CA2669347A1 - Methods for treating pompe disease - Google Patents

Methods for treating pompe disease Download PDF

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Publication number
CA2669347A1
CA2669347A1 CA002669347A CA2669347A CA2669347A1 CA 2669347 A1 CA2669347 A1 CA 2669347A1 CA 002669347 A CA002669347 A CA 002669347A CA 2669347 A CA2669347 A CA 2669347A CA 2669347 A1 CA2669347 A1 CA 2669347A1
Authority
CA
Canada
Prior art keywords
gaa
human
fusion protein
targeting domain
mannose
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.)
Abandoned
Application number
CA002669347A
Other languages
English (en)
French (fr)
Inventor
Jonathan Lebowitz
John Maga
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.)
Biomarin Pharmaceutical Inc
Original Assignee
Individual
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 Individual filed Critical Individual
Publication of CA2669347A1 publication Critical patent/CA2669347A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • 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
    • 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)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)
CA002669347A 2006-11-13 2007-11-13 Methods for treating pompe disease Abandoned CA2669347A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US85851406P 2006-11-13 2006-11-13
US60/858,514 2006-11-13
US87925507P 2007-01-05 2007-01-05
US60/879,255 2007-01-05
US90018707P 2007-02-07 2007-02-07
US60/900,187 2007-02-07
PCT/US2007/023881 WO2008063511A2 (en) 2006-11-13 2007-11-13 Methods for treating pompe disease

Publications (1)

Publication Number Publication Date
CA2669347A1 true CA2669347A1 (en) 2008-05-29

Family

ID=39333078

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002669347A Abandoned CA2669347A1 (en) 2006-11-13 2007-11-13 Methods for treating pompe disease

Country Status (7)

Country Link
US (2) US20090117091A1 (cg-RX-API-DMAC10.html)
EP (1) EP2099523A2 (cg-RX-API-DMAC10.html)
JP (1) JP2010509344A (cg-RX-API-DMAC10.html)
AU (1) AU2007322123A1 (cg-RX-API-DMAC10.html)
CA (1) CA2669347A1 (cg-RX-API-DMAC10.html)
IL (1) IL198692A0 (cg-RX-API-DMAC10.html)
WO (1) WO2008063511A2 (cg-RX-API-DMAC10.html)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3782655A1 (en) 2005-05-17 2021-02-24 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
US8536148B2 (en) 2009-09-04 2013-09-17 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Disabling autophagy as a treatment for lysosomal storage diseases
WO2012061597A1 (en) * 2010-11-03 2012-05-10 The United States Of America As Represented By The Secretary, Department Of Health And Human Services Office Of Technology Transfer, National Institutes Of Health Alpha-glucosidase binders and methods of their use
SG10201605874TA (en) * 2011-04-22 2016-09-29 Genzyme Corp Modified acid alpha glucosidase with accelerated processing
KR20200032244A (ko) 2012-03-07 2020-03-25 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
JP2016514096A (ja) * 2013-02-20 2016-05-19 バレリオン セラピューティクス， エルエルシー ポンぺ病の処置のための方法および組成物
JP6851964B2 (ja) 2014-09-30 2021-03-31 アミカス セラピューティックス インコーポレイテッド 炭水化物が増大している非常に強力な酸性アルファ−グルコシダーゼ
IL299470B2 (en) 2015-12-30 2026-05-01 Amicus Therapeutics Inc Improved acid alpha-glucosidase for the treatment of Pompe disease
CA3019354A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Method for selection of high m6p recombinant proteins
KR102618519B1 (ko) 2016-03-30 2023-12-28 아미쿠스 세라퓨틱스, 인코포레이티드 재조합 산 알파-글루코시다제를 포함하는 제형
CN119113091A (zh) 2017-05-15 2024-12-13 阿米库斯治疗学公司 重组人类酸性α-葡萄糖苷酶
AU2018281280B2 (en) 2017-06-07 2024-12-19 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
CN112040985A (zh) 2018-02-07 2020-12-04 瑞泽恩制药公司 用于递送治疗性蛋白质的方法和组合物
TW202344691A (zh) 2018-04-30 2023-11-16 美商阿米庫斯醫療股份有限公司 基因治療構築體及使用方法
EP3793591A1 (en) 2018-05-17 2021-03-24 Regeneron Pharmaceuticals, Inc. Anti-cd63 antibodies, conjugates, and uses thereof
JP7577650B2 (ja) 2018-10-10 2024-11-05 アミカス セラピューティックス インコーポレイテッド ジスルフィド結合により安定したポリペプチド組成物及びその使用方法
US20220193261A1 (en) * 2019-04-30 2022-06-23 The Trustees Of The University Of Pennsylvania Compositions useful for treatment of pompe disease
EP3871687A1 (en) 2020-02-27 2021-09-01 eleva GmbH Enzyme replacement therapy for treating pompe disease
EP4149519A4 (en) * 2020-05-14 2024-06-12 The Trustees of The University of Pennsylvania COMPOSITIONS FOR THE TREATMENT OF POMPE DISEASE
WO2023204202A1 (ja) * 2022-04-19 2023-10-26 Ｊｃｒファーマ株式会社 マンノース－６－リン酸の数を減少させた糖蛋白質
TW202542190A (zh) 2023-12-20 2025-11-01 美商戴納立製藥公司 包含酸性α—葡萄糖苷酶之融合蛋白及其方法

