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.)
- Pending
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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
- 230000007850 degeneration Effects 0.000 claims 2
- 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 (enExample) | 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 (enExample) |
| EP (1) | EP3256588A2 (enExample) |
| JP (2) | JP2018506304A (enExample) |
| KR (1) | KR102670852B1 (enExample) |
| CN (1) | CN107438671B (enExample) |
| AR (1) | AR103646A1 (enExample) |
| AU (3) | AU2016219396B2 (enExample) |
| CA (2) | CA3255657A1 (enExample) |
| CL (1) | CL2017002027A1 (enExample) |
| CO (1) | CO2017009083A2 (enExample) |
| CR (1) | CR20170406A (enExample) |
| EA (1) | EA201791805A1 (enExample) |
| EC (1) | ECSP17059343A (enExample) |
| HK (1) | HK1247641A1 (enExample) |
| IL (1) | IL253893B (enExample) |
| MA (1) | MA40819B1 (enExample) |
| MX (2) | MX2017010369A (enExample) |
| MY (2) | MY181458A (enExample) |
| PE (1) | PE20171382A1 (enExample) |
| PH (1) | PH12017501432A1 (enExample) |
| SG (3) | SG11201706444TA (enExample) |
| TN (1) | TN2017000354A1 (enExample) |
| TW (1) | TWI781079B (enExample) |
| UY (1) | UY36554A (enExample) |
| WO (1) | WO2016130589A2 (enExample) |
Families Citing this family (44)
| Publication number | Priority date | Publication date | Assignee | Title |
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| US9394333B2 (en) | 2008-12-02 | 2016-07-19 | Wave Life Sciences Japan | Method for the synthesis of phosphorus atom modified nucleic acids |
| MX342945B (es) | 2009-07-06 | 2016-10-18 | Ontorii Inc * | Profármacos de ácido nucleico novedosos y métodos de uso de los mismos. |
| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| KR20240148947A (ko) | 2012-07-13 | 2024-10-11 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
| SG11201500239VA (en) | 2012-07-13 | 2015-03-30 | Wave Life Sciences Japan | Asymmetric auxiliary group |
| SG11201500243WA (en) | 2012-07-13 | 2015-04-29 | Shin Nippon Biomedical Lab Ltd | Chiral nucleic acid adjuvant |
| WO2015108047A1 (ja) | 2014-01-15 | 2015-07-23 | 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 |
| WO2015108048A1 (ja) | 2014-01-15 | 2015-07-23 | 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 |
| EP3095460A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent |
| ES2917473T3 (es) | 2014-01-16 | 2022-07-08 | Wave Life Sciences Ltd | Diseño quiral |
| MX2017006217A (es) | 2014-11-14 | 2018-05-02 | Voyager Therapeutics Inc | Polinucleotidos moduladores. |
| RU2020108189A (ru) | 2014-11-14 | 2020-03-11 | Вояджер Терапьютикс, Инк. | Композиции и способы лечения бокового амиотрофического склероза (als) |
| SG11201706444TA (en) | 2015-02-10 | 2017-09-28 | Genzyme Corp | VARIANT RNAi |
| WO2016161388A1 (en) | 2015-04-03 | 2016-10-06 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| CN108025089A (zh) | 2015-07-22 | 2018-05-11 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| EP3334499A4 (en) | 2015-08-14 | 2019-04-17 | University of Massachusetts | BIOACTIVE CONJUGATES FOR THE RELEASE OF OLIGONUCLEOTIDES |
| WO2017068791A1 (ja) * | 2015-10-23 | 2017-04-27 | 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 |
| CN109477108A (zh) | 2016-05-04 | 2019-03-15 | 波涛生命科学有限公司 | 寡核苷酸组合物和其方法 |
| BR112018073472A2 (pt) | 2016-05-18 | 2019-08-27 | Voyager Therapeutics, Inc. | composições e métodos de tratamento da doença de huntington |
| AU2017267665C1 (en) | 2016-05-18 | 2023-10-05 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| CN111108198A (zh) | 2017-05-05 | 2020-05-05 | 沃雅戈治疗公司 | 治疗亨廷顿病的组合物和方法 |
| 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 |
| MY205041A (en) * | 2017-09-22 | 2024-09-29 | Genzyme Corp | Variant rnai |
| US20200377887A1 (en) * | 2017-09-22 | 2020-12-03 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| US10870855B2 (en) * | 2017-12-06 | 2020-12-22 | Ovid Therapeutics Inc. | Use of MIR101 or MIR128 in the treatment of seizure disorders |
