BR112022011760A2 - Fragmento de alfa glicosidase ácida recombinante e/ou de alfa glicosidase ácida recombinante biologicamente ativa, alfa glicosidase ácida recombinante, composição, sequência polinucleotídica recombinante, vetor de expressão, vetor de expressão pdh, célula hospedeira, método para produzir uma variante de alfa glicosidase ácida recombinante, variante de alfa glicosidase ácida recombinante, composição farmacêutica para o tratamento da doença de pompe, composição farmacêutica, método para tratar e/ou prevenir os sintomas da doença de pompe em um indivíduo, e, uso das composições - Google Patents

Fragmento de alfa glicosidase ácida recombinante e/ou de alfa glicosidase ácida recombinante biologicamente ativa, alfa glicosidase ácida recombinante, composição, sequência polinucleotídica recombinante, vetor de expressão, vetor de expressão pdh, célula hospedeira, método para produzir uma variante de alfa glicosidase ácida recombinante, variante de alfa glicosidase ácida recombinante, composição farmacêutica para o tratamento da doença de pompe, composição farmacêutica, método para tratar e/ou prevenir os sintomas da doença de pompe em um indivíduo, e, uso das composições

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Publication number
BR112022011760A2
BR112022011760A2 BR112022011760A BR112022011760A BR112022011760A2 BR 112022011760 A2 BR112022011760 A2 BR 112022011760A2 BR 112022011760 A BR112022011760 A BR 112022011760A BR 112022011760 A BR112022011760 A BR 112022011760A BR 112022011760 A2 BR112022011760 A2 BR 112022011760A2
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BR
Brazil
Prior art keywords
recombinant
acid alpha
glicosidase
treatment
expression vector
Prior art date
Application number
BR112022011760A
Other languages
English (en)
Inventor
Casey HALLOWS William
Cathleen Botham Rachel
Zhu Yu
Chng Chinping
DELLAS Nikki
W Huisman Gjalt
Hicham Alaoui Ismaili Moulay
William HOMAN David
P Silverman Adam
Vroom Jonathan
P Lao Jessica
Original Assignee
Codexis 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 Codexis Inc filed Critical Codexis Inc
Publication of BR112022011760A2 publication Critical patent/BR112022011760A2/pt

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    • 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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • C12N9/2411—Amylases
    • C12N9/2428—Glucan 1,4-alpha-glucosidase (3.2.1.3), i.e. glucoamylase
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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)
    • C12Y302/01003—Glucan 1,4-alpha-glucosidase (3.2.1.3), i.e. glucoamylase
    • 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)
    • 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
    • Y02E50/00—Technologies for the production of fuel of non-fossil origin
    • Y02E50/10—Biofuels, e.g. bio-diesel

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

FRAGMENTO DE ALFA GLICOSIDASE ÁCIDA RECOMBINANTE E/OU DE ALFA GLICOSIDASE ÁCIDA RECOMBINANTE BIOLOGICAMENTE ATIVA, ALFA GLICOSIDASE ÁCIDA RECOMBINANTE, COMPOSIÇÃO, SEQUÊNCIA POLINUCLEOTÍDICA RECOMBINANTE, VETOR DE EXPRESSÃO, VETOR DE EXPRESSÃO PDH, CÉLULA HOSPEDEIRA, MÉTODO PARA PRODUZIR UMA VARIANTE DE ALFA GLICOSIDASE ÁCIDA RECOMBINANTE, VARIANTE DE ALFA GLICOSIDASE ÁCIDA RECOMBINANTE, COMPOSIÇÃO FARMACÊUTICA PARA O TRATAMENTO DA DOENÇA DE POMPE, COMPOSIÇÃO FARMACÊUTICA, MÉTODO PARA TRATAR E/OU PREVENIR OS SINTOMAS DA DOENÇA DE POMPE EM UM INDIVÍDUO, E, USO DAS COMPOSIÇÕES. A presente invenção provê polipeptídeos de alfa glicosidase ácida (GAA) manipulados e suas composições. Em algumas modalidades, os polipeptídeos GAA manipulados foram otimizados para prover expressão aumentada, estabilidade em pH neutro e atividade em lisados de células. A invenção também provê métodos para utilização das composições que compreendem os polipeptídeos GAA manipulados para fins terapêuticos e outros.
BR112022011760A 2019-12-20 2020-12-18 Fragmento de alfa glicosidase ácida recombinante e/ou de alfa glicosidase ácida recombinante biologicamente ativa, alfa glicosidase ácida recombinante, composição, sequência polinucleotídica recombinante, vetor de expressão, vetor de expressão pdh, célula hospedeira, método para produzir uma variante de alfa glicosidase ácida recombinante, variante de alfa glicosidase ácida recombinante, composição farmacêutica para o tratamento da doença de pompe, composição farmacêutica, método para tratar e/ou prevenir os sintomas da doença de pompe em um indivíduo, e, uso das composições BR112022011760A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962951625P 2019-12-20 2019-12-20
PCT/US2020/066041 WO2021127457A1 (en) 2019-12-20 2020-12-18 Engineered acid alpha-glucosidase variants

Publications (1)

Publication Number Publication Date
BR112022011760A2 true BR112022011760A2 (pt) 2022-08-30

Family

ID=76438815

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022011760A BR112022011760A2 (pt) 2019-12-20 2020-12-18 Fragmento de alfa glicosidase ácida recombinante e/ou de alfa glicosidase ácida recombinante biologicamente ativa, alfa glicosidase ácida recombinante, composição, sequência polinucleotídica recombinante, vetor de expressão, vetor de expressão pdh, célula hospedeira, método para produzir uma variante de alfa glicosidase ácida recombinante, variante de alfa glicosidase ácida recombinante, composição farmacêutica para o tratamento da doença de pompe, composição farmacêutica, método para tratar e/ou prevenir os sintomas da doença de pompe em um indivíduo, e, uso das composições

Country Status (17)

Country Link
US (1) US11970722B2 (pt)
EP (1) EP4077679A4 (pt)
JP (1) JP2023507575A (pt)
KR (1) KR20220129547A (pt)
CN (1) CN115427574A (pt)
AR (1) AR120858A1 (pt)
AU (1) AU2020408066A1 (pt)
BR (1) BR112022011760A2 (pt)
CA (1) CA3165484A1 (pt)
CL (1) CL2022001655A1 (pt)
CO (1) CO2022010224A2 (pt)
EC (1) ECSP22056585A (pt)
IL (1) IL294082A (pt)
MX (1) MX2022007399A (pt)
PE (1) PE20221266A1 (pt)
TW (1) TW202136506A (pt)
WO (1) WO2021127457A1 (pt)

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Publication number Publication date
WO2021127457A1 (en) 2021-06-24
EP4077679A4 (en) 2024-04-24
US11970722B2 (en) 2024-04-30
CO2022010224A2 (es) 2022-08-09
KR20220129547A (ko) 2022-09-23
CL2022001655A1 (es) 2023-02-24
AR120858A1 (es) 2022-03-23
ECSP22056585A (es) 2022-11-30
AU2020408066A1 (en) 2022-06-30
CN115427574A (zh) 2022-12-02
US20210189365A1 (en) 2021-06-24
JP2023507575A (ja) 2023-02-24
MX2022007399A (es) 2022-09-19
TW202136506A (zh) 2021-10-01
PE20221266A1 (es) 2022-08-16
CA3165484A1 (en) 2021-06-24
EP4077679A1 (en) 2022-10-26
IL294082A (en) 2022-08-01

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