JP6884268B2 - 糖原病Ｉａ型治療薬 - Google Patents

糖原病Ｉａ型治療薬 Download PDF

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JP6884268B2
JP6884268B2 JP2020505066A JP2020505066A JP6884268B2 JP 6884268 B2 JP6884268 B2 JP 6884268B2 JP 2020505066 A JP2020505066 A JP 2020505066A JP 2020505066 A JP2020505066 A JP 2020505066A JP 6884268 B2 JP6884268 B2 JP 6884268B2
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compound
mmol
g6pc
synthesis
oligonucleotide
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JPWO2019172286A1 (ja
Inventor
小泉　誠
誠 小泉
朗之 大西
朗之 大西
剛 益田
剛 益田
充広 岩本
充広 岩本
幸子 関口
幸子 関口
伊藤　健太郎
健太郎 伊藤
真之介 辻
真之介 辻
雅文 松尾
雅文 松尾
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Daiichi Sankyo Co Ltd
Kobe Gakuin Educational Foundation
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Daiichi Sankyo Co Ltd
Kobe Gakuin Educational Foundation
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
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    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
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    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/3341—5-Methylcytosine
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    • C12N2320/33—Alteration of splicing
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JP2020505066A 2018-03-09 2019-03-05 糖原病Ｉａ型治療薬 Active JP6884268B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
JP2018043524 2018-03-09
JP2018043524 2018-03-09
JP2018128015 2018-07-05
JP2018128015 2018-07-05
PCT/JP2019/008713 WO2019172286A1 (ja) 2018-03-09 2019-03-05 糖原病Ｉａ型治療薬

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JP2021002759A Division JP2021072800A (ja) 2018-03-09 2021-01-12 糖原病Ｉａ型治療薬

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JPWO2019172286A1 JPWO2019172286A1 (ja) 2021-03-18
JP6884268B2 true JP6884268B2 (ja) 2021-06-09

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JP2021002759A Pending JP2021072800A (ja) 2018-03-09 2021-01-12 糖原病Ｉａ型治療薬
JP2023137828A Pending JP2023159397A (ja) 2018-03-09 2023-08-28 糖原病Ｉａ型治療薬

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US (1) US11958878B2 (https=)
EP (1) EP3763815A4 (https=)
JP (3) JP6884268B2 (https=)
KR (2) KR20210130854A (https=)
CN (2) CN111511915A (https=)
TW (3) TWI760600B (https=)
WO (1) WO2019172286A1 (https=)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021049504A1 (ja) * 2019-09-10 2021-03-18 第一三共株式会社 肝臓送達用ＧａｌＮＡｃ－オリゴヌクレオチドコンジュゲートおよび製造方法
IL292286A (en) * 2019-10-18 2022-06-01 Daiichi Sankyo Co Ltd Production method for bicyclic phosphoramidite
CN114763367B (zh) * 2021-01-14 2024-08-06 施能康生物科技有限公司 化合物、缀合物及其用途
CA3224134A1 (en) * 2021-06-24 2022-12-29 Patrick Joseph ANTONELLIS Novel therapeutic delivery moieties and uses thereof
JP2024532271A (ja) 2021-08-30 2024-09-05 ホンジーン バイオテック コーポレイション 官能化ｎ－アセチルガラクトサミンアナログ
WO2023114746A1 (en) 2021-12-15 2023-06-22 Hongene Biotech Corporation Functionalized n-acetylgalactosamine analogs
KR20240161967A (ko) 2022-03-16 2024-11-13 다이이찌 산쿄 가부시키가이샤 트랜스페린 수용체 2 의 발현을 억제하는 siRNA
JPWO2023176863A1 (https=) 2022-03-16 2023-09-21
US20230372508A1 (en) * 2022-05-19 2023-11-23 Olix Us, Inc. Linkers coupling functional ligands to macromolecules
JPWO2024075760A1 (https=) 2022-10-05 2024-04-11
WO2024118503A1 (en) 2022-11-28 2024-06-06 Hongene Biotech Corporation Functionalized n-acetylgalactosamine analogs