Family Cites Families (60)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4309776A (en) * 1980-05-13 1982-01-12 Ramon Berguer Intravascular implantation device and method of using the same
US4749570A (en) * 1981-12-31 1988-06-07 The Governors Of The University Of Alberta Targeting conjugates of albumin and therapeutic agents
US4522811A (en) * 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
EP0189481B1 (en) * 1984-07-13 1991-01-23 Chiron Corporation Insulin-like growth factor ii
US4801575A (en) * 1986-07-30 1989-01-31 The Regents Of The University Of California Chimeric peptides for neuropeptide delivery through the blood-brain barrier
US4902505A (en) * 1986-07-30 1990-02-20 Alkermes Chimeric peptides for neuropeptide delivery through the blood-brain barrier
US5470828A (en) * 1987-12-24 1995-11-28 Gropep Pty. Ltd. Peptide analogs of insulin-like growth factor II
US5817623A (en) * 1995-03-06 1998-10-06 Univ Colorado State Res Found Method for treating diabetic peripheral neuropathy with IGF-I or IGF-II
US6451600B1 (en) * 1989-12-22 2002-09-17 Genzyme Corporation Enzymatically active recombinant glucocerebrosidase
US5549892A (en) * 1988-12-23 1996-08-27 Genzyme Corporation Enhanced in vivo uptake of glucocerebrosidase
US5236838A (en) * 1988-12-23 1993-08-17 Genzyme Corporation Enzymatically active recombinant glucocerebrosidase
US5399346A (en) * 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
US5977307A (en) * 1989-09-07 1999-11-02 Alkermes, Inc. Transferrin receptor specific ligand-neuropharmaceutical agent fusion proteins
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
US5401650A (en) * 1990-10-24 1995-03-28 The Mount Sinai School Of Medicine Of The City University Of New York Cloning and expression of biologically active α-galactosidase A
US5633235A (en) * 1991-04-19 1997-05-27 Regents Of The University Of Michigan Triciribine and analogs as antiviral drugs
US5736363A (en) * 1991-07-29 1998-04-07 British Bio-Technology Limited IGF-II analogues
US6270989B1 (en) * 1991-11-05 2001-08-07 Transkaryotic Therapies, Inc. Protein production and delivery
US5258453A (en) * 1992-01-21 1993-11-02 University Of Utah Drug delivery system for the simultaneous delivery of drugs activatable by enzymes and light
EP0646178A1 (en) * 1992-06-04 1995-04-05 The Regents Of The University Of California expression cassette with regularoty regions functional in the mammmlian host
US5981194A (en) * 1992-07-10 1999-11-09 University Of British Columbia Use of p97 and iron binding proteins as diagnostic and therapeutic agents
US5476779A (en) * 1992-09-30 1995-12-19 University Of Maryland At College Park DNA encoding insulin-like growth factor II isolated from rainbow trout
US5633234A (en) * 1993-01-22 1997-05-27 The Johns Hopkins University Lysosomal targeting of immunogens
US5798366A (en) * 1993-05-13 1998-08-25 Monsanto Company Method for treatment of CNS-involved lysosomal storage diseases
US5704910A (en) * 1995-06-05 1998-01-06 Nephros Therapeutics, Inc. Implantable device and use therefor
US6281010B1 (en) * 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
US5817789A (en) * 1995-06-06 1998-10-06 Transkaryotic Therapies, Inc. Chimeric proteins for use in transport of a selected substance into cells
US6118045A (en) * 1995-08-02 2000-09-12 Pharming B.V. Lysosomal proteins produced in the milk of transgenic animals
US20020013953A1 (en) * 1995-08-02 2002-01-31 Reuser Arnold J. Compositions and methods for treating enzyme deficiency
CA2233343A1 (en) * 1995-09-29 1997-04-03 Biomarin Pharmaceutical, Inc. Use of heparinases to decrease inflammatory responses
US6348194B1 (en) * 1995-11-13 2002-02-19 Ixsys Incorporated Tumor specific internalizing antigens and methods for targeting therapeutic agents
US6344436B1 (en) * 1996-01-08 2002-02-05 Baylor College Of Medicine Lipophilic peptides for macromolecule delivery