| KR20200135433A (ko) * | 2018-03-23 | 2020-12-02 | 유니버시티 오브 매사추세츠 | 골 장애 치료를 위한 유전자 치료제 |
| AU2019268330A1 (en) | 2018-05-15 | 2020-11-26 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of Parkinson's disease |
| MX2021000810A (es) | 2018-07-24 | 2021-04-28 | Voyager Therapeutics Inc | Sistemas y metodos para producir formulaciones de terapia genetica. |
| MX2021001395A (es) * | 2018-08-03 | 2021-08-11 | Genzyme Corp | Arni variante contra alfa-sinucleína. |
| EP3840759A4 (en) | 2018-08-23 | 2022-06-01 | University Of Massachusetts | O-METHYL RICH FULLY STABILIZED OLIGONUCLEOTIDES |
| EP4461814A3 (en) * | 2018-10-12 | 2025-02-26 | Genzyme Corporation | Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy |
| EP3883954A4 (en) * | 2018-11-21 | 2022-08-10 | Stridebio, Inc. | Recombinant viral vectors and nucleic acids for producing the same |
| MX2021008331A (es) * | 2019-01-09 | 2021-08-05 | Univ De Coimbra | Arn bicatenario y usos del mismo. |
| AU2020207935A1 (en) | 2019-01-18 | 2021-08-26 | University Of Massachusetts | Dynamic pharmacokinetic-modifying anchors |
| EP3983077A4 (en) * | 2019-06-17 | 2023-12-20 | Alnylam Pharmaceuticals, Inc. | DELIVERY OF OLIGONUCLEOTIDES TO THE STRIATUM |
| 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 |
| US20210355491A1 (en) * | 2020-04-20 | 2021-11-18 | University Of Massachusetts | Oligonucleotides for msh3 modulation |
| US12534724B2 (en) | 2020-05-26 | 2026-01-27 | University Of Massachusetts | Synthetic oligonucleotides having regions of block and cluster modifications |
| EP4442830A4 (en) | 2021-11-29 | 2025-04-30 | Shanghai Regenelead Therapies Co., Ltd. | AADC/GDNF polynucleotide and use thereof in the treatment of Parkinson's disease |
| AU2023220237A1 (en) | 2022-02-21 | 2024-08-15 | Shanghai Regenelead Therapies Co., Ltd | Vegf-binding molecule and pharmaceutical use thereof |
| EP4677091A2 (en) * | 2023-03-09 | 2026-01-14 | Leal Therapeutics, Inc. | Compositions and methods for modulating grin2a |
| WO2025178406A1 (ko) * | 2024-02-21 | 2025-08-28 | 서울대학교산학협력단 | Drosha 절단을 촉진시키는 구조적 모티프를 포함하는 rna 간섭 유도 핵산 분자의 변형 전구체 |
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| SMT201900347T1 (it) | 2014-12-24 | 2019-07-11 | Uniqure Ip Bv | Soppressione del gene huntingtina indotta da rnai |
| SG11201706444TA (en) | 2015-02-10 | 2017-09-28 | Genzyme Corp | VARIANT RNAi |
| MY205041A (en) | 2017-09-22 | 2024-09-29 | Genzyme Corp | Variant rnai |
-
2016
- 2016-02-09 SG SG11201706444TA patent/SG11201706444TA/en unknown
- 2016-02-09 MY MYPI2017702831A patent/MY181458A/en unknown
- 2016-02-09 SG SG10201907396XA patent/SG10201907396XA/en unknown
- 2016-02-09 CA CA3255657A patent/CA3255657A1/en active Pending
- 2016-02-09 PE PE2017001371A patent/PE20171382A1/es unknown
- 2016-02-09 KR KR1020177025286A patent/KR102670852B1/ko active Active
- 2016-02-09 JP JP2017560478A patent/JP2018506304A/ja active Pending
- 2016-02-09 MA MA40819A patent/MA40819B1/fr unknown
- 2016-02-09 MX MX2017010369A patent/MX2017010369A/es unknown
- 2016-02-09 WO PCT/US2016/017207 patent/WO2016130589A2/en not_active Ceased
- 2016-02-09 HK HK18107116.4A patent/HK1247641A1/zh unknown
- 2016-02-09 CA CA2976075A patent/CA2976075C/en active Active
- 2016-02-09 TN TNP/2017/000354A patent/TN2017000354A1/en unknown
- 2016-02-09 CR CR20170406A patent/CR20170406A/es unknown
- 2016-02-09 CN CN201680020699.5A patent/CN107438671B/zh active Active
- 2016-02-09 EP EP16710035.3A patent/EP3256588A2/en active Pending
- 2016-02-09 EA EA201791805A patent/EA201791805A1/ru unknown
- 2016-02-09 AU AU2016219396A patent/AU2016219396B2/en active Active
- 2016-02-09 MY MYPI2020002819A patent/MY194175A/en unknown
- 2016-02-09 US US15/549,895 patent/US10450563B2/en active Active
- 2016-02-10 AR ARP160100356A patent/AR103646A1/es unknown
- 2016-02-10 UY UY0001036554A patent/UY36554A/es not_active Application Discontinuation
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2017
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2019
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