JP2021072800A5 - - Google Patents
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- 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) | KR102398295B1 (https=) |
| CN (2) | CN111511915A (https=) |
| TW (3) | TW202202622A (https=) |
| WO (1) | WO2019172286A1 (https=) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL291251B1 (en) * | 2019-09-10 | 2026-02-01 | Daiichi Sankyo Co Ltd | Oligonucleotide-galnac conjugate for targeted delivery to the liver and method for its production |
| IL292286A (en) | 2019-10-18 | 2022-06-01 | Daiichi Sankyo Co Ltd | Production method for bicyclic phosphoramidite |
| CN118852286A (zh) * | 2021-01-14 | 2024-10-29 | 施能康生物科技有限公司 | 化合物、缀合物及其用途 |
| AU2022299173A1 (en) * | 2021-06-24 | 2024-01-18 | Eli Lilly And Company | Novel therapeutic delivery moieties and uses thereof |
| EP4396193A4 (en) * | 2021-08-30 | 2025-07-02 | Hongene Biotech Corp | FUNCTIONALIZED N-ACETYLGALACTOSAMINE ANALOGUES |
| JP2024546585A (ja) | 2021-12-15 | 2024-12-26 | ホンジーン バイオテック コーポレイション | 官能基化ｎ－アセチルガラクトサミンアナログ |
| EP4495240A1 (en) | 2022-03-16 | 2025-01-22 | Daiichi Sankyo Company, Limited | Sirna for suppressing expression of transferrin receptor-2 |
| US20250188477A1 (en) | 2022-03-16 | 2025-06-12 | Daiichi Sankyo Company, Limited | CHEMICALLY MODIFIED OLIGONUCLEOTIDE HAVING RNAi ACTIVITY |
| US20230372508A1 (en) * | 2022-05-19 | 2023-11-23 | Olix Us, Inc. | Linkers coupling functional ligands to macromolecules |
| TW202430499A (zh) | 2022-10-05 | 2024-08-01 | 日商第一三共股份有限公司 | 具有２分支型之ｎ－乙醯基－ｄ－半乳糖胺結構之化合物的製造方法 |
| 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 |
| DE69435005T2 (de) | 1993-05-11 | 2008-04-17 | The University Of North Carolina At Chapel Hill | Antisense Oligonukleotide die anomales Splicing verhindern und deren Verwendung |
| US6096881A (en) | 1997-05-30 | 2000-08-01 | Hybridon, Inc. | Sulfur transfer reagents for oligonucleotide synthesis |
| JP4236812B2 (ja) | 1997-09-12 | 2009-03-11 | エクシコン エ／エス | オリゴヌクレオチド類似体 |
| ES2234563T5 (es) | 1999-02-12 | 2018-01-17 | Daiichi Sankyo Company, Limited | Nuevos análogos de nucleósidos y oligonucleótidos |
| 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 |
| EP2392660A3 (en) * | 2002-11-25 | 2012-03-28 | Masafumi Matsuo | ENA Nucleic Acid Drugs Modifying Splicing in mRNA Precursor |
| 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 |
| WO2009073809A2 (en) | 2007-12-04 | 2009-06-11 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugates as delivery agents for oligonucleotides |
| US12129514B2 (en) * | 2009-04-30 | 2024-10-29 | Molecular Loop Biosolutions, Llc | Methods and compositions for evaluating genetic markers |
| ES2699827T3 (es) | 2009-06-17 | 2019-02-13 | Biogen Ma Inc | Composiciones y métodos para la modulación de corte y empalme de SMN2 en un sujeto |
| 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 |
| WO2012168435A1 (en) * | 2011-06-10 | 2012-12-13 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods for the treatment of leber congenital amaurosis |
| ES2935606T3 (es) * | 2011-12-08 | 2023-03-08 | Sarepta Therapeutics Inc | Análogos de oligonucleótidos dirigidos a LMNA humana |
