JP2018516091A5 - - Google Patents
Download PDFInfo
- Publication number
- 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
Links
- 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 (https=) | 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 |
| 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 |
| 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 |
| 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 |
|---|---|---|---|
| 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 |
| 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 |
| 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 (https=) |
| EP (2) | EP3766973A1 (https=) |
| JP (5) | JP7009356B2 (https=) |
| CN (2) | CN107980062B (https=) |
| AU (4) | AU2016244027B2 (https=) |
| CA (1) | CA2980337A1 (https=) |
| CY (1) | CY1123289T1 (https=) |
| DK (1) | DK3277814T3 (https=) |
| ES (1) | ES2808750T3 (https=) |
| HR (1) | HRP20201200T1 (https=) |
| HU (1) | HUE050441T2 (https=) |
| LT (1) | LT3277814T (https=) |
| PL (1) | PL3277814T3 (https=) |
| PT (1) | PT3277814T (https=) |
| RS (1) | RS60762B1 (https=) |
| SI (1) | SI3277814T1 (https=) |
| SM (1) | SMT202000454T1 (https=) |
| WO (1) | WO2016161374A1 (https=) |
Families Citing this family (86)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2715289C (en) | 2008-02-11 | 2019-12-24 | Rxi Pharmaceuticals Corporation | Modified rnai polynucleotides and uses thereof |
| EP2342340A1 (en) | 2008-09-22 | 2011-07-13 | Rxi Pharmaceuticals Corporation | Rna interference in skin indications |
| WO2010059226A2 (en) | 2008-11-19 | 2010-05-27 | Rxi Pharmaceuticals Corporation | Inhibition of map4k4 through rnai |
| CN102282155B (zh) | 2008-12-02 | 2017-06-09 | 日本波涛生命科学公司 | 磷原子修饰的核酸的合成方法 |
| 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 |
| RU2612521C2 (ru) | 2009-07-06 | 2017-03-09 | Онтории, Инк. | Новые пролекарства нуклеиновых кислот и способы их применения |
| RU2615143C2 (ru) | 2010-03-24 | 2017-04-04 | Адвирна | Самодоставляющие PHKi соединения уменьшенного размера |
| CN103200945B (zh) | 2010-03-24 | 2016-07-06 | 雷克西制药公司 | 眼部症候中的rna干扰 |
| EP3560503B1 (en) | 2010-03-24 | 2021-11-17 | Phio Pharmaceuticals Corp. | Rna interference in dermal and fibrotic indications |
| JP5868324B2 (ja) | 2010-09-24 | 2016-02-24 | 株式会社Wave Life Sciences Japan | 不斉補助基 |
| CN103796657B (zh) | 2011-07-19 | 2017-07-11 | 波涛生命科学有限公司 | 合成官能化核酸的方法 |
| CA2879066C (en) | 2012-07-13 | 2019-08-13 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant |
| PL2872485T3 (pl) | 2012-07-13 | 2021-05-31 | Wave Life Sciences Ltd. | Asymetryczna grupa pomocnicza |
| KR102213609B1 (ko) | 2012-07-13 | 2021-02-08 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
| CN106061488B (zh) | 2013-12-02 | 2021-04-09 | 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 |
| JPWO2015108047A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤 |
| 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 |
| BR112016016400A2 (pt) | 2014-01-16 | 2017-10-03 | Wave Life Sciences Ltd | Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos |
| WO2015168108A2 (en) | 2014-04-28 | 2015-11-05 | Rxi Pharmaceuticals Corporation | Methods for treating cancer using nucleic targeting mdm2 or mycn |
