ES2523024T3 - Tratamiento de glucogenosis de tipo II - Google Patents

Tratamiento de glucogenosis de tipo II Download PDF

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Publication number
ES2523024T3
ES2523024T3 ES07001091.3T ES07001091T ES2523024T3 ES 2523024 T3 ES2523024 T3 ES 2523024T3 ES 07001091 T ES07001091 T ES 07001091T ES 2523024 T3 ES2523024 T3 ES 2523024T3
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treatment
type
glycogenosis
months
storage disease
Prior art date
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Expired - Lifetime
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ES07001091.3T
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English (en)
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Yuan-Tsong Chen
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Duke University
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Duke University
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Application filed by Duke University filed Critical Duke University
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Publication of ES2523024T3 publication Critical patent/ES2523024T3/es
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    • 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)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/02—Non-specific cardiovascular stimulants, e.g. drugs for syncope, antihypotensives
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Diabetes (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Transplantation (AREA)
  • Hospice & Palliative Care (AREA)
  • Emergency Medicine (AREA)
  • Endocrinology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

α-Glucosidasa ácida (GAA) humana recombinante producida en un cultivo celular de ovario de hámster chino para uso en un método de tratamiento de la glucogenosis de tipo II en un ser humano, o para uso en un método de tratamiento de la cardiomiopatía asociada con la glucogenosis de tipo II en un ser humano, donde el ser humano es CRIM (material immunoreactivo de reacción cruzada)-positivo para la α-glucosidasa ácida endógena.

Description

imagen1
imagen2
imagen3
imagen4
imagen5
E07001091
03-11-2014
Tabla 2. Datos clínicos de referencia en 3 pacientes con enfermedad de Pompe infantil
Número depaciente/sexo
Origen étnico Edad inicioGAArh Estado cardiaco Función pulmonar Desarrollomotor(puntuaciónAIMS) Actividad GAAen fibroblastosde la piel (% denormal) EstadoCRIM* Edadactual
Paciente1/masculino
Caucásico 4 meses Cardiomiopatíasevera; estadopost ataquecardiaco Límite normal,compresiónbronquio principalizquierdo por unamarcadacardiomegalia,desaturación O2 <<5º% 0,84% Negativo 29 meses
Paciente2/masculino
Afro-Americano 3 meses Cardiomiopatíamoderada Desaturación O2durante el llanto <5º% 0,57% Negativo 25 meses
Paciente3/masculino
Caucásico 2½ meses Cardiomiopatíalímite normal <<5º% 0,69% Positivo 23 meses
*CRIM = material inmunoreactivo de reacción cruzada
7
imagen6
imagen7
imagen8
imagen9

Claims (1)

  1. imagen1
ES07001091.3T 2000-07-18 2001-07-10 Tratamiento de glucogenosis de tipo II Expired - Lifetime ES2523024T3 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US21923700P 2000-07-18 2000-07-18
US219237P 2000-07-18

Publications (1)

Publication Number Publication Date
ES2523024T3 true ES2523024T3 (es) 2014-11-20

Family

ID=22818451

Family Applications (5)

Application Number Title Priority Date Filing Date
ES01951000T Expired - Lifetime ES2277931T3 (es) 2000-07-18 2001-07-10 Tratamiento de la glucogenesis de tipo ii.
ES07001091.3T Expired - Lifetime ES2523024T3 (es) 2000-07-18 2001-07-10 Tratamiento de glucogenosis de tipo II
ES14167882.1T Expired - Lifetime ES2599401T3 (es) 2000-07-18 2001-07-10 Tratamiento de glucogenosis de tipo II
ES18194771T Expired - Lifetime ES2799882T3 (es) 2000-07-18 2001-07-10 Tratamiento de glucogenosis de tipo II
ES16178390T Expired - Lifetime ES2700865T3 (es) 2000-07-18 2001-07-10 Tratamiento de glucogenosis de tipo II

Family Applications Before (1)

Application Number Title Priority Date Filing Date
ES01951000T Expired - Lifetime ES2277931T3 (es) 2000-07-18 2001-07-10 Tratamiento de la glucogenesis de tipo ii.

