CL2020002973A1 - Casetes de expresión de a- glucosidasa de ácido optimizado con codón y métodos de uso del mismo - Google Patents
Casetes de expresión de a- glucosidasa de ácido optimizado con codón y métodos de uso del mismoInfo
- Publication number
- CL2020002973A1 CL2020002973A1 CL2020002973A CL2020002973A CL2020002973A1 CL 2020002973 A1 CL2020002973 A1 CL 2020002973A1 CL 2020002973 A CL2020002973 A CL 2020002973A CL 2020002973 A CL2020002973 A CL 2020002973A CL 2020002973 A1 CL2020002973 A1 CL 2020002973A1
- Authority
- CL
- Chile
- Prior art keywords
- methods
- glucosidase
- expression cassettes
- nucleic acids
- same
- Prior art date
Links
- 102100024295 Maltase-glucoamylase Human genes 0.000 title abstract 4
- 108010028144 alpha-Glucosidases Proteins 0.000 title abstract 4
- 239000002253 acid Substances 0.000 title abstract 3
- 108020004705 Codon Proteins 0.000 title 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 4
- 102000039446 nucleic acids Human genes 0.000 abstract 4
- 108020004707 nucleic acids Proteins 0.000 abstract 4
- 239000013598 vector Substances 0.000 abstract 1
Classifications
-
- 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
-
- 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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- 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
- 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
-
- 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)
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
-
- 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
- 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
-
- 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
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
-
- 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
- C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Microbiology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Immunology (AREA)
- Physical Education & Sports Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Neurology (AREA)
- Gastroenterology & Hepatology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862672419P | 2018-05-16 | 2018-05-16 | |
| US201862734454P | 2018-09-21 | 2018-09-21 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2020002973A1 true CL2020002973A1 (es) | 2021-07-19 |
Family
ID=68540790
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2020002973A CL2020002973A1 (es) | 2018-05-16 | 2020-11-16 | Casetes de expresión de a- glucosidasa de ácido optimizado con codón y métodos de uso del mismo |
Country Status (17)
| Country | Link |
|---|---|
| US (2) | US12084693B2 (enExample) |
| EP (1) | EP3794043A4 (enExample) |
| JP (2) | JP2021523718A (enExample) |
| KR (1) | KR20210021310A (enExample) |
| CN (1) | CN112334489A (enExample) |
| AU (1) | AU2019268363A1 (enExample) |
| BR (1) | BR112020023393A2 (enExample) |
| CA (1) | CA3100213A1 (enExample) |
| CL (1) | CL2020002973A1 (enExample) |
| CO (1) | CO2020015707A2 (enExample) |
| MX (1) | MX2020012332A (enExample) |
| MY (1) | MY207355A (enExample) |
| PE (1) | PE20210337A1 (enExample) |
| PH (1) | PH12020551957A1 (enExample) |
| SG (1) | SG11202011191WA (enExample) |
| TW (1) | TW202016310A (enExample) |
| WO (1) | WO2019222411A1 (enExample) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2018281280B2 (en) | 2017-06-07 | 2024-12-19 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for internalizing enzymes |
| 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 |
| EP3793591A1 (en) | 2018-05-17 | 2021-03-24 | Regeneron Pharmaceuticals, Inc. | 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 |
| CN113226352B (zh) | 2018-08-24 | 2025-08-26 | 星火治疗有限公司 | 最优化启动子序列、无内含子的表达结构及使用方法 |
| EP3996696A1 (en) * | 2019-07-09 | 2022-05-18 | Genethon | Treatment of glycogen storage disease (gsd) |
| MX2022007399A (es) | 2019-12-20 | 2022-09-19 | Codexis Inc | Variantes de alfa-glucosidasa ácida diseñadas. |
| PE20240915A1 (es) * | 2021-01-14 | 2024-04-30 | Spark Therapeutics Inc | Composiciones y metodos para tratar la enfermedad de fabry |
| CA3229580A1 (en) * | 2021-08-20 | 2023-02-23 | Anna Nikolaevna STRELKOVA | Method for producing modified adeno-associated virus capsid |
| WO2023028567A2 (en) * | 2021-08-25 | 2023-03-02 | Canbridge Pharmaceuticals, Inc. | Aav particles comprising a liver-tropic capsid protein and acid alpha-glucosidase (gaa) and their use to treat pompe disease |