| 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 |
| US20150273016A1 (en) | 2012-10-17 | 2015-10-01 | Fondazione Telethon | Gene therapy for glycogen storage diseases |
| CN104837996A (zh) | 2012-11-15 | 2015-08-12 | 罗氏创新中心哥本哈根有限公司 | 抗apob反义缀合物化合物 |
| WO2014109384A1 (ja) | 2013-01-10 | 2014-07-17 | 塩野義製薬株式会社 | 架橋型核酸誘導体の製造方法 |
| AU2014211406B2 (en) | 2013-01-30 | 2019-07-18 | Roche Innovation Center Copenhagen A/S | LNA oligonucleotide carbohydrate conjugates |
| DK2992098T3 (da) | 2013-05-01 | 2019-06-17 | Ionis Pharmaceuticals Inc | Sammensætninger og fremgangsmåder til modulering af hbv- og ttr-ekspression |
| TW201534578A (zh) | 2013-07-08 | 2015-09-16 | Daiichi Sankyo Co Ltd | 新穎脂質 |
| WO2015006740A2 (en) | 2013-07-11 | 2015-01-15 | Alnylam Pharmaceuticals, Inc. | Oligonucleotide-ligand conjugates and process for their preparation |
| MX2016005855A (es) * | 2013-11-14 | 2016-07-13 | Roche Innovation Ct Copenhagen As | Compuestos de conjugados antisentido de apolipoproteina b (apob). |
| AU2014354839B2 (en) | 2013-11-26 | 2020-06-04 | 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 |
| CN106795200B (zh) | 2014-10-10 | 2020-06-19 | 豪夫迈·罗氏有限公司 | Galnac亚磷酰胺、其核酸缀合物及其用途 |
| WO2016106303A1 (en) | 2014-12-23 | 2016-06-30 | THE UNITED STATES OF AMERICA, as representd by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES | Adeno-associated virus vectors encoding modified g6pc and uses thereof |
| KR20180031025A (ko) | 2015-07-31 | 2018-03-27 | 아크투루스 쎄라퓨틱스, 인크. | 약물 전달을 위한 다중 리간드 제제 |
| BR112018008971A2 (pt) * | 2015-11-06 | 2018-11-27 | Crispr Therapeutics Ag | materiais e métodos para tratamento de doença de armazenamento de glicogênio tipo 1a |
| BR112018003910B1 (pt) | 2015-11-16 | 2022-03-22 | F. Hoffmann-La Roche Ag | Derivado de fosforamidita galnac, processo para a preparação de um derivado de fosforamidita galnac de fórmula i e uso dos derivados de fosforamidita galnac de fórmula i |
| 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 |
| EP3594346A4 (en) | 2017-03-10 | 2020-12-16 | National Center For Child Health And Development | ANTISENS OLIGONUCLEOTIDE AND COMPOSITION FOR THE PREVENTION OR TREATMENT OF TYPE IA GLYCOGEN STORAGE DISEASE |
| 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 KR KR1020207018195A patent/KR102398295B1/ko active Active
- 2019-03-05 CN CN201980006746.4A patent/CN111511915A/zh active Pending
- 2019-03-05 WO PCT/JP2019/008713 patent/WO2019172286A1/ja not_active Ceased
- 2019-03-05 KR KR1020217034642A patent/KR20210130854A/ko not_active Withdrawn
- 2019-03-05 US US16/966,132 patent/US11958878B2/en active Active
- 2019-03-05 JP JP2020505066A patent/JP6884268B2/ja active Active
- 2019-03-05 CN CN202310034281.1A patent/CN115976028B/zh active Active
- 2019-03-05 EP EP19763548.5A patent/EP3763815A4/en not_active Withdrawn
- 2019-03-08 TW TW110137469A patent/TW202202622A/zh unknown
- 2019-03-08 TW TW112150508A patent/TW202417628A/zh unknown
- 2019-03-08 TW TW108107814A patent/TWI760600B/zh active
-
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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