DK3449934T3 - Behandling af glycogenlagringssygdom type II - Google Patents

Behandling af glycogenlagringssygdom type II Download PDF

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Publication number
DK3449934T3
DK3449934T3 DK18194771.4T DK18194771T DK3449934T3 DK 3449934 T3 DK3449934 T3 DK 3449934T3 DK 18194771 T DK18194771 T DK 18194771T DK 3449934 T3 DK3449934 T3 DK 3449934T3
Authority
DK
Denmark
Prior art keywords
storage disease
disease type
treatment
glycogen storage
glycogen
Prior art date
Application number
DK18194771.4T
Other languages
English (en)
Inventor
Yuan-Tsong Chen
Original Assignee
Univ Duke
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=22818451&utm_source=***_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=DK3449934(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Univ Duke filed Critical Univ Duke
Application granted granted Critical
Publication of DK3449934T3 publication Critical patent/DK3449934T3/da

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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)
    • A61K38/47—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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Cardiology (AREA)
  • Diabetes (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • General Engineering & Computer Science (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Hospice & Palliative Care (AREA)
  • Emergency Medicine (AREA)
  • Endocrinology (AREA)
  • Transplantation (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)
DK18194771.4T 2000-07-18 2001-07-10 Behandling af glycogenlagringssygdom type II DK3449934T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US21923700P 2000-07-18 2000-07-18
EP16178390.7A EP3108895B1 (en) 2000-07-18 2001-07-10 Treatment of glycogen storage disease type ii

Publications (1)

Publication Number Publication Date
DK3449934T3 true DK3449934T3 (da) 2020-06-29

Family

ID=22818451

Family Applications (5)

Application Number Title Priority Date Filing Date
DK07001091.3T DK1782825T3 (da) 2000-07-18 2001-07-10 Behandling af glycogenlagringssygdom type II
DK14167882.1T DK2767291T3 (da) 2000-07-18 2001-07-10 Behandling af glycogenlagringssygdom type II
DK01951000T DK1301201T3 (da) 2000-07-18 2001-07-10 Behandling af glycogenosis type II
DK16178390.7T DK3108895T3 (da) 2000-07-18 2001-07-10 Behandling af glycogenlagringssygdom type II
DK18194771.4T DK3449934T3 (da) 2000-07-18 2001-07-10 Behandling af glycogenlagringssygdom type II

Family Applications Before (4)

Application Number Title Priority Date Filing Date
DK07001091.3T DK1782825T3 (da) 2000-07-18 2001-07-10 Behandling af glycogenlagringssygdom type II
DK14167882.1T DK2767291T3 (da) 2000-07-18 2001-07-10 Behandling af glycogenlagringssygdom type II
DK01951000T DK1301201T3 (da) 2000-07-18 2001-07-10 Behandling af glycogenosis type II
DK16178390.7T DK3108895T3 (da) 2000-07-18 2001-07-10 Behandling af glycogenlagringssygdom type II

Country Status (18)

Country Link
US (10) US7056712B2 (da)
EP (4) EP3449934B1 (da)
JP (10) JP5113312B2 (da)
AT (1) ATE355075T1 (da)
AU (3) AU2001271941B2 (da)
BR (1) BR0113006A (da)
CA (1) CA2416492C (da)
CY (2) CY1120977T1 (da)
DE (1) DE60126947T2 (da)
DK (5) DK1782825T3 (da)
ES (5) ES2799882T3 (da)
HK (3) HK1054690A1 (da)
LT (3) LT3108895T (da)
MX (1) MXPA03000474A (da)
PT (5) PT3108895T (da)
SI (3) SI3449934T1 (da)
TW (1) TWI280882B (da)
WO (1) WO2002005841A1 (da)

Families Citing this family (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2799882T3 (es) * 2000-07-18 2020-12-22 Univ Duke Tratamiento de glucogenosis de tipo II
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
CA2445577C (en) * 2001-04-30 2012-07-03 Symbiontics, Inc. Subcellular targeting of therapeutic proteins
US20040005309A1 (en) * 2002-05-29 2004-01-08 Symbiontics, Inc. Targeted therapeutic proteins
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
ATE521701T1 (de) 2003-01-22 2011-09-15 Univ Duke Verbesserte konstrukte zur expression lysosomaler polypeptide
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
WO2005078077A2 (en) * 2004-02-10 2005-08-25 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
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
JP2010509344A (ja) * 2006-11-13 2010-03-25 ザイストール セラピューティクス， インコーポレイテッド ポンペ病を治療するための方法
WO2009075815A1 (en) 2007-12-07 2009-06-18 Duke University Immunomodulating gene therapy
DK2279210T3 (da) 2008-05-07 2017-07-24 Biomarin Pharm Inc Lysosomale targeting peptider og anvendelser deraf
WO2010005565A2 (en) 2008-07-08 2010-01-14 Duke University Method of treating glycogen storage disease
ES2569514T3 (es) 2009-06-17 2016-05-11 Biomarin Pharmaceutical Inc. Formulaciones para enzimas lisosómicas
US8809282B2 (en) 2010-05-06 2014-08-19 Duke University Method of reducing titers of antibodies specific for a therapeutic agent
AU2014218854B2 (en) 2013-02-20 2019-01-24 Valerion Therapeutics, Llc Methods and compositions for treatment of Pompe disease
CN115120745A (zh) * 2013-05-15 2022-09-30 明尼苏达大学董事会 腺相关病毒介导的基因向中枢神经***转移
FI3201320T3 (fi) 2014-09-30 2024-01-08 Amicus Therapeutics Inc Erittäin tehokas hapan alfa-glukosidaasi parannetuilla hiilihydraateilla
JP6821192B2 (ja) * 2015-05-07 2021-01-27 国立大学法人 東京大学 ポリイオンコンプレックス型ポリマーソームを用いたナノリアクタとその製造方法
MA44747A (fr) * 2015-08-31 2019-03-06 Univ Duke Méthodes et compositions pour le traitement de troubles associés à une accumulation du glycogène cytoplasmique
US10940125B2 (en) 2015-09-18 2021-03-09 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
KR20220145918A (ko) 2016-03-30 2022-10-31 아미쿠스 세라퓨틱스, 인코포레이티드 고 m6p 재조합 단백질의 선택 방법
CA3019128A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
IL305449A (en) 2016-04-15 2023-10-01 Univ Pennsylvania Gene therapy for the treatment of type II mucositis
CA3076036A1 (en) 2017-09-22 2019-03-28 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
WO2020163480A1 (en) * 2019-02-05 2020-08-13 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase and uses thereof

