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CO2019002248A2 - Variantes de alfa-glucosidasa ácida y usos de las mismas - Google Patents

Variantes de alfa-glucosidasa ácida y usos de las mismas

Info

Publication number
CO2019002248A2
CO2019002248A2 CONC2019/0002248A CO2019002248A CO2019002248A2 CO 2019002248 A2 CO2019002248 A2 CO 2019002248A2 CO 2019002248 A CO2019002248 A CO 2019002248A CO 2019002248 A2 CO2019002248 A2 CO 2019002248A2
Authority
CO
Colombia
Prior art keywords
glucosidase
variants
acid alpha
alpha
acid
Prior art date
Application number
CONC2019/0002248A
Other languages
English (en)
Inventor
Federico Mingozzi
Giuseppe Ronzitti
Pasqualina Colella
Francesco Puzzo
Original Assignee
Genethon
Univ Sorbonne
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 Genethon, Univ Sorbonne filed Critical Genethon
Publication of CO2019002248A2 publication Critical patent/CO2019002248A2/es

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    • 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
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    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
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    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
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    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2405Glucanases
    • C12N9/2408Glucanases acting on alpha -1,4-glucosidic bonds
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    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102Alpha-glucosidase (3.2.1.20)

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  • Health & Medical Sciences (AREA)
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Abstract

La presente invención se refiere a variantes de alfa-glucosidasa ácida y usos de las mismas.
CONC2019/0002248A 2016-09-12 2019-03-12 Variantes de alfa-glucosidasa ácida y usos de las mismas CO2019002248A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP16306149.2A EP3293203A1 (en) 2016-09-12 2016-09-12 Acid-alpha glucosidase variants and uses thereof
PCT/EP2017/072945 WO2018046775A1 (en) 2016-09-12 2017-09-12 Acid-alpha glucosidase variants and uses thereof

Publications (1)

Publication Number Publication Date
CO2019002248A2 true CO2019002248A2 (es) 2019-05-21

Family

ID=56990394

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2019/0002248A CO2019002248A2 (es) 2016-09-12 2019-03-12 Variantes de alfa-glucosidasa ácida y usos de las mismas

Country Status (23)

Country Link
US (3) US20190390224A1 (es)
EP (3) EP3293203A1 (es)
JP (2) JP7601554B2 (es)
KR (2) KR20190056388A (es)
CN (2) CN109843930B (es)
AU (2) AU2017322377B2 (es)
BR (1) BR112019004783A2 (es)
CA (1) CA3036368A1 (es)
CL (1) CL2019000610A1 (es)
CO (1) CO2019002248A2 (es)
DK (1) DK3510049T5 (es)
ES (1) ES2970293T3 (es)
FI (1) FI3510049T3 (es)
IL (2) IL265170B2 (es)
MX (2) MX2019002841A (es)
MY (1) MY205583A (es)
PE (2) PE20241128A1 (es)
PH (1) PH12019500376A1 (es)
PL (1) PL3510049T3 (es)
PT (1) PT3510049T (es)
SG (1) SG11201901430WA (es)
WO (1) WO2018046775A1 (es)
ZA (1) ZA201902140B (es)

