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WO2013013017A3 - Compositions et procédés pour modifier la glycosylation de substances thérapeutiques pour les troubles du stockage lysosomal - Google Patents

Compositions et procédés pour modifier la glycosylation de substances thérapeutiques pour les troubles du stockage lysosomal Download PDF

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Publication number
WO2013013017A3
WO2013013017A3 PCT/US2012/047354 US2012047354W WO2013013017A3 WO 2013013017 A3 WO2013013017 A3 WO 2013013017A3 US 2012047354 W US2012047354 W US 2012047354W WO 2013013017 A3 WO2013013017 A3 WO 2013013017A3
Authority
WO
WIPO (PCT)
Prior art keywords
methods
modifying
compositions
glycosylation
lysosomal storage
Prior art date
Application number
PCT/US2012/047354
Other languages
English (en)
Other versions
WO2013013017A2 (fr
Inventor
Anthony Rossomando
Brian Bettencourt
Original Assignee
Alnylam Pharmaceuticals, 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
Application filed by Alnylam Pharmaceuticals, Inc. filed Critical Alnylam Pharmaceuticals, Inc.
Publication of WO2013013017A2 publication Critical patent/WO2013013017A2/fr
Publication of WO2013013017A3 publication Critical patent/WO2013013017A3/fr

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/81—Protease inhibitors
    • C07K14/8107—Endopeptidase (E.C. 3.4.21-99) inhibitors
    • C07K14/811—Serine protease (E.C. 3.4.21) inhibitors
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/81—Protease inhibitors
    • C07K14/8107—Endopeptidase (E.C. 3.4.21-99) inhibitors
    • C07K14/811—Serine protease (E.C. 3.4.21) inhibitors
    • C07K14/8114—Kunitz type inhibitors
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/81—Protease inhibitors
    • C07K14/8107—Endopeptidase (E.C. 3.4.21-99) inhibitors
    • C07K14/811—Serine protease (E.C. 3.4.21) inhibitors
    • C07K14/8121—Serpins
    • 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
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • 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
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface 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)
    • C12N9/24—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)
    • C12N9/2405—Glucanases
    • C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
    • 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)
    • C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P21/00—Preparation of peptides or proteins
    • C12P21/005—Glycopeptides, glycoproteins
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]

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  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Virology (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)

Abstract

L'invention concerne de manière générale des compositions et des procédés de production de protéines lysosomales qui présentent une structure glycane modifiée, de telle sorte que la protéine peut être délivrée efficacement dans les lysosomes de cellules cibles (par exemple par endocytose médiée par le mannose-6-phosphate). Les protéines lysosomales sont produites en modifiant les voies de glycosylation et/ou le ciblage lysosomal dans une cellule hôte en utilisant une molécule effectrice d'ARN, comme un ARNsi. Les protéines lysosomales à glycane modifié produites au moyen des procédés décrits ici présentent des propriétés améliorées.
PCT/US2012/047354 2011-07-21 2012-07-19 Compositions et procédés pour modifier la glycosylation de substances thérapeutiques pour les troubles du stockage lysosomal WO2013013017A2 (fr)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201161510437P 2011-07-21 2011-07-21
US61/510,437 2011-07-21
US201161532999P 2011-09-09 2011-09-09
US61/532,999 2011-09-09
US201261617322P 2012-03-29 2012-03-29
US61/617,322 2012-03-29

Publications (2)

Publication Number Publication Date
WO2013013017A2 WO2013013017A2 (fr) 2013-01-24
WO2013013017A3 true WO2013013017A3 (fr) 2013-04-18

Family

ID=47558721

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2012/047354 WO2013013017A2 (fr) 2011-07-21 2012-07-19 Compositions et procédés pour modifier la glycosylation de substances thérapeutiques pour les troubles du stockage lysosomal

Country Status (1)

Country Link
WO (1) WO2013013017A2 (fr)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2639833T3 (es) 2013-03-15 2017-10-30 Shire Viropharma Incorporated Composiciones C1-INH para uso en la prevención y tratamiento del angioedema hereditario (AEH)
US20150104445A1 (en) * 2013-10-10 2015-04-16 Viropharma Holdings Limited Methods of inhibiting the alternative pathway of complement immune system activation and compositions used therein
WO2015095568A1 (fr) * 2013-12-18 2015-06-25 Kelvin Lee Réduction de l'activité d'une lipase dans des formulations de produits
SI3201320T1 (sl) 2014-09-30 2024-02-29 Amicus Therapeutics, Inc. Zelo močna kislinska alfa-glukozidaza z okrepljenimi ogljikovimi hidrati
TWI789758B (zh) * 2014-09-30 2023-01-11 美商阿米庫斯醫療股份有限公司 高效經修飾之酸性α-葡萄糖苷酶及其製造與使用方法
EP3334753B1 (fr) * 2015-08-14 2020-10-07 Oxford University Innovation Limited Agents de liaison de facteur de croissance analogue à l'insuline 2 (igf2)
KR102510941B1 (ko) 2015-12-30 2023-03-20 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
JP7046003B2 (ja) 2016-03-30 2022-04-01 アミカス セラピューティックス インコーポレイテッド 高ｍ６ｐ組換えタンパク質の選択方法
EP3436053B1 (fr) * 2016-03-30 2022-04-06 Amicus Therapeutics, Inc. Formulations comprenant une alpha-glucosidase acide recombinante
US10512676B2 (en) 2016-03-30 2019-12-24 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
EP3293203A1 (fr) * 2016-09-12 2018-03-14 Genethon Variants d'alpha-glucosidase acide et leurs utilisations
CN117384890A (zh) 2016-09-12 2024-01-12 吉尼松公司 酸性α-葡萄糖苷酶变体及其用途
AU2017368050A1 (en) 2016-11-29 2019-06-20 Puretech Lyt, Inc. Exosomes for delivery of therapeutic agents
TWI870336B (zh) 2017-05-15 2025-01-21 美商阿米庫斯醫療股份有限公司 重組人類酸性α-葡萄糖苷酶

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5641670A (en) * 1991-11-05 1997-06-24 Transkaryotic Therapies, Inc. Protein production and protein delivery
US20060188975A1 (en) * 2003-08-21 2006-08-24 Mani Ramaswami Genetically modified somatic cells for sustained secretion of lysosomal proenzymes deficient in lysosomal storage disorders
WO2011005786A2 (fr) * 2009-07-06 2011-01-13 Alnylam Pharmaceuticals, Inc. Compositions et procédés pour améliorer la production d'un produit biologique

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5641670A (en) * 1991-11-05 1997-06-24 Transkaryotic Therapies, Inc. Protein production and protein delivery
US20060188975A1 (en) * 2003-08-21 2006-08-24 Mani Ramaswami Genetically modified somatic cells for sustained secretion of lysosomal proenzymes deficient in lysosomal storage disorders
WO2011005786A2 (fr) * 2009-07-06 2011-01-13 Alnylam Pharmaceuticals, Inc. Compositions et procédés pour améliorer la production d'un produit biologique

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
FREEZE, H.: "Towards a therapy for phosphomannomutase 2 defeciency, the defect in CDG-la patients.", BIOCHIM BIOPHYS ACTA, vol. 1792, no. 9, September 2009 (2009-09-01), pages 835 - 840 *

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