Family Cites Families (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH0787982A (ja) 1993-01-29 1995-04-04 Sankyo Co Ltd 修飾オリゴデオキシリボヌクレオチド
CN1123038A (zh) 1993-05-11 1996-05-22 北卡罗来纳大学查珀尔希尔分校 阻止异常剪接的反义寡核苷酸及其使用方法
US6096881A (en) 1997-05-30 2000-08-01 Hybridon, Inc. Sulfur transfer reagents for oligonucleotide synthesis
AU9063398A (en) 1997-09-12 1999-04-05 Exiqon A/S Oligonucleotide analogues
CZ296576B6 (cs) 1999-02-12 2006-04-12 Sankyo Company Limited Nukleosidový analog a oligonukleotidový analog a farmaceutický prostredek, sonda a primer s jeho obsahem
US6261840B1 (en) 2000-01-18 2001-07-17 Isis Pharmaceuticals, Inc. Antisense modulation of PTP1B expression
JP2001267074A (ja) 2000-03-22 2001-09-28 Fuji Photo Film Co Ltd 有機発光素子
JP4777777B2 (ja) 2002-11-25 2011-09-21 雅文 松尾 ｍＲＮＡ前駆体のスプライシングを修飾するＥＮＡ核酸医薬
US20050261233A1 (en) 2004-04-21 2005-11-24 Sanjay Bhanot Modulation of glucose-6-phosphatase translocase expression
US7323308B2 (en) 2004-09-03 2008-01-29 Affymetrix, Inc. Methods of genetic analysis of E. coli
US8178503B2 (en) 2006-03-03 2012-05-15 International Business Machines Corporation Ribonucleic acid interference molecules and binding sites derived by analyzing intergenic and intronic regions of genomes
WO2008109366A2 (en) 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting ccnd1 gene expression and uses thereof
WO2009082607A2 (en) 2007-12-04 2009-07-02 Alnylam Pharmaceuticals, Inc. Targeting lipids
US12129514B2 (en) * 2009-04-30 2024-10-29 Molecular Loop Biosolutions, Llc Methods and compositions for evaluating genetic markers
PT3305302T (pt) 2009-06-17 2018-12-14 Biogen Ma Inc Composições e métodos de modulação de excisões de smn2 em um sujeito
WO2011053614A1 (en) 2009-10-30 2011-05-05 Merck Sharp & Dohme Corp. Crystalline cdm-nag and methods for producing same
CA2812046A1 (en) 2010-09-15 2012-03-22 Alnylam Pharmaceuticals, Inc. Modified irna agents
DK2718437T3 (en) * 2011-06-10 2018-08-06 Inst Nat Sante Rech Med Methods for the Treatment of Liver's Congenital Amaurosis
EP2788487B1 (en) * 2011-12-08 2018-04-04 Sarepta Therapeutics, Inc. Oligonucleotide analogues targeting human lmna
AU2013240200B2 (en) * 2012-03-28 2016-10-20 Longhorn Vaccines And Diagnostics, Llc Compositions and methods for the collection and isolation of nucleic acids from biological specimens
BR112015008673A2 (pt) 2012-10-17 2017-09-26 Fond Telethon composto, composição farmacêutica e método
US10077443B2 (en) 2012-11-15 2018-09-18 Roche Innovation Center Copenhagen A/S Oligonucleotide conjugates
JP6270742B2 (ja) 2013-01-10 2018-01-31 塩野義製薬株式会社 架橋型核酸誘導体の製造方法
CN104955952A (zh) 2013-01-30 2015-09-30 弗·哈夫曼-拉罗切有限公司 Lna寡核苷酸碳水化合物缀合物
DK2991656T3 (da) 2013-05-01 2020-03-23 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression
TW201534578A (zh) 2013-07-08 2015-09-16 Daiichi Sankyo Co Ltd 新穎脂質
JP6702862B2 (ja) 2013-07-11 2020-06-03 アルニラム ファーマスーティカルズ インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． オリゴヌクレオチド−リガンドコンジュゲートおよびそれらの調製方法
KR20160083876A (ko) 2013-11-14 2016-07-12 로슈 이노베이션 센터 코펜하겐 에이/에스 ApoB 안티센스 접합체 화합물
WO2015081101A1 (en) 2013-11-26 2015-06-04 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
JP6482475B2 (ja) 2014-01-07 2019-03-13 レナセラピューティクス株式会社 アンチセンスオリゴヌクレオチド及び糖誘導体を含む二本鎖オリゴヌクレオチド
WO2016055601A1 (en) 2014-10-10 2016-04-14 F. Hoffmann-La Roche Ag Galnac phosphoramidites, nucleic acid conjugates thereof and their use
JP6824169B2 (ja) 2014-12-23 2021-02-03 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
EP3328439B1 (en) 2015-07-31 2020-04-08 Arcturus Therapeutics, Inc. Multiligand agent for drug delivery
CA2999649A1 (en) * 2015-11-06 2017-05-11 Crispr Therapeutics Ag Materials and methods for treatment of glycogen storage disease type 1a
MX378999B (es) 2015-11-16 2025-03-10 Hoffmann La Roche Fosforamidita de agrupacion de n-acetilgalactosamina (galnac).
CA3005245A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of alagille syndrome
AU2017213404A1 (en) 2016-01-29 2018-09-20 Kyowa Kirin Co., Ltd. Nucleic acid conjugate
US10731546B2 (en) 2017-02-06 2020-08-04 Borgwarner Inc. Diffuser in wastegate turbine housings
US11963974B2 (en) 2017-03-10 2024-04-23 National Center For Child Health And Development Antisense oligonucleotide and composition for prevention or treatment of glycogen storage disease type Ia
JP6591515B2 (ja) 2017-12-07 2019-10-16 東芝テック株式会社 印刷装置、制御方法及びコンピュータプログラム

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