MA40791A - Ciblage lysosomal d'enzymes et utilisations associées - Google Patents

Ciblage lysosomal d'enzymes et utilisations associées

Info

Publication number
MA40791A
MA40791A MA040791A MA40791A MA40791A MA 40791 A MA40791 A MA 40791A MA 040791 A MA040791 A MA 040791A MA 40791 A MA40791 A MA 40791A MA 40791 A MA40791 A MA 40791A
Authority
MA
Morocco
Prior art keywords
enzymes
lysosomal targeting
lysosomal
related uses
compositions
Prior art date
Application number
MA040791A
Other languages
English (en)
Inventor
Michael F Concino
Bohong Zhang
Original Assignee
Shire Human Genetic Therapies
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
Application filed by Shire Human Genetic Therapies filed Critical Shire Human Genetic Therapies
Publication of MA40791A publication Critical patent/MA40791A/fr

Links

Classifications

    • 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
    • A61K47/65—Peptidic linkers, binders or spacers, e.g. peptidic enzyme-labile linkers
    • 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
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/6454—Dibasic site splicing serine proteases, e.g. kexin (3.4.21.61); furin (3.4.21.75) and other proprotein convertases
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/96—Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0105—Alpha-N-acetylglucosaminidase (3.2.1.50)
    • 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
    • C07K2319/50—Fusion polypeptide containing protease site

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)

Abstract

L'invention fournit des compositions et des procédés pour un ciblage lysosomal efficace médié par pcsk9. En particulier, les compositions et les procédés fournis par l'invention peuvent être utilisés pour traiter des maladies de stockage lysosomales telles que pompe disease et sanfilippo syndrome type b, et ils peuvent être utilisés pour cibler des enzymes lysosomales sur les différents muscles du corps humain.
MA040791A 2014-10-24 2015-10-23 Ciblage lysosomal d'enzymes et utilisations associées MA40791A (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201462068033P 2014-10-24 2014-10-24

Publications (1)

Publication Number Publication Date
MA40791A true MA40791A (fr) 2017-09-27

Family

ID=54478975

Family Applications (1)

Application Number Title Priority Date Filing Date
MA040791A MA40791A (fr) 2014-10-24 2015-10-23 Ciblage lysosomal d'enzymes et utilisations associées

Country Status (4)

Country Link
US (1) US10556015B2 (fr)
EP (1) EP3220958A1 (fr)
MA (1) MA40791A (fr)
WO (1) WO2016065319A1 (fr)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104857504A (zh) 2010-06-25 2015-08-26 夏尔人类遗传性治疗公司 芳基硫酸酯酶a cns递送的方法和组合物
US20220133863A1 (en) * 2010-06-25 2022-05-05 Shire Human Genetic Therapies, Inc. Treatment of sanfilippo syndrome type b
IL287019B2 (en) * 2015-12-08 2025-10-01 Regeneron Pharma Preparations and methods for internalizing enzymes
EP3293259A1 (fr) * 2016-09-12 2018-03-14 Genethon Variants d'alpha-glucosidase acide et leurs utilisations
IL299325B2 (en) 2016-09-12 2025-08-01 Inst Nat Sante Rech Med Acid-alpha glucosidase variants and uses thereof
EP3293203A1 (fr) * 2016-09-12 2018-03-14 Genethon Variants d'alpha-glucosidase acide et leurs utilisations
EP3635009B1 (fr) 2017-06-07 2026-02-25 Regeneron Pharmaceuticals, Inc. Compositions et méthodes pour l'internalisation d'enzymes
WO2019051297A1 (fr) * 2017-09-08 2019-03-14 The Nemours Foundation Agent, dispositif et système de circulation sanguine pour traiter des maladies lysosomales, et méthodes de traitement de maladies lysosomales
IL276464B2 (en) 2018-02-07 2026-01-01 Regeneron Pharma Methods and compositions for administering therapeutic protein
IL278244B2 (en) 2018-04-30 2026-01-01 Regeneron Pharma Antibodies and bispecific antigen-binding molecules that bind HER2 and/or APLP2, conjugates and uses thereof
EP4714496A2 (fr) * 2018-05-01 2026-03-25 OrfoNeuro ApS Traitement de la céroïde-lipofuscinose neuronale
MA52626A (fr) 2018-05-17 2021-03-24 Regeneron Pharma Anticorps anti-cd63, conjugués et leurs utilisations
WO2024151982A1 (fr) * 2023-01-13 2024-07-18 Amicus Therapeutics, Inc. Constructions de thérapie génique pour le traitement de la maladie de pompe

Family Cites Families (9)

* 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
AU2003237314B2 (en) * 2002-05-29 2010-03-04 Biomarin Pharmaceutical Inc. Targeted therapeutic proteins
JP4914224B2 (ja) 2004-02-10 2012-04-11 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 酸性αグルコシダーゼおよびそのフラグメント
CA2900402C (fr) * 2006-05-08 2018-01-16 Adaerata, Limited Partnership Polypeptide pcsk9, ligand associe audit polypeptide et necessaires et procedes utilisant ledit ligand
US20110223147A1 (en) 2008-05-07 2011-09-15 Zystor Therapeutics, Inc. Lysosomal targeting peptides and uses thereof
ES2758827T3 (es) 2009-06-17 2020-05-06 Biomarin Pharm Inc Formulaciones para enzimas lisosómicas
NZ702801A (en) * 2010-06-25 2016-08-26 Shire Human Genetic Therapies Treatment of sanfilippo syndrome type b
EP2471929A1 (fr) * 2010-12-29 2012-07-04 Algenics Production de protéines glycosylées à forte teneur en mannose stockées dans les plastides de micro-algues
DK3115372T3 (da) * 2012-11-27 2019-06-11 Biomarin Pharm Inc Målrettede terapeutiske lysosomale enzymfusionsproteiner og anvendelser deraf

Also Published As

Publication number Publication date
US20170333569A1 (en) 2017-11-23
EP3220958A1 (fr) 2017-09-27
US10556015B2 (en) 2020-02-11
WO2016065319A1 (fr) 2016-04-28

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