AU2002347910A1 - Methods and compositions for targeting proteins across the blood brain barrier - Google Patents

Methods and compositions for targeting proteins across the blood brain barrier

Info

Publication number
AU2002347910A1
AU2002347910A1 AU2002347910A AU2002347910A AU2002347910A1 AU 2002347910 A1 AU2002347910 A1 AU 2002347910A1 AU 2002347910 A AU2002347910 A AU 2002347910A AU 2002347910 A AU2002347910 A AU 2002347910A AU 2002347910 A1 AU2002347910 A1 AU 2002347910A1
Authority
AU
Australia
Prior art keywords
compositions
methods
brain barrier
blood brain
targeting proteins
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
AU2002347910A
Other languages
English (en)
Inventor
Jonathan H. Lebowitz
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.)
Symbiontics Inc
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 AU2002347910A1 publication Critical patent/AU2002347910A1/en
Abandoned 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)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Urology & Nephrology (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Diabetes (AREA)
  • Endocrinology (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Vascular Medicine (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Toxicology (AREA)
  • Neurosurgery (AREA)
  • Plant Pathology (AREA)
  • Neurology (AREA)
  • Cardiology (AREA)
AU2002347910A 2001-10-16 2002-10-16 Methods and compositions for targeting proteins across the blood brain barrier Abandoned AU2002347910A1 (en)

Applications Claiming Priority (13)

Application Number Priority Date Filing Date Title
US32965001P 2001-10-16 2001-10-16
US60/329,650 2001-10-16
US10/136,639 US20030072761A1 (en) 2001-10-16 2002-04-30 Methods and compositions for targeting proteins across the blood brain barrier
US10/136,639 2002-04-30
US10/136,841 2002-04-30
US10/136,841 US7396811B2 (en) 2001-04-30 2002-04-30 Subcellular targeting of therapeutic proteins
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/US2002/032996 WO2003032913A2 (en) 2001-10-16 2002-10-16 Methods and compositions for targeting proteins across the blood brain barrier

Publications (1)

Publication Number Publication Date
AU2002347910A1 true AU2002347910A1 (en) 2003-04-28

Family

ID=27558195

Family Applications (3)

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
AU2002362930A Abandoned AU2002362930A2 (en) 2001-10-16 2002-10-16 Methods and compositions for targeting underglycosylated 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
AU2002362930A Abandoned AU2002362930A2 (en) 2001-10-16 2002-10-16 Methods and compositions for targeting underglycosylated 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 (https=)
JP (2) JP2005506340A (https=)
AU (3) AU2002347910A1 (https=)
CA (1) CA2463473A1 (https=)
IL (1) IL161352A0 (https=)
WO (2) WO2003032913A2 (https=)