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3063911A (en) * 1961-04-03 1962-11-13 Taisho Pharmaceutical Co Ltd Acid-stable digestive enzyme preparation and process of making same
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
US5356804A (en) * 1990-10-24 1994-10-18 Mount Sinai School Of Medicine Of The City Of New York Cloning and expression of biologically active human α-galactosidase A
US6118045A (en) 1995-08-02 2000-09-12 Pharming B.V. Lysosomal proteins produced in the milk of transgenic animals
IL139616A0 (en) 1998-05-13 2002-02-10 Harbor Ucla Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating, diseases caused by deficiencies thereof
KR20010101131A (ko) * 1998-12-07 2001-11-14 추후기재 폼페병의 치료 방법
US6534300B1 (en) 1999-09-14 2003-03-18 Genzyme Glycobiology Research Institute, Inc. Methods for producing highly phosphorylated lysosomal hydrolases
ES2799882T3 (es) * 2000-07-18 2020-12-22 Univ Duke Tratamiento de glucogenosis de tipo II
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
WO2005078077A2 (en) * 2004-02-10 2005-08-25 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
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
SI3108895T1 (sl) 2019-01-31
JP2020019822A (ja) 2020-02-06
EP2767291B1 (en) 2016-09-14
BR0113006A (pt) 2004-10-19
US9907839B2 (en) 2018-03-06
JP5792774B2 (ja) 2015-10-14
HK1103363A1 (en) 2007-12-21
DK1782825T3 (da) 2014-11-03
MXPA03000474A (es) 2003-10-06
US20020110551A1 (en) 2002-08-15
ATE355075T1 (de) 2006-03-15
DK1301201T3 (da) 2007-04-02
LT2767291T (lt) 2016-11-25
EP1301201A1 (en) 2003-04-16
PT3449934T (pt) 2020-06-25
US20150004152A1 (en) 2015-01-01
US20050123531A1 (en) 2005-06-09
US8900552B2 (en) 2014-12-02
JP2014185187A (ja) 2014-10-02
JP2016056206A (ja) 2016-04-21
EP3108895B1 (en) 2018-09-19
PT1782825E (pt) 2014-11-04
CY1123084T1 (el) 2021-10-29
ES2523024T3 (es) 2014-11-20
WO2002005841A1 (en) 2002-01-24
JP2012006953A (ja) 2012-01-12
ES2700865T3 (es) 2019-02-19
US20210169996A1 (en) 2021-06-10
US20130195834A1 (en) 2013-08-01
JP2008081507A (ja) 2008-04-10
EP3449934A1 (en) 2019-03-06
DK2767291T3 (da) 2016-12-05
JP2013231081A (ja) 2013-11-14
ES2277931T3 (es) 2007-08-01
JP6163216B2 (ja) 2017-07-12
US20080175833A1 (en) 2008-07-24
EP2767291A2 (en) 2014-08-20
EP3108895A1 (en) 2016-12-28
PT3108895T (pt) 2018-12-18
US20120058132A1 (en) 2012-03-08
DE60126947D1 (de) 2007-04-12
US20180185457A1 (en) 2018-07-05
US9370556B2 (en) 2016-06-21
ES2799882T3 (es) 2020-12-22
EP1301201B1 (en) 2007-02-28
DK3108895T3 (da) 2018-11-26
CA2416492C (en) 2008-04-29
ES2599401T3 (es) 2017-02-01
US7056712B2 (en) 2006-06-06
US20100254966A1 (en) 2010-10-07
SI2767291T1 (sl) 2016-11-30
AU2001271941B2 (en) 2007-03-01
SI3449934T1 (sl) 2020-09-30
AU2010200487A1 (en) 2010-03-04
US20160184410A1 (en) 2016-06-30
JP5113312B2 (ja) 2013-01-09
AU7194101A (en) 2002-01-30
EP3449934B1 (en) 2020-05-20
DE60126947T2 (de) 2007-10-31
CY1120977T1 (el) 2019-12-11
HK1054690A1 (en) 2003-12-12
CA2416492A1 (en) 2002-01-24
JP2004503598A (ja) 2004-02-05
PT2767291T (pt) 2016-10-25
JP2018002749A (ja) 2018-01-11
LT3108895T (lt) 2018-11-26
HK1197656A1 (zh) 2015-02-06
JP2017137357A (ja) 2017-08-10
LT3449934T (lt) 2020-08-10
EP2767291A3 (en) 2014-10-08
US10792341B2 (en) 2020-10-06
PT1301201E (pt) 2007-03-30
JP2018199721A (ja) 2018-12-20
TWI280882B (en) 2007-05-11

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