PE20190802A1 - Variantes de alfa-glucosidasa acida y usos de las mismas - Google Patents
Variantes de alfa-glucosidasa acida y usos de las mismasInfo
- Publication number
- PE20190802A1 PE20190802A1 PE2019000499A PE2019000499A PE20190802A1 PE 20190802 A1 PE20190802 A1 PE 20190802A1 PE 2019000499 A PE2019000499 A PE 2019000499A PE 2019000499 A PE2019000499 A PE 2019000499A PE 20190802 A1 PE20190802 A1 PE 20190802A1
- Authority
- PE
- Peru
- Prior art keywords
- gaa
- nucleic acid
- glucosidase
- refers
- protein
- Prior art date
Links
- 102100024295 Maltase-glucoamylase Human genes 0.000 title abstract 2
- 239000002253 acid Substances 0.000 title abstract 2
- 108010028144 alpha-Glucosidases Proteins 0.000 title abstract 2
- 108020004707 nucleic acids Proteins 0.000 abstract 3
- 102000039446 nucleic acids Human genes 0.000 abstract 3
- 150000007523 nucleic acids Chemical class 0.000 abstract 3
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 abstract 2
- 108010076504 Protein Sorting Signals Proteins 0.000 abstract 2
- 102000004169 proteins and genes Human genes 0.000 abstract 2
- 108090000623 proteins and genes Proteins 0.000 abstract 2
- 229920002527 Glycogen Polymers 0.000 abstract 1
- 206010018464 Glycogen storage disease type I Diseases 0.000 abstract 1
- 125000003275 alpha amino acid group Chemical group 0.000 abstract 1
- 229940096919 glycogen Drugs 0.000 abstract 1
- 208000007345 glycogen storage disease Diseases 0.000 abstract 1
- 201000004541 glycogen storage disease I Diseases 0.000 abstract 1
- 230000007331 pathological accumulation Effects 0.000 abstract 1
Classifications
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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
- 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
- C12N15/86—Viral vectors
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/34—Muscles; Smooth muscle cells; Heart; Cardiac stem cells; Myoblasts; Myocytes; Cardiomyocytes
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/37—Digestive system
- A61K35/407—Liver; Hepatocytes
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
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- 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
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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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
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
- C12N5/0658—Skeletal muscle cells, e.g. myocytes, myotubes, myoblasts
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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
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/067—Hepatocytes
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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)
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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)
- C12N9/2405—Glucanases
- C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/06—Fusion polypeptide containing a localisation/targetting motif containing a lysosomal/endosomal localisation signal
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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
- C12N2510/00—Genetically modified cells
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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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
- C12N2740/15041—Use of virus, viral particle or viral elements as a vector
- C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
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- 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)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Cell Biology (AREA)
- Molecular Biology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Developmental Biology & Embryology (AREA)
- Immunology (AREA)
- Epidemiology (AREA)
- Diabetes (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Nutrition Science (AREA)
- Cardiology (AREA)
- Vascular Medicine (AREA)
- Physiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
Abstract
