JP2022554141A - 糖原病を治療するための組成物及び方法 - Google Patents

糖原病を治療するための組成物及び方法 Download PDF

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JP2022554141A
JP2022554141A JP2022523856A JP2022523856A JP2022554141A JP 2022554141 A JP2022554141 A JP 2022554141A JP 2022523856 A JP2022523856 A JP 2022523856A JP 2022523856 A JP2022523856 A JP 2022523856A JP 2022554141 A JP2022554141 A JP 2022554141A
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ティー． グレイ，ジョン
カニンガム，ジャスティン
リコ，サルバドール
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オーデンツ セラピューティクス，インコーポレーテッド
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    • A—HUMAN NECESSITIES
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    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
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    • 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
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    • A—HUMAN NECESSITIES
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    • 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
    • A61K48/0083—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 administration regime
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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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    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • C—CHEMISTRY; METALLURGY
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    • 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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
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  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Biomedical Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Pulmonology (AREA)
  • Neurology (AREA)
JP2022523856A 2019-10-25 2020-10-23 糖原病を治療するための組成物及び方法 Pending JP2022554141A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962926282P 2019-10-25 2019-10-25
US62/926,282 2019-10-25
US202063083349P 2020-09-25 2020-09-25
US63/083,349 2020-09-25
PCT/US2020/057081 WO2021081338A1 (en) 2019-10-25 2020-10-23 Compositions and methods for treating glycogen storage disorders

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JP2022554141A true JP2022554141A (ja) 2022-12-28
JPWO2021081338A5 JPWO2021081338A5 (https=) 2023-10-31
JP2022554141A5 JP2022554141A5 (https=) 2023-10-31

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JP2022523856A Pending JP2022554141A (ja) 2019-10-25 2020-10-23 糖原病を治療するための組成物及び方法

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US (1) US20220387562A1 (https=)
EP (1) EP4048286A4 (https=)
JP (1) JP2022554141A (https=)
KR (1) KR20220105643A (https=)
CN (1) CN114828858A (https=)
AU (1) AU2020372429A1 (https=)
BR (1) BR112022007674A2 (https=)
CA (1) CA3158281A1 (https=)
CO (1) CO2022006772A2 (https=)
IL (1) IL292401A (https=)
MX (1) MX2022004799A (https=)
PH (1) PH12022550977A1 (https=)
TW (2) TWI885000B (https=)
WO (1) WO2021081338A1 (https=)

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KR20240145491A (ko) * 2022-02-03 2024-10-07 아스텔라스 진 테라피스, 인크. 폼페병의 개선된 치료를 위한 조성물 및 방법
JP2025527694A (ja) 2022-08-23 2025-08-22 マストバイオ株式会社 Ｉｌ２変異体及びそれを含むタンパク質複合体

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019153009A1 (en) * 2018-02-05 2019-08-08 Audentes Therapeutics, Inc. Transcription regulatory elements and uses thereof
WO2019154939A1 (en) * 2018-02-07 2019-08-15 Genethon Hybrid regulatory elements

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ATE437944T1 (de) * 2000-04-28 2009-08-15 Asklepios Biopharmaceutical In Für das dystrophin-minigen kodierende dna- sequenzen und verfahren zu deren verwendung
ATE400656T1 (de) * 2000-10-06 2008-07-15 Univ Michigan Mini-dystrophin nukleinsäure- und peptidsequenzen
RS61055B1 (sr) * 2012-06-19 2020-12-31 Univ Florida Kompozicije i postupci za lečenje bolesti
EA201890543A1 (ru) * 2015-09-17 2018-08-31 Рисёрч Инститьют Эт Нэйшнвайд Чилдрен'С Хоспитэл Способы и материалы для генной терапии galgt2
EP3293260A1 (en) * 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
WO2019012336A2 (en) * 2017-03-17 2019-01-17 Newcastle University ADENO-ASSOCIATED VIRAL VECTOR DELIVERY OF A MICRO-DYSTROPHIN FRAGMENT FOR TREATING MUSCLE DYSTROPHY
EP3648776A4 (en) * 2017-07-06 2021-04-07 The Medical College of Wisconsin, Inc. NEW IN-VITRO AND IN-VIVO ENRICHMENT STRATEGY AGAINST LYMPHOCYTE FROM VECTOR-TRANSDUCED HSC FOR THE TREATMENT OF DISEASES

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019153009A1 (en) * 2018-02-05 2019-08-08 Audentes Therapeutics, Inc. Transcription regulatory elements and uses thereof
WO2019154939A1 (en) * 2018-02-07 2019-08-15 Genethon Hybrid regulatory elements

Non-Patent Citations (8)

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Title
"新医薬品の臨床評価に関する一般指針について", 厚生省薬務局新医薬品課長通知, vol. 薬新薬第四三号, JPN6023035220, 29 June 1992 (1992-06-29), ISSN: 0005781085 *
GUIDANCE FOR INDUSTRY, JPN6012064675, July 2005 (2005-07-01), ISSN: 0005781084 *
HUM. GENE THER., vol. 26, no. 3, JPN6024039673, 2015, pages 185 - 193, ISSN: 0005572032 *
HUM. GENE THER., vol. 27, no. 1, JPN6024039675, 2015, pages 43 - 59, ISSN: 0005572033 *
MOL. THER. METHOD CLIN. DEV., vol. 12, JPN6024039672, March 2019 (2019-03-01), pages 85 - 101, ISSN: 0005572031 *
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Publication number Publication date
EP4048286A1 (en) 2022-08-31
IL292401A (en) 2022-06-01
CN114828858A (zh) 2022-07-29
TWI885000B (zh) 2025-06-01
CA3158281A1 (en) 2021-04-29
TW202600177A (zh) 2026-01-01
PH12022550977A1 (en) 2023-10-09
BR112022007674A2 (pt) 2022-08-09
TW202116359A (zh) 2021-05-01
CO2022006772A2 (es) 2022-08-09
MX2022004799A (es) 2022-07-19
WO2021081338A1 (en) 2021-04-29
KR20220105643A (ko) 2022-07-27
US20220387562A1 (en) 2022-12-08
AU2020372429A1 (en) 2022-04-28
EP4048286A4 (en) 2023-12-06

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