| US10900039B2 (en) | 2014-09-05 | 2021-01-26 | Phio Pharmaceuticals Corp. | Methods for treating aging and skin disorders using nucleic acids targeting Tyr or MMP1 |
| KR20230145206A (ko) | 2014-11-14 | 2023-10-17 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| WO2016077687A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| PL3277814T3 (pl) | 2015-04-03 | 2020-11-30 | University Of Massachusetts | Związki oligonukleotydowe ukierunkowane na mrna huntingtyny |
| RS62672B1 (sr) | 2015-04-03 | 2021-12-31 | Univ Massachusetts | Oligonukleotidna jedinjenja za tretman preeklampsije i drugih angiogenskih poremećaja |
| US20160319278A1 (en) | 2015-04-03 | 2016-11-03 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| US10808247B2 (en) | 2015-07-06 | 2020-10-20 | Phio Pharmaceuticals Corp. | Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach |
| EP3862005A1 (en) | 2015-07-06 | 2021-08-11 | Phio Pharmaceuticals Corp. | Nucleic acid molecules targeting superoxide dismutase 1 (sod1) |
| IL320434A (en) | 2015-07-22 | 2025-06-01 | Wave Life Sciences Ltd | Oligonucleotide preparations and methods |
| WO2017030973A1 (en) * | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| WO2017070151A1 (en) | 2015-10-19 | 2017-04-27 | Rxi Pharmaceuticals Corporation | Reduced size self-delivering nucleic acid compounds targeting long non-coding rna |
| US10478503B2 (en) * | 2016-01-31 | 2019-11-19 | University Of Massachusetts | Branched oligonucleotides |
| JP2019513371A (ja) | 2016-04-01 | 2019-05-30 | アビディティー バイオサイエンシーズ エルエルシー | 核酸ポリペプチド組成物とその使用 |
| EP3452596A4 (en) | 2016-05-04 | 2020-03-18 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS |
| KR102427379B1 (ko) * | 2016-05-18 | 2022-08-02 | 보이저 테라퓨틱스, 인크. | 헌팅톤 질환을 치료하기 위한 조성물 및 방법 |
| KR20240056729A (ko) | 2016-05-18 | 2024-04-30 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| US11299733B2 (en) | 2016-07-15 | 2022-04-12 | 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 |
| ES2912270T3 (es) * | 2016-11-23 | 2022-05-25 | Mayo Found Medical Education & Res | Transporte de productos biológicos por medio de partículas |
| CN110121558B (zh) * | 2016-11-28 | 2023-06-13 | 日商那帕洁制药股份有限公司 | 化学修饰siRNA |
| DK3607069T3 (da) * | 2017-04-05 | 2022-11-21 | Silence Therapeutics Gmbh | Produkter og sammensætninger |
| US12544344B2 (en) | 2017-04-19 | 2026-02-10 | Phio Pharmaceuticals Corp. | Topical delivery of nucleic acid compounds |
| JP2020518258A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法 |
| JP2020518259A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | ハンチントン病治療組成物および方法 |
| CA3065523A1 (en) | 2017-06-02 | 2018-12-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| JP2020522510A (ja) * | 2017-06-02 | 2020-07-30 | ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． | オリゴヌクレオチド組成物及びその使用方法 |
| JP7406793B2 (ja) * | 2017-06-23 | 2023-12-28 | ユニバーシティー オブ マサチューセッツ | ２テイル自己デリバリー型ｓｉＲＮＡおよび関連方法 |
| EP3691657A4 (en) | 2017-10-04 | 2021-07-21 | Avidity Biosciences, Inc. | NUCLEIC ACID POLYPEPTIDE COMPOSITIONS AND USES THEREOF |
| TW202413649A (zh) | 2017-10-16 | 2024-04-01 | 美商航海家醫療公司 | 肌萎縮性脊髓側索硬化症(als)之治療 |
| EP3697908A1 (en) | 2017-10-16 | 2020-08-26 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| EP3735252A4 (en) * | 2018-01-04 | 2021-10-06 | Avidity Biosciences, Inc. | HETERODUPLEX NUCLEIC ACID MOLECULES AND USES THEREOF |
