CA2669347A1 - Methods for treating pompe disease - Google Patents

Methods for treating pompe disease Download PDF

Info

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

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/62Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs 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)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Diabetes (AREA)
  • Neurology (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 (enExample)
EP (1) EP2099523A2 (enExample)
JP (1) JP2010509344A (enExample)
AU (1) AU2007322123A1 (enExample)
CA (1) CA2669347A1 (enExample)
IL (1) IL198692A0 (enExample)
WO (1) WO2008063511A2 (enExample)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2572148T3 (es) 2005-05-17 2016-05-30 Amicus Therapeutics Inc Un método para el tratamiento de la enfermedad de Pompe usando 1-desoxinojirimicina y derivados
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
PE20140617A1 (es) * 2011-04-22 2014-05-28 Genzyme Corp Alfa glucosidasa acida modificada con procesamiento acelerado
KR20140135222A (ko) 2012-03-07 2014-11-25 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
WO2014130723A1 (en) * 2013-02-20 2014-08-28 Valerion Therapeutics, Llc Methods and compositions for treatment of pompe disease
DK3201320T5 (da) 2014-09-30 2024-10-14 Amicus Therapeutics Inc Højpotent sur alfa-glucosidase med forbedrede carbohydrater
MX394968B (es) 2015-12-30 2025-03-24 Amicus Therapeutics Inc Alfa-glucosidasa con mayor cantidad de acido para el tratamiento de la enfermedad de pompe.
CA3019128A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
KR102343162B1 (ko) 2016-03-30 2021-12-23 아미쿠스 세라퓨틱스, 인코포레이티드 고 m6p 재조합 단백질의 선택 방법
SMT202300187T1 (it) 2017-05-15 2023-07-20 Amicus Therapeutics Inc Alfa-glucosidasi acida umana ricombinante
CN110809583A (zh) 2017-06-07 2020-02-18 瑞泽恩制药公司 用于内化酶的组合物和方法
KR20200118151A (ko) 2018-02-07 2020-10-14 리제너론 파마슈티칼스 인코포레이티드 치료 단백질 전달을 위한 방법 및 조성물
BR112020021962A2 (pt) 2018-04-30 2021-01-26 Amicus Therapeutics, Inc. construtos para terapia gênica e métodos de uso
MX2020012350A (es) 2018-05-17 2021-01-29 Regeneron Pharma Anticuerpos anti-cd63, conjugados y usos de estos.
EP3863663A2 (en) 2018-10-10 2021-08-18 Amicus Therapeutics, Inc. Disulfide bond stabilized polypeptide compositions and methods of use
BR112021021720A2 (pt) * 2019-04-30 2021-12-28 Univ Pennsylvania Composições úteis para tratamento de doença de pompe
EP3871687A1 (en) 2020-02-27 2021-09-01 eleva GmbH Enzyme replacement therapy for treating pompe disease
WO2021231863A1 (en) * 2020-05-14 2021-11-18 The Trustees Of The University Of Pennsylvania Compositions useful for treatment of pompe disease
JP2023159049A (ja) * 2022-04-19 2023-10-31 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
WO2025137213A1 (en) 2023-12-20 2025-06-26 Denali Therapeutics Inc. Fusion proteins comprising acid alpha-glucosidase enzymes and methods thereof

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
WO1986000619A1 (en) * 1984-07-13 1986-01-30 Chiron Corporation Prepro insulin-like growth factors i and ii
US4902505A (en) * 1986-07-30 1990-02-20 Alkermes Chimeric peptides for neuropeptide delivery through the blood-brain barrier
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
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
EP0596969A1 (en) * 1991-07-29 1994-05-18 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
AU4528493A (en) * 1992-06-04 1994-01-04 Regents Of The University Of California, The In vivo gene therapy with intron-free sequence of interest
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
JP3713276B2 (ja) * 1995-09-29 2005-11-09 バイオマリン ファーマシューティカル インコーポレイテッド 炎症反応の軽減のためのヘパリナーゼの使用
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
JP2000501744A (ja) * 1996-01-22 2000-02-15 クリエイティブ バイオモレキゥルズ,インコーポレーテッド モルホゲン類似体およびその製法
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
DE69837555T2 (de) * 1997-06-02 2007-12-20 The Johns Hopkins University 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
AU759813B2 (en) * 1998-08-11 2003-05-01 Kentucky Bioprocessing, Llc Method for recovering proteins from the interstitial fluid of plant tissues
WO2000034451A1 (en) * 1998-12-07 2000-06-15 Pharming Intellectual Property B.V. Treatment of pompe's 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
ES2799882T3 (es) * 2000-07-18 2020-12-22 Univ Duke Tratamiento de glucogenosis de tipo 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
US20040005309A1 (en) * 2002-05-29 2004-01-08 Symbiontics, Inc. Targeted therapeutic proteins
ATE384736T1 (de) * 2001-04-30 2008-02-15 Zystor Therapeutics Inc Subzelluläres targeting von therapeutischen proteinen
US7629309B2 (en) * 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US7560424B2 (en) * 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
AU2003224880A1 (en) * 2002-04-05 2003-10-27 Genzyme Corporation Methods of enhancing lysosomal storage disease therapy
US20040248262A1 (en) * 2003-01-22 2004-12-09 Koeberl Dwight D. Constructs for expressing lysomal polypeptides
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
ES2344302T3 (es) * 2004-02-10 2010-08-24 Zystor Therapeutics , Inc. Alfa glucosidasa acida y fragmentos de la misma.

Also Published As

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

Similar Documents

Publication Publication Date Title
US20090117091A1 (en) Methods for treating pompe disease
KR102521039B1 (ko) 표적화된 치료적 리소좀 효소 융합 단백질들 및 그들의 용도들
EP3272773B1 (en) Lyosomal targeting peptides and uses thereof
Boado et al. Reduction in brain heparan sulfate with systemic administration of an IgG Trojan Horse–sulfamidase fusion protein in the mucopolysaccharidosis type IIIA mouse
EP1981546A2 (en) Enzyme replacement therapy for treating lysosomal storage diseases
CN101636200A (zh) 用于治疗庞贝氏症的方法
HK1232896A1 (en) Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof
HK1250042B (en) Lysosomal targeting peptides and uses thereof
HK1240250A1 (en) Lysosomal targeting peptides and uses thereof
HK1232896B (en) Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof
HK1216026B (en) Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof
HK1153204A (en) Lysosomal targeting peptides and uses thereof
HK1153204B (en) Lysosomal targeting peptides and uses thereof

Legal Events

Date Code Title Description
EEER Examination request
FZDE Discontinued

Effective date: 20141113