010039650 imiglucerase Proteins 0.000 claims description 3
- 230000002757 inflammatory effect Effects 0.000 claims description 3
- 230000002401 inhibitory effect Effects 0.000 claims description 3
- 229960002486 laronidase Drugs 0.000 claims description 3
- 239000000758 substrate Substances 0.000 claims description 3
- 229940079593 drug Drugs 0.000 claims description 2
- 230000004064 dysfunction Effects 0.000 claims description 2
- UQRORFVVSGFNRO-UTINFBMNSA-N miglustat Chemical compound CCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO UQRORFVVSGFNRO-UTINFBMNSA-N 0.000 claims description 2
- 229960001512 miglustat Drugs 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 16
- ZCYVEMRRCGMTRW-UHFFFAOYSA-N 7553-56-2 Chemical compound [I] ZCYVEMRRCGMTRW-UHFFFAOYSA-N 0.000 claims 1
- 108090000790 Enzymes Proteins 0.000 claims 1
- 102000004190 Enzymes Human genes 0.000 claims 1
- QAOWNCQODCNURD-UHFFFAOYSA-L Sulfate Chemical compound [O-]S([O-])(=O)=O QAOWNCQODCNURD-UHFFFAOYSA-L 0.000 claims 1
- 108060008682 Tumor Necrosis Factor Proteins 0.000 claims 1
- 102000000852 Tumor Necrosis Factor-alpha Human genes 0.000 claims 1
- 229940088598 enzyme Drugs 0.000 claims 1
- 229910052740 iodine Inorganic materials 0.000 claims 1
- 239000011630 iodine Substances 0.000 claims 1
- 208000005340 mucopolysaccharidosis III Diseases 0.000 claims 1
- 208000011045 mucopolysaccharidosis type 3 Diseases 0.000 claims 1
- 238000009256 replacement therapy Methods 0.000 claims 1
- 238000000034 method Methods 0.000 description 49
- 201000008977 glycoproteinosis Diseases 0.000 description 4
- 230000000366 juvenile effect Effects 0.000 description 4
- 230000002159 abnormal effect Effects 0.000 description 3
- 230000004853 protein function Effects 0.000 description 3
- 201000001828 Sly syndrome Diseases 0.000 description 2
- 239000003795 chemical substances by application Substances 0.000 description 2
- 208000020084 Bone disease Diseases 0.000 description 1
- 210000000845 cartilage Anatomy 0.000 description 1
- 208000015100 cartilage disease Diseases 0.000 description 1
- 230000000474 nursing effect Effects 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161498946P | 2011-06-20 | 2011-06-20 | |
| US61/498,946 | 2011-06-20 | ||
| US201161569452P | 2011-12-12 | 2011-12-12 | |
| US61/569,452 | 2011-12-12 | ||
| PCT/US2012/043369 WO2012177778A1 (en) | 2011-06-20 | 2012-06-20 | Anti-tnf- therapy for the mucopolysaccharidoses and other lysosomal disorders |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2014518222A JP2014518222A (ja) | 2014-07-28 |
| JP2014518222A5 true JP2014518222A5 (https=) | 2015-11-05 |
| JP6175431B2 JP6175431B2 (ja) | 2017-08-02 |
Family
ID=47422922
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014517125A Active JP6175431B2 (ja) | 2011-06-20 | 2012-06-20 | ムコ多糖症およびその他のリソソーム障害に対する抗ｔｎｆ療法 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US9155784B2 (https=) |
| EP (1) | EP2721151B1 (https=) |
| JP (1) | JP6175431B2 (https=) |
| CN (1) | CN103764824B (https=) |
| ES (1) | ES2664844T3 (https=) |
| WO (1) | WO2012177778A1 (https=) |
Families Citing this family (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6849257B2 (en) | 2000-02-04 | 2005-02-01 | Children's Hospital Research Foundation | Lipid hydrolysis therapy for atherosclerosis and related diseases |