| CN111954548A (zh) * | 2018-02-21 | 2020-11-17 | 亚克安娜生命科学有限公司 | 流体递送系统和方法 |
| EP3769769A4 (en) * | 2018-03-19 | 2022-05-04 | National University Corporation Tokyo Medical and Dental University | NUCLEIC ACID WITH REDUCED TOXICITY |
| SG11202009877XA (en) | 2018-04-12 | 2020-11-27 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods of use thereof |
| EP3781677A4 (en) | 2018-04-16 | 2022-01-19 | University of Massachusetts | COMPOSITIONS AND METHODS FOR IMPROVED GENE EDITTING |
| US12391942B2 (en) | 2018-05-11 | 2025-08-19 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| WO2020033899A1 (en) | 2018-08-10 | 2020-02-13 | University Of Massachusetts | Modified oligonucleotides targeting snps |
| US11279930B2 (en) | 2018-08-23 | 2022-03-22 | University Of Massachusetts | O-methyl rich fully stabilized oligonucleotides |
| CN113614232A (zh) | 2019-01-18 | 2021-11-05 | 马萨诸塞大学 | 动态药代动力学修饰锚 |
| CN113924364A (zh) * | 2019-03-08 | 2022-01-11 | 罗格斯新泽西州立大学 | 治疗亨廷顿病的组合物和方法 |
| US11820985B2 (en) * | 2019-03-26 | 2023-11-21 | University Of Massachusetts | Modified oligonucleotides with increased stability |
| US11629347B2 (en) * | 2019-05-06 | 2023-04-18 | University Of Massachusetts | Anti-C9ORF72 oligonucleotides and related methods |
| US12006499B2 (en) | 2019-06-06 | 2024-06-11 | Avidity Biosciences, Inc. | Una amidites and uses thereof |
| CN114375296A (zh) | 2019-06-06 | 2022-04-19 | 艾维迪提生物科学公司 | 核酸多肽组合物及其用途 |
| EP3983077A4 (en) * | 2019-06-17 | 2023-12-20 | Alnylam Pharmaceuticals, Inc. | DELIVERY OF OLIGONUCLEOTIDES TO THE STRIATUM |
| US20220348912A1 (en) | 2019-06-20 | 2022-11-03 | University Of Massachusetts | Compositions and methods for improved gene editing |
| KR20220047989A (ko) | 2019-08-09 | 2022-04-19 | 유니버시티 오브 매사추세츠 | Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드 |
| US12365894B2 (en) | 2019-09-16 | 2025-07-22 | University Of Massachusetts | Branched lipid conjugates of siRNA for specific tissue delivery |
| EP4051795A1 (en) * | 2019-11-01 | 2022-09-07 | Alnylam Pharmaceuticals, Inc. | Huntingtin (htt) irna agent compositions and methods of use thereof |
| CN112980837B (zh) * | 2019-12-13 | 2023-07-04 | 深圳艾码生物科技有限公司 | 一种抑制HTT基因表达的siRNA及其前体和应用 |
| US20210363524A1 (en) * | 2020-03-18 | 2021-11-25 | University Of Massachusetts | Oligonucleotides for snca modulation |
| JP7837560B2 (ja) | 2020-03-26 | 2026-03-31 | ユニバーシティー オブ マサチューセッツ | 安定性の向上を有する修飾オリゴヌクレオチドの合成 |
| US20210355491A1 (en) * | 2020-04-20 | 2021-11-18 | University Of Massachusetts | Oligonucleotides for msh3 modulation |
| WO2021242883A1 (en) | 2020-05-26 | 2021-12-02 | University Of Massachusetts | Synthetic oligonucleotides having regions of block and cluster modifications |
| WO2022031591A2 (en) * | 2020-08-03 | 2022-02-10 | University Of Massachusetts | Oligonucleotides for htt-1a modulation |
| US20240200063A1 (en) * | 2021-03-24 | 2024-06-20 | Atalanta Therapeutics, Inc. | Microglial gene silencing using double-stranded sirna |
| EP4314296A2 (en) | 2021-03-29 | 2024-02-07 | Alnylam Pharmaceuticals, Inc. | Huntingtin (htt) irna agent compositions and methods of use thereof |
| CN117677699A (zh) | 2021-06-23 | 2024-03-08 | 马萨诸塞大学 | 用于治疗先兆子痫和其他血管生成病症的优化抗flt1寡核苷酸化合物 |
| US20230340475A1 (en) * | 2021-12-17 | 2023-10-26 | University Of Massachusetts | Oligonucleotides for mlh1 modulation |
