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)
  • Wood Science & Technology (AREA)
  • Zoology (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)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (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
BR112012033214B1 (pt) 2010-06-25 2020-11-03 Shire Human Genetic Therapies, Inc. uso de uma formulação estável compreendendo uma proteína de iduronato-2-sulfatase (i2s), formulação compreendendo a mesma e recipiente
US20220133863A1 (en) * 2010-06-25 2022-05-05 Shire Human Genetic Therapies, Inc. Treatment of sanfilippo syndrome type b
MY207684A (en) * 2015-12-08 2025-03-12 Regeneron Pharma Compositions and methods for internalizing enzymes
KR102617502B1 (ko) 2016-09-12 2023-12-26 제네똥 산-알파 글루코시다제 변이체 및 이의 용도
EP3293259A1 (fr) * 2016-09-12 2018-03-14 Genethon Variants d'alpha-glucosidase acide et leurs utilisations
EP3293203A1 (fr) * 2016-09-12 2018-03-14 Genethon Variants d'alpha-glucosidase acide et leurs utilisations
AU2018281280B2 (en) 2017-06-07 2024-12-19 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing 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
CN112040985A (zh) 2018-02-07 2020-12-04 瑞泽恩制药公司 用于递送治疗性蛋白质的方法和组合物
JP7328990B2 (ja) 2018-04-30 2023-08-17 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Ｈｅｒ２及び／またはａｐｌｐ２に結合する抗体及び二重特異性抗原結合分子、ならびにそれらのコンジュゲート及び使用
EP4714496A3 (fr) * 2018-05-01 2026-04-29 OrfoNeuro ApS Traitement de la céroïde-lipofuscinose neuronale
EP3793591A1 (fr) 2018-05-17 2021-03-24 Regeneron Pharmaceuticals, Inc. Anticorps anti-cd63, conjugués et leurs utilisations
US20260216373A1 (en) * 2023-01-13 2026-07-30 Amicus Therapeutics, Inc. Gene Therapy Constructs for the Treatment of Pompe Disease

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003102583A1 (fr) 2002-05-29 2003-12-11 Symbiontics, Inc. Proteines therapeutiques ciblees
US7629309B2 (en) * 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
DE602005020745D1 (de) 2004-02-10 2010-06-02 Zystor Therapeutics Inc Saure alpha-glukosidase und fragmente davon
EP2348106B1 (fr) * 2006-05-08 2014-07-02 Adaerata, Limited Partnership Protéines chimères, cellules les comprenant et analyses les utilisant
WO2009137721A2 (fr) * 2008-05-07 2009-11-12 Zystor Therapeutics, Inc. Peptides de ciblage lysosomial et leurs utilisations
US8785168B2 (en) 2009-06-17 2014-07-22 Biomarin Pharmaceutical Inc. Formulations for lysosomal enzymes
CN103269709A (zh) * 2010-06-25 2013-08-28 夏尔人类遗传性治疗公司 Sanfilippo综合症类型B的治疗
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
ES2679374T3 (es) * 2012-11-27 2018-08-24 Biomarin Pharmaceutical Inc. Proteínas de fusión terapéuticas dirigidas de enzima lisosómica y usos de las mismas

Also Published As

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

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