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
Other languages
English (en)
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 US10/136,639 external-priority patent/US20030072761A1/en
Priority claimed from US10/136,841 external-priority patent/US7396811B2/en
Application filed by Symbiontics Inc filed Critical Symbiontics Inc
Publication of IL161352A0 publication Critical patent/IL161352A0/xx

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62DNA sequences coding for fusion proteins
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/22Hormones
    • A61K38/30Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
    • 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
    • A61K47/64Drug-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
    • A61K47/642Drug-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
    • 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
    • A61K47/64Drug-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
    • A61K47/6425Drug-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575Hormones
    • C07K14/65Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/06Fusion polypeptide containing a localisation/targetting motif containing a lysosomal/endosomal localisation signal
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/20Fusion polypeptide containing a tag with affinity for a non-protein ligand
    • C07K2319/21Fusion polypeptide containing a tag with affinity for a non-protein ligand containing a His-tag
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/50Fusion polypeptide containing protease site
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/61Fusion polypeptide containing an enzyme fusion for detection (lacZ, luciferase)
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/70Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/74Fusion polypeptide containing domain for protein-protein interaction containing a fusion for binding to a cell surface receptor
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/70Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/74Fusion polypeptide containing domain for protein-protein interaction containing a fusion for binding to a cell surface receptor
    • C07K2319/75Fusion 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)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Diabetes (AREA)
  • Urology & Nephrology (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Endocrinology (AREA)
  • Wood Science & Technology (AREA)
  • Plant Pathology (AREA)
  • Neurology (AREA)
  • Vascular Medicine (AREA)
  • Microbiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Physics & Mathematics (AREA)
  • Cardiology (AREA)
  • Neurosurgery (AREA)
  • Cell Biology (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
US10/136,639 US20030072761A1 (en) 2001-10-16 2002-04-30 Methods and compositions for targeting proteins across the blood brain barrier
US10/136,841 US7396811B2 (en) 2001-04-30 2002-04-30 Subcellular targeting of therapeutic proteins
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 (enExample)
JP (2) JP2005506340A (enExample)
AU (3) AU2002347910A1 (enExample)
CA (1) CA2463473A1 (enExample)
IL (1) IL161352A0 (enExample)
WO (2) WO2003032727A1 (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
EP1974752B1 (en) 2001-04-30 2012-09-26 BioMarin Pharmaceutical Inc. Subcellular targeting of therapeutic proteins
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, 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
IL161352A0 (en) * 2001-10-16 2004-09-27 Symbiontics Inc Methods and compositions for targeting underglycosylated proteins across the blood brain barrier
CA2553955C (en) 2004-02-10 2012-08-28 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
ES2511844T3 (es) * 2007-12-21 2014-10-23 F. Hoffmann-La Roche Ag Formulación de anticuerpo
ES2629853T3 (es) 2008-05-07 2017-08-16 Biomarin Pharmaceutical Inc. Péptidos de dirección lisosómica y usos de los mismos
EP2475376B1 (en) 2009-06-17 2016-03-30 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
SI2485761T1 (sl) 2009-10-09 2019-05-31 Armagen, Inc. Postopki in sestavki za povečanje aktivnosti iduronat-2-sulfataze v CŽS
NZ702801A (en) * 2010-06-25 2016-08-26 Shire Human Genetic Therapies Treatment of sanfilippo syndrome type b
EP2588130B1 (en) 2010-06-25 2016-08-17 Shire Human Genetic Therapies, Inc. Cns delivery of therapeutic agents
AU2011270670B2 (en) 2010-06-25 2017-01-12 Takeda Pharmaceutical Company Limited Methods and compositions for CNS delivery of arylsulfatase A
UA115649C2 (uk) 2010-06-25 2017-12-11 Шае Хюмен Дженетік Терапіс, Інк. Способи та композиції для доставки до цнс ідуронат-2-сульфатази
ES2896060T3 (es) 2010-06-25 2022-02-23 Shire Human Genetic Therapies Métodos y composiciones para la administración al SNC de arilsulfatasa A
CA2805413A1 (en) 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc. Methods and compositions for cns delivery of heparan n-sulfatase
WO2013078564A2 (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
LT3115372T (lt) 2012-11-27 2019-06-25 Biomarin Pharmaceutical Inc. Tiksliniai terapiniai lizosominio fermento sulieti baltymai ir jų panaudojimas
KR20150132332A (ko) 2013-03-15 2015-11-25 글락소스미스클라인 인털렉츄얼 프로퍼티 (넘버 2) 리미티드 저농도 항체 제형
HRP20181507T1 (hr) 2014-04-01 2018-11-16 Swedish Orphan Biovitrum Ab (Publ) Modificirana sulfamidaza i njena proizvodnja
US10980892B2 (en) 2015-06-15 2021-04-20 Angiochem Inc. Methods for the treatment of leptomeningeal carcinomatosis
IL261246B2 (en) 2016-02-24 2023-03-01 Biomarin Pharm Inc Therapeutic fusion proteins targeting lysosomal enzymes, their formulations and their uses
JP7749322B2 (ja) 2018-04-30 2025-10-06 アミカス セラピューティックス インコーポレイテッド 遺伝子治療構築物及び使用方法
EA202190999A1 (ru) 2018-10-10 2021-10-15 Амикус Терапьютикс, Инк. Композиции на основе полипептидов, стабилизированных с помощью дисульфидной связи, и способы применения
US20220002688A1 (en) 2018-12-20 2022-01-06 Armagen, Inc. Purification of iduronate-2-sulfatase immunoglobulin fusion protein
US20240148866A1 (en) * 2021-02-11 2024-05-09 Denali Therapeutics Inc. Anti-transferrin receptor fusion proteins and methods of use thereof

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
CA2025907A1 (en) * 1989-09-21 1991-03-22 Franklin D. Collins Method of 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.
WO2003102583A1 (en) * 2002-05-29 2003-12-11 Symbiontics, Inc. Targeted therapeutic proteins
EP1974752B1 (en) * 2001-04-30 2012-09-26 BioMarin Pharmaceutical Inc. Subcellular targeting of therapeutic proteins
IL161352A0 (en) * 2001-10-16 2004-09-27 Symbiontics Inc Methods and compositions for targeting underglycosylated proteins across the blood brain barrier

Also Published As

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

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