| AU2023354001A1 (en) * | 2022-09-28 | 2025-04-17 | Atalanta Therapeutics, Inc. | Compositions and methods for treatment of huntington's disease |
| IL325355A (en) * | 2023-06-30 | 2026-02-01 | Spark Therapeutics Inc | RNA inhibitor targeting huntingtin expression |
| WO2025073871A1 (en) | 2023-10-03 | 2025-04-10 | Bicycletx Limited | Bicyclic peptide ligand complexes specific for tfr1 |
| WO2025160434A1 (en) * | 2024-01-26 | 2025-07-31 | Genzyme Corporation | Artificial micrornas targeting huntington's disease |
| WO2025265013A2 (en) * | 2024-06-20 | 2025-12-26 | Arrowhead Pharmaceuticals, Inc. | Rnai agents for inhibiting expression of huntingtin (htt), compositions thereof, and methods of use |
Family Cites Families (65)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4522811A (en) | 1982-07-08 | 1985-06-11 | Syntex (U.S.A.) Inc. | Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides |
| US5328470A (en) | 1989-03-31 | 1994-07-12 | The Regents Of The University Of Michigan | Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor |
| ES2113371T3 (es) | 1989-11-16 | 1998-05-01 | Univ Duke | Transformacion de celulas epidermicas de tejidos animales con la ayuda de particulas. |
| US5252479A (en) * | 1991-11-08 | 1993-10-12 | Research Corporation Technologies, Inc. | Safe vector for gene therapy |
| US5587308A (en) * | 1992-06-02 | 1996-12-24 | The United States Of America As Represented By The Department Of Health & Human Services | Modified adeno-associated virus vector capable of expression from a novel promoter |
| US6291438B1 (en) | 1993-02-24 | 2001-09-18 | Jui H. Wang | Antiviral anticancer poly-substituted phenyl derivatized oligoribonucleotides and methods for their use |
| US5858988A (en) | 1993-02-24 | 1999-01-12 | Wang; Jui H. | Poly-substituted-phenyl-oligoribo nucleotides having enhanced stability and membrane permeability and methods of use |
| TW360548B (en) | 1993-04-08 | 1999-06-11 | Powderject Res Ltd | Products for therapeutic use |
| WO1996033761A1 (en) | 1995-04-28 | 1996-10-31 | Medtronic, Inc. | Intraparenchymal infusion catheter system |
| US5684143A (en) | 1996-02-21 | 1997-11-04 | Lynx Therapeutics, Inc. | Oligo-2'-fluoronucleotide N3'->P5' phosphoramidates |
| US5735814A (en) | 1996-04-30 | 1998-04-07 | Medtronic, Inc. | Techniques of treating neurodegenerative disorders by brain infusion |
| US20080119427A1 (en) | 1996-06-06 | 2008-05-22 | Isis Pharmaceuticals, Inc. | Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation |
| US5898031A (en) | 1996-06-06 | 1999-04-27 | Isis Pharmaceuticals, Inc. | Oligoribonucleotides for cleaving RNA |
| US6177403B1 (en) | 1996-10-21 | 2001-01-23 | The Trustees Of The University Of Pennsylvania | Compositions, methods, and apparatus for delivery of a macromolecular assembly to an extravascular tissue of an animal |
| US6042820A (en) | 1996-12-20 | 2000-03-28 | Connaught Laboratories Limited | Biodegradable copolymer containing α-hydroxy acid and α-amino acid units |
| US6472375B1 (en) | 1998-04-16 | 2002-10-29 | John Wayne Cancer Institute | DNA vaccine and methods for its use |
| EP1386004A4 (en) | 2001-04-05 | 2005-02-16 | Ribozyme Pharm Inc | MODULATION OF GENE EXPRESSION ASSOCIATED WITH INFLAMMATORY PROLIFERATION AND GROWTH OF NEURITIES BY METHODS INVOLVING NUCLEIC ACID |
