JP2021072800A5 - - Google Patents
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- Publication number
- JP2021072800A5 JP2021072800A5 JP2021002759A JP2021002759A JP2021072800A5 JP 2021072800 A5 JP2021072800 A5 JP 2021072800A5 JP 2021002759 A JP2021002759 A JP 2021002759A JP 2021002759 A JP2021002759 A JP 2021002759A JP 2021072800 A5 JP2021072800 A5 JP 2021072800A5
- Authority
- JP
- Japan
- Prior art keywords
- oligonucleotide
- solvate
- acceptable salt
- pharmaceutically acceptable
- oligonucleotide according
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
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- 108091034117 Oligonucleotide Proteins 0.000 claims 32
- 150000003839 salts Chemical class 0.000 claims 19
- 239000012453 solvate Substances 0.000 claims 19
- 101000930910 Homo sapiens Glucose-6-phosphatase catalytic subunit 1 Proteins 0.000 claims 9
- 230000000295 complement effect Effects 0.000 claims 9
- 230000035772 mutation Effects 0.000 claims 9
- 239000002773 nucleotide Substances 0.000 claims 6
- 125000003729 nucleotide group Chemical group 0.000 claims 6
- 230000004048 modification Effects 0.000 claims 5
- 238000012986 modification Methods 0.000 claims 5
- 125000000824 D-ribofuranosyl group Chemical group [H]OC([H])([H])[C@@]1([H])OC([H])(*)[C@]([H])(O[H])[C@]1([H])O[H] 0.000 claims 4
- HMFHBZSHGGEWLO-SOOFDHNKSA-N D-ribofuranose Chemical compound OC[C@H]1OC(O)[C@H](O)[C@@H]1O HMFHBZSHGGEWLO-SOOFDHNKSA-N 0.000 claims 2
- OVRNDRQMDRJTHS-KEWYIRBNSA-N N-acetyl-D-galactosamine Chemical group CC(=O)N[C@H]1C(O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-KEWYIRBNSA-N 0.000 claims 2
- 238000010934 O-alkylation reaction Methods 0.000 claims 2
- 206010018464 Glycogen storage disease type I Diseases 0.000 claims 1
- 229910000147 aluminium phosphate Inorganic materials 0.000 claims 1
- 239000002299 complementary DNA Substances 0.000 claims 1
- 238000004132 cross linking Methods 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 208000011460 glycogen storage disease due to glucose-6-phosphatase deficiency type IA Diseases 0.000 claims 1
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims 1
- 238000001727 in vivo Methods 0.000 claims 1
- 239000008177 pharmaceutical agent Substances 0.000 claims 1
- NBIIXXVUZAFLBC-UHFFFAOYSA-N phosphoric acid Substances OP(O)(O)=O NBIIXXVUZAFLBC-UHFFFAOYSA-N 0.000 claims 1
- 229940124597 therapeutic agent Drugs 0.000 claims 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023137828A JP2023159397A (ja) | 2018-03-09 | 2023-08-28 | 糖原病Ｉａ型治療薬 |
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2018043524 | 2018-03-09 | ||
| JP2018043524 | 2018-03-09 | ||
| JP2018128015 | 2018-07-05 | ||
| JP2018128015 | 2018-07-05 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020505066A Division JP6884268B2 (ja) | 2018-03-09 | 2019-03-05 | 糖原病Ｉａ型治療薬 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023137828A Division JP2023159397A (ja) | 2018-03-09 | 2023-08-28 | 糖原病Ｉａ型治療薬 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021072800A JP2021072800A (ja) | 2021-05-13 |
| JP2021072800A5 true JP2021072800A5 (https=) | 2022-02-08 |
Family
ID=67845726
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020505066A Active JP6884268B2 (ja) | 2018-03-09 | 2019-03-05 | 糖原病Ｉａ型治療薬 |
| 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 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020505066A Active JP6884268B2 (ja) | 2018-03-09 | 2019-03-05 | 糖原病Ｉａ型治療薬 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023137828A Pending JP2023159397A (ja) | 2018-03-09 | 2023-08-28 | 糖原病Ｉａ型治療薬 |
Country Status (7)
| Country | Link |
|---|---|
| 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)
| 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)
| 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 |
-
2019
- 2019-03-05 CN CN201980006746.4A patent/CN111511915A/zh active Pending
- 2019-03-05 CN CN202310034281.1A patent/CN115976028B/zh active Active
- 2019-03-05 WO PCT/JP2019/008713 patent/WO2019172286A1/ja not_active Ceased
- 2019-03-05 JP JP2020505066A patent/JP6884268B2/ja active Active
- 2019-03-05 KR KR1020217034642A patent/KR20210130854A/ko not_active Withdrawn
- 2019-03-05 EP EP19763548.5A patent/EP3763815A4/en not_active Withdrawn
- 2019-03-05 US US16/966,132 patent/US11958878B2/en active Active
- 2019-03-05 KR KR1020207018195A patent/KR102398295B1/ko active Active
- 2019-03-08 TW TW108107814A patent/TWI760600B/zh active
- 2019-03-08 TW TW110137469A patent/TW202202622A/zh unknown
- 2019-03-08 TW TW112150508A patent/TW202417628A/zh unknown
-
2021
- 2021-01-12 JP JP2021002759A patent/JP2021072800A/ja active Pending
-
2023
- 2023-08-28 JP JP2023137828A patent/JP2023159397A/ja active Pending
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