JP2018506304A5 - - Google Patents
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- JP2018506304A5 JP2018506304A5 JP2017560478A JP2017560478A JP2018506304A5 JP 2018506304 A5 JP2018506304 A5 JP 2018506304A5 JP 2017560478 A JP2017560478 A JP 2017560478A JP 2017560478 A JP2017560478 A JP 2017560478A JP 2018506304 A5 JP2018506304 A5 JP 2018506304A5
- Authority
- JP
- Japan
- Prior art keywords
- rnai
- vector
- guide region
- sequence
- strand
- 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.)
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- 230000009368 gene silencing by RNA Effects 0.000 claims 46
- 108091030071 RNAI Proteins 0.000 claims 45
- 239000013598 vector Substances 0.000 claims 35
- 239000002245 particle Substances 0.000 claims 33
- 150000007523 nucleic acids Chemical group 0.000 claims 25
- 241000702421 Dependoparvovirus Species 0.000 claims 18
- 241000124008 Mammalia Species 0.000 claims 13
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 13
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 12
- 108020004707 nucleic acids Proteins 0.000 claims 12
- 102000039446 nucleic acids Human genes 0.000 claims 12
- 239000002773 nucleotide Substances 0.000 claims 11
- 125000003729 nucleotide group Chemical group 0.000 claims 11
- 241000700605 Viruses Species 0.000 claims 9
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 8
- 230000008488 polyadenylation Effects 0.000 claims 8
- 210000000234 capsid Anatomy 0.000 claims 7
- 230000002401 inhibitory effect Effects 0.000 claims 7
- 208000023105 Huntington disease Diseases 0.000 claims 6
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 6
- 229920001184 polypeptide Polymers 0.000 claims 6
- 108090000765 processed proteins & peptides Proteins 0.000 claims 6
- 102000004196 processed proteins & peptides Human genes 0.000 claims 6
- 230000003612 virological effect Effects 0.000 claims 6
- 239000013608 rAAV vector Substances 0.000 claims 5
- 241000711975 Vesicular stomatitis virus Species 0.000 claims 4
- 208000035475 disorder Diseases 0.000 claims 4
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 3
- 241000283690 Bos taurus Species 0.000 claims 3
- 241000701022 Cytomegalovirus Species 0.000 claims 3
- 241000712899 Lymphocytic choriomeningitis mammarenavirus Species 0.000 claims 3
- 241000829100 Macaca mulatta polyomavirus 1 Species 0.000 claims 3
- 108700011259 MicroRNAs Proteins 0.000 claims 3
- 241000714474 Rous sarcoma virus Species 0.000 claims 3
- 108010006025 bovine growth hormone Proteins 0.000 claims 3
- 210000004027 cell Anatomy 0.000 claims 3
- 239000002679 microRNA Substances 0.000 claims 3
- 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 2
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 2
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 2
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 2
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 2
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 2
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 2
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 2
- 241000649047 Adeno-associated virus 12 Species 0.000 claims 2
- 241001115402 Ebolavirus Species 0.000 claims 2
- 206010066919 Epidemic polyarthritis Diseases 0.000 claims 2
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 2
- 108010043121 Green Fluorescent Proteins Proteins 0.000 claims 2
- 102000004144 Green Fluorescent Proteins Human genes 0.000 claims 2
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 2
- 208000015439 Lysosomal storage disease Diseases 0.000 claims 2
- 241001115401 Marburgvirus Species 0.000 claims 2
- 208000001089 Multiple system atrophy Diseases 0.000 claims 2
- 241000711798 Rabies lyssavirus Species 0.000 claims 2
- 241000710942 Ross River virus Species 0.000 claims 2
- 108091027967 Small hairpin RNA Proteins 0.000 claims 2
- 238000009825 accumulation Methods 0.000 claims 2
- 210000004227 basal ganglia Anatomy 0.000 claims 2
- 210000003169 central nervous system Anatomy 0.000 claims 2
- 210000003710 cerebral cortex Anatomy 0.000 claims 2
- 230000000295 complement effect Effects 0.000 claims 2
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- 201000010099 disease Diseases 0.000 claims 2
- 239000005090 green fluorescent protein Substances 0.000 claims 2
