JP2024002985A5 - - Google Patents
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- Publication number
- JP2024002985A5 JP2024002985A5 JP2023103614A JP2023103614A JP2024002985A5 JP 2024002985 A5 JP2024002985 A5 JP 2024002985A5 JP 2023103614 A JP2023103614 A JP 2023103614A JP 2023103614 A JP2023103614 A JP 2023103614A JP 2024002985 A5 JP2024002985 A5 JP 2024002985A5
- Authority
- JP
- Japan
- Prior art keywords
- oligonucleotide
- positions
- rnai oligonucleotide
- nucleotides
- rnai
- 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.)
- Granted
Links
- 108091034117 Oligonucleotide Proteins 0.000 claims 24
- 108091030071 RNAI Proteins 0.000 claims 19
- 230000009368 gene silencing by RNA Effects 0.000 claims 19
- 125000003729 nucleotide group Chemical group 0.000 claims 17
- 239000002773 nucleotide Substances 0.000 claims 15
- 108091081021 Sense strand Proteins 0.000 claims 12
- 230000000692 anti-sense effect Effects 0.000 claims 12
- 101000798696 Homo sapiens Transmembrane protease serine 6 Proteins 0.000 claims 3
- 102100032452 Transmembrane protease serine 6 Human genes 0.000 claims 3
- 230000004048 modification Effects 0.000 claims 3
- 238000012986 modification Methods 0.000 claims 3
- NYHBQMYGNKIUIF-UUOKFMHZSA-N Guanosine Chemical compound C1=NC=2C(=O)NC(N)=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O NYHBQMYGNKIUIF-UUOKFMHZSA-N 0.000 claims 2
- OVRNDRQMDRJTHS-CBQIKETKSA-N N-Acetyl-D-Galactosamine Chemical compound CC(=O)N[C@H]1[C@@H](O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-CBQIKETKSA-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
- MBLBDJOUHNCFQT-UHFFFAOYSA-N N-acetyl-D-galactosamine Natural products CC(=O)NC(C=O)C(O)C(O)C(O)CO MBLBDJOUHNCFQT-UHFFFAOYSA-N 0.000 claims 2
- 230000000295 complement effect Effects 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 239000003446 ligand Substances 0.000 claims 2
- 125000000956 methoxy group Chemical group [H]C([H])([H])O* 0.000 claims 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 2
- 239000002342 ribonucleoside Substances 0.000 claims 2
- 230000008685 targeting Effects 0.000 claims 2
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 2
- 229940126836 transmembrane protease serine 6 synthesis reducer Drugs 0.000 claims 2
- 208000018565 Hemochromatosis Diseases 0.000 claims 1
- 208000033981 Hereditary haemochromatosis Diseases 0.000 claims 1
- 208000005980 beta thalassemia Diseases 0.000 claims 1
- -1 delivery agent Substances 0.000 claims 1
- 229940124447 delivery agent Drugs 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 239000000126 substance Substances 0.000 claims 1
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202263355210P | 2022-06-24 | 2022-06-24 | |
| US63/355,210 | 2022-06-24 | ||
| EP22209113 | 2022-11-23 | ||
| EP22209113 | 2022-11-23 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2024002985A JP2024002985A (ja) | 2024-01-11 |
| JP2024002985A5 true JP2024002985A5 (enExample) | 2024-03-22 |
| JP7560616B2 JP7560616B2 (ja) | 2024-10-02 |
Family
ID=87036742
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023103614A Active JP7560616B2 (ja) | 2022-06-24 | 2023-06-23 | 膜貫通型セリンプロテアーゼ６（ｔｍｐｒｓｓ６）発現を阻害するための組成物および方法 |
Country Status (14)
| Country | Link |
|---|---|
| US (2) | US11952574B2 (enExample) |
