EP1446007A4 - Methods and compositions for targeting underglycosylated proteins across the blood brain barrier - Google Patents

Methods and compositions for targeting underglycosylated proteins across the 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
targeting
compositions
methods
brain barrier
blood brain
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Withdrawn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)
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Withdrawn legal-status Critical Current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 (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 (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
EP02801739A 2001-10-16 2002-10-16 Methods and compositions for targeting underglycosylated proteins across the 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
US10/136,841 US7396811B2 (en) 2001-04-30 2002-04-30 Subcellular targeting of therapeutic proteins
US136639 2002-04-30
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 Methods and compositions for targeting underglycosylated proteins across the blood brain barrier

Country Status (6)

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

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE60224816T2 (en) 2001-04-30 2009-01-22 ZyStor Therapeutics, Inc., Milwaukee Subcellular targetting of therapeutic proteins
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
WO2003102583A1 (en) * 2002-05-29 2003-12-11 Symbiontics, Inc. Targeted therapeutic proteins
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
CA2463473A1 (en) * 2001-10-16 2003-04-24 Symbiontics 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
EP1716232B9 (en) 2004-02-10 2010-10-13 ZyStor Therapeutics , Inc. Acid alpha-glucosidase and fragments thereof
US8974791B2 (en) 2007-07-27 2015-03-10 Armagen Technologies, Inc. Methods and compositions for increasing α-L-iduronidase activity in the CNS
KR101247418B1 (en) * 2007-12-21 2013-03-25 에프. 호프만-라 로슈 아게 Antibody formulation
EP3778652A1 (en) 2008-05-07 2021-02-17 BioMarin Pharmaceutical Inc. Lysosomal targeting peptides and uses thereof
US8785168B2 (en) 2009-06-17 2014-07-22 Biomarin Pharmaceutical Inc. Formulations for lysosomal enzymes
EP3964229A1 (en) 2010-06-25 2022-03-09 Shire Human Genetic Therapies, Inc. Methods and compositions for cns delivery of iduronate-2-sulfatase
NZ605871A (en) * 2010-06-25 2015-02-27 Shire Human Genetic Therapies Treatment of sanfilippo syndrome type b
CN103282046B (en) 2010-06-25 2015-05-13 夏尔人类遗传性治疗公司 Methods and compositions for CNS delivery of arylsulfatase A
ES2650689T3 (en) 2010-06-25 2018-01-19 Shire Human Genetic Therapies, Inc. Administration of therapeutic agents to the central nervous system
CA3171308A1 (en) 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc. Methods and compositions for cns delivery of iduronate-2-sulfatase
JP6073783B2 (en) 2010-06-25 2017-02-01 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 Methods and compositions for CNS delivery of heparan N-sulfatase
CA2857567A1 (en) * 2011-12-01 2013-06-06 Angiochem Inc. Targeted lysosomal enzyme compounds
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
HUE039334T2 (en) 2012-11-27 2018-12-28 Biomarin Pharm Inc Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof
SG11201506499YA (en) 2013-03-15 2015-09-29 Glaxosmithkline Ip No 2 Ltd Low concentration antibody formulations
ES2687262T3 (en) 2014-04-01 2018-10-24 Swedish Orphan Biovitrum Ab (Publ) Modified sulfamidase and production thereof
CA2989400A1 (en) 2015-06-15 2016-12-22 Angiochem Inc. Ang1005 for the treatment of leptomeningeal carcinomatosis
AU2017222620B2 (en) 2016-02-24 2022-06-16 Biomarin Pharmaceutical Inc. Targeted therapeutic lysosomal enzyme fusion proteins, associated formulations and uses thereof
AU2019261982A1 (en) * 2018-04-30 2020-10-15 Amicus Therapeutics, Inc. Gene therapy constructs and methods of use
EP3863663A2 (en) 2018-10-10 2021-08-18 Amicus Therapeutics, Inc. Disulfide bond stabilized polypeptide compositions and methods of use
JP7458404B2 (en) 2018-12-20 2024-03-29 アーマジェン・インコーポレイテッド Purification of iduronic acid-2-sulfatase immunoglobulin fusion protein

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

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
JP2005506340A (en) 2005-03-03
WO2003032913A3 (en) 2004-08-12
JP2009203241A (en) 2009-09-10
IL161352A0 (en) 2004-09-27
WO2003032727A1 (en) 2003-04-24
AU2002362930A2 (en) 2003-04-28
AU2010214643A1 (en) 2010-09-16
WO2003032913A9 (en) 2004-04-29
CA2463473A1 (en) 2003-04-24
WO2003032913A2 (en) 2003-04-24
AU2002347910A1 (en) 2003-04-28
EP1446007A1 (en) 2004-08-18

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Legal Events

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Owner name: ZYSTOR THERAPEUTICS , INC.

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Inventor name: SLY, WILIAN, S.

Inventor name: LEBOWITZ, JONATHAN, H.

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Inventor name: SLY, WILIAN, S.

Inventor name: DR JONATHAN LEBOWITZ

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