ES2664844T3 - Tratamiento dirigido contra TNF para las mucopolisacaridosis y otros trastornos lisosómicos - Google Patents

Tratamiento dirigido contra TNF para las mucopolisacaridosis y otros trastornos lisosómicos Download PDF

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Publication number
ES2664844T3
ES2664844T3 ES12803458.4T ES12803458T ES2664844T3 ES 2664844 T3 ES2664844 T3 ES 2664844T3 ES 12803458 T ES12803458 T ES 12803458T ES 2664844 T3 ES2664844 T3 ES 2664844T3
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ES
Spain
Prior art keywords
mps
treatment
pps
disease
rats
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.)
Active
Application number
ES12803458.4T
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English (en)
Spanish (es)
Inventor
Edward H. Schuchman
Calogera M. Simonaro
Gary E. Striker
Helen Vlassara
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Icahn School of Medicine at Mount Sinai
Original Assignee
Mount Sinai School of Medicine
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 Mount Sinai School of Medicine filed Critical Mount Sinai School of Medicine
Application granted granted Critical
Publication of ES2664844T3 publication Critical patent/ES2664844T3/es
Active legal-status Critical Current
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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/715—Polysaccharides, i.e. having more than five saccharide radicals attached to each other by glycosidic linkages; Derivatives thereof, e.g. ethers, esters
    • A61K31/737—Sulfated polysaccharides, e.g. chondroitin sulfate, dermatan sulfate
    • 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)
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Molecular Biology (AREA)
  • Dermatology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Endocrinology (AREA)
  • Emergency Medicine (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
ES12803458.4T 2011-06-20 2012-06-20 Tratamiento dirigido contra TNF para las mucopolisacaridosis y otros trastornos lisosómicos Active ES2664844T3 (es)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201161498946P 2011-06-20 2011-06-20
US201161498946P 2011-06-20
US201161569452P 2011-12-12 2011-12-12
US201161569452P 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 (1)

Publication Number Publication Date
ES2664844T3 true ES2664844T3 (es) 2018-04-23

Family

ID=47422922

Family Applications (1)

Application Number Title Priority Date Filing Date
ES12803458.4T Active ES2664844T3 (es) 2011-06-20 2012-06-20 Tratamiento dirigido contra TNF para las mucopolisacaridosis y otros trastornos lisosómicos

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)

* Cited by examiner, † Cited by third party
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)

* Cited by examiner, † Cited by third party
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

Also Published As

Publication number Publication date
JP6175431B2 (ja) 2017-08-02
CN103764824A (zh) 2014-04-30
WO2012177778A1 (en) 2012-12-27
US20140205584A1 (en) 2014-07-24
CN103764824B (zh) 2018-04-24
US9155784B2 (en) 2015-10-13
EP2721151A4 (en) 2015-04-22
EP2721151B1 (en) 2017-12-06
EP2721151A1 (en) 2014-04-23
JP2014518222A (ja) 2014-07-28

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