| EP2447370B1 (en) | 2001-09-28 | 2018-07-18 | Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. | MicroRNA molecules |
| US20050096284A1 (en) | 2002-02-20 | 2005-05-05 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA) |
| US7858367B2 (en) | 2002-04-30 | 2010-12-28 | Duke University | Viral vectors and methods for producing and using the same |
| KR101215701B1 (ko) | 2002-07-19 | 2012-12-26 | 베스 이스라엘 데코니스 메디칼 센터 | 자간전증 또는 자간의 진단 및 치료 방법 |
| AU2003291755A1 (en) | 2002-11-05 | 2004-06-07 | Isis Pharmaceuticals, Inc. | Oligomers comprising modified bases for binding cytosine and uracil or thymine and their use |
| US7250496B2 (en) * | 2002-11-14 | 2007-07-31 | Rosetta Genomics Ltd. | Bioinformatically detectable group of novel regulatory genes and uses thereof |
| AU2003295600A1 (en) * | 2002-11-14 | 2004-06-15 | Dharmacon, Inc. | Functional and hyperfunctional sirna |
| US7750144B2 (en) | 2003-06-02 | 2010-07-06 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNA silencing |
| PT1633767T (pt) | 2003-06-02 | 2019-02-27 | Univ Massachusetts | Métodos e composições para controlar a eficácia do silenciamento de arn |
| EP1633890B2 (en) | 2003-06-02 | 2020-11-18 | University of Massachusetts | METHODS AND COMPOSITIONS FOR ENHANCING THE EFFICACY AND SPECIFICITY OF FNAi |
| WO2008005562A2 (en) | 2006-07-07 | 2008-01-10 | University Of Massachusetts | Rna silencing compositions and methods for the treatment of huntington's disease |
| EP1709195B1 (en) | 2003-12-19 | 2014-01-22 | Novartis Vaccines and Diagnostics, Inc. | Cell transfecting formulations of small interfering rna, related compositions and methods of making and use |
| WO2005077333A2 (en) | 2004-02-10 | 2005-08-25 | University Of Florida Research Foundation, Inc. | Gel-based delivery of recombinant adeno-associated virus vectors |
| ES2423060T3 (es) | 2004-03-12 | 2013-09-17 | Alnylam Pharmaceuticals, Inc. | Agentes iRNA que tienen como diana al VEGF |
| US20060014289A1 (en) | 2004-04-20 | 2006-01-19 | Nastech Pharmaceutical Company Inc. | Methods and compositions for enhancing delivery of double-stranded RNA or a double-stranded hybrid nucleic acid to regulate gene expression in mammalian cells |
| WO2005116204A1 (ja) * | 2004-05-11 | 2005-12-08 | Rnai Co., Ltd. | Ｒｎａ干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法 |
| JP5136766B2 (ja) | 2004-12-15 | 2013-02-06 | ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | キメラベクター |
| EP1937066A4 (en) | 2005-08-18 | 2008-12-24 | Alnylam Pharmaceuticals Inc | METHOD AND COMPOSITIONS FOR THE TREATMENT OF NERVOUS DISEASES |
| JP5111385B2 (ja) * | 2005-10-28 | 2013-01-09 | 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 | ハンチンチン遺伝子の発現を抑制するための組成物および方法 |
| US20070259827A1 (en) | 2006-01-25 | 2007-11-08 | University Of Massachusetts | Compositions and methods for enhancing discriminatory RNA interference |
| US9198981B2 (en) | 2006-02-01 | 2015-12-01 | The University Of Kentucky | Modulation of angiogenesis |
| WO2007094218A1 (ja) | 2006-02-16 | 2007-08-23 | Gifu University | 修飾オリゴヌクレオチド |
| US20090306178A1 (en) | 2006-03-27 | 2009-12-10 | Balkrishen Bhat | Conjugated double strand compositions for use in gene modulation |
| US20080039415A1 (en) | 2006-08-11 | 2008-02-14 | Gregory Robert Stewart | Retrograde transport of sirna and therapeutic uses to treat neurologic disorders |
