JP2021523718A - コドン最適化された酸性α−グルコシダーゼ発現カセット及びそれを使用する方法 - Google Patents

コドン最適化された酸性α−グルコシダーゼ発現カセット及びそれを使用する方法 Download PDF

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JP2021523718A
JP2021523718A JP2020564115A JP2020564115A JP2021523718A JP 2021523718 A JP2021523718 A JP 2021523718A JP 2020564115 A JP2020564115 A JP 2020564115A JP 2020564115 A JP2020564115 A JP 2020564115A JP 2021523718 A JP2021523718 A JP 2021523718A
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nucleic acid
gaa
acid encoding
aav
sequence
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JP2021523718A5 (enExample
Inventor
サビエル アングエラ，
サビエル アングエラ，
ショーン アーマー，
ショーン アーマー，
ジェイミー ノーディン，
ジェイミー ノーディン，
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Spark Therapeutics Inc
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Spark Therapeutics Inc
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Priority to JP2025006785A priority Critical patent/JP2025066765A/ja
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    • 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
    • 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
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    • 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
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    • 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
    • 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
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    • 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
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    • 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
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    • 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

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  • Health & Medical Sciences (AREA)
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  • Wood Science & Technology (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Biophysics (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • 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)
JP2020564115A 2018-05-16 2019-05-15 コドン最適化された酸性α−グルコシダーゼ発現カセット及びそれを使用する方法 Ceased JP2021523718A (ja)

Priority Applications (1)

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JP2025006785A JP2025066765A (ja) 2018-05-16 2025-01-17 コドン最適化された酸性α－グルコシダーゼ発現カセット及びそれを使用する方法

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862672419P 2018-05-16 2018-05-16
US62/672,419 2018-05-16
US201862734454P 2018-09-21 2018-09-21
US62/734,454 2018-09-21
PCT/US2019/032502 WO2019222411A1 (en) 2018-05-16 2019-05-15 Codon-optimized acid αlpha-glucosidase expression cassettes and methods of using same

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JP2025006785A Division JP2025066765A (ja) 2018-05-16 2025-01-17 コドン最適化された酸性α－グルコシダーゼ発現カセット及びそれを使用する方法

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JP2021523718A true JP2021523718A (ja) 2021-09-09
JP2021523718A5 JP2021523718A5 (enExample) 2022-04-11

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JP2025006785A Withdrawn JP2025066765A (ja) 2018-05-16 2025-01-17 コドン最適化された酸性α－グルコシダーゼ発現カセット及びそれを使用する方法

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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)

* Cited by examiner, † Cited by third party
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

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013151666A2 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of biologics and proteins associated with human disease
WO2018046772A1 (en) * 2016-09-12 2018-03-15 Genethon Acid-alpha glucosidase variants and uses thereof
WO2018046775A1 (en) * 2016-09-12 2018-03-15 Genethon Acid-alpha glucosidase variants and uses thereof

Family Cites Families (9)

* Cited by examiner, † Cited by third party
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
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
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

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013151666A2 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of biologics and proteins associated with human disease
WO2018046772A1 (en) * 2016-09-12 2018-03-15 Genethon Acid-alpha glucosidase variants and uses thereof
WO2018046775A1 (en) * 2016-09-12 2018-03-15 Genethon Acid-alpha glucosidase variants and uses thereof

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CO2020015707A2 (es) 2021-03-19
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
CL2020002973A1 (es) 2021-07-19
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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