JP2022530359A - バッテン病の視覚効果を治療または予防するための遺伝子療法 - Google Patents

バッテン病の視覚効果を治療または予防するための遺伝子療法 Download PDF

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JP2022530359A
JP2022530359A JP2021561810A JP2021561810A JP2022530359A JP 2022530359 A JP2022530359 A JP 2022530359A JP 2021561810 A JP2021561810 A JP 2021561810A JP 2021561810 A JP2021561810 A JP 2021561810A JP 2022530359 A JP2022530359 A JP 2022530359A
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JP2022530359A5 (enExample
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ジル エム． ワイマー，
キャスリン メイヤー，
シビ リキッテ，
ブライアン ケー． カスパー，
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サンフォード リサーチ
リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P27/02—Ophthalmic agents
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    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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    • 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
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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
    • 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
    • C12N2750/14171—Demonstrated in vivo effect

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  • General Chemical & Material Sciences (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2021561810A 2019-04-15 2020-04-15 バッテン病の視覚効果を治療または予防するための遺伝子療法 Ceased JP2022530359A (ja)

Priority Applications (1)

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JP2025156997A JP2025181954A (ja) 2019-04-15 2025-09-22 バッテン病の視覚効果を治療または予防するための遺伝子療法

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US201962834340P 2019-04-15 2019-04-15
US62/834,340 2019-04-15
PCT/US2020/028366 WO2020214737A1 (en) 2019-04-15 2020-04-15 Gene therapy for treating or preventing visual effects in batten disease

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JP2025156997A Division JP2025181954A (ja) 2019-04-15 2025-09-22 バッテン病の視覚効果を治療または予防するための遺伝子療法

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JP2022530359A true JP2022530359A (ja) 2022-06-29
JP2022530359A5 JP2022530359A5 (enExample) 2023-04-25

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JP2021561810A Ceased JP2022530359A (ja) 2019-04-15 2020-04-15 バッテン病の視覚効果を治療または予防するための遺伝子療法
JP2025156997A Pending JP2025181954A (ja) 2019-04-15 2025-09-22 バッテン病の視覚効果を治療または予防するための遺伝子療法

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US (1) US20220193259A1 (enExample)
EP (1) EP3955969A1 (enExample)
JP (2) JP2022530359A (enExample)
AU (1) AU2020257208A1 (enExample)
CA (1) CA3137080A1 (enExample)
WO (1) WO2020214737A1 (enExample)

Families Citing this family (2)

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Publication number Priority date Publication date Assignee Title
CN119213133B (zh) * 2022-05-16 2026-01-06 上海天泽云泰生物医药有限公司 用于治疗i型戊二酸尿症的重组aav载体
CN120842306B (zh) * 2025-07-17 2026-01-06 渤海大学 一种仿刺参精抗氧化肽及其制备方法和应用

Citations (1)

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JP2015525565A (ja) * 2012-07-04 2015-09-07 フィリップ・モーリス・プロダクツ・ソシエテ・アノニム 改善された結合剤を備える可燃性熱源

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DE69433922T2 (de) 1993-11-09 2005-07-28 Medical College Of Ohio, Toledo Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
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WO1996017947A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
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EP3955969A1 (en) 2022-02-23
US20220193259A1 (en) 2022-06-23
WO2020214737A1 (en) 2020-10-22
CA3137080A1 (en) 2020-10-22
AU2020257208A1 (en) 2021-11-11
JP2025181954A (ja) 2025-12-11

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