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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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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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475Growth factors; Growth regulators
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0002Antibodies with enzymatic activity, e.g. abzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2405Glucanases
    • C12N9/2434Glucanases acting on beta-1,4-glucosidic bonds
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/036Fusion polypeptide containing a localisation/targetting motif targeting to the medium outside of the cell, e.g. type III secretion
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022Alpha-galactosidase (3.2.1.22)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01045Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • YGENERAL 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
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02EREDUCTION OF GREENHOUSE GAS [GHG] EMISSIONS, RELATED TO ENERGY GENERATION, TRANSMISSION OR DISTRIBUTION
    • Y02E60/00Enabling technologies; Technologies with a potential or indirect contribution to GHG emissions mitigation
    • Y02E60/10Energy 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)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (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

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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)

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US (1) US9279007B2 (enExample)
EP (2) EP2643468B1 (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) DK2643468T3 (enExample)
ES (2) ES2687415T3 (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
ES2763526T3 (es) 2014-07-03 2020-05-29 Hoffmann La Roche Sistemas de expresión de polipéptidos
EP3443001B1 (en) 2016-04-11 2025-04-30 Obsidian Therapeutics, Inc. REGULATED BIOCIRCUIT SYSTEMS
CN110831961B (zh) 2017-03-03 2024-01-02 黑曜石疗法公司 用于免疫疗法的cd19组合物和方法
ES3065816T3 (en) 2017-06-07 2026-05-08 Regeneron Pharma Compositions and methods for internalizing enzymes
KR20200090889A (ko) 2017-11-30 2020-07-29 아미쿠스 세라퓨틱스, 인코포레이티드 Cdkl5 발현 변이체 및 cdkl5 융합 단백질
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
JP7749322B2 (ja) 2018-04-30 2025-10-06 アミカス セラピューティックス インコーポレイテッド 遺伝子治療構築物及び使用方法
BR112020023145A2 (pt) 2018-05-17 2021-02-02 Regeneron Pharmaceuticals, Inc. anticorpo anti-cd63 ou fragmento de ligação ao antígeno do mesmo, molécula de ligação ao antígeno biespecífica, proteína terapêutica de múltiplos domínios, polinucleotídeo composição farmacêutica, e, composto
US20220403001A1 (en) 2018-06-12 2022-12-22 Obsidian Therapeutics, Inc. Pde5 derived regulatory constructs and methods of use in immunotherapy
EA202190999A1 (ru) 2018-10-10 2021-10-15 Амикус Терапьютикс, Инк. Композиции на основе полипептидов, стабилизированных с помощью дисульфидной связи, и способы применения
WO2020086742A1 (en) 2018-10-24 2020-04-30 Obsidian Therapeutics, Inc. Er tunable protein regulation
SG11202105267VA (en) * 2018-12-19 2021-07-29 Versameb Ag Rna encoding a protein
KR20210149251A (ko) 2019-03-08 2021-12-08 옵시디안 테라퓨틱스, 인크. 조율가능한 조절을 위한 인간 탄산 무수화효소 2 조성물 및 방법
CA3134485A1 (en) * 2019-04-30 2020-11-05 James M. Wilson Compositions useful for treatment of pompe disease
JP2022537670A (ja) 2019-06-12 2022-08-29 オブシディアン セラピューティクス, インコーポレイテッド Ca2の組成物および調整可能な制御方法
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
CA3156506A1 (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
MX2023006445A (es) 2020-12-01 2023-08-10 Univ Pennsylvania Composiciones y usos de estas para el tratamiento del síndrome de angelman.
WO2023086939A1 (en) 2021-11-12 2023-05-19 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
CA2352286C (en) * 1998-11-24 2011-07-12 Bristol-Myers Squibb Company Intracellular targeted delivery of compounds by 70 kd heat shock protein
US7279564B2 (en) * 2003-11-06 2007-10-09 Genencor International, Inc. Expression in filamentous fungi of protease inhibitors and variants thereof
DE602007007923D1 (de) * 2006-04-11 2010-09-02 Csl Behring Gmbh Verfahren zur erhöhung der in-vivo-gewinnung therapeutischer polypeptide

Also Published As

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

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