Referido a una molecula de acido nucleico que codifica una proteina de alfa-glucosidasa acida (GAA) quimerica, que comprende un resto de peptido senal y un resto de GAA funcional; donde el resto de GAA corresponde a una forma truncada de una GAA parenteral; y el resto de peptido senal tiene una secuencia de aminoacidos seleccionada entre el grupo que consiste en SEQ ID NO: 2 a 4, preferentemente SEQ ID NO:2. Dicha molecula expresa y secreta niveles mas altos en comparacion con la proteina de GAA de tipo silvestre y producen una correccion mejorada de la acumulacion patologica de glucogeno en todo el cuerpo. Tambien se refiere a una celula transformada con la molecula de acido nucleico, una construccion y un vector que comprenden el citado acido nucleico; siendo util para tratar una enfermedad de almacenamiento de glucogeno, tal como enfermedad de von Gierke (FSDI), entre otros
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 |
|---|---|
| PE20190802A1 true PE20190802A1 (es) | 2019-06-10 |
Family
ID=56990394
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PE2024000616A PE20241128A1 (es) | 2016-09-12 | 2017-09-12 | Variantes de alfa-glucosidasa acida y usos de las mismas |
| PE2019000499A PE20190802A1 (es) | 2016-09-12 | 2017-09-12 | Variantes de alfa-glucosidasa acida y usos de las mismas |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PE2024000616A PE20241128A1 (es) | 2016-09-12 | 2017-09-12 | Variantes de alfa-glucosidasa acida 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) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| EP3293259A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| 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 |
Family Cites Families (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6858425B1 (en) | 1998-12-04 | 2005-02-22 | Genzyme Corporation | Human acid alpha glucosidase gene and bovine alpha-S1 casein gene sequences |
| JP4990434B2 (ja) | 1998-12-07 | 2012-08-01 | ジェンザイム・コーポレーション | ポンペ病の処置 |
| US7629309B2 (en) * | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| JP5433133B2 (ja) | 2003-01-22 | 2014-03-05 | デューク・ユニヴァーシティ | リソソームポリペプチドを発現するための改善された構築物 |
| CN1922313B (zh) * | 2004-02-10 | 2011-10-26 | 生物马林医药公司 | 酸性α-糖苷酶及其片段 |
| MX360727B (es) | 2004-06-01 | 2018-11-14 | Genzyme Corp | Composiciones y metodos para evitar la agregacion del vector aav. |
| EP1769070A4 (en) * | 2004-06-29 | 2007-12-12 | Novozymes Inc | POLYPEPTIDES WITH ALPHA-GLUCOSIDASE ACTIVITY AND POLYNUCLEOTIDES THAT CODE |
| PT2318037E (pt) | 2008-07-08 | 2015-05-20 | Univ Duke | Método de tratamento da doença de armazenamento de glicogénio |
| WO2012085622A1 (en) * | 2010-12-22 | 2012-06-28 | Fondazione Telethon | Therapeutic strategies to treat cns pathology in mucopolysaccharidoses |
| WO2012145644A1 (en) | 2011-04-22 | 2012-10-26 | Genzyme Corporation | Modified acid alpha glucosidase with accelerated processing |
| WO2013013017A2 (en) * | 2011-07-21 | 2013-01-24 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for modifying the glycosylation of lysosomal storage disorder therapeutics |
| US9540657B2 (en) * | 2012-05-25 | 2017-01-10 | California Institute Of Technology | Expression of secreted and cell-surface polypeptides |
| MX2016001044A (es) | 2013-07-22 | 2016-09-09 | The Children's Hospital Of Philadelphia | Aav variante y composiciones, metodos y usos para transferencia genica a celulas, organos y tejidos. |
| CA3205555A1 (en) | 2014-04-25 | 2015-10-29 | Genethon | Treatment of hyperbilirubinemia |
| US10202591B2 (en) * | 2014-06-13 | 2019-02-12 | Valerion Therapeutics, Llc | Methods and compositions for treatment of glycogen storage diseases and glycogen metabolism disorders |
| WO2015196179A1 (en) | 2014-06-20 | 2015-12-23 | University Of Florida Research Foundation, Inc. | Methods of packaging multiple adeno-associated virus vectors |
| MA40791A (fr) * | 2014-10-24 | 2017-09-27 | Shire Human Genetic Therapies | Ciblage lysosomal d'enzymes et utilisations associées |
-
2016
- 2016-09-12 EP EP16306149.2A patent/EP3293203A1/en not_active Withdrawn
-
2017
- 2017-09-12 US US16/332,376 patent/US20190390224A1/en not_active Abandoned
- 2017-09-12 SG SG11201901430WA patent/SG11201901430WA/en unknown
- 2017-09-12 JP JP2019513381A patent/JP7601554B2/ja active Active
- 2017-09-12 AU AU2017322377A patent/AU2017322377B2/en active Active
- 2017-09-12 CA CA3036368A patent/CA3036368A1/en active Pending
- 2017-09-12 PE PE2024000616A patent/PE20241128A1/es unknown
- 2017-09-12 PL PL17768082.4T patent/PL3510049T3/pl unknown
- 2017-09-12 PT PT177680824T patent/PT3510049T/pt unknown
- 2017-09-12 MY MYPI2019000888A patent/MY205583A/en unknown
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2022
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