JP2021534814A5 - - Google Patents
Info
- Publication number
- JP2021534814A5 JP2021534814A5 JP2021529252A JP2021529252A JP2021534814A5 JP 2021534814 A5 JP2021534814 A5 JP 2021534814A5 JP 2021529252 A JP2021529252 A JP 2021529252A JP 2021529252 A JP2021529252 A JP 2021529252A JP 2021534814 A5 JP2021534814 A5 JP 2021534814A5
- Authority
- JP
- Japan
- Prior art keywords
- promoter
- rnai
- nucleic acid
- aav
- seq
- 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
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024115452A JP2024156715A (ja) | 2018-08-03 | 2024-07-19 | アルファ－シヌクレインに対するバリアントＲＮＡｉ |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862714616P | 2018-08-03 | 2018-08-03 | |
| US62/714,616 | 2018-08-03 | ||
| PCT/US2019/044924 WO2020028816A1 (en) | 2018-08-03 | 2019-08-02 | Variant rnai against alpha-synuclein |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024115452A Division JP2024156715A (ja) | 2018-08-03 | 2024-07-19 | アルファ－シヌクレインに対するバリアントＲＮＡｉ |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021534814A JP2021534814A (ja) | 2021-12-16 |
| JP2021534814A5 true JP2021534814A5 (enExample) | 2022-08-04 |
Family
ID=67587976
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021529252A Pending JP2021534814A (ja) | 2018-08-03 | 2019-08-02 | アルファ−シヌクレインに対するバリアントＲＮＡｉ |
| JP2024115452A Pending JP2024156715A (ja) | 2018-08-03 | 2024-07-19 | アルファ－シヌクレインに対するバリアントＲＮＡｉ |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024115452A Pending JP2024156715A (ja) | 2018-08-03 | 2024-07-19 | アルファ－シヌクレインに対するバリアントＲＮＡｉ |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US12365895B2 (enExample) |
| EP (2) | EP3830265A1 (enExample) |
| JP (2) | JP2021534814A (enExample) |
| KR (1) | KR102931619B1 (enExample) |
| CN (1) | CN112805382A (enExample) |
| AR (1) | AR114540A1 (enExample) |
| AU (1) | AU2019314529B2 (enExample) |
| BR (1) | BR112021001980A2 (enExample) |
| CA (1) | CA3108526A1 (enExample) |
| CO (1) | CO2021002637A2 (enExample) |
| IL (1) | IL280581B1 (enExample) |
| MX (1) | MX2021001395A (enExample) |
| PH (1) | PH12021550254A1 (enExample) |
| SG (1) | SG11202101072UA (enExample) |
| TW (2) | TW202509215A (enExample) |
| WO (1) | WO2020028816A1 (enExample) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX2020003095A (es) | 2017-09-22 | 2020-10-15 | Genzyme Corp | Arni variante. |
| TW202509215A (zh) | 2018-08-03 | 2025-03-01 | 美商健臻公司 | 針對α-突觸核蛋白的變體RNAi |
| JP2023518268A (ja) * | 2020-03-18 | 2023-04-28 | ユニバーシティー オブ マサチューセッツ | Ｓｎｃａ調節のためのオリゴヌクレオチド |
| WO2022072447A1 (en) * | 2020-10-01 | 2022-04-07 | Alnylam Pharmaceuticals, Inc. | Snca irna compositions and methods of use thereof for treating or preventing snca-associated neurodegenerative diseases |
| JP2024523445A (ja) * | 2021-06-21 | 2024-06-28 | ユニキュアー バイオファーマ ビー．ブイ． | α－シヌクレインをサイレンシングするための遺伝子構築物及びその使用 |
| KR20240166577A (ko) * | 2022-04-01 | 2024-11-26 | 알닐람 파마슈티칼스 인코포레이티드 | Snca-연관된 질환을 치료하기 위한 snca-표적화 sirna 조성물 |
| KR20240169111A (ko) * | 2022-04-06 | 2024-12-02 | 젠자임 코포레이션 | Dm-1 근긴장성 이영양증에 대한 표적화된 유전자 요법 |
| JP2025531512A (ja) * | 2022-09-29 | 2025-09-19 | アタランタ セラピューティクス，インコーポレーテッド | アルファシヌクレイン核酸を標的化するｓｉＲＮＡ組成物及び方法 |
| WO2024211480A2 (en) * | 2023-04-05 | 2024-10-10 | Genzyme Corporation | Targeted gene therapy for dm-1 myotonic dystrophy |
| WO2025160429A1 (en) * | 2024-01-26 | 2025-07-31 | Genzyme Corporation | Artificial micrornas targeting snca |
| TW202529779A (zh) * | 2024-01-30 | 2025-08-01 | 大陸商上海舶望製藥有限公司 | 用於抑制突觸核蛋白α（SNCA）基因表達的組合物和方法 |
| WO2025170027A1 (ja) | 2024-02-08 | 2025-08-14 | テルモ株式会社 | オリゴヌクレオチド |
