JP2018516091A5 - - Google Patents
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- JP2018516091A5 JP2018516091A5 JP2018502624A JP2018502624A JP2018516091A5 JP 2018516091 A5 JP2018516091 A5 JP 2018516091A5 JP 2018502624 A JP2018502624 A JP 2018502624A JP 2018502624 A JP2018502624 A JP 2018502624A JP 2018516091 A5 JP2018516091 A5 JP 2018516091A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- nucleotides
- oligonucleotide
- dsrna
- complementary
- 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
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- 125000003729 nucleotide group Chemical group 0.000 claims 23
- 239000002773 nucleotide Substances 0.000 claims 18
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 17
- 108091034117 Oligonucleotide Proteins 0.000 claims 15
- 230000000295 complement effect Effects 0.000 claims 14
- 101150043003 Htt gene Proteins 0.000 claims 8
- 230000000692 anti-sense effect Effects 0.000 claims 7
- 239000002336 ribonucleotide Substances 0.000 claims 7
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 6
- 108020004999 messenger RNA Proteins 0.000 claims 5
- 230000015556 catabolic process Effects 0.000 claims 4
- 238000006731 degradation reaction Methods 0.000 claims 4
- 230000002401 inhibitory effect Effects 0.000 claims 4
- 210000004556 brain Anatomy 0.000 claims 3
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol group Chemical group [C@@H]1(CC[C@H]2[C@@H]3CC=C4C[C@@H](O)CC[C@]4(C)[C@H]3CC[C@]12C)[C@H](C)CCCC(C)C HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims 3
- 238000000034 method Methods 0.000 claims 3
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 3
- 101710163270 Nuclease Proteins 0.000 claims 2
- 108091081021 Sense strand Proteins 0.000 claims 2
- RYYWUUFWQRZTIU-UHFFFAOYSA-N Thiophosphoric acid Chemical group OP(O)(S)=O RYYWUUFWQRZTIU-UHFFFAOYSA-N 0.000 claims 2
- 230000002209 hydrophobic effect Effects 0.000 claims 2
- 102000040650 (ribonucleotides)n+m Human genes 0.000 claims 1
- 208000035657 Abasia Diseases 0.000 claims 1
- 108020004394 Complementary RNA Proteins 0.000 claims 1
- 208000023105 Huntington disease Diseases 0.000 claims 1
- 102100034343 Integrase Human genes 0.000 claims 1
- 101710203526 Integrase Proteins 0.000 claims 1
- 239000002202 Polyethylene glycol Substances 0.000 claims 1
- 108091028664 Ribonucleotide Proteins 0.000 claims 1
- 108091027568 Single-stranded nucleotide Proteins 0.000 claims 1
- 239000000074 antisense oligonucleotide Substances 0.000 claims 1
- 238000012230 antisense oligonucleotides Methods 0.000 claims 1
- 235000012000 cholesterol Nutrition 0.000 claims 1
- 239000003184 complementary RNA Substances 0.000 claims 1
- 150000001875 compounds Chemical class 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 125000001921 locked nucleotide group Chemical group 0.000 claims 1
- 230000001404 mediated effect Effects 0.000 claims 1
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims 1
- ONKSSDKXDIVIHK-UHFFFAOYSA-N n,n-didecyldodecanamide Chemical group CCCCCCCCCCCC(=O)N(CCCCCCCCCC)CCCCCCCCCC ONKSSDKXDIVIHK-UHFFFAOYSA-N 0.000 claims 1
- 210000001577 neostriatum Anatomy 0.000 claims 1
- 108020004707 nucleic acids Proteins 0.000 claims 1
- 102000039446 nucleic acids Human genes 0.000 claims 1
- 150000007523 nucleic acids Chemical class 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 150000008105 phosphatidylcholines Chemical class 0.000 claims 1
- 150000008298 phosphoramidates Chemical class 0.000 claims 1
- 229920001223 polyethylene glycol Polymers 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
- 125000002652 ribonucleotide group Chemical group 0.000 claims 1