| US8889127B2 (en) | 2004-07-01 | 2014-11-18 | Icahn School Of Medicine At Mount Sinai | Targeted protein replacement for the treatment of lysosomal storage disorders |
| SI3998078T1 (sl) * | 2009-08-28 | 2026-02-27 | Genzyme Corporation | Encimsko nadomestno zdravljenje z naraščajočim odmerkom za zdravljenje pomanjkanja kisle sfingomielinaze |
| FI3205351T3 (fi) | 2010-04-23 | 2023-07-06 | Alexion Pharma Inc | Lysosomaalisen kertymätaudin entsyymi |
| KR20150038636A (ko) | 2010-09-09 | 2015-04-08 | 시나게바 바이오파르마, 코포레이션 | 환자에서 리소좀 산 리파제 결핍증의 치료를 위한 리소좀 산 리파제의 용도 |
| WO2012112681A1 (en) | 2011-02-15 | 2012-08-23 | Shire Human Genetic Therapies, Inc. | Methods for treating lysosomal acid lipase deficiency |
| LT2753346T (lt) | 2011-09-07 | 2020-08-10 | Mount Sinai School Of Medicine | Ceramidazė ir ląstelių diferenciacija |
| ES2897825T3 (es) | 2012-06-01 | 2022-03-02 | Icahn School Med Mount Sinai | Niveles de ceramida en el tratamiento y prevención de infecciones |
| PT2968479T (pt) | 2013-03-14 | 2019-08-07 | Icahn School Med Mount Sinai | Composições terapêuticas de ceramidase ácida e métodos de fabrico e utilização |
| SG11201509030TA (en) | 2013-05-07 | 2015-11-27 | Bio Blast Pharma Ltd | Treatment of protein aggregation myopathic and neurodegenerative diseases by parenteral administration of trehalose |
| NZ720499A (en) * | 2013-12-23 | 2020-04-24 | Bcn Peptides Sa | Bicalutamide analogs or (s)-bicalutamide as exocytosis activating compounds for use in the treatment of a lysosomal storage disorder or glycogenosis |
| SI2970413T1 (sl) * | 2014-04-01 | 2018-10-30 | Swedish Orphan Biovitrum Ab (Publ) | Modificirana sulfamidaza in proizvodja le-te |
| US20150348259A1 (en) * | 2014-06-03 | 2015-12-03 | Carestream Health, Inc. | Quantitative method for 3-d bone mineral density visualization and monitoring |
| WO2017019612A1 (en) * | 2015-07-27 | 2017-02-02 | Mary Kay Inc. | Topical skin formulations |
| WO2020039480A1 (ja) | 2018-08-20 | 2020-02-27 | 株式会社レクメド | 新規ポリ硫酸ペントサンナトリウム製剤 |
| FI3863647T3 (fi) * | 2018-10-10 | 2024-03-01 | Paradigm Biopharmaceuticals Ltd | Pahanlaatuisen luustokivun hoitaminen pentosaanipolysulfaatilla |
| IL291799A (en) * | 2019-10-01 | 2022-06-01 | Seelos Therapeutics Inc | Trehalose formulations and uses thereof |
| WO2021179047A1 (en) * | 2020-03-12 | 2021-09-16 | Cullis Hill Sydney David | Treatment for coronavirus infection and associated cytokine toxicity |
| IT202200007808A1 (it) | 2022-04-20 | 2023-10-20 | Luigi Michele Pavone | Composizioni terapeutiche per malattie causate da accumulo di eparan solfato |
Family Cites Families (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5180715A (en) | 1980-11-17 | 1993-01-19 | The Regents Of The University Of California | Irrigation of internal bladder surfaces in mammals with sodium pentosanpolysulfate |
| US5672347A (en) | 1984-07-05 | 1997-09-30 | Genentech, Inc. | Tumor necrosis factor antagonists and their use |
| US5668116A (en) | 1987-03-19 | 1997-09-16 | Anthropharm Pty. Limited | Anti-inflammatory compounds and compositions |
| US6451983B2 (en) | 1989-08-07 | 2002-09-17 | Peptech Limited | Tumor necrosis factor antibodies |
| JP3443119B2 (ja) | 1989-08-07 | 2003-09-02 | ペプテック リミテッド | 腫瘍壊死因子結合リガンド |