| EP (1) | EP4544049A1 (enExample) |
| JP (1) | JP7560616B2 (enExample) |
| KR (1) | KR102759742B1 (enExample) |
| CN (1) | CN119403927A (enExample) |
| AR (1) | AR129710A1 (enExample) |
| AU (1) | AU2023288906A1 (enExample) |
| CA (1) | CA3257962A1 (enExample) |
| CO (1) | CO2024017422A2 (enExample) |
| IL (1) | IL317012A (enExample) |
| MA (1) | MA71238A (enExample) |
| MX (1) | MX2024015763A (enExample) |
| TW (1) | TWI868755B (enExample) |
| WO (1) | WO2023247738A1 (enExample) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| TWI868755B (zh) | 2022-06-24 | 2025-01-01 | 丹麥商諾佛 儂迪克股份有限公司 | 抑制跨膜絲胺酸蛋白酶６（ｔｍｐｒｓｓ６）表現的組成物及方法 |
| TW202527968A (zh) * | 2024-01-08 | 2025-07-16 | 大陸商上海舶望製藥有限公司 | 用於抑制跨膜絲氨酸蛋白酶6（tmprss6）表達的組合物和方法 |
Family Cites Families (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6469158B1 (en) | 1992-05-14 | 2002-10-22 | Ribozyme Pharmaceuticals, Incorporated | Synthesis, deprotection, analysis and purification of RNA and ribozymes |
| US5804683A (en) | 1992-05-14 | 1998-09-08 | Ribozyme Pharmaceuticals, Inc. | Deprotection of RNA with alkylamine |
| US5977343A (en) | 1992-05-14 | 1999-11-02 | Ribozyme Pharmaceuticals, Inc. | Synthesis, deprotection, analysis and purification of RNA and ribozymes |
| DE69431669T2 (de) | 1993-09-02 | 2003-10-23 | Ribozyme Pharmaceuticals, Inc. | Enzymatische nukleiksaüre die nicht-nukleotide enthaltet |
| US5889136A (en) | 1995-06-09 | 1999-03-30 | The Regents Of The University Of Colorado | Orthoester protecting groups in RNA synthesis |
| US5998203A (en) | 1996-04-16 | 1999-12-07 | Ribozyme Pharmaceuticals, Inc. | Enzymatic nucleic acids containing 5'-and/or 3'-cap structures |
| US6111086A (en) | 1998-02-27 | 2000-08-29 | Scaringe; Stephen A. | Orthoester protecting groups |
| DK2796553T3 (da) | 2000-03-30 | 2019-09-30 | Whitehead Inst Biomedical Res | Rna-sekvensspecifikke formidlere af rna-interferens |
| CZ302719B6 (cs) | 2000-12-01 | 2011-09-21 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | Izolovaná molekula dvouretezcové RNA, zpusob její výroby a její použití |
| US20050159378A1 (en) | 2001-05-18 | 2005-07-21 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of Myc and/or Myb gene expression using short interfering nucleic acid (siNA) |
| EP1442137A4 (en) | 2001-11-07 | 2005-08-31 | Applera Corp | UNIVERSAL NUCLEOTIDES FOR NUCLEIC ACID ANALYSIS |
| US20070265220A1 (en) | 2004-03-15 | 2007-11-15 | City Of Hope | Methods and compositions for the specific inhibition of gene expression by double-stranded RNA |
| US20090018097A1 (en) | 2005-09-02 | 2009-01-15 | Mdrna, Inc | Modification of double-stranded ribonucleic acid molecules |
| US20100331389A1 (en) | 2008-09-22 | 2010-12-30 | Bob Dale Brown | Compositions and methods for the specific inhibition of gene expression by dsRNA containing modified nucleotides |
| US8513207B2 (en) | 2008-12-18 | 2013-08-20 | Dicerna Pharmaceuticals, Inc. | Extended dicer substrate agents and methods for the specific inhibition of gene expression |
| WO2010093788A2 (en) | 2009-02-11 | 2010-08-19 | Dicerna Pharmaceuticals, Inc. | Multiplex dicer substrate rna interference molecules having joining sequences |
| WO2011005860A2 (en) | 2009-07-07 | 2011-01-13 | Alnylam Pharmaceuticals, Inc. | 5' phosphate mimics |