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IL319481A (en) 2016-09-12 2025-05-01 Inst Nat Sante Rech Med Variants of acid alpha-glucosidase and their uses
BR112019025888A2 (pt) 2017-06-07 2020-06-30 Regeneron Pharmaceuticals, Inc. polinucleotídeo que codifica uma proteína terapêutica multidomínio, vetor de terapia gênica, proteína terapêutica multidomínio recombinante, método de expressão, métodos para reduzir o acúmulo de glicogênio em um tecido em um paciente em necessidade, para reduzir o acúmulo de glicogênio em um tecido em um paciente em necessidade epara tratar a deficiência enzimática em um paciente em necessidade e/ou tolerar o paciente à enzima para a qual é deficiente, anticorpo anti-cd63 ou fragmento de ligação a antígeno, e, composição farmacêutica
AU2019218892B2 (en) 2018-02-07 2025-08-14 Regeneron Pharmaceuticals, Inc. Methods and compositions for therapeutic protein delivery
WO2019222411A1 (en) * 2018-05-16 2019-11-21 Spark Therapeutics, Inc. Codon-optimized acid αlpha-glucosidase expression cassettes and methods of using same
SG11202011232VA (en) 2018-05-17 2020-12-30 Regeneron Pharma Anti-cd63 antibodies, conjugates, and uses thereof
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
MX2021002198A (es) 2018-08-24 2021-07-15 Spark Therapeutics Inc Secuencias de promotores optimizadas, construcciones de expresion libres de intrones y metodos de uso.
KR20210148273A (ko) 2019-04-08 2021-12-07 제네똥 근육 발현을 위한 하이브리드 프로모터
EP3996696A1 (en) 2019-07-09 2022-05-18 Genethon Treatment of glycogen storage disease (gsd)
WO2021078834A1 (en) 2019-10-22 2021-04-29 Genethon Chimeric acid-alpha glucosidase polypeptides and uses thereof
US20230033268A1 (en) 2019-10-22 2023-02-02 Genethon Chimeric polypeptides and uses thereof
MX2022007399A (es) * 2019-12-20 2022-09-19 Codexis Inc Variantes de alfa-glucosidasa ácida diseñadas.
EP3913060A1 (en) * 2020-05-22 2021-11-24 Genethon Vectors encoding a glucose-6-phosphatase (g6pase-a) for gene therapy
JP2024529540A (ja) 2021-08-04 2024-08-06 ジェネトン 筋肉及びcnsにおける遺伝子発現のためのハイブリッドプロモーター

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IL265170B2 (en) 2023-10-01
AU2017322377A1 (en) 2019-03-28
IL302354A (en) 2023-06-01
IL265170A (en) 2019-05-30
IL302354B2 (en) 2024-09-01
SG11201901430WA (en) 2019-03-28
CN109843930A (zh) 2019-06-04
PH12019500376A1 (en) 2019-05-15
MX2019002841A (es) 2019-08-29
EP4361178A3 (en) 2024-07-17
ZA201902140B (en) 2024-12-18
JP2019533991A (ja) 2019-11-28
IL302354B1 (en) 2024-05-01
PL3510049T3 (pl) 2024-08-19
PE20190802A1 (es) 2019-06-10
RU2019110772A (ru) 2020-10-12
PE20241128A1 (es) 2024-05-24
US20240043867A1 (en) 2024-02-08
JP7601554B2 (ja) 2024-12-17
CN117512014A (zh) 2024-02-06
ES2970293T3 (es) 2024-05-27
FI3510049T3 (fi) 2024-02-02
DK3510049T5 (da) 2024-09-02
IL265170B1 (en) 2023-06-01
RU2019110772A3 (es) 2020-11-25
KR20230049763A (ko) 2023-04-13
KR20190056388A (ko) 2019-05-24
EP3510049B1 (en) 2023-11-08
DK3510049T3 (da) 2024-01-29
EP3510049A1 (en) 2019-07-17
JP2022180543A (ja) 2022-12-06
PT3510049T (pt) 2024-01-30
BR112019004783A2 (pt) 2019-06-04
EP3293203A1 (en) 2018-03-14
EP4361178A2 (en) 2024-05-01
CL2019000610A1 (es) 2019-07-05
AU2017322377B2 (en) 2024-05-16
CA3036368A1 (en) 2018-03-15
CN109843930B (zh) 2023-11-17
MY205583A (en) 2024-10-28
EP3510049B8 (en) 2023-12-20
AU2024205629A1 (en) 2024-08-29
WO2018046775A1 (en) 2018-03-15
MX2024004419A (es) 2024-05-03
US20190390224A1 (en) 2019-12-26
JP7667122B2 (ja) 2025-04-22
US20210040503A1 (en) 2021-02-11

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