CA2487815A1 - Proteines therapeutiques ciblees - Google Patents

Proteines therapeutiques ciblees Download PDF

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Publication number
CA2487815A1
CA2487815A1 CA002487815A CA2487815A CA2487815A1 CA 2487815 A1 CA2487815 A1 CA 2487815A1 CA 002487815 A CA002487815 A CA 002487815A CA 2487815 A CA2487815 A CA 2487815A CA 2487815 A1 CA2487815 A1 CA 2487815A1
Authority
CA
Canada
Prior art keywords
igf
therapeutic
human
receptor
leu
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
CA002487815A
Other languages
English (en)
Inventor
Jonathan H. Lebowitz
Stephen M. Beverley
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.)
Biomarin Pharmaceutical Inc
Original Assignee
Individual
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 US10/272,531 external-priority patent/US20040005309A1/en
Application filed by Individual filed Critical Individual
Publication of CA2487815A1 publication Critical patent/CA2487815A1/fr
Abandoned legal-status Critical Current

Links

Classifications

    • 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
    • A61K47/54—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 an organic compound
    • A61K47/55—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 an organic compound the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug, i.e. a dimer, oligomer or polymer of pharmacologically or therapeutically active compounds
    • A61K47/551—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 an organic compound the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug, i.e. a dimer, oligomer or polymer of pharmacologically or therapeutically active compounds one of the codrug's components being a vitamin, e.g. niacinamide, vitamin B3, cobalamin, vitamin B12, folate, vitamin A or retinoic acid
    • 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
    • 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
    • C07K2319/50—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)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Endocrinology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Diabetes (AREA)
  • Immunology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Toxicology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Genetics & Genomics (AREA)
  • Cell Biology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)

Abstract

L'invention concerne des produits thérapeutiques qui se localisent dans un compartiment infracellulaire spécifique, tel que le lysosome. Les produits thérapeutiques ciblés comprennent un agent thérapeutique et un fragment de ciblage qui se lie à un récepteur sur une surface extérieure de la cellule, ce qui assure une localisation infracellulaire appropriée des produits thérapeutiques après internalisation du récepteur. L'invention concerne en outre des acides nucléiques, des cellules et des méthodes se rapportant à la mise en oeuvre des produits de l'invention.
CA002487815A 2002-05-29 2003-05-29 Proteines therapeutiques ciblees Abandoned CA2487815A1 (fr)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
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
US10/272,531 US20040005309A1 (en) 2002-05-29 2002-10-16 Targeted therapeutic proteins
US10/272,531 2002-10-16
US44573403P 2003-02-06 2003-02-06
US60/445,734 2003-02-06
PCT/US2003/017211 WO2003102583A1 (fr) 2002-05-29 2003-05-29 Proteines therapeutiques ciblees

Publications (1)

Publication Number Publication Date
CA2487815A1 true CA2487815A1 (fr) 2003-12-11

Family

ID=29716364

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002487815A Abandoned CA2487815A1 (fr) 2002-05-29 2003-05-29 Proteines therapeutiques ciblees

Country Status (4)

Country Link
EP (1) EP1514106A4 (fr)
AU (1) AU2003237314B2 (fr)
CA (1) CA2487815A1 (fr)
WO (1) WO2003102583A1 (fr)

Families Citing this family (36)

