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 (https=) | 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 (https=) |
| EP (1) | EP3830265A1 (https=) |
| JP (2) | JP2021534814A (https=) |
| KR (1) | KR102931619B1 (https=) |
| CN (1) | CN112805382A (https=) |
| AR (1) | AR114540A1 (https=) |
| AU (1) | AU2019314529B2 (https=) |
| BR (1) | BR112021001980A2 (https=) |
| CA (1) | CA3108526A1 (https=) |
| CO (1) | CO2021002637A2 (https=) |
| IL (1) | IL280581A (https=) |
| MX (1) | MX2021001395A (https=) |
| PH (1) | PH12021550254A1 (https=) |
| SG (1) | SG11202101072UA (https=) |
| TW (2) | TW202509215A (https=) |
| WO (1) | WO2020028816A1 (https=) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN111356763B (zh) * | 2017-09-22 | 2024-03-12 | 建新公司 | 变体RNAi |
| KR102931619B1 (ko) | 2018-08-03 | 2026-02-26 | 젠자임 코포레이션 | 알파-시뉴클레인에 대한 변이형 RNAi |
| US20210363524A1 (en) * | 2020-03-18 | 2021-11-25 | University Of Massachusetts | Oligonucleotides for snca modulation |
| EP4221838A4 (en) * | 2020-10-01 | 2025-04-16 | Alnylam Pharmaceuticals, Inc. | Snca irna compositions and methods of use thereof for treating or preventing snca-associated neurodegenerative diseases |
| WO2022268835A1 (en) * | 2021-06-21 | 2022-12-29 | Uniqure Biopharma B.V. | Gene constructs for silencing alpha-synuclein and uses thereof |
| WO2023196862A1 (en) * | 2022-04-06 | 2023-10-12 | Genzyme Corporation | Targeted gene therapy for dm-1 myotonic dystrophy |
| US20260103703A1 (en) * | 2022-09-29 | 2026-04-16 | Atalanta Therapeutics, Inc. | siRNA COMPOSITIONS AND METHODS TARGETING ALPHA-SYNUCLEIN NUCLEIC ACIDS |
| US20240384271A1 (en) * | 2023-04-05 | 2024-11-21 | Genzyme Corporation | Targeted gene therapy for dm-1 myotonic dystrophy |
| WO2025160429A1 (en) * | 2024-01-26 | 2025-07-31 | Genzyme Corporation | Artificial micrornas targeting snca |
| WO2025162214A1 (en) * | 2024-01-30 | 2025-08-07 | Shanghai Argo Biopharmaceutical Co., Ltd. | Compositions and methods for inhibiting expression of synuclein alpha (snca) gene |
Family Cites Families (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0931158A1 (en) | 1996-09-06 | 1999-07-28 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| CA2379166C (en) | 1999-08-09 | 2013-03-26 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs |
| ATE318923T1 (de) | 2000-06-01 | 2006-03-15 | Univ North Carolina | Doppelsträngige parvovirus-vektoren |
| 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 |
| MX359371B (es) | 2001-11-13 | 2018-09-25 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| WO2006119432A2 (en) | 2005-04-29 | 2006-11-09 | The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & 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 |
| EP2561073B1 (en) | 2010-04-23 | 2016-08-24 | University of Massachusetts | Cns targeting aav vectors 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 |
| CA2817960C (en) | 2010-11-17 | 2020-06-09 | Ionis Pharmaceuticals, Inc. | Modulation of alpha synuclein expression |
| TR201908020T4 (tr) | 2011-02-17 | 2019-06-21 | Univ Pennsylvania | Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler. |
| WO2013130824A1 (en) | 2012-02-29 | 2013-09-06 | 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 |
| CR20170406A (es) | 2015-02-10 | 2017-11-14 | Genzyme Corp | Arni variante |
| KR102931619B1 (ko) | 2018-08-03 | 2026-02-26 | 젠자임 코포레이션 | 알파-시뉴클레인에 대한 변이형 RNAi |
-
2019
- 2019-08-02 KR KR1020217006255A patent/KR102931619B1/ko active Active
- 2019-08-02 BR BR112021001980-4A patent/BR112021001980A2/pt unknown
- 2019-08-02 JP JP2021529252A patent/JP2021534814A/ja active Pending
- 2019-08-02 SG SG11202101072UA patent/SG11202101072UA/en unknown
- 2019-08-02 TW TW113117876A patent/TW202509215A/zh unknown
- 2019-08-02 MX MX2021001395A patent/MX2021001395A/es unknown
- 2019-08-02 WO PCT/US2019/044924 patent/WO2020028816A1/en not_active Ceased
- 2019-08-02 AU AU2019314529A patent/AU2019314529B2/en active Active
- 2019-08-02 US US17/265,489 patent/US12365895B2/en active Active
- 2019-08-02 CA CA3108526A patent/CA3108526A1/en active Pending
- 2019-08-02 TW TW108127564A patent/TW202022116A/zh unknown
- 2019-08-02 EP EP19752626.2A patent/EP3830265A1/en active Pending
- 2019-08-02 CN CN201980063972.6A patent/CN112805382A/zh active Pending
- 2019-08-02 PH PH1/2021/550254A patent/PH12021550254A1/en unknown
- 2019-08-02 AR ARP190102204A patent/AR114540A1/es unknown
-
2021
- 2021-02-02 IL IL280581A patent/IL280581A/en unknown
- 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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