Family Applications After (3)

Application Number Title Priority Date Filing Date
ES14167882.1T Expired - Lifetime ES2599401T3 (es) 2000-07-18 2001-07-10 Tratamiento de glucogenosis de tipo II
ES18194771T Expired - Lifetime ES2799882T3 (es) 2000-07-18 2001-07-10 Tratamiento de glucogenosis de tipo II
ES16178390T Expired - Lifetime ES2700865T3 (es) 2000-07-18 2001-07-10 Tratamiento de glucogenosis de tipo II

Country Status (18)

Country Link
US (10) US7056712B2 (es)
EP (4) EP1301201B1 (es)
JP (10) JP5113312B2 (es)
AT (1) ATE355075T1 (es)
AU (3) AU7194101A (es)
BR (1) BR0113006A (es)
CA (1) CA2416492C (es)
CY (2) CY1120977T1 (es)
DE (1) DE60126947T2 (es)
DK (5) DK3108895T3 (es)
ES (5) ES2277931T3 (es)
HK (3) HK1054690A1 (es)
LT (3) LT3108895T (es)
MX (1) MXPA03000474A (es)
PT (5) PT1301201E (es)
SI (3) SI3108895T1 (es)
TW (1) TWI280882B (es)
WO (1) WO2002005841A1 (es)

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DK3108895T3 (en) * 2000-07-18 2018-11-26 Univ Duke Treatment of glycogen storage disease type II
US7560424B2 (en) * 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US20040005309A1 (en) * 2002-05-29 2004-01-08 Symbiontics, Inc. Targeted therapeutic proteins
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US7629309B2 (en) * 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US20030072761A1 (en) * 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
JP5433133B2 (ja) 2003-01-22 2014-03-05 デューク・ユニヴァーシティ リソソームポリペプチドを発現するための改善された構築物
US7442372B2 (en) 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
US7129049B2 (en) * 2003-12-22 2006-10-31 Regents Of The University Of Minnesota Method of detecting equine glycogen storage disease IV
ATE465250T1 (de) * 2004-02-10 2010-05-15 Zystor Therapeutics Inc Saure alpha-glukosidase und fragmente davon
US20050208090A1 (en) * 2004-03-18 2005-09-22 Medtronic, Inc. Methods and systems for treatment of neurological diseases of the central nervous system
EP1773401B1 (en) * 2004-06-21 2013-01-02 Medtronic, Inc. Medical systems and methods for delivering compositions to cells
AU2007322123A1 (en) * 2006-11-13 2008-05-29 Biomarin Pharmaceutical Inc. Methods for treating Pompe disease
WO2009075815A1 (en) 2007-12-07 2009-06-18 Duke University Immunomodulating gene therapy
EP2279210B1 (en) 2008-05-07 2017-04-12 BioMarin Pharmaceutical Inc. Lysosomal targeting peptides and uses thereof
SI2318037T1 (sl) * 2008-07-08 2015-07-31 Duke University Metoda za zdravljenje bolezni glikogenoze
ES2569514T3 (es) * 2009-06-17 2016-05-11 Biomarin Pharmaceutical Inc. Formulaciones para enzimas lisosómicas
WO2011139379A2 (en) 2010-05-06 2011-11-10 Duke University A method of treating patients undergoing protein replacement therapy, gene replacement therapy, or other therapeutic modalities
AU2014218854B2 (en) 2013-02-20 2019-01-24 Valerion Therapeutics, Llc Methods and compositions for treatment of Pompe disease
SG11201509419QA (en) * 2013-05-15 2015-12-30 Univ Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
PT3201320T (pt) 2014-09-30 2024-01-12 Amicus Therapeutics Inc Alfa-glucosidase ácida altamente potente com hidratos de carbono intensificados
EP3292871B1 (en) * 2015-05-07 2021-06-23 The University of Tokyo Nanoreactor using polyion complex polymersomes, and method for producing same
WO2017040647A1 (en) * 2015-08-31 2017-03-09 Duke University Methods and compositions for the treatment of cytopplasmic glycogen storage disorders
WO2017049157A1 (en) 2015-09-18 2017-03-23 Duke University Methods and compositions for the treatment of steatosis-associated disorders
KR102510941B1 (ko) 2015-12-30 2023-03-20 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
KR102444612B1 (ko) 2016-03-30 2022-09-21 아미쿠스 세라퓨틱스, 인코포레이티드 재조합 산 알파-글루코시다제를 포함하는 제형
MX2018011951A (es) 2016-03-30 2019-02-13 Amicus Therapeutics Inc Metodo para seleccionar proteinas recombinantes ricas en m6p.
IL262211B2 (en) 2016-04-15 2024-01-01 Univ Pennsylvania Gene therapy for the treatment of type II mucositis
KR20200104852A (ko) 2017-09-22 2020-09-04 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 Ii형 점액다당류증의 치료를 위한 유전자 요법
WO2020163480A1 (en) * 2019-02-05 2020-08-13 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase and uses thereof