| US9725479B2 (en) | 2010-04-22 | 2017-08-08 | Ionis Pharmaceuticals, Inc. | 5′-end derivatives |
| EP3674409A1 (en) | 2011-03-29 | 2020-07-01 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of tmprss6 gene |
| US8901098B2 (en) | 2011-10-25 | 2014-12-02 | Isis Pharmaceuticals, Inc. | Antisense modulation of GCCR expression |
| ES2761343T3 (es) | 2011-11-07 | 2020-05-19 | Ionis Pharmaceuticals Inc | Modulación de la expresión de TMPRSS6 |
| BR112015029276B1 (pt) * | 2013-05-22 | 2022-07-12 | Alnylam Pharmaceuticals, Inc | Agente de irna fita dupla capaz de inibir a expressão de tmprss6, composição farmacêutica e uso dos mesmos |
| EP3134732A2 (en) * | 2014-04-24 | 2017-03-01 | Alnylam Pharmaceuticals, Inc. | Methods and compositions for detecting anti-drug antibodies |
| WO2016085852A1 (en) * | 2014-11-24 | 2016-06-02 | Alnylam Pharmaceuticals, Inc. | Tmprss6 irna compositions and methods of use thereof |
| JP7105065B2 (ja) * | 2014-12-15 | 2022-07-22 | ダイセルナ ファーマシューティカルズ， インコーポレイテッド | リガンド修飾二本鎖核酸 |
| JP6833705B2 (ja) | 2015-04-03 | 2021-02-24 | アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． | Ｔｍｐｒｓｓ６発現を調節するための化合物及び方法 |
| WO2018045317A1 (en) | 2016-09-02 | 2018-03-08 | Dicerna Pharmaceuticals, Inc. | 4'-phosphate analogs and oligonucleotides comprising the same |
| RS63887B1 (sr) | 2017-04-05 | 2023-02-28 | Silence Therapeutics Gmbh | Inhibicija tmprss6 posredovana rnk interferencijom |
| WO2019006375A1 (en) * | 2017-06-29 | 2019-01-03 | Dicerna Pharmaceuticals, Inc. | COMPOSITIONS AND METHODS FOR INHIBITING HMGB1 EXPRESSION |
| EP3598995A1 (en) | 2018-07-26 | 2020-01-29 | Silence Therapeutics GmbH | Products and compositions |
| EP3883581A4 (en) * | 2018-12-28 | 2023-03-29 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for inhibiting hmgb1 expression |
| TW202221120A (zh) * | 2020-08-04 | 2022-06-01 | 美商黛瑟納製藥公司 | 用於治療代謝症候群之組成物及方法 |
| US20250002908A1 (en) | 2021-04-20 | 2025-01-02 | AstraZeneca Ireland Limited | Compositions and methods for inhibiting complement component 3 expression |
| JP2024517686A (ja) | 2021-04-26 | 2024-04-23 | アルナイラム ファーマシューティカルズ， インコーポレイテッド | 膜貫通プロテアーゼ、セリン６（ＴＭＰＲＳＳ６）ｉＲＮＡ組成物およびその使用方法 |
| TWI868755B (zh) | 2022-06-24 | 2025-01-01 | 丹麥商諾佛 儂迪克股份有限公司 | 抑制跨膜絲胺酸蛋白酶６（ｔｍｐｒｓｓ６）表現的組成物及方法 |
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2023
- 2023-06-21 TW TW112123278A patent/TWI868755B/zh active
- 2023-06-23 CN CN202380048101.3A patent/CN119403927A/zh active Pending
- 2023-06-23 EP EP23735271.1A patent/EP4544049A1/en active Pending
- 2023-06-23 IL IL317012A patent/IL317012A/en unknown
- 2023-06-23 CA CA3257962A patent/CA3257962A1/en active Pending
- 2023-06-23 AR ARP230101626A patent/AR129710A1/es unknown
- 2023-06-23 WO PCT/EP2023/067070 patent/WO2023247738A1/en not_active Ceased
- 2023-06-23 JP JP2023103614A patent/JP7560616B2/ja active Active
- 2023-06-23 US US18/213,289 patent/US11952574B2/en active Active
- 2023-06-23 AU AU2023288906A patent/AU2023288906A1/en active Pending
- 2023-06-23 KR KR1020230080886A patent/KR102759742B1/ko active Active
- 2023-06-23 MA MA71238A patent/MA71238A/fr unknown
-
2024
- 2024-02-14 US US18/441,001 patent/US12503701B2/en active Active
- 2024-12-17 MX MX2024015763A patent/MX2024015763A/es unknown
- 2024-12-18 CO CONC2024/0017422A patent/CO2024017422A2/es unknown