| WO2008154482A2 (en) | 2007-06-08 | 2008-12-18 | Sirnaomics, Inc. | Sirna compositions and methods of use in treatment of ocular diseases |
| CN101199858A (zh) | 2007-10-18 | 2008-06-18 | 广州拓谱基因技术有限公司 | 治疗眼科疾病的多靶点小干扰rna鸡尾酒制剂及制备方法 |
| TW200927177A (en) | 2007-10-24 | 2009-07-01 | Nat Inst Of Advanced Ind Scien | Lipid-modified double-stranded RNA having potent RNA interference effect |
| WO2009099991A2 (en) * | 2008-01-31 | 2009-08-13 | The Brigham And Women's Hospital, Inc. | Treatment of cancer |
| US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| EP2342340A1 (en) | 2008-09-22 | 2011-07-13 | Rxi Pharmaceuticals Corporation | Rna interference in skin indications |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| EP2470656B1 (en) | 2009-08-27 | 2015-05-06 | Idera Pharmaceuticals, Inc. | Composition for inhibiting gene expression and uses thereof |
| RU2615143C2 (ru) | 2010-03-24 | 2017-04-04 | Адвирна | Самодоставляющие PHKi соединения уменьшенного размера |
| WO2012005898A2 (en) | 2010-06-15 | 2012-01-12 | Alnylam Pharmaceuticals, Inc. | Chinese hamster ovary (cho) cell transcriptome, corresponding sirnas and uses thereof |
| CA2828544A1 (en) | 2011-03-03 | 2012-09-07 | Quark Pharmaceuticals, Inc. | Oligonucleotide modulators of the toll-like receptor pathway |
| US20140275211A1 (en) * | 2011-06-21 | 2014-09-18 | Alnylam Pharmaceuticals, Inc. | Assays and methods for determining activity of a therapeutic agent in a subject |
| US20150299696A1 (en) | 2012-05-02 | 2015-10-22 | Sirna Therapeutics, Inc. | SHORT INTERFERING NUCLEIC ACID (siNA) COMPOSITIONS |
| KR101567576B1 (ko) | 2012-05-22 | 2015-11-10 | 올릭스 주식회사 | 세포 내 관통능을 가지고 ｒｎａ 간섭을 유도하는 핵산 분자 및 그 용도 |
| US9480752B2 (en) | 2012-07-10 | 2016-11-01 | Baseclick Gmbh | Anandamide-modified nucleic acid molecules |
| US20150247141A1 (en) | 2012-09-14 | 2015-09-03 | Rana Therapeutics, Inc. | Multimeric oligonucleotide compounds |
| 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 |
| US10513710B2 (en) * | 2014-04-18 | 2019-12-24 | University Of Massachusetts | Exosomal loading using hydrophobically modified oligonucleotides |
| RS62672B1 (sr) | 2015-04-03 | 2021-12-31 | Univ Massachusetts | Oligonukleotidna jedinjenja za tretman preeklampsije i drugih angiogenskih poremećaja |
| PL3277814T3 (pl) | 2015-04-03 | 2020-11-30 | University Of Massachusetts | Związki oligonukleotydowe ukierunkowane na mrna huntingtyny |
| US20160319278A1 (en) | 2015-04-03 | 2016-11-03 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| IL320434A (en) | 2015-07-22 | 2025-06-01 | Wave Life Sciences Ltd | Oligonucleotide preparations and methods |
| WO2017030973A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| US10478503B2 (en) | 2016-01-31 | 2019-11-19 | University Of Massachusetts | Branched oligonucleotides |
-
2016
- 2016-04-01 PL PL16716412T patent/PL3277814T3/pl unknown
- 2016-04-01 CN CN201680031524.4A patent/CN107980062B/zh active Active
- 2016-04-01 CA CA2980337A patent/CA2980337A1/en active Pending
- 2016-04-01 PT PT167164128T patent/PT3277814T/pt unknown
- 2016-04-01 JP JP2018502624A patent/JP7009356B2/ja active Active
- 2016-04-01 EP EP20164108.1A patent/EP3766973A1/en active Pending