Family Cites Families (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2264482A1 (en) | 1996-09-06 | 1998-03-12 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| US6995006B2 (en) | 1997-09-05 | 2006-02-07 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6596535B1 (en) | 1999-08-09 | 2003-07-22 | Targeted Genetics Corporation | Metabolically activated recombinant viral vectors and methods for the preparation and use |
| US7465583B2 (en) | 2000-06-01 | 2008-12-16 | The University Of North Carolina At Chapel Hill | Duplexed parvovirus vectors |
| US20050137155A1 (en) * | 2001-05-18 | 2005-06-23 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of Parkinson disease using short interfering nucleic acid (siNA) |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| CN103589692B (zh) | 2001-11-13 | 2018-07-24 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
| US8283151B2 (en) | 2005-04-29 | 2012-10-09 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| DK2164967T3 (en) | 2007-05-31 | 2015-10-19 | Univ Iowa Res Found | Reduction of off-target rna interferenstoksicitet |
| GB0816778D0 (en) | 2008-09-12 | 2008-10-22 | Isis Innovation | Gene silencing |
| DK2561073T3 (en) | 2010-04-23 | 2016-12-12 | Univ Massachusetts | Aav vectors targeted to central nervous system and methods of use thereof |
| WO2012027713A2 (en) * | 2010-08-26 | 2012-03-01 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibition of snca |
| US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| EP3521451A1 (en) * | 2010-11-17 | 2019-08-07 | Ionis Pharmaceuticals, Inc. | Modulation of alpha synuclein expression |
| CN103502458B (zh) | 2011-02-17 | 2016-11-16 | 宾夕法尼亚大学托管会 | 用于改变组织特异性和改善aav9-介导的基因转移的组合物和方法 |
| CA2865011C (en) | 2012-02-29 | 2021-06-15 | Sangamo Biosciences, Inc. | Methods and compositions for treating huntington's disease |
| LT3137497T (lt) | 2014-05-02 | 2021-07-26 | Genzyme Corporation | Aav vektoriai, skirti tinklainės ir cns genų terapijai |
| CA3255657A1 (en) * | 2015-02-10 | 2025-12-01 | Genzyme Corporation | VARIANT RNAi |
| TW202509215A (zh) | 2018-08-03 | 2025-03-01 | 美商健臻公司 | 針對α-突觸核蛋白的變體RNAi |
-
2019
- 2019-08-02 TW TW113117876A patent/TW202509215A/zh unknown
- 2019-08-02 EP EP19752626.2A patent/EP3830265A1/en active Pending
- 2019-08-02 BR BR112021001980-4A patent/BR112021001980A2/pt not_active Application Discontinuation
- 2019-08-02 IL IL280581A patent/IL280581B1/en unknown
- 2019-08-02 JP JP2021529252A patent/JP2021534814A/ja active Pending
- 2019-08-02 PH PH1/2021/550254A patent/PH12021550254A1/en unknown
- 2019-08-02 EP EP26170091.8A patent/EP4763987A3/en active Pending
- 2019-08-02 CN CN201980063972.6A patent/CN112805382A/zh active Pending
- 2019-08-02 CA CA3108526A patent/CA3108526A1/en active Pending
- 2019-08-02 TW TW108127564A patent/TW202022116A/zh unknown
- 2019-08-02 AU AU2019314529A patent/AU2019314529B2/en active Active
- 2019-08-02 MX MX2021001395A patent/MX2021001395A/es unknown
- 2019-08-02 KR KR1020217006255A patent/KR102931619B1/ko active Active
- 2019-08-02 US US17/265,489 patent/US12365895B2/en active Active
- 2019-08-02 AR ARP190102204A patent/AR114540A1/es unknown
- 2019-08-02 WO PCT/US2019/044924 patent/WO2020028816A1/en not_active Ceased
- 2019-08-02 SG SG11202101072UA patent/SG11202101072UA/en unknown
-
2021
- 2021-02-26 CO CONC2021/0002637A patent/CO2021002637A2/es unknown
-
2024
- 2024-07-19 JP JP2024115452A patent/JP2024156715A/ja active Pending
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