| US5656272A (en) | 1991-03-18 | 1997-08-12 | New York University Medical Center | Methods of treating TNF-α-mediated Crohn's disease using chimeric anti-TNF antibodies |
| ES2121907T3 (es) | 1992-08-28 | 1998-12-16 | Bayer Ag | Uso de anticuerpos monoclonales anti-tnf para el tratamiento de meningitis bacterianas. |
| CA2146647C (en) | 1992-10-08 | 2009-05-05 | Marc Feldmann | Treatment of autoimmune and inflammatory disorders |
| US5643892A (en) | 1995-06-07 | 1997-07-01 | Baker Norton Pharmaceuticals, Inc. | Method of treating chronic progressive vascular diseases |
| EP1005376B1 (en) | 1997-03-14 | 2010-11-03 | The Children's Hospital of Philadelphia | Compositions for use in gene therapy for treatment of hemophilia |
| CA2319212A1 (en) | 1998-02-17 | 1999-08-19 | Genzyme Corporation | Methods for purified aav vector production |
| WO1999057296A1 (en) | 1998-05-01 | 1999-11-11 | Genzyme Corporation | Partially deleted adenoviral vectors |
| US7927587B2 (en) | 1999-08-05 | 2011-04-19 | Regents Of The University Of Minnesota | MAPC administration for the treatment of lysosomal storage disorders |
| US20010034328A1 (en) | 2000-02-25 | 2001-10-25 | Cartt Stephen Lahue | Treatment of male chronic pelvic pain syndrome |
| AU2001269923A1 (en) | 2000-06-19 | 2002-01-02 | Genzyme Corporation | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
| US7951545B2 (en) | 2002-04-30 | 2011-05-31 | Seikagaku Corporation | Method for detecting lysosomal storage diseases |
| US7446098B2 (en) | 2003-02-18 | 2008-11-04 | Mount Sinai School Of Medicine Of New York University | Combination therapy for treating protein deficiencies |
| US20050208090A1 (en) | 2004-03-18 | 2005-09-22 | Medtronic, Inc. | Methods and systems for treatment of neurological diseases of the central nervous system |
| WO2007095688A1 (en) * | 2006-02-23 | 2007-08-30 | The Australian National University | Methods for increasing the number of circulating cells |
| US20080076723A1 (en) | 2006-09-27 | 2008-03-27 | Sylvan Pharmaceuticals Pty Ltd. | Inhibition of cathepsin K activity and the treatment and prevention of disease |
| EP3486653A1 (en) * | 2007-05-24 | 2019-05-22 | The United States Government as represented by The Department of Veterans Affairs | Treatment of skeletal muscle disorder using ent2 |
| NZ601148A (en) * | 2007-05-31 | 2012-08-31 | Glycan Biosciences | Use of pentosan polysulfate for treatment or prophylaxis of allergic rhinitis |
| US8540985B2 (en) * | 2008-06-26 | 2013-09-24 | Orphazyme Aps | Use of Hsp70 as a regulator of enzymatic activity |
| SI2995306T1 (sl) * | 2009-10-19 | 2019-04-30 | Amicus Therapeutics, Inc. | Novi sestavki za preprečevanje in/ali zdravljenje motenj lizosomskega shranjevanja |
| CA3140959A1 (en) * | 2009-11-27 | 2011-06-03 | Genzyme Corporation | An amorphous and a crystalline form of genz 112638 hemitartrate as inhibitor of glucosylceramide synthase |
-
2012
- 2012-06-20 US US14/128,411 patent/US9155784B2/en active Active
- 2012-06-20 EP EP12803458.4A patent/EP2721151B1/en active Active
- 2012-06-20 CN CN201280037341.5A patent/CN103764824B/zh active Active
- 2012-06-20 WO PCT/US2012/043369 patent/WO2012177778A1/en not_active Ceased
- 2012-06-20 ES ES12803458.4T patent/ES2664844T3/es active Active
- 2012-06-20 JP JP2014517125A patent/JP6175431B2/ja active Active
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