JP7486274B2 - 糖原病を処置するための方法および組成物 - Google Patents

糖原病を処置するための方法および組成物 Download PDF

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JP7486274B2
JP7486274B2 JP2021534702A JP2021534702A JP7486274B2 JP 7486274 B2 JP7486274 B2 JP 7486274B2 JP 2021534702 A JP2021534702 A JP 2021534702A JP 2021534702 A JP2021534702 A JP 2021534702A JP 7486274 B2 JP7486274 B2 JP 7486274B2
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クリストファー ティッパー，
ケリー リード クラーク，
サミュエル ウォズワース，
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ウルトラジェニックス ファーマシューティカル インコーポレイテッド
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    • 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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    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
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    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
    • C12Y301/03009—Glucose-6-phosphatase (3.1.3.9)
    • 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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    • 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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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • 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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  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
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  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2021534702A 2018-12-18 2019-12-18 糖原病を処置するための方法および組成物 Active JP7486274B2 (ja)

Applications Claiming Priority (3)

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US201862781380P 2018-12-18 2018-12-18
US62/781,380 2018-12-18
PCT/US2019/067247 WO2020132115A1 (en) 2018-12-18 2019-12-18 Methods and compositions for treating glycogen storage diseases

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JP2022514271A JP2022514271A (ja) 2022-02-10
JP2022514271A5 JP2022514271A5 (OSRAM) 2022-05-20
JP7486274B2 true JP7486274B2 (ja) 2024-05-17

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US (1) US20220017922A1 (OSRAM)
EP (1) EP3898981B1 (OSRAM)
JP (1) JP7486274B2 (OSRAM)
CN (1) CN113454226B (OSRAM)
AU (1) AU2019405758B2 (OSRAM)
BR (1) BR112021012104A2 (OSRAM)
CA (1) CA3123841A1 (OSRAM)
DK (1) DK3898981T5 (OSRAM)
ES (1) ES2969222T3 (OSRAM)
IL (1) IL283956A (OSRAM)
MX (1) MX2021007379A (OSRAM)
PL (1) PL3898981T3 (OSRAM)
PT (1) PT3898981T (OSRAM)
WO (1) WO2020132115A1 (OSRAM)

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WO2023069891A1 (en) * 2021-10-19 2023-04-27 University Of Connecticut Compositions and methods for the treatment of glycogen storage disorders

Citations (2)

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JP2016510221A (ja) 2013-02-15 2016-04-07 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 眼の遺伝子関連疾患の治療のための方法及び組成物
JP2018501791A (ja) 2014-12-23 2018-01-25 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 改変ｇ６ｐｃをコードするアデノ随伴ウイルスベクターおよびその使用

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US6759050B1 (en) 1998-12-03 2004-07-06 Avigen, Inc. Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
JP2002191363A (ja) * 1999-07-29 2002-07-09 Herikkusu Kenkyusho:Kk 全長ｃＤＮＡ合成用プライマー、およびその用途
US7261544B2 (en) 2003-05-21 2007-08-28 Genzyme Corporation Methods for producing preparations of recombinant AAV virions substantially free of empty capsids
CA2713338C (en) 2008-01-29 2021-10-26 Applied Genetic Technologies Corporation Recombinant virus production using mammalian cells in suspension
US8679837B2 (en) 2009-04-02 2014-03-25 University Of Florida Research Foundation, Inc. Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors
ES2680915T3 (es) 2010-01-28 2018-09-11 The Children's Hospital Of Philadelphia Plataforma de fabricación escalable para la purificación de vectores virales y vectores virales purificados de este modo para su utilización en terapia génica
US9644216B2 (en) * 2013-11-26 2017-05-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Adeno-associated virus vectors for treatment of glycogen storage disease
LT3436593T (lt) 2016-03-28 2023-03-10 Ultragenyx Pharmaceutical Inc. Adenoviruso šiluminio inaktyvavimo būdai
JP2020506695A (ja) * 2017-01-30 2020-03-05 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 糖原病を処置するための組換えウイルスベクター

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JP2016510221A (ja) 2013-02-15 2016-04-07 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 眼の遺伝子関連疾患の治療のための方法及び組成物
JP2018501791A (ja) 2014-12-23 2018-01-25 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 改変ｇ６ｐｃをコードするアデノ随伴ウイルスベクターおよびその使用

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Publication number Publication date
KR20210105390A (ko) 2021-08-26
ES2969222T3 (es) 2024-05-17
DK3898981T3 (da) 2024-01-22
MX2021007379A (es) 2021-07-15
AU2019405758A1 (en) 2021-07-01
EP3898981A1 (en) 2021-10-27
PL3898981T3 (pl) 2024-04-02
AU2019405758A2 (en) 2022-05-12
WO2020132115A8 (en) 2021-05-14
CA3123841A1 (en) 2020-06-25
CN113454226B (zh) 2025-10-31
WO2020132115A1 (en) 2020-06-25
PT3898981T (pt) 2023-12-28
AU2019405758B2 (en) 2026-02-05
US20220017922A1 (en) 2022-01-20
EP3898981B1 (en) 2023-10-18
IL283956A (en) 2021-07-29
DK3898981T5 (da) 2024-08-05
JP2022514271A (ja) 2022-02-10
CN113454226A (zh) 2021-09-28
BR112021012104A2 (pt) 2021-09-08

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