| EP4392569A4 (en) * | 2021-08-25 | 2025-07-16 | Canbridge Pharmaceuticals Inc | AAV PARTICLES COMPRISING A LIVER-TROPIC CAPSID PROTEIN AND AN ALPHA-GALACTOSIDASE AND THEIR USE FOR TREATING FABRY DISEASE |
| IL314482A (en) * | 2022-02-02 | 2024-09-01 | Regeneron Pharma | Anti-tfr:gaa and anti-cd63:gaa insertions for treatment of pompe disease |
| CN118667797A (zh) * | 2023-03-14 | 2024-09-20 | 北京据德医药科技有限公司 | 一种重组酸性α-葡萄糖苷酶及其用途 |
| WO2024231820A1 (en) * | 2023-05-05 | 2024-11-14 | Takeda Pharmaceutical Company Limited | Treatment of pompe disease |
| IL326018A (en) * | 2023-07-28 | 2026-03-01 | Regeneron Pharma | Introduction of anti-TFR:GAA and anti-CD63:GAA for the treatment of Pompe disease |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2687597B1 (en) | 2011-03-16 | 2018-12-26 | Amano Enzyme Inc. | Modified alpha-glucosidase and applications of same |
| WO2013151664A1 (en) | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins |
| CN104411338A (zh) * | 2012-04-02 | 2015-03-11 | 现代治疗公司 | 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸 |
| JP6342886B2 (ja) * | 2012-04-18 | 2018-06-13 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ | Ａａｖカプシドバリアントを使用した高効率な遺伝子導入のための組成物及び方法 |
| US10077436B2 (en) * | 2013-09-17 | 2018-09-18 | Edeniq, Inc. | Beta-glucosidase enzymes for increased biomass saccharification |
| JP6649265B2 (ja) * | 2013-11-26 | 2020-02-19 | ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ | 糖原病の処置のためのアデノ随伴ウイルスベクター |
| US10781459B2 (en) * | 2014-06-20 | 2020-09-22 | University Of Florida Research Foundation, Incorporated | Methods of packaging multiple adeno-associated virus vectors |
| WO2016063331A1 (ja) | 2014-10-20 | 2016-04-28 | 昭和産業株式会社 | 新規α－グルコシダーゼ |
| EP3293259A1 (en) * | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| EP3293203A1 (en) * | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| KR102617502B1 (ko) * | 2016-09-12 | 2023-12-26 | 제네똥 | 산-알파 글루코시다제 변이체 및 이의 용도 |
| WO2020102645A1 (en) * | 2018-11-16 | 2020-05-22 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus for treating pompe disease |
-
2019
- 2019-05-15 SG SG11202011191WA patent/SG11202011191WA/en unknown
- 2019-05-15 US US17/055,523 patent/US12084693B2/en active Active
- 2019-05-15 KR KR1020207036017A patent/KR20210021310A/ko active Pending
- 2019-05-15 MY MYPI2020005992A patent/MY207355A/en unknown
- 2019-05-15 BR BR112020023393-5A patent/BR112020023393A2/pt not_active Application Discontinuation
- 2019-05-15 PE PE2020001867A patent/PE20210337A1/es unknown
- 2019-05-15 CA CA3100213A patent/CA3100213A1/en active Pending
- 2019-05-15 WO PCT/US2019/032502 patent/WO2019222411A1/en not_active Ceased
- 2019-05-15 EP EP19803504.0A patent/EP3794043A4/en active Pending
- 2019-05-15 MX MX2020012332A patent/MX2020012332A/es unknown
- 2019-05-15 AU AU2019268363A patent/AU2019268363A1/en not_active Abandoned
- 2019-05-15 CN CN201980040391.0A patent/CN112334489A/zh active Pending
- 2019-05-15 JP JP2020564115A patent/JP2021523718A/ja not_active Ceased
- 2019-05-16 TW TW108116960A patent/TW202016310A/zh unknown
-
2020
- 2020-11-16 PH PH12020551957A patent/PH12020551957A1/en unknown
- 2020-11-16 CL CL2020002973A patent/CL2020002973A1/es unknown
- 2020-12-15 CO CONC2020/0015707A patent/CO2020015707A2/es unknown
-
2024
- 2024-09-09 US US18/828,493 patent/US20250101396A1/en active Pending
-
2025
- 2025-01-17 JP JP2025006785A patent/JP2025066765A/ja not_active Withdrawn
Also Published As
| Publication number | Publication date |
|---|---|
| CO2020015707A2 (es) | 2021-03-19 |
| JP2021523718A (ja) | 2021-09-09 |
| KR20210021310A (ko) | 2021-02-25 |
| EP3794043A4 (en) | 2022-02-23 |
| MY207355A (en) | 2025-02-21 |
| PE20210337A1 (es) | 2021-02-19 |
| BR112020023393A2 (pt) | 2021-02-09 |
| MX2020012332A (es) | 2021-02-26 |
| EP3794043A1 (en) | 2021-03-24 |
| TW202016310A (zh) | 2020-05-01 |
| SG11202011191WA (en) | 2020-12-30 |
| WO2019222411A1 (en) | 2019-11-21 |
| US20210222141A1 (en) | 2021-07-22 |
| PH12020551957A1 (en) | 2021-09-20 |
| AU2019268363A1 (en) | 2021-01-07 |
| CN112334489A (zh) | 2021-02-05 |
| CA3100213A1 (en) | 2019-11-21 |
| US20250101396A1 (en) | 2025-03-27 |
| JP2025066765A (ja) | 2025-04-23 |
| US12084693B2 (en) | 2024-09-10 |
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