CA2818689C - Novel signal sequences to improve protein expression and secretion of recombinant enzymes and other proteins - Google Patents

Novel signal sequences to improve protein expression and secretion of recombinant enzymes and other proteins Download PDF

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Publication number
CA2818689C
CA2818689C CA2818689A CA2818689A CA2818689C CA 2818689 C CA2818689 C CA 2818689C CA 2818689 A CA2818689 A CA 2818689A CA 2818689 A CA2818689 A CA 2818689A CA 2818689 C CA2818689 C CA 2818689C
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polypeptide
bip
seq
protein
heterologous polypeptide
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CA2818689A
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French (fr)
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CA2818689A1 (en
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Hung Do
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Amicus Therapeutics Inc
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Amicus Therapeutics Inc
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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
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C07K14/475—Growth factors; Growth regulators
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • 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
    • C12N9/0002—Antibodies with enzymatic activity, e.g. abzymes
    • 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
    • C12N9/2434—Glucanases acting on beta-1,4-glucosidic bonds
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03—Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/036—Fusion polypeptide containing a localisation/targetting motif targeting to the medium outside of the cell, e.g. type III secretion
    • 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)
    • 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)
    • C12Y302/01022—Alpha-galactosidase (3.2.1.22)
    • 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)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02E—REDUCTION OF GREENHOUSE GAS [GHG] EMISSIONS, RELATED TO ENERGY GENERATION, TRANSMISSION OR DISTRIBUTION
    • Y02E60/00—Enabling technologies; Technologies with a potential or indirect contribution to GHG emissions mitigation
    • Y02E60/10—Energy storage using batteries

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA2818689A 2010-11-22 2011-11-22 Novel signal sequences to improve protein expression and secretion of recombinant enzymes and other proteins Active CA2818689C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US41592610P 2010-11-22 2010-11-22
US61/415,926 2010-11-22
PCT/US2011/061862 WO2012071422A2 (en) 2010-11-22 2011-11-22 Novel signal sequences to improve protein expressions and secretion of recombinant enzymes and other proteins

Publications (2)

Publication Number Publication Date
CA2818689A1 CA2818689A1 (en) 2012-05-31
CA2818689C true CA2818689C (en) 2021-09-21

Family

ID=46146394

Family Applications (1)

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CA2818689A Active CA2818689C (en) 2010-11-22 2011-11-22 Novel signal sequences to improve protein expression and secretion of recombinant enzymes and other proteins

Country Status (19)