- 229920002477 rna polymer Polymers 0.000 claims 1
- 125000006850 spacer group Chemical group 0.000 claims 1
- 230000008685 targeting Effects 0.000 claims 1
- ZIBGPFATKBEMQZ-UHFFFAOYSA-N triethylene glycol Chemical compound OCCOCCOCCO ZIBGPFATKBEMQZ-UHFFFAOYSA-N 0.000 claims 1
Priority Applications (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022003059A JP7504482B2 (ja) | 2015-04-03 | 2022-01-12 | ハンチンチンｍＲＮＡをターゲティングするオリゴヌクレオチド化合物 |
| JP2022003057A JP2022064900A (ja) | 2015-04-03 | 2022-01-12 | ハンチンチンｍＲＮＡをターゲティングするオリゴヌクレオチド化合物 |
| JP2023206985A JP2024028909A (ja) | 2015-04-03 | 2023-12-07 | ハンチンチンｍＲＮＡをターゲティングするオリゴヌクレオチド化合物 |
| JP2024091478A JP2024152725A (ja) | 2015-04-03 | 2024-06-05 | ハンチンチンｍＲＮＡをターゲティングするオリゴヌクレオチド化合物 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562142731P | 2015-04-03 | 2015-04-03 | |
| US62/142,731 | 2015-04-03 | ||
| US201662289274P | 2016-01-31 | 2016-01-31 | |
| US62/289,274 | 2016-01-31 | ||
| PCT/US2016/025722 WO2016161374A1 (en) | 2015-04-03 | 2016-04-01 | Oligonucleotide compounds for targeting huntingtin mrna |
Related Child Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022003057A Division JP2022064900A (ja) | 2015-04-03 | 2022-01-12 | ハンチンチンｍＲＮＡをターゲティングするオリゴヌクレオチド化合物 |
| JP2022003059A Division JP7504482B2 (ja) | 2015-04-03 | 2022-01-12 | ハンチンチンｍＲＮＡをターゲティングするオリゴヌクレオチド化合物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018516091A JP2018516091A (ja) | 2018-06-21 |
| JP2018516091A5 true JP2018516091A5 (enExample) | 2019-03-07 |
| JP7009356B2 JP7009356B2 (ja) | 2022-01-25 |
Family
ID=55752785
Family Applications (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018502624A Active JP7009356B2 (ja) | 2015-04-03 | 2016-04-01 | ハンチンチンｍＲＮＡをターゲティングするオリゴヌクレオチド化合物 |
| JP2022003059A Active JP7504482B2 (ja) | 2015-04-03 | 2022-01-12 | ハンチンチンｍＲＮＡをターゲティングするオリゴヌクレオチド化合物 |
| JP2022003057A Pending JP2022064900A (ja) | 2015-04-03 | 2022-01-12 | ハンチンチンｍＲＮＡをターゲティングするオリゴヌクレオチド化合物 |
| JP2023206985A Pending JP2024028909A (ja) | 2015-04-03 | 2023-12-07 | ハンチンチンｍＲＮＡをターゲティングするオリゴヌクレオチド化合物 |
| JP2024091478A Revoked JP2024152725A (ja) | 2015-04-03 | 2024-06-05 | ハンチンチンｍＲＮＡをターゲティングするオリゴヌクレオチド化合物 |
Family Applications After (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022003059A Active JP7504482B2 (ja) | 2015-04-03 | 2022-01-12 | ハンチンチンｍＲＮＡをターゲティングするオリゴヌクレオチド化合物 |
| JP2022003057A Pending JP2022064900A (ja) | 2015-04-03 | 2022-01-12 | ハンチンチンｍＲＮＡをターゲティングするオリゴヌクレオチド化合物 |
| JP2023206985A Pending JP2024028909A (ja) | 2015-04-03 | 2023-12-07 | ハンチンチンｍＲＮＡをターゲティングするオリゴヌクレオチド化合物 |
| JP2024091478A Revoked JP2024152725A (ja) | 2015-04-03 | 2024-06-05 | ハンチンチンｍＲＮＡをターゲティングするオリゴヌクレオチド化合物 |
Country Status (18)
| Country | Link |
|---|---|
| US (5) | US9809817B2 (enExample) |
| EP (2) | EP3277814B1 (enExample) |
| JP (5) | JP7009356B2 (enExample) |
| CN (2) | CN116004624A (enExample) |
| AU (4) | AU2016244027B2 (enExample) |
| CA (1) | CA2980337A1 (enExample) |
| CY (1) | CY1123289T1 (enExample) |
| DK (1) | DK3277814T3 (enExample) |
| ES (1) | ES2808750T3 (enExample) |
| HR (1) | HRP20201200T1 (enExample) |
| HU (1) | HUE050441T2 (enExample) |
| LT (1) | LT3277814T (enExample) |
| PL (1) | PL3277814T3 (enExample) |
| PT (1) | PT3277814T (enExample) |
| RS (1) | RS60762B1 (enExample) |
| SI (1) | SI3277814T1 (enExample) |
| SM (1) | SMT202000454T1 (enExample) |
| WO (1) | WO2016161374A1 (enExample) |
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| EP3643782A1 (en) | 2008-02-11 | 2020-04-29 | Phio Pharmaceuticals Corp. | Modified rnai polynucleotides and uses thereof |
| CN108165548B (zh) | 2008-09-22 | 2022-10-14 | 菲奥医药公司 | 减小大小的自递送RNAi化合物 |
