EP1446007A4 - METHOD AND COMPOSITION FOR TARGETING HYPOGLYCOSYLATED PROTEINS VIA BLOOD-BRAIN BARRIER - Google Patents

METHOD AND COMPOSITION FOR TARGETING HYPOGLYCOSYLATED PROTEINS VIA BLOOD-BRAIN BARRIER

Info

Publication number
EP1446007A4
EP1446007A4 EP02801739A EP02801739A EP1446007A4 EP 1446007 A4 EP1446007 A4 EP 1446007A4 EP 02801739 A EP02801739 A EP 02801739A EP 02801739 A EP02801739 A EP 02801739A EP 1446007 A4 EP1446007 A4 EP 1446007A4
Authority
EP
European Patent Office
Prior art keywords
subglycosylated
hemato
targeting
proteins
compositions
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.)
Withdrawn
Application number
EP02801739A
Other languages
German (de)
English (en)
French (fr)
Other versions
EP1446007A1 (en
Inventor
Jonathan H Lebowitz
Wilian S Sly
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
Zystor Therapeutics Inc
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
Priority claimed from US10/136,841 external-priority patent/US7396811B2/en
Priority claimed from US10/136,639 external-priority patent/US20030072761A1/en
Application filed by Zystor Therapeutics Inc filed Critical Zystor Therapeutics Inc
Publication of EP1446007A1 publication Critical patent/EP1446007A1/en
Publication of EP1446007A4 publication Critical patent/EP1446007A4/en
Withdrawn legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62—DNA sequences coding for fusion proteins
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/22—Hormones
    • A61K38/30—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
    • 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
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/642—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent the peptide or protein in the drug conjugate being a cytokine, e.g. IL2, chemokine, growth factors or interferons being the inactive part of the conjugate
    • 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
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/6425—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent the peptide or protein in the drug conjugate being a receptor, e.g. CD4, a cell surface antigen, i.e. not a peptide ligand targeting the antigen, or a cell surface determinant, i.e. a part of the surface of a cell
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous 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
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575—Hormones
    • C07K14/65—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/06—Fusion polypeptide containing a localisation/targetting motif containing a lysosomal/endosomal localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/20—Fusion polypeptide containing a tag with affinity for a non-protein ligand
    • C07K2319/21—Fusion polypeptide containing a tag with affinity for a non-protein ligand containing a His-tag
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/50—Fusion polypeptide containing protease site
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/61—Fusion polypeptide containing an enzyme fusion for detection (lacZ, luciferase)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/74—Fusion polypeptide containing domain for protein-protein interaction containing a fusion for binding to a cell surface receptor
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/74—Fusion polypeptide containing domain for protein-protein interaction containing a fusion for binding to a cell surface receptor
    • C07K2319/75—Fusion polypeptide containing domain for protein-protein interaction containing a fusion for binding to a cell surface receptor containing a fusion for activation of a cell surface receptor, e.g. thrombopoeitin, NPY and other peptide hormones

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Genetics & Genomics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Endocrinology (AREA)
  • Biotechnology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Urology & Nephrology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Neurosurgery (AREA)
  • Vascular Medicine (AREA)
  • Cardiology (AREA)
  • Physics & Mathematics (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cell Biology (AREA)
  • Neurology (AREA)
  • Toxicology (AREA)
EP02801739A 2001-10-16 2002-10-16 METHOD AND COMPOSITION FOR TARGETING HYPOGLYCOSYLATED PROTEINS VIA BLOOD-BRAIN BARRIER Withdrawn EP1446007A4 (en)

Applications Claiming Priority (13)

Application Number Priority Date Filing Date Title
US32965001P 2001-10-16 2001-10-16
US329650P 2001-10-16
US136639 2002-04-30
US10/136,841 US7396811B2 (en) 2001-04-30 2002-04-30 Subcellular targeting of therapeutic proteins
US10/136,639 US20030072761A1 (en) 2001-10-16 2002-04-30 Methods and compositions for targeting proteins across the blood brain barrier
US136841 2002-04-30
US38445202P 2002-05-29 2002-05-29
US384452P 2002-05-29
US38601902P 2002-06-05 2002-06-05
US386019P 2002-06-05
US40881602P 2002-09-06 2002-09-06
US408816P 2002-09-06
PCT/US2002/032968 WO2003032727A1 (en) 2001-10-16 2002-10-16 Methods and compositions for targeting underglycosylated proteins across the blood brain barrier