Country Link
US (1) US9279007B2 (enExample)
EP (2) EP3461905B1 (enExample)
JP (1) JP2014503198A (enExample)
KR (2) KR20190060888A (enExample)
CN (1) CN103328649B (enExample)
BR (1) BR112013012671B1 (enExample)
CA (1) CA2818689C (enExample)
CY (1) CY1121049T1 (enExample)
DK (2) DK3461905T3 (enExample)
ES (2) ES2829199T3 (enExample)
HR (1) HRP20181315T1 (enExample)
HU (1) HUE039070T2 (enExample)
LT (1) LT2643468T (enExample)
PL (1) PL2643468T3 (enExample)
PT (1) PT2643468T (enExample)
RS (1) RS57601B1 (enExample)
SI (1) SI2643468T1 (enExample)
SM (1) SMT201800466T1 (enExample)
WO (1) WO2012071422A2 (enExample)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HRP20192243T1 (hr) * 2014-07-03 2020-03-06 F. Hoffmann - La Roche Ag Polipeptidni ekspresijski sustavi
WO2017180587A2 (en) 2016-04-11 2017-10-19 Obsidian Therapeutics, Inc. Regulated biocircuit systems
AU2018227583B2 (en) 2017-03-03 2023-06-01 Obsidian Therapeutics, Inc. CD19 compositions and methods for immunotherapy
AU2018281280B2 (en) 2017-06-07 2024-12-19 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
CN111936624A (zh) * 2017-11-30 2020-11-13 阿米库斯治疗学公司 Cdkl5表达变体和cdkl5融合蛋白
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
TW202344691A (zh) 2018-04-30 2023-11-16 美商阿米庫斯醫療股份有限公司 基因治療構築體及使用方法
EP3793591A1 (en) 2018-05-17 2021-03-24 Regeneron Pharmaceuticals, Inc. Anti-cd63 antibodies, conjugates, and uses thereof
US20220403001A1 (en) 2018-06-12 2022-12-22 Obsidian Therapeutics, Inc. Pde5 derived regulatory constructs and methods of use in immunotherapy
JP7577650B2 (ja) 2018-10-10 2024-11-05 アミカス セラピューティックス インコーポレイテッド ジスルフィド結合により安定したポリペプチド組成物及びその使用方法
US20210386788A1 (en) 2018-10-24 2021-12-16 Obsidian Therapeutics, Inc. Er tunable protein regulation
JP2022514863A (ja) * 2018-12-19 2022-02-16 ヴェルサメブ アーゲー タンパク質をコードするrna
BR112021017744A2 (pt) 2019-03-08 2021-11-16 Obsidian Therapeutics Inc Composições e métodos de cd40l para regulação ajustável
US20220193261A1 (en) * 2019-04-30 2022-06-23 The Trustees Of The University Of Pennsylvania Compositions useful for treatment of pompe disease
CA3140668A1 (en) 2019-06-12 2020-12-17 Obsidian Therapeutics, Inc. Ca2 compositions and methods for tunable regulation
WO2020252404A1 (en) 2019-06-12 2020-12-17 Obsidian Therapeutics, Inc. Ca2 compositions and methods for tunable regulation
WO2021046451A1 (en) 2019-09-06 2021-03-11 Obsidian Therapeutics, Inc. Compositions and methods for dhfr tunable protein regulation
WO2021087282A1 (en) * 2019-10-30 2021-05-06 Amicus Therapeutics, Inc. Recombinant cdkl5 proteins, gene therapy and production methods
CN112342247B (zh) * 2020-11-10 2022-03-11 江苏科技大学 一种提高家蚕细胞表达外源蛋白分泌率的方法
WO2022119890A1 (en) 2020-12-01 2022-06-09 The Trustees Of The University Of Pennsylvania Compositions and uses thereof for treatment of angelman syndrome
US20250034533A1 (en) 2021-11-12 2025-01-30 Amicus Therapeutics, Inc. Compositions And Methods For Treating Mucopolysaccharidosis IIIA
WO2025264978A2 (en) 2024-06-20 2025-12-26 Amicus Therapeutics, Inc. Recombinant alpha-galactosidase a proteins and gene therapy

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000031113A1 (en) * 1998-11-24 2000-06-02 Bristol-Myers Squibb Company INTRACELLULAR TARGETED DELIVERY OF COMPOUNDS BY 70 kD HEAT SHOCK PROTEIN
EP1689766B1 (en) * 2003-11-06 2012-04-25 Danisco US Inc. Expression in filamentous fungi of protease inhibitors and variants thereof
ATE474917T1 (de) * 2006-04-11 2010-08-15 Csl Behring Gmbh Verfahren zur erhöhung der in-vivo-gewinnung therapeutischer polypeptide

Also Published As

Publication number Publication date
KR20130121878A (ko) 2013-11-06
CN103328649A (zh) 2013-09-25
ES2829199T3 (es) 2021-05-31
DK3461905T3 (da) 2020-11-02
CA2818689A1 (en) 2012-05-31
CN103328649B (zh) 2016-06-22
EP2643468B1 (en) 2018-07-04
EP3461905B1 (en) 2020-08-05
SMT201800466T1 (it) 2018-11-09
KR101984318B1 (ko) 2019-05-31
EP2643468A2 (en) 2013-10-02
LT2643468T (lt) 2018-09-10
US9279007B2 (en) 2016-03-08
RS57601B1 (sr) 2018-11-30
KR20190060888A (ko) 2019-06-03
ES2687415T3 (es) 2018-10-25
JP2014503198A (ja) 2014-02-13
EP3461905A1 (en) 2019-04-03
CY1121049T1 (el) 2019-12-11
WO2012071422A2 (en) 2012-05-31
BR112013012671A2 (pt) 2016-10-11
EP2643468A4 (en) 2014-12-24
US20140045216A1 (en) 2014-02-13
PT2643468T (pt) 2018-10-11
PL2643468T3 (pl) 2018-11-30
HRP20181315T1 (hr) 2018-10-19
SI2643468T1 (sl) 2018-10-30
HUE039070T2 (hu) 2018-12-28
WO2012071422A3 (en) 2012-08-02
BR112013012671B1 (pt) 2022-03-03
DK2643468T3 (en) 2018-09-24

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