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US5464778A (en) 1989-03-08 1995-11-07 Board Of Regents Of The University Of Oklahoma Glycoprotein ligand for P-selectin and methods of use thereof
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DK3108895T3 (en) * 2000-07-18 2018-11-26 Univ Duke Treatment of glycogen storage disease type II
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
ATE465250T1 (de) * 2004-02-10 2010-05-15 Zystor Therapeutics Inc Saure alpha-glukosidase und fragmente davon
US8470552B2 (en) 2009-10-12 2013-06-25 Keck Graduate Institute Strategy to reduce lactic acid production and control PH in animal cell culture

Also Published As

Publication number Publication date
US20100254966A1 (en) 2010-10-07
US8900552B2 (en) 2014-12-02
EP3108895B1 (en) 2018-09-19
ATE355075T1 (de) 2006-03-15
JP6163216B2 (ja) 2017-07-12
EP2767291A3 (en) 2014-10-08
US20080175833A1 (en) 2008-07-24
LT3108895T (lt) 2018-11-26
DK2767291T3 (en) 2016-12-05
DK3449934T3 (da) 2020-06-29
EP2767291A2 (en) 2014-08-20
US7056712B2 (en) 2006-06-06
PT3449934T (pt) 2020-06-25
JP2016056206A (ja) 2016-04-21
TWI280882B (en) 2007-05-11
EP3108895A1 (en) 2016-12-28
SI2767291T1 (sl) 2016-11-30
DE60126947D1 (de) 2007-04-12
SI3449934T1 (sl) 2020-09-30
BR0113006A (pt) 2004-10-19
US20050123531A1 (en) 2005-06-09
ES2700865T3 (es) 2019-02-19
AU7194101A (en) 2002-01-30
US20160184410A1 (en) 2016-06-30
CY1123084T1 (el) 2021-10-29
HK1197656A1 (zh) 2015-02-06
CY1120977T1 (el) 2019-12-11
US9907839B2 (en) 2018-03-06
HK1103363A1 (en) 2007-12-21
PT3108895T (pt) 2018-12-18
JP2008081507A (ja) 2008-04-10
JP2017137357A (ja) 2017-08-10
WO2002005841A1 (en) 2002-01-24
EP3449934B1 (en) 2020-05-20
JP2018199721A (ja) 2018-12-20
EP1301201B1 (en) 2007-02-28
AU2010200487A1 (en) 2010-03-04
DK3108895T3 (en) 2018-11-26
ES2799882T3 (es) 2020-12-22
JP2012006953A (ja) 2012-01-12
DE60126947T2 (de) 2007-10-31
SI3108895T1 (sl) 2019-01-31
JP2013231081A (ja) 2013-11-14
JP2014185187A (ja) 2014-10-02
JP2018002749A (ja) 2018-01-11
CA2416492A1 (en) 2002-01-24
EP2767291B1 (en) 2016-09-14
JP2004503598A (ja) 2004-02-05
EP1301201A1 (en) 2003-04-16
DK1301201T3 (da) 2007-04-02
US20150004152A1 (en) 2015-01-01
LT2767291T (lt) 2016-11-25
PT1301201E (pt) 2007-03-30
CA2416492C (en) 2008-04-29
US20180185457A1 (en) 2018-07-05
ES2599401T3 (es) 2017-02-01
DK1782825T3 (da) 2014-11-03
US20210169996A1 (en) 2021-06-10
US20130195834A1 (en) 2013-08-01
US9370556B2 (en) 2016-06-21
AU2001271941B2 (en) 2007-03-01
PT1782825E (pt) 2014-11-04
HK1054690A1 (en) 2003-12-12
JP2020019822A (ja) 2020-02-06
MXPA03000474A (es) 2003-10-06
US20120058132A1 (en) 2012-03-08
US10792341B2 (en) 2020-10-06
EP3449934A1 (en) 2019-03-06
LT3449934T (lt) 2020-08-10
ES2277931T3 (es) 2007-08-01
PT2767291T (pt) 2016-10-25
JP5113312B2 (ja) 2013-01-09
JP5792774B2 (ja) 2015-10-14
US20020110551A1 (en) 2002-08-15

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