JPWO2020028816A5 - - Google Patents
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- Publication number
- JPWO2020028816A5 JPWO2020028816A5 JP2021529252A JP2021529252A JPWO2020028816A5 JP WO2020028816 A5 JPWO2020028816 A5 JP WO2020028816A5 JP 2021529252 A JP2021529252 A JP 2021529252A JP 2021529252 A JP2021529252 A JP 2021529252A JP WO2020028816 A5 JPWO2020028816 A5 JP WO2020028816A5
- Authority
- JP
- Japan
- Prior art keywords
- promoter
- rnai
- aav
- nucleic acid
- optionally
- 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
- 241000702421 Dependoparvovirus Species 0.000 claims 18
- 108091030071 RNAI Proteins 0.000 claims 16
- 230000009368 gene silencing by RNA Effects 0.000 claims 16
- 150000007523 nucleic acids Chemical class 0.000 claims 15
- 239000002245 particle Substances 0.000 claims 10
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 9
- 239000013598 vector Substances 0.000 claims 9
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 7
- 108020004707 nucleic acids Proteins 0.000 claims 6
- 102000039446 nucleic acids Human genes 0.000 claims 6
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 5
- 210000000234 capsid Anatomy 0.000 claims 5
- 230000008488 polyadenylation Effects 0.000 claims 5
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 4
- 239000002773 nucleotide Substances 0.000 claims 4
- 125000003729 nucleotide group Chemical group 0.000 claims 4
- 241000701022 Cytomegalovirus Species 0.000 claims 3
- 241000829100 Macaca mulatta polyomavirus 1 Species 0.000 claims 3
- 108700011259 MicroRNAs Proteins 0.000 claims 3
- 208000032859 Synucleinopathies Diseases 0.000 claims 3
- 239000002679 microRNA Substances 0.000 claims 3
- 230000000626 neurodegenerative effect Effects 0.000 claims 3
- 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
- 241000283690 Bos taurus Species 0.000 claims 2
- 241000283707 Capra Species 0.000 claims 2
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 2
- 101000834898 Homo sapiens Alpha-synuclein Proteins 0.000 claims 2
- 241000124008 Mammalia Species 0.000 claims 2
- 208000001089 Multiple system atrophy Diseases 0.000 claims 2
- 208000018737 Parkinson disease Diseases 0.000 claims 2
- 102100028251 Phosphoglycerate kinase 1 Human genes 0.000 claims 2
- 101710139464 Phosphoglycerate kinase 1 Proteins 0.000 claims 2
- 241000714474 Rous sarcoma virus Species 0.000 claims 2
- 108091027967 Small hairpin RNA Proteins 0.000 claims 2
- 