JPWO2020077412A5 - - Google Patents
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- Publication number
- JPWO2020077412A5 JPWO2020077412A5 JP2021546400A JP2021546400A JPWO2020077412A5 JP WO2020077412 A5 JPWO2020077412 A5 JP WO2020077412A5 JP 2021546400 A JP2021546400 A JP 2021546400A JP 2021546400 A JP2021546400 A JP 2021546400A JP WO2020077412 A5 JPWO2020077412 A5 JP WO2020077412A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- set forth
- effector
- sequence set
- shmir
- 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
Links
- 239000012636 effector Substances 0.000 claims description 189
- 230000000295 complement effect Effects 0.000 claims description 102
- 108020004707 nucleic acids Proteins 0.000 claims description 101
- 102000039446 nucleic acids Human genes 0.000 claims description 101
- 150000007523 nucleic acids Chemical class 0.000 claims description 101
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 94
- 101000609211 Homo sapiens Polyadenylate-binding protein 2 Proteins 0.000 claims description 41
- 102100039427 Polyadenylate-binding protein 2 Human genes 0.000 claims description 38
- 238000000034 method Methods 0.000 claims description 36
- 239000002679 microRNA Substances 0.000 claims description 29
- 201000009110 Oculopharyngeal muscular dystrophy Diseases 0.000 claims description 17
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 15
- 210000003205 muscle Anatomy 0.000 claims description 14
- 239000013604 expression vector Substances 0.000 claims description 13
- 239000013598 vector Substances 0.000 claims description 12
- 239000000203 mixture Substances 0.000 claims description 9
- 108020005065 3' Flanking Region Proteins 0.000 claims description 8
- 108020005029 5' Flanking Region Proteins 0.000 claims description 8
- 210000001184 pharyngeal muscle Anatomy 0.000 claims description 8
- 108020004999 messenger RNA Proteins 0.000 claims description 7
- 238000002347 injection Methods 0.000 claims description 5
- 239000007924 injection Substances 0.000 claims description 5
- -1 minicircle Substances 0.000 claims description 5
- 108700011259 MicroRNAs Proteins 0.000 claims description 4
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 3
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 3
- 102000051773 human PABPN1 Human genes 0.000 claims description 3
- 239000002773 nucleotide Substances 0.000 claims description 3
- 125000003729 nucleotide group Chemical group 0.000 claims description 3
- 239000013612 plasmid Substances 0.000 claims description 3
- 230000001177 retroviral effect Effects 0.000 claims description 3
- 230000009747 swallowing Effects 0.000 claims description 3
- 238000011144 upstream manufacturing Methods 0.000 claims description 3
- 239000013603 viral vector Substances 0.000 claims description 3
- 230000003612 virological effect Effects 0.000 claims description 3
- 239000003937 drug carrier Substances 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 17
- 238000012228 RNA interference-mediated gene silencing Methods 0.000 claims 2
- 230000009368 gene silencing by RNA Effects 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 1
- 210000003800 pharynx Anatomy 0.000 claims 1
- 238000011321 prophylaxis Methods 0.000 claims 1
- 238000011282 treatment Methods 0.000 claims 1
- 230000002401 inhibitory effect Effects 0.000 description 1
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862747089P | 2018-10-17 | 2018-10-17 | |
| US62/747,089 | 2018-10-17 | ||
| PCT/AU2019/051134 WO2020077412A1 (en) | 2018-10-17 | 2019-10-17 | Methods for treating oculopharyngeal muscular dystrophy (opmd) |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022508835A JP2022508835A (ja) | 2022-01-19 |
| JPWO2020077412A5 true JPWO2020077412A5 (enExample) | 2022-10-24 |
Family
ID=70282838
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021546400A Pending JP2022508835A (ja) | 2018-10-17 | 2019-10-17 | 眼咽頭筋ジストロフィー（ｏｐｍｄ）を治療するための方法 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20220106594A1 (enExample) |
| EP (1) | EP3866859A4 (enExample) |
| JP (1) | JP2022508835A (enExample) |
| KR (1) | KR20210081361A (enExample) |
| CN (1) | CN112867511A (enExample) |
| AU (1) | AU2019362925A1 (enExample) |
| BR (1) | BR112021007256A2 (enExample) |
| CA (1) | CA3114945A1 (enExample) |
| IL (1) | IL282323A (enExample) |
| MX (1) | MX2021004225A (enExample) |
| SG (1) | SG11202102185RA (enExample) |
| WO (1) | WO2020077412A1 (enExample) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN115838725B (zh) * | 2022-12-30 | 2023-09-08 | 广州派真生物技术有限公司 | 在哺乳动物心脏中特异性启动基因的启动子序列及其应用 |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2014117050A2 (en) * | 2013-01-26 | 2014-07-31 | Mirimus, Inc. | Modified mirna as a scaffold for shrna |
| KR102353847B1 (ko) * | 2016-04-14 | 2022-01-21 | 베니텍 바이오파마 리미티드 | 안구인두 근이영양증(opmd)의 치료용 시약 및 이의 용도 |
| WO2018107228A1 (en) * | 2016-12-14 | 2018-06-21 | Benitec Biopharma Limited | Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof |
| EP3931317A4 (en) * | 2019-02-28 | 2023-01-04 | Benitec IP Holdings Inc. | COMPOSITIONS AND METHODS FOR TREATMENT OF OCULOPHARYNGEAL MUSCULAR DYSTROPHY (OPMD) |
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2019
- 2019-10-17 JP JP2021546400A patent/JP2022508835A/ja active Pending
- 2019-10-17 BR BR112021007256-0A patent/BR112021007256A2/pt not_active IP Right Cessation
- 2019-10-17 SG SG11202102185RA patent/SG11202102185RA/en unknown
- 2019-10-17 CN CN201980066632.9A patent/CN112867511A/zh active Pending
- 2019-10-17 AU AU2019362925A patent/AU2019362925A1/en not_active Abandoned
- 2019-10-17 WO PCT/AU2019/051134 patent/WO2020077412A1/en not_active Ceased
- 2019-10-17 KR KR1020217014239A patent/KR20210081361A/ko not_active Withdrawn
- 2019-10-17 EP EP19872588.9A patent/EP3866859A4/en not_active Withdrawn
- 2019-10-17 CA CA3114945A patent/CA3114945A1/en active Pending
- 2019-10-17 MX MX2021004225A patent/MX2021004225A/es unknown
- 2019-10-17 US US17/285,569 patent/US20220106594A1/en not_active Abandoned
-
2021
- 2021-04-13 IL IL282323A patent/IL282323A/en unknown