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 (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
WO2002087510A2 (en) 2001-04-30 2002-11-07 Symbiontics, Inc. Subcellular targeting of therapeutic proteins
AU2002347910A1 (en) * 2001-10-16 2003-04-28 Symbiontics Inc. Methods and compositions for targeting 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
CA2553955C (en) * 2004-02-10 2012-08-28 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
AU2008282496B2 (en) 2007-07-27 2013-04-04 Armagen Technologies, Inc. Methods and compositions for increasing alpha-iduronidase activity in the CNS
BRPI0820819B1 (pt) * 2007-12-21 2021-06-08 F. Hoffmann-La Roche Ag formulação de anticorpos humanizados b-ly 1 e seu uso
JP5627571B2 (ja) 2008-05-07 2014-11-19 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 リソソーム標的化ペプチドおよびその使用
EP3679942A1 (en) 2009-06-17 2020-07-15 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
EP2485761B1 (en) 2009-10-09 2019-02-27 Armagen, Inc. Methods and compositions for increasing iduronate 2-sulfatase activity in the cns
JP6073783B2 (ja) 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 ヘパランｎ−スルファターゼのｃｎｓ送達のための方法および組成物
AU2011270672B2 (en) * 2010-06-25 2017-01-12 Shire Human Genetic Therapies, Inc. Treatment of Sanfilippo Syndrome type B
LT2588130T (lt) 2010-06-25 2016-12-12 Shire Human Genetic Therapies, Inc. Terapinės priemonės pristatymas cns
PT3103469T (pt) 2010-06-25 2021-03-04 Shire Human Genetic Therapies Administração de agentes terapêuticos no snc
KR20230159646A (ko) 2010-06-25 2023-11-21 샤이어 휴먼 지네틱 테라피즈 인크. 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들
HRP20191923T1 (hr) 2010-06-25 2020-01-10 Shire Human Genetic Therapies, Inc. Postupci i sastavi za isporuku arilsulfataze a u cns
CN104145015A (zh) * 2011-12-01 2014-11-12 安吉奥开米公司 靶向的溶酶体酶化合物
US20140377244A1 (en) 2011-12-23 2014-12-25 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
JP2015526434A (ja) 2012-08-14 2015-09-10 アンジオケム インコーポレーテッド ペプチド−デンドリマーコンジュゲート及びその使用
ES2729997T3 (es) 2012-11-27 2019-11-07 Biomarin Pharm Inc Proteínas de fusión terapéuticas dirigidas de enzima lisosómica y usos de las mismas
AU2014229282B2 (en) * 2013-03-15 2017-02-02 Glaxosmithkline Intellectual Property (No.2) Limited Low concentration antibody formulations
SI2970413T1 (sl) 2014-04-01 2018-10-30 Swedish Orphan Biovitrum Ab (Publ) Modificirana sulfamidaza in proizvodja le-te
EP3307326B9 (en) 2015-06-15 2021-02-24 Angiochem Inc. Methods for the treatment of leptomeningeal carcinomatosis
MX2018010196A (es) 2016-02-24 2018-11-19 Biomarin Pharm Inc Proteinas de fusion de enzimas lisosomales terapeuticas dirigidas, formulaciones asociadas y usos de las mismas.
BR112020021962A2 (pt) * 2018-04-30 2021-01-26 Amicus Therapeutics, Inc. construtos para terapia gênica e métodos de uso
CN113631182B (zh) 2018-10-10 2025-03-21 阿米库斯治疗学公司 二硫键稳定的多肽组合物和使用方法
US20220002688A1 (en) 2018-12-20 2022-01-06 Armagen, Inc. Purification of iduronate-2-sulfatase immunoglobulin fusion protein
EP4291246A4 (en) * 2021-02-11 2024-12-11 Denali Therapeutics Inc. ANTI-TRANSFERRIN RECEPTOR FUSION PROTEINS AND METHODS OF USE THEREOF

Family Cites Families (7)

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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
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.
WO2002087510A2 (en) * 2001-04-30 2002-11-07 Symbiontics, Inc. Subcellular targeting of therapeutic proteins
AU2002347910A1 (en) * 2001-10-16 2003-04-28 Symbiontics Inc. Methods and compositions for targeting proteins across the blood brain barrier
EP1514106A4 (en) * 2002-05-29 2007-05-09 Zystor Therapeutics 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
EP1446007A4 (en) 2005-12-07
WO2003032913A2 (en) 2003-04-24
AU2002362930A2 (en) 2003-04-28
EP1446007A1 (en) 2004-08-18
WO2003032727A1 (en) 2003-04-24
WO2003032913A3 (en) 2004-08-12
JP2009203241A (ja) 2009-09-10
IL161352A0 (en) 2004-09-27
WO2003032913A9 (en) 2004-04-29
JP2005506340A (ja) 2005-03-03
CA2463473A1 (en) 2003-04-24
AU2010214643A1 (en) 2010-09-16

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MK6 Application lapsed section 142(2)(f)/reg. 8.3(3) - pct applic. not entering national phase