AU2002362930A2 - 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 Download PDF

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Publication number
AU2002362930A2
AU2002362930A2 AU2002362930A AU2002362930A AU2002362930A2 AU 2002362930 A2 AU2002362930 A2 AU 2002362930A2 AU 2002362930 A AU2002362930 A AU 2002362930A AU 2002362930 A AU2002362930 A AU 2002362930A AU 2002362930 A2 AU2002362930 A2 AU 2002362930A2
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AU
Australia
Prior art keywords
igf
therapeutic
protein
fusion protein
cell
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
AU2002362930A
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English (en)
Inventor
Jonathan H Lebowitz
William 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.)
Zystor Therapeutics 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 AU2002362930A2 publication Critical patent/AU2002362930A2/en
Assigned to ZYSTOR THERAPEUTICS, INC. reassignment ZYSTOR THERAPEUTICS, INC. Request for Assignment Assignors: SYMBIONTICS, INC.
Priority to AU2008200706A priority Critical patent/AU2008200706B2/en
Abandoned legal-status Critical Current

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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)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Endocrinology (AREA)
  • Diabetes (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Urology & Nephrology (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Cardiology (AREA)
  • Plant Pathology (AREA)
  • Vascular Medicine (AREA)
  • Cell Biology (AREA)
  • Physics & Mathematics (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Neurosurgery (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
AU2002362930A 2001-10-16 2002-10-16 Methods and compositions for targeting underglycosylated proteins across the blood brain barrier Abandoned AU2002362930A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2008200706A AU2008200706B2 (en) 2001-10-16 2008-02-14 Methods and compositions for targeting underglycosylated proteins across the blood brain barrier

Applications Claiming Priority (13)

Application Number Priority Date Filing Date Title
US32965001P 2001-10-16 2001-10-16
US60/329,650 2001-10-16
US10/136,841 2002-04-30
US10/136,841 US7396811B2 (en) 2001-04-30 2002-04-30 Subcellular targeting of therapeutic proteins
US10/136,639 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
US38445202P 2002-05-29 2002-05-29
US60/384,452 2002-05-29
US38601902P 2002-06-05 2002-06-05
US60/386,019 2002-06-05
US40881602P 2002-09-06 2002-09-06
US60/408,816 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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2008200706A Division AU2008200706B2 (en) 2001-10-16 2008-02-14 Methods and compositions for targeting underglycosylated proteins across the blood brain barrier

Publications (1)

Publication Number Publication Date
AU2002362930A2 true AU2002362930A2 (en) 2003-04-28

Family

ID=27558195

Family Applications (3)

Application Number Title Priority Date Filing Date
AU2002362930A Abandoned AU2002362930A2 (en) 2001-10-16 2002-10-16 Methods and compositions for targeting underglycosylated proteins across the blood brain barrier
AU2002347910A Abandoned AU2002347910A1 (en) 2001-10-16 2002-10-16 Methods and compositions for targeting proteins across the blood brain barrier
AU2010214643A Abandoned AU2010214643A1 (en) 2001-10-16 2010-08-25 Methods and compositions for targeting underglycosylated proteins across the blood brain barrier

Family Applications After (2)

Application Number Title Priority Date Filing Date
AU2002347910A Abandoned AU2002347910A1 (en) 2001-10-16 2002-10-16 Methods and compositions for targeting proteins across the blood brain barrier
AU2010214643A Abandoned AU2010214643A1 (en) 2001-10-16 2010-08-25 Methods and compositions for targeting underglycosylated proteins across the blood brain barrier

Country Status (6)

Country Link
EP (1) EP1446007A4 (enExample)
JP (2) JP2005506340A (enExample)
AU (3) AU2002362930A2 (enExample)
CA (1) CA2463473A1 (enExample)
IL (1) IL161352A0 (enExample)
WO (2) WO2003032913A2 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2003237314B2 (en) * 2002-05-29 2010-03-04 Biomarin Pharmaceutical Inc. Targeted therapeutic proteins