| WO2010059226A2 (en) | 2008-11-19 | 2010-05-27 | Rxi Pharmaceuticals Corporation | Inhibition of map4k4 through rnai |
| RU2015151857A (ru) | 2008-12-02 | 2019-01-15 | Уэйв Лайф Сайенсес Джапан, Инк. | Способ синтеза модифицированных по атому фосфора нуклеиновых кислот |
| WO2010078536A1 (en) | 2009-01-05 | 2010-07-08 | Rxi Pharmaceuticals Corporation | Inhibition of pcsk9 through rnai |
| US9745574B2 (en) | 2009-02-04 | 2017-08-29 | Rxi Pharmaceuticals Corporation | RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality |
| MX342945B (es) | 2009-07-06 | 2016-10-18 | Ontorii Inc * | Profármacos de ácido nucleico novedosos y métodos de uso de los mismos. |
| CN103200945B (zh) | 2010-03-24 | 2016-07-06 | 雷克西制药公司 | 眼部症候中的rna干扰 |
| US9080171B2 (en) | 2010-03-24 | 2015-07-14 | RXi Parmaceuticals Corporation | Reduced size self-delivering RNAi compounds |
| KR102453078B1 (ko) | 2010-03-24 | 2022-10-11 | 피오 파마슈티칼스 코프. | 진피 및 섬유증성 적응증에서의 ｒｎａ 간섭 |
| US10428019B2 (en) | 2010-09-24 | 2019-10-01 | Wave Life Sciences Ltd. | Chiral auxiliaries |
| EP3248982A1 (en) | 2011-07-19 | 2017-11-29 | Wave Life Sciences Ltd. | Thiosulfonate reagents for the synthesis of functionalized nucleic acids |
| CN104661664B (zh) | 2012-07-13 | 2020-07-03 | 波涛生命科学有限公司 | 手性控制 |
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| CA2879023C (en) | 2012-07-13 | 2017-03-28 | Wave Life Sciences Japan | Asymmetric auxiliary group |
| CN113151180A (zh) | 2013-12-02 | 2021-07-23 | 菲奥医药公司 | 癌症的免疫治疗 |
| JPWO2015108048A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
| US10144933B2 (en) | 2014-01-15 | 2018-12-04 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator |
| US10322173B2 (en) | 2014-01-15 | 2019-06-18 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent |
| MX2016009290A (es) | 2014-01-16 | 2017-02-28 | Wave Life Sciences Ltd | Diseño quiral. |
| CA2947270A1 (en) | 2014-04-28 | 2015-11-05 | Rxi Pharmaceuticals Corporation | Methods for treating cancer using nucleic acids targeting mdm2 or mycn |
| KR102689262B1 (ko) | 2014-09-05 | 2024-07-30 | 피오 파마슈티칼스 코프. | Tyr 또는 mmp1을 표적화하는 핵산을 사용한 노화 및 피부 장애의 치료 방법 |
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| WO2016161388A1 (en) | 2015-04-03 | 2016-10-06 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| CN116004624A (zh) | 2015-04-03 | 2023-04-25 | 马萨诸塞大学 | 用于靶向亨廷汀mRNA的寡核苷酸化合物 |
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| WO2017030973A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| US11021707B2 (en) | 2015-10-19 | 2021-06-01 | Phio Pharmaceuticals Corp. | Reduced size self-delivering nucleic acid compounds targeting long non-coding RNA |
| JP7749201B6 (ja) * | 2016-01-31 | 2025-10-21 | ユニバーシティー オブ マサチューセッツ | 分岐オリゴヌクレオチド |
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| RU2758488C2 (ru) | 2016-05-18 | 2021-10-28 | Вояджер Терапьютикс, Инк. | Модулирующие полинуклеотиды |
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| WO2018013640A1 (en) | 2016-07-15 | 2018-01-18 | University Of Massachusetts | Process of delivering small rnas to sperm |
| CA3033368A1 (en) | 2016-08-12 | 2018-02-15 | University Of Massachusetts | Conjugated oligonucleotides |
| US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| CA3044703A1 (en) * | 2016-11-23 | 2018-05-31 | Mayo Foundation For Medical Education And Research | Particle-mediated delivery of biologics |
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| CN110799647A (zh) | 2017-06-23 | 2020-02-14 | 马萨诸塞大学 | 双尾自递送sirna及相关的方法 |
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| EP4454654A3 (en) | 2017-10-16 | 2025-02-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
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