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
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 (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
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 US7396811B2 (en) 2001-04-30 2002-04-30 Subcellular targeting of therapeutic proteins
US10/136,841 2002-04-30
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 (en)
JP (2) JP2005506340A (en)
AU (3) AU2002347910A1 (en)
CA (1) CA2463473A1 (en)
IL (1) IL161352A0 (en)
WO (2) WO2003032913A2 (en)

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 (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
EP1436316B1 (en) 2001-04-30 2008-01-23 ZyStor Therapeutics , Inc. Subcellular targeting of therapeutic proteins
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted 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
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
ATE465250T1 (en) * 2004-02-10 2010-05-15 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
ES2511844T3 (en) * 2007-12-21 2014-10-23 F. Hoffmann-La Roche Ag Antibody formulation
US20110223147A1 (en) 2008-05-07 2011-09-15 Zystor Therapeutics, Inc. Lysosomal targeting peptides and uses thereof
WO2010148253A2 (en) 2009-06-17 2010-12-23 Zystor Therapeutics, Inc. Formulations for lysosomal enzymes
CA2803003C (en) 2010-06-25 2022-11-22 Shire Human Genetic Therapies, Inc. Methods and compositions for cns delivery of arylsulfatase a
NZ605865A (en) 2010-06-25 2015-07-31 Shire Human Genetic Therapies Cns delivery of therapeutic agents
KR20140005842A (en) 2010-06-25 2014-01-15 샤이어 휴먼 지네틱 테라피즈 인크. Methods and compositions for cns delivery of heparan n-sulfatase
UA115649C2 (en) 2010-06-25 2017-12-11 Шае Хюмен Дженетік Терапіс, Інк. Methods and compositions for cns delivery of iduronate-2-sulfatase
ES2895655T3 (en) 2010-06-25 2022-02-22 Shire Human Genetic Therapies Methods and compositions for CNS administration of iduronate-2-sulfatase
NZ605871A (en) 2010-06-25 2015-02-27 Shire Human Genetic Therapies Treatment of sanfilippo syndrome type b
MX2014006594A (en) * 2011-12-01 2015-09-16 Angiochem Inc Targeted lysosomal enzyme compounds.
EP2793922B1 (en) 2011-12-23 2019-10-30 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
EP2885318A4 (en) 2012-08-14 2016-03-30 Angiochem Inc 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
JP6541581B2 (en) 2013-03-15 2019-07-10 グラクソスミスクライン、インテレクチュアル、プロパティー、（ナンバー２）、リミテッドＧｌａｘｏｓｍｉｔｈｋｌｉｎｅ Ｉｎｔｅｌｌｅｃｔｕａｌ Ｐｒｏｐｅｒｔｙ （Ｎｏ．２） Ｌｉｍｉｔｅｄ Low concentration antibody preparation
KR20160138282A (en) 2014-04-01 2016-12-02 스위디쉬 오르펀 바이오비트럼 에이비(피유비엘) Modified sulfamidase and production thereof
US10980892B2 (en) 2015-06-15 2021-04-20 Angiochem Inc. Methods for the treatment of leptomeningeal carcinomatosis
EP3419651B1 (en) 2016-02-24 2023-10-25 BioMarin Pharmaceutical Inc. Improved naglu fusion protein formulation
CA3098674A1 (en) * 2018-04-30 2019-11-07 Amicus Therapeutics, Inc. Gene therapy constructs and methods of use
BR112021006829A2 (en) 2018-10-10 2021-07-20 Amicus Therapeutics, Inc. polypeptide compositions stabilized with disulfide bonds and methods of use
EP3898689A1 (en) 2018-12-20 2021-10-27 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.
WO2003102583A1 (en) * 2002-05-29 2003-12-11 Symbiontics, Inc. Targeted therapeutic proteins
EP1436316B1 (en) * 2001-04-30 2008-01-23 ZyStor Therapeutics , Inc. 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

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
AU2002362930A2 (en) 2003-04-28
WO2003032727A1 (en) 2003-04-24
EP1446007A1 (en) 2004-08-18
AU2010214643A1 (en) 2010-09-16
WO2003032913A9 (en) 2004-04-29
IL161352A0 (en) 2004-09-27
JP2009203241A (en) 2009-09-10
WO2003032913A3 (en) 2004-08-12
CA2463473A1 (en) 2003-04-24
JP2005506340A (en) 2005-03-03
WO2003032913A2 (en) 2003-04-24

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Legal Events

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