IL161352A0 - 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
IL161352A0
IL161352A0 IL16135202A IL16135202A IL161352A0 IL 161352 A0 IL161352 A0 IL 161352A0 IL 16135202 A IL16135202 A IL 16135202A IL 16135202 A IL16135202 A IL 16135202A IL 161352 A0 IL161352 A0 IL 161352A0
Authority
IL
Israel
Prior art keywords
targeting
compositions
methods
brain barrier
blood brain
Prior art date
Application number
IL16135202A
Original Assignee
Symbiontics 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 Symbiontics Inc filed Critical Symbiontics Inc
Publication of IL161352A0 publication Critical patent/IL161352A0/en

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)
  • 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)
IL16135202A 2001-10-16 2002-10-16 Methods and compositions for targeting underglycosylated proteins across the blood brain barrier IL161352A0 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US32965001P 2001-10-16 2001-10-16
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
US38445202P 2002-05-29 2002-05-29
US38601902P 2002-06-05 2002-06-05
US40881602P 2002-09-06 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 (1)

Publication Number Publication Date
IL161352A0 true IL161352A0 (en) 2004-09-27

Family

ID=27558195

Family Applications (1)

Application Number Title Priority Date Filing Date
IL16135202A IL161352A0 (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 (29)

* 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
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
JP4641705B2 (en) 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 Subcellular targeting of therapeutic proteins
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
JP2005506340A (en) * 2001-10-16 2005-03-03 シムバイオンティクス インコーポレイテッド Methods and compositions for targeting underglycosylated proteins across the blood brain barrier
EP1514106A4 (en) * 2002-05-29 2007-05-09 Zystor Therapeutics Inc Targeted therapeutic proteins
WO2005078077A2 (en) * 2004-02-10 2005-08-25 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
CA3184105A1 (en) 2007-07-27 2009-02-05 Armagen Inc. Methods and compositions for increasing alpha-l-iduronidase activity in the cns
MX2010006395A (en) * 2007-12-21 2010-07-01 Hoffmann La Roche Antibody formulation.
PL2279210T3 (en) 2008-05-07 2017-10-31 Biomarin Pharm Inc Lysosomal targeting peptides and uses thereof
EP3679942A1 (en) 2009-06-17 2020-07-15 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
PL2485761T3 (en) 2009-10-09 2019-10-31 Armagen Inc Methods and compositions for increasing iduronate 2-sulfatase activity in the cns
NZ702808A (en) 2010-06-25 2016-08-26 Shire Human Genetic Therapies Methods and compositions for cns delivery of iduronate-2-sulfatase
LT2585104T (en) 2010-06-25 2019-10-25 Shire Human Genetic Therapies Methods and compositions for cns delivery of arylsulfatase a
KR102211960B1 (en) 2010-06-25 2021-02-05 샤이어 휴먼 지네틱 테라피즈 인크. CNS delivery of therapeutic agents
ES2643015T3 (en) 2010-06-25 2017-11-21 Shire Human Genetic Therapies, Inc. Treatment of Sanfilippo Syndrome Type B
KR20140005842A (en) 2010-06-25 2014-01-15 샤이어 휴먼 지네틱 테라피즈 인크. Methods and compositions for cns delivery of heparan n-sulfatase
HUE031036T2 (en) 2010-06-25 2017-06-28 Shire Human Genetic Therapies Cns delivery of therapeutic agents
BR112014013161A2 (en) * 2011-12-01 2019-09-24 Angiochem Inc lysosomal enzyme target compounds
EP2793922B1 (en) 2011-12-23 2019-10-30 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
JP2015526434A (en) 2012-08-14 2015-09-10 アンジオケム インコーポレーテッド Peptide-dendrimer conjugates and uses thereof
DK2925776T3 (en) 2012-11-27 2018-09-03 Biomarin Pharm Inc TARGETED THERAPEUTIC LYSOSOMAL ENZYMUS FUSION PROTEINS AND APPLICATIONS THEREOF
US10537638B2 (en) 2013-03-15 2020-01-21 Glaxosmithkline Intellectual Property (No.2) Limited Low concentration antibody formulations
LT2970413T (en) 2014-04-01 2018-10-10 Swedish Orphan Biovitrum Ab (Publ) Modified sulfamidase and production thereof
JP6823055B2 (en) 2015-06-15 2021-01-27 アンジオケム インコーポレーテッド How to treat soft meningeal carcinomatosis
RU2751235C2 (en) 2016-02-24 2021-07-12 Байомарин Фармасьютикал Инк. Therapeutic targeted fusion proteins based on lysosomal enzymes, relevant compositions and their applications
SG11202010533WA (en) * 2018-04-30 2020-11-27 Amicus Therapeutics Inc Gene therapy constructs and methods of use
SG11202103640VA (en) 2018-10-10 2021-05-28 Amicus Therapeutics Inc Disulfide bond stabilized polypeptide compositions and methods of use
US20220002688A1 (en) 2018-12-20 2022-01-06 Armagen, 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
WO1991004014A1 (en) * 1989-09-21 1991-04-04 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.
JP4641705B2 (en) * 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 Subcellular targeting of therapeutic proteins
JP2005506340A (en) * 2001-10-16 2005-03-03 シムバイオンティクス インコーポレイテッド Methods and compositions for targeting underglycosylated proteins across the blood brain barrier
EP1514106A4 (en) * 2002-05-29 2007-05-09 Zystor Therapeutics Inc Targeted therapeutic proteins

Also Published As

Publication number Publication date
WO2003032913A2 (en) 2003-04-24
AU2002362930A2 (en) 2003-04-28
CA2463473A1 (en) 2003-04-24
JP2005506340A (en) 2005-03-03
WO2003032727A1 (en) 2003-04-24
WO2003032913A3 (en) 2004-08-12
AU2010214643A1 (en) 2010-09-16
EP1446007A1 (en) 2004-08-18
JP2009203241A (en) 2009-09-10
EP1446007A4 (en) 2005-12-07
AU2002347910A1 (en) 2003-04-28
WO2003032913A9 (en) 2004-04-29

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