- 2016-04-01 HU HUE16716412A patent/HUE050441T2/hu unknown
- 2016-04-01 US US15/089,319 patent/US9809817B2/en active Active
- 2016-04-01 HR HRP20201200TT patent/HRP20201200T1/hr unknown
- 2016-04-01 AU AU2016244027A patent/AU2016244027B2/en active Active
- 2016-04-01 SM SM20200454T patent/SMT202000454T1/it unknown
- 2016-04-01 DK DK16716412.8T patent/DK3277814T3/da active
- 2016-04-01 WO PCT/US2016/025722 patent/WO2016161374A1/en not_active Ceased
- 2016-04-01 EP EP16716412.8A patent/EP3277814B1/en active Active
- 2016-04-01 ES ES16716412T patent/ES2808750T3/es active Active
- 2016-04-01 SI SI201630878T patent/SI3277814T1/sl unknown
- 2016-04-01 LT LTEP16716412.8T patent/LT3277814T/lt unknown
- 2016-04-01 RS RS20200929A patent/RS60762B1/sr unknown
- 2016-04-01 CN CN202211430603.6A patent/CN116004624A/zh active Pending
-
2017
- 2017-09-06 US US15/697,120 patent/US10435688B2/en active Active
-
2019
- 2019-01-31 US US16/263,200 patent/US10774327B2/en active Active
-
2020
- 2020-03-06 US US16/811,580 patent/US11230713B2/en active Active
- 2020-08-24 CY CY20201100791T patent/CY1123289T1/el unknown
-
2021
- 2021-11-29 US US17/536,647 patent/US20220251554A1/en not_active Abandoned
-
2022
- 2022-01-12 JP JP2022003057A patent/JP2022064900A/ja active Pending
- 2022-01-12 JP JP2022003059A patent/JP7504482B2/ja active Active
- 2022-06-30 AU AU2022204719A patent/AU2022204719A1/en not_active Abandoned
- 2022-06-30 AU AU2022204722A patent/AU2022204722A1/en not_active Abandoned
-
2023
- 2023-12-07 JP JP2023206985A patent/JP2024028909A/ja active Pending
-
2024
- 2024-06-05 JP JP2024091478A patent/JP2024152725A/ja not_active Revoked
-
2025
- 2025-06-20 AU AU2025204686A patent/AU2025204686A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2018516091A5 (https=) | ||
| JP2022525208A5 (https=) | ||
| US10538763B2 (en) | Compounds and methods for modulation of DUX4 | |
| JP2022528487A5 (https=) | ||
| EP3484524B1 (en) | Compounds and methods for modulation of smn2 | |
| CN105452461B (zh) | 生长激素受体的调节剂 | |
| JP2021533762A5 (https=) | ||
| EP3724206B1 (en) | Conjugated antisense compounds and their use | |
| KR20200131287A (ko) | 마이크로-rna 22의 억제제 | |
| US20210315918A1 (en) | Compounds and Methods for Modulation of Transcript Processing | |
| WO2021157730A1 (ja) | 核酸医薬とその使用 | |
| KR20250120382A (ko) | 유전자 침묵 기술로서 산재된 데옥시리보뉴클레오타이드를 갖는 짧은 이중체 rna 및 이의 용도 | |
| TW202309286A (zh) | 作為新穎基因靜默技術的非對稱短雙股ｄｎａ及其應用 | |
| TW202313977A (zh) | 作為新穎基因靜默技術的短雙股ｄｎａ及其應用 | |
| JP6934695B2 (ja) | 核酸医薬とその使用 | |
| JP2024519204A (ja) | 複数の標的を介して疼痛を処置するための治療組成物 | |
| CN116670276A (zh) | 用于通过多个靶标治疗疼痛的治疗组合物 | |
| KR102138495B1 (ko) | 이중 가닥의 올리고뉴클레오티드 및 그 제조 방법 | |
| Riasi et al. | Gene Silencing Method Based on DNA. | |
| HK40129500A (en) | Translation enhancing nucleic acid compounds: aso coupled translation - upregulation 1 (act-up1) and uses thereof | |
| EP4671372A2 (en) | Translation-enhancing nucleic acid compounds: Upward regulation 1 coupled with ASO (ACT-UP1) and their uses | |
| KR20240110595A (ko) | 안정화된 rna 제제 | |
| WO2025165891A1 (en) | Translation enhancing nucleic acid compounds: aso coupled translation - upregulation 1 (act-up1) and uses thereof | |
| KR20250116748A (ko) | 유전자 침묵 기술로서 산재된 데옥시리보뉴클레오타이드를 갖는 비대칭 짧은 이중체 rna 및 이의 용도 | |
| WO2023159138A2 (en) | Use of dinucleotide repeat rnas to treat cancer |