- 230000001697 htt accumulation Effects 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- 201000002212 progressive supranuclear palsy Diseases 0.000 claims 2
- 239000004055 small Interfering RNA Substances 0.000 claims 2
- 208000024827 Alzheimer disease Diseases 0.000 claims 1
- 101710081722 Antitrypsin Proteins 0.000 claims 1
- 208000003174 Brain Neoplasms Diseases 0.000 claims 1
- 241000282465 Canis Species 0.000 claims 1
- 206010012289 Dementia Diseases 0.000 claims 1
- 102100021519 Hemoglobin subunit beta Human genes 0.000 claims 1
- 108091005904 Hemoglobin subunit beta Proteins 0.000 claims 1
- 101000933465 Homo sapiens Beta-glucuronidase Proteins 0.000 claims 1
- 241000713666 Lentivirus Species 0.000 claims 1
- 201000009906 Meningitis Diseases 0.000 claims 1
- 108091033773 MiR-155 Proteins 0.000 claims 1
- 241000713869 Moloney murine leukemia virus Species 0.000 claims 1
- 241000283973 Oryctolagus cuniculus Species 0.000 claims 1
- 208000018737 Parkinson disease Diseases 0.000 claims 1
- 241001494479 Pecora Species 0.000 claims 1
- 102000010292 Peptide Elongation Factor 1 Human genes 0.000 claims 1
- 108010077524 Peptide Elongation Factor 1 Proteins 0.000 claims 1
- 108010071690 Prealbumin Proteins 0.000 claims 1
- 238000012228 RNA interference-mediated gene silencing Methods 0.000 claims 1
- 208000006011 Stroke Diseases 0.000 claims 1
- 108010017842 Telomerase Proteins 0.000 claims 1
- 239000004098 Tetracycline Substances 0.000 claims 1
- 108010022394 Threonine synthase Proteins 0.000 claims 1
- 102000009190 Transthyretin Human genes 0.000 claims 1
- 239000002253 acid Substances 0.000 claims 1
- 230000001475 anti-trypsic effect Effects 0.000 claims 1
- 208000015114 central nervous system disease Diseases 0.000 claims 1
- 210000003161 choroid Anatomy 0.000 claims 1
- 210000000349 chromosome Anatomy 0.000 claims 1
- 230000037430 deletion Effects 0.000 claims 1
- 238000012217 deletion Methods 0.000 claims 1
- 102000004419 dihydrofolate reductase Human genes 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 239000003623 enhancer Substances 0.000 claims 1
- 206010015037 epilepsy Diseases 0.000 claims 1
- 230000030279 gene silencing Effects 0.000 claims 1
- 238000012226 gene silencing method Methods 0.000 claims 1
- AWUCVROLDVIAJX-UHFFFAOYSA-N glycerol 1-phosphate Chemical compound OCC(O)COP(O)(O)=O AWUCVROLDVIAJX-UHFFFAOYSA-N 0.000 claims 1
- 230000001939 inductive effect Effects 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 210000004698 lymphocyte Anatomy 0.000 claims 1
- 210000005171 mammalian brain Anatomy 0.000 claims 1
- 210000004962 mammalian cell Anatomy 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 239000013646 rAAV2 vector Substances 0.000 claims 1
- 230000001177 retroviral effect Effects 0.000 claims 1
- 230000001568 sexual effect Effects 0.000 claims 1
- 230000002123 temporal effect Effects 0.000 claims 1
- 229960002180 tetracycline Drugs 0.000 claims 1
- 229930101283 tetracycline Natural products 0.000 claims 1
- 235000019364 tetracycline Nutrition 0.000 claims 1
- 150000003522 tetracyclines Chemical class 0.000 claims 1
- 230000001988 toxicity Effects 0.000 claims 1
- 231100000419 toxicity Toxicity 0.000 claims 1
- 239000002753 trypsin inhibitor Substances 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021078464A JP7526710B2 (ja) | 2015-02-10 | 2021-05-06 | バリアントＲＮＡｉ |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562114578P | 2015-02-10 | 2015-02-10 | |
| US62/114,578 | 2015-02-10 | ||
| PCT/US2016/017207 WO2016130589A2 (en) | 2015-02-10 | 2016-02-09 | VARIANT RNAi |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021078464A Division JP7526710B2 (ja) | 2015-02-10 | 2021-05-06 | バリアントＲＮＡｉ |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2018506304A JP2018506304A (ja) | 2018-03-08 |
| JP2018506304A5 true JP2018506304A5 (https=) | 2019-03-22 |
Family
ID=56614905
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017560478A Pending JP2018506304A (ja) | 2015-02-10 | 2016-02-09 | バリアントＲＮＡｉ |
| JP2021078464A Active JP7526710B2 (ja) | 2015-02-10 | 2021-05-06 | バリアントＲＮＡｉ |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021078464A Active JP7526710B2 (ja) | 2015-02-10 | 2021-05-06 | バリアントＲＮＡｉ |
Country Status (25)
| Country | Link |
|---|---|
| US (4) | US10450563B2 (https=) |
| EP (1) | EP3256588A2 (https=) |