* 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
ES2300439T3 (es) 2001-04-30 2008-06-16 Zystor Therapeutics , Inc. Reconocimiento subcelular de proteinas terapeuticas.
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
JP2005506340A (ja) * 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 血液脳関門をわたる過小グリコシル化タンパク質の標的化のための方法および組成物
US9221867B2 (en) 2003-01-06 2015-12-29 Angiochem Inc. Method for transporting a compound across the blood-brain barrier
US20050244400A1 (en) * 2004-02-10 2005-11-03 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
EP1818395A1 (fr) 2006-02-08 2007-08-15 Diatos Compositions et méthodes pour le traitement de maladies lysosomales
US9365634B2 (en) 2007-05-29 2016-06-14 Angiochem Inc. Aprotinin-like polypeptides for delivering agents conjugated thereto to tissues
EP2182980A4 (fr) 2007-07-27 2012-04-18 Armagen Technologies Inc Procédés et compositions destinés à augmenter une activité a-iduronidase dans le snc
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
MX2011004017A (es) 2008-10-15 2011-06-24 Angiochem Inc Conjugados de agonistas glp-1 y usos de los mismos.
CA2745524C (fr) 2008-12-05 2020-06-09 Angiochem Inc. Conjugues de neurotensine ou d'analogues de neurotensine et leurs applications
JP2012512185A (ja) 2008-12-17 2012-05-31 アンジオケム インコーポレーテッド 膜１型マトリックス金属タンパク質阻害剤およびその使用
US9173891B2 (en) 2009-04-20 2015-11-03 Angiochem, Inc. Treatment of ovarian cancer using an anticancer agent conjugated to an angiopep-2 analog
ES2569514T3 (es) 2009-06-17 2016-05-11 Biomarin Pharmaceutical Inc. Formulaciones para enzimas lisosómicas
IN2012DN00248A (fr) 2009-07-02 2015-05-01 Angiochem Inc
PL2588130T3 (pl) 2010-06-25 2017-09-29 Shire Human Genetic Therapies, Inc. Dostarczanie środków terapeutycznych do OUN
IL291554B2 (en) 2010-06-25 2024-02-01 Shire Human Genetic Therapies Administering therapeutic agents to the central nervous system
WO2011163652A2 (fr) 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc. Traitement du syndrome de sanfilippo de type b
JP6045492B2 (ja) 2010-06-25 2016-12-14 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 イズロン酸−２−スルファターゼのｃｎｓ送達のための方法および組成物
ME03647B (fr) 2010-06-25 2020-07-20 Shire Human Genetic Therapies Procédés et compositions pour l'administration au snc d'arylsulfatase a
RU2012154576A (ru) 2010-06-25 2014-07-27 Шир Хьюман Дженетик Терапис, Инк. Способы и композиции для доставки к цнс гепаран-n-сульфатазы
AU2012344700A1 (en) * 2011-12-01 2014-06-19 Angiochem Inc. Targeted iduronate-2-sulfatase compounds
WO2013078564A2 (fr) * 2011-12-01 2013-06-06 Angiochem Inc. Composés d'enzyme lysosomale vectorisée
EP2793922B1 (fr) 2011-12-23 2019-10-30 Shire Human Genetic Therapies, Inc. Formulations stables pour l'administration au système nerveux central d'arylsulfatase a
WO2013185235A1 (fr) * 2012-06-15 2013-12-19 Angiochem Inc. Composés d'iduronidase ciblés
CA2892146A1 (fr) 2012-11-27 2014-06-05 Biomarin Pharmaceutical Inc. Proteines hybrides d'enzyme lysosomale therapeutiques ciblees et leurs utilisations
WO2014082184A1 (fr) * 2012-11-30 2014-06-05 Angiochem Inc. Composés d'iduronate-2-sulfatase ciblés
PL2991683T3 (pl) 2013-05-02 2020-03-31 Glykos Finland Oy Koniugaty glikoproteiny lub glikanu z toksycznym ładunkiem
WO2014194428A1 (fr) * 2013-06-06 2014-12-11 Angiochem Inc. Composés d'héparane sulfatase ciblés
ES2785551T3 (es) 2014-06-30 2020-10-07 Glykos Finland Oy Derivado de sacárido de una carga útil tóxica y sus conjugados con anticuerpos
US10556015B2 (en) 2014-10-24 2020-02-11 Criteo S.A. Lysosomal targeting of enzymes, and uses thereof
EP3307326B9 (fr) 2015-06-15 2021-02-24 Angiochem Inc. Procédés de traitement d'une carcinomatose leptoméningée
BR112018017322A2 (pt) 2016-02-24 2019-01-02 Biomarin Pharm Inc proteínas de fusão de enzima lisossômica terapêuticas direcionadas, formulações associadas e usos das mesmas
JP7458404B2 (ja) 2018-12-20 2024-03-29 アーマジェン・インコーポレイテッド イズロン酸－２－スルファターゼ免疫グロブリン融合タンパク質の精製

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2300439T3 (es) * 2001-04-30 2008-06-16 Zystor Therapeutics , Inc. Reconocimiento subcelular de proteinas terapeuticas.
JP2005506340A (ja) * 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 血液脳関門をわたる過小グリコシル化タンパク質の標的化のための方法および組成物

Also Published As

Publication number Publication date
WO2003102583A1 (fr) 2003-12-11
EP1514106A1 (fr) 2005-03-16
EP1514106A4 (fr) 2007-05-09
AU2003237314A1 (en) 2003-12-19
AU2003237314B2 (en) 2010-03-04

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Effective date: 20130529