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL158623A0 (en) 2001-04-30 2004-05-12 Symbiontics Inc Subcellular targeting of therapeutic proteins
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
AU2002362930A2 (en) * 2001-10-16 2003-04-28 Zystor Therapeutics, Inc. Methods and compositions for targeting underglycosylated proteins across the blood brain barrier
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
US20050244400A1 (en) * 2004-02-10 2005-11-03 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
EP2997976A1 (en) 2007-07-27 2016-03-23 Armagen Technologies, Inc. Methods and compositions for increasing alpha-l-iduronidase activity in the cns
EP2234600B1 (en) * 2007-12-21 2014-08-20 F. Hoffmann-La Roche AG Antibody formulation
HUE034850T2 (en) 2008-05-07 2018-03-28 Biomarin Pharm Inc Lysosomal targeting peptides and their use
EP3679942A1 (en) 2009-06-17 2020-07-15 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
WO2011044542A1 (en) 2009-10-09 2011-04-14 Armagen Technologies, Inc. Methods and compositions for increasing iduronate 2-sulfatase activity in the cns
IL291554B2 (en) 2010-06-25 2024-02-01 Shire Human Genetic Therapies Administering therapeutic agents to the central nervous system
WO2011163652A2 (en) 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc. Treatment of sanfilippo syndrome type b
MX2013000320A (es) 2010-06-25 2013-06-05 Shire Human Genetic Therapies Composiciones y metodos para suministro al sistema nervioso central de heparan n-sulfatasa.
ME03649B (me) 2010-06-25 2020-07-20 Shire Human Genetic Therapies Postupci i kompozicije za isporuku iduronat-2-sulfataze u cns
MX382443B (es) 2010-06-25 2025-03-11 Shire Human Genetic Therapies Una formulación estable para usarse en el tratamiento de la enfermedad por leucodistrofia metacromática (mld).
PE20130589A1 (es) 2010-06-25 2013-05-19 Shire Human Genetic Therapies Composiciones y metodos para suministro al sistema nervioso central de arilsulfatasa a
HK1200189A1 (en) * 2011-12-01 2015-07-31 安吉奥开米公司 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
US9687561B2 (en) 2012-08-14 2017-06-27 Angiochem Inc. Peptide-dendrimer conjugates and uses thereof
KR102521039B1 (ko) 2012-11-27 2023-04-12 바이오마린 파머수티컬 인크. 표적화된 치료적 리소좀 효소 융합 단백질들 및 그들의 용도들
KR20150132332A (ko) 2013-03-15 2015-11-25 글락소스미스클라인 인털렉츄얼 프로퍼티 (넘버 2) 리미티드 저농도 항체 제형
CA2943140A1 (en) 2014-04-01 2015-10-08 Swedish Orphan Biovitrum Ab (Publ) Modified sulfamidase and production thereof
CN107921143B (zh) 2015-06-15 2021-11-19 安吉奥开米公司 用于治疗软脑膜癌病的方法
AU2017222620B2 (en) 2016-02-24 2022-06-16 Biomarin Pharmaceutical Inc. Targeted therapeutic lysosomal enzyme fusion proteins, associated formulations and uses thereof
US10874750B2 (en) 2018-04-30 2020-12-29 Amicus Therapeutics, Inc. Gene therapy constructs and methods of use
WO2020077114A2 (en) 2018-10-10 2020-04-16 Amicus Therapeutics, Inc. Disulfide bond stabilized polypeptide compositions and methods of use
WO2020132452A1 (en) 2018-12-20 2020-06-25 Shire Human Genetic Therapies, Inc. Purification of iduronate-2-sulfatase immunoglobulin fusion protein

Family Cites Families (7)

* 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
US5672683A (en) * 1989-09-07 1997-09-30 Alkermes, Inc. Transferrin neuropharmaceutical agent fusion protein
AU6501390A (en) * 1989-09-21 1991-04-18 Synergen, Inc. Method for transporting compositions across 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.
IL158623A0 (en) * 2001-04-30 2004-05-12 Symbiontics Inc Subcellular targeting of therapeutic proteins
AU2002362930A2 (en) * 2001-10-16 2003-04-28 Zystor Therapeutics, Inc. Methods and compositions for targeting underglycosylated proteins across the blood brain barrier
AU2003237314B2 (en) * 2002-05-29 2010-03-04 Biomarin Pharmaceutical Inc. Targeted therapeutic proteins

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2003237314B2 (en) * 2002-05-29 2010-03-04 Biomarin Pharmaceutical Inc. Targeted therapeutic proteins

Also Published As

Publication number Publication date
WO2003032913A9 (en) 2004-04-29
EP1446007A1 (en) 2004-08-18
AU2010214643A1 (en) 2010-09-16
JP2009203241A (ja) 2009-09-10
WO2003032913A3 (en) 2004-08-12
IL161352A0 (en) 2004-09-27
JP2005506340A (ja) 2005-03-03
WO2003032727A1 (en) 2003-04-24
CA2463473A1 (en) 2003-04-24
EP1446007A4 (en) 2005-12-07
WO2003032913A2 (en) 2003-04-24
AU2002347910A1 (en) 2003-04-28

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Legal Events

Date Code Title Description
DA3 Amendments made section 104

Free format text: THE NATURE OF THE AMENDMENT IS AS SHOWN IN THE STATEMENT(S) FILED 16 APR 2004

DA3 Amendments made section 104

Free format text: THE NATURE OF THE AMENDMENT IS: AMEND THE NAME OF THE CO-INVENTOR FROM SLY, WILIAN S TO SLY, WILLIAM S.

PC1 Assignment before grant (sect. 113)

Owner name: ZYSTOR THERAPEUTICS, INC.

Free format text: FORMER APPLICANT(S): SYMBIONTICS, INC.

MK5 Application lapsed section 142(2)(e) - patent request and compl. specification not accepted