US6273598B1 (en) * 1996-01-22 2001-08-14 Creative Biomolecules, Inc. Computer system and methods for producing morphogen analogs of human OP-1
US6020144A (en) * 1996-09-12 2000-02-01 Symbiontics, Inc. Sustained delivery device comprising a Leishmania protozoa and methods of making and using the same
US6458574B1 (en) * 1996-09-12 2002-10-01 Transkaryotic Therapies, Inc. Treatment of a α-galactosidase a deficiency
US6083725A (en) * 1996-09-13 2000-07-04 Transkaryotic Therapies, Inc. Tranfected human cells expressing human α-galactosidase A protein
US6235874B1 (en) * 1997-01-10 2001-05-22 Academia Sinica Production of biologically active recombinant insulin-like growth factor II polypeptides
US6329501B1 (en) * 1997-05-29 2001-12-11 Auburn University Methods and compositions for targeting compounds to muscle
ATE359561T1 (de) * 1997-06-02 2007-05-15 Univ Johns Hopkins Rechnerverfahren freie energieberechnung für ligandenentwurf verwendend und die voraussage von bindenden zielen
US6472140B1 (en) * 1997-09-05 2002-10-29 The General Hospital Corporation α-2- macroglobulin therapies and drug screening methods for Alzheimer's disease.
EP1027069B1 (en) * 1997-10-29 2006-07-26 Genzyme Corporation Gene therapy for gaucher disease
DE19832057C1 (de) * 1998-07-16 2000-03-16 Siemens Ag Verfahren zur Regenerierung eines desaktivierten Katalysators
NZ510358A (en) * 1998-08-11 2002-09-27 Large Scale Biology Corp Method for recovering proteins from the interstitial fluid of plant tissues
DK2020438T3 (en) * 1998-12-07 2018-07-23 Genzyme Corp Treatment of Pompe disease
US6596500B1 (en) * 1998-12-08 2003-07-22 The General Hospital Corporation Binding of retinoids to M6P/IGF-II receptor
US6638727B1 (en) * 1999-01-26 2003-10-28 Cytyc Health Corporation Methods for identifying treating or monitoring asymptomatic patients for risk reduction or therapeutic treatment of breast cancer
US6534300B1 (en) * 1999-09-14 2003-03-18 Genzyme Glycobiology Research Institute, Inc. Methods for producing highly phosphorylated lysosomal hydrolases
US20020081654A1 (en) * 2000-04-07 2002-06-27 Sandrin Mauro Sergio Targeting hydrolase enzymes
US7056712B2 (en) * 2000-07-18 2006-06-06 Duke University Treatment of glycogen storage disease type II
US20020142299A1 (en) * 2001-01-09 2002-10-03 Davidson Beverly L. PTD-modified proteins
US7723296B2 (en) * 2001-01-18 2010-05-25 Genzyme Corporation Methods for introducing mannose-6-phosphate and other oligosaccharides onto glycoproteins and its application thereof
US20030004236A1 (en) * 2001-04-20 2003-01-02 Meade Thomas J. Magnetic resonance imaging agents for detection and delivery of therapeutic agents and detection of physiological substances
US7629309B2 (en) * 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
EP1436316B1 (en) * 2001-04-30 2008-01-23 ZyStor Therapeutics , Inc. Subcellular targeting of therapeutic proteins
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
US7658916B2 (en) * 2002-04-05 2010-02-09 Genzyme Corporation Methods of enhancing lysosomal storage disease therapy by modulation of cell surface receptor density
PT1587923E (pt) * 2003-01-22 2011-12-07 Univ Duke Construções melhoradas para a expressão de polipéptidos lisossomais
US20050026823A1 (en) * 2003-06-20 2005-02-03 Biomarin Pharmaceutical Inc. Use of the chaperone receptor-associated protein (RAP) for the delivery of therapeutic compounds to the brain and other tissues
DE602005020745D1 (de) * 2004-02-10 2010-06-02 Zystor Therapeutics Inc Saure alpha-glukosidase und fragmente davon

Also Published As

Publication number Publication date
IL198692A0 (en) 2011-07-31
JP2010509344A (ja) 2010-03-25
US20120093794A1 (en) 2012-04-19
US20090117091A1 (en) 2009-05-07
EP2099523A2 (en) 2009-09-16
AU2007322123A1 (en) 2008-05-29
WO2008063511A2 (en) 2008-05-29
WO2008063511A3 (en) 2009-04-09

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Legal Events

Date Code Title Description
EEER Examination request
FZDE Discontinued

Effective date: 20141113