Publications (2)

Publication Number Publication Date
EP1446007A1 EP1446007A1 (en) 2004-08-18
EP1446007A4 true EP1446007A4 (en) 2005-12-07

Family

ID=27558195

Family Applications (1)

Application Number Title Priority Date Filing Date
EP02801739A Withdrawn EP1446007A4 (en) 2001-10-16 2002-10-16 METHOD AND COMPOSITION FOR TARGETING HYPOGLYCOSYLATED PROTEINS VIA BLOOD-BRAIN BARRIER

Country Status (6)

Country Link
EP (1) EP1446007A4 (enExample)
JP (2) JP2005506340A (enExample)
AU (3) AU2002347910A1 (enExample)
CA (1) CA2463473A1 (enExample)
IL (1) IL161352A0 (enExample)
WO (2) WO2003032913A2 (enExample)

Families Citing this family (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
WO2003102583A1 (en) * 2002-05-29 2003-12-11 Symbiontics, Inc. Targeted therapeutic proteins
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
JP4641705B2 (ja) 2001-04-30 2011-03-02 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 治療的タンパク質の亜細胞標的化
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
CA2463473A1 (en) * 2001-10-16 2003-04-24 Symbiontics Inc. Targeted therapeutic proteins
ES2344302T3 (es) * 2004-02-10 2010-08-24 Zystor Therapeutics , Inc. Alfa glucosidasa acida y fragmentos de la misma.
AU2008282496B2 (en) 2007-07-27 2013-04-04 Armagen Technologies, Inc. Methods and compositions for increasing alpha-iduronidase activity in the CNS
KR101247418B1 (ko) * 2007-12-21 2013-03-25 에프. 호프만-라 로슈 아게 항체 제형
CN102066422B (zh) 2008-05-07 2015-07-22 宝玛瑞制药公司 溶酶体靶向肽及其应用
WO2010148253A2 (en) 2009-06-17 2010-12-23 Zystor Therapeutics, Inc. Formulations for lysosomal enzymes
PL2485761T3 (pl) 2009-10-09 2019-10-31 Armagen Inc Sposoby i kompozycje do zwiększania aktywności 2-sulfatazy iduronianu w cns
EP3875107A1 (en) 2010-06-25 2021-09-08 Shire Human Genetic Therapies, Inc. Pharmaceutical formulation comprising a replacement enzyme for a lysosomal enzyme for use in treating lysosomal storage disease intrathecally
DK2585104T3 (da) 2010-06-25 2019-10-14 Shire Human Genetic Therapies Fremgangsmåder og sammensætninger tilcns-levering af arylsulfatase a
ES2858726T3 (es) * 2010-06-25 2021-09-30 Shire Human Genetic Therapies Administración de agentes terapéuticos al SNC
ES2754234T3 (es) 2010-06-25 2020-04-16 Shire Human Genetic Therapies Métodos y composiciones para la administración SNC de iduronato-2-sulfatasa
CN103260637B (zh) 2010-06-25 2016-04-06 夏尔人类遗传性治疗公司 乙酰肝素n-硫酸酯酶cns递送的方法和组合物
JP6063380B2 (ja) 2010-06-25 2017-01-18 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド サンフィリポ症候群ｂ型の処置
JP2015505824A (ja) * 2011-12-01 2015-02-26 アンジオケム インコーポレーテッド 標的化リソソーム酵素化合物
WO2013096899A2 (en) 2011-12-23 2013-06-27 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
WO2014026283A1 (en) 2012-08-14 2014-02-20 Angiochem Inc. Peptide-dendrimer conjugates and uses thereof
MX377150B (es) 2012-11-27 2025-03-07 Biomarin Pharm Inc Proteinas de fusion de enzima lisosomal terapeutica objetivo y uso de las mismas.
WO2014141152A2 (en) 2013-03-15 2014-09-18 Glaxosmithkline Intellectual Property (No.2) Limited Low concentration antibody formulations
LT2970413T (lt) 2014-04-01 2018-10-10 Swedish Orphan Biovitrum Ab (Publ) Modifikuota sulfamidazė ir jos gamyba
DK3307326T3 (da) 2015-06-15 2020-10-19 Angiochem Inc Fremgangsmåder til behandling af leptomeningeal karcinomatose
JP7109369B2 (ja) 2016-02-24 2022-07-29 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 ターゲティングされた治療用リソソーム酵素融合タンパク質、関連する製剤、およびその使用
EP3788064A1 (en) * 2018-04-30 2021-03-10 Amicus Therapeutics, Inc. Gene therapy constructs and methods of use
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
EP3898689A1 (en) 2018-12-20 2021-10-27 Armagen, Inc. Purification of iduronate-2-sulfatase immunoglobulin fusion protein
CN116997364A (zh) * 2021-02-11 2023-11-03 戴纳立制药公司 抗转铁蛋白受体融合蛋白及其使用方法