| JP (2) | JP2018506304A (https=) |
| KR (1) | KR102670852B1 (https=) |
| CN (1) | CN107438671B (https=) |
| AR (1) | AR103646A1 (https=) |
| AU (3) | AU2016219396B2 (https=) |
| CA (2) | CA2976075C (https=) |
| CL (1) | CL2017002027A1 (https=) |
| CO (1) | CO2017009083A2 (https=) |
| CR (1) | CR20170406A (https=) |
| EA (1) | EA201791805A1 (https=) |
| EC (1) | ECSP17059343A (https=) |
| HK (1) | HK1247641A1 (https=) |
| IL (1) | IL253893B (https=) |
| MA (1) | MA40819B1 (https=) |
| MX (2) | MX2017010369A (https=) |
| MY (2) | MY194175A (https=) |
| PE (1) | PE20171382A1 (https=) |
| PH (1) | PH12017501432A1 (https=) |
| SG (3) | SG10201907396XA (https=) |
| TN (1) | TN2017000354A1 (https=) |
| TW (1) | TWI781079B (https=) |
| UY (1) | UY36554A (https=) |
| WO (1) | WO2016130589A2 (https=) |
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| 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 |
| RU2612521C2 (ru) | 2009-07-06 | 2017-03-09 | Онтории, Инк. | Новые пролекарства нуклеиновых кислот и способы их применения |
| 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 |
| KR102213609B1 (ko) | 2012-07-13 | 2021-02-08 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
| PL2872485T3 (pl) | 2012-07-13 | 2021-05-31 | Wave Life Sciences Ltd. | Asymetryczna grupa pomocnicza |
| 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 |
| 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) |
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| 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 |
| JPWO2017068791A1 (ja) * | 2015-10-23 | 2018-08-09 | レナセラピューティクス株式会社 | 少なくとも１つのバルジ構造を有する核酸複合体 |
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| 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 |
| 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 |
| 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 |
| CN111356763B (zh) | 2017-09-22 | 2024-03-12 | 建新公司 | 变体RNAi |
| US20200377887A1 (en) * | 2017-09-22 | 2020-12-03 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| KR20200119782A (ko) * | 2017-12-06 | 2020-10-20 | 오비드 테라퓨틱스 인크. | 발작 장애 치료 시 mir101 또는 mir128의 용도 |
| EP4169576A1 (en) * | 2018-03-23 | 2023-04-26 | University of Massachusetts | Gene therapeutics for treating bone disorders |
| KR20210019996A (ko) | 2018-05-15 | 2021-02-23 | 보이저 테라퓨틱스, 인크. | 파킨슨병의 치료를 위한 조성물 및 방법 |
| CN112770812A (zh) | 2018-07-24 | 2021-05-07 | 沃雅戈治疗公司 | 产生基因治疗制剂的系统和方法 |
| KR102931619B1 (ko) * | 2018-08-03 | 2026-02-26 | 젠자임 코포레이션 | 알파-시뉴클레인에 대한 변이형 RNAi |
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| AU2019357039B2 (en) * | 2018-10-12 | 2025-10-09 | Genzyme Corporation | Generation of improved human pah for treatment of severe PKU by liver-directed gene replacement therapy |
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| CA3125310A1 (en) * | 2019-01-09 | 2020-07-16 | Universidade De Coimbra | Double stranded rna and uses thereof |
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| US12365894B2 (en) | 2019-09-16 | 2025-07-22 | University Of Massachusetts | Branched lipid conjugates of siRNA for specific tissue delivery |
| 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 |
| TW202325850A (zh) | 2021-11-29 | 2023-07-01 | 大陸商上海瑞宏迪醫藥有限公司 | Aadc、gdnf多核苷酸及其用於治療帕金森病 |
| AU2023220237A1 (en) | 2022-02-21 | 2024-08-15 | Shanghai Regenelead Therapies Co., Ltd | Vegf-binding molecule and pharmaceutical use thereof |
| WO2024187106A2 (en) * | 2023-03-09 | 2024-09-12 | Leal Therapeutics, Inc. | Compositions and methods for modulating grin2a |
| TW202508610A (zh) | 2023-08-23 | 2025-03-01 | 大陸商上海瑞宏迪醫藥有限公司 | 醫藥組成物及其用途 |
| KR20250129583A (ko) * | 2024-02-21 | 2025-08-29 | 서울대학교산학협력단 | Drosha 절단을 촉진시키는 구조적 모티프를 포함하는 rna 간섭 유도 핵산 분자의 변형 전구체 |
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-
2016
- 2016-02-09 CR CR20170406A patent/CR20170406A/es unknown
- 2016-02-09 SG SG10201907396XA patent/SG10201907396XA/en unknown
- 2016-02-09 CN CN201680020699.5A patent/CN107438671B/zh active Active
- 2016-02-09 CA CA2976075A patent/CA2976075C/en active Active
- 2016-02-09 MY MYPI2020002819A patent/MY194175A/en unknown
- 2016-02-09 PE PE2017001371A patent/PE20171382A1/es unknown
- 2016-02-09 MX MX2017010369A patent/MX2017010369A/es unknown
- 2016-02-09 MY MYPI2017702831A patent/MY181458A/en unknown
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