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1991004014A1 (en) * 1989-09-21 1991-04-04 Synergen, Inc. Method for transporting compositions across the blood brain barrier
WO1995002421A1 (en) * 1993-07-16 1995-01-26 Alkermes, Inc. Transferrin receptor specific ligand-neuropharmaceutical agent fusion proteins
WO2002087510A2 (en) * 2001-04-30 2002-11-07 Symbiontics, Inc. Subcellular targeting of therapeutic proteins
WO2003032913A2 (en) * 2001-10-16 2003-04-24 Symbiontics Inc. Methods and compositions for targeting proteins across the blood brain barrier
WO2003102583A1 (en) * 2002-05-29 2003-12-11 Symbiontics, Inc. Targeted therapeutic proteins

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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
US6472140B1 (en) * 1997-09-05 2002-10-29 The General Hospital Corporation α-2- macroglobulin therapies and drug screening methods for Alzheimer's disease.

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1991004014A1 (en) * 1989-09-21 1991-04-04 Synergen, Inc. Method for transporting compositions across the blood brain barrier
WO1995002421A1 (en) * 1993-07-16 1995-01-26 Alkermes, Inc. Transferrin receptor specific ligand-neuropharmaceutical agent fusion proteins
WO2002087510A2 (en) * 2001-04-30 2002-11-07 Symbiontics, Inc. Subcellular targeting of therapeutic proteins
WO2003032913A2 (en) * 2001-10-16 2003-04-24 Symbiontics Inc. Methods and compositions for targeting proteins across the blood brain barrier
WO2003102583A1 (en) * 2002-05-29 2003-12-11 Symbiontics, Inc. Targeted therapeutic proteins

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
DUFFY K R ET AL: "Human blood-brain barrier insulin-like growth factor receptor", METABOLISM, CLINICAL AND EXPERIMENTAL, W.B. SAUNDERS CO., PHILADELPHIA, PA, US, vol. 37, no. 2, 1 February 1988 (1988-02-01), pages 136 - 140, XP026359542, ISSN: 0026-0495, [retrieved on 19880201] *
See also references of WO03032727A1 *

Also Published As

Publication number Publication date
WO2003032913A3 (en) 2004-08-12
IL161352A0 (en) 2004-09-27
WO2003032913A2 (en) 2003-04-24
JP2009203241A (ja) 2009-09-10
AU2002362930A2 (en) 2003-04-28
WO2003032727A1 (en) 2003-04-24
JP2005506340A (ja) 2005-03-03
AU2002347910A1 (en) 2003-04-28
EP1446007A1 (en) 2004-08-18
AU2010214643A1 (en) 2010-09-16
WO2003032913A9 (en) 2004-04-29
CA2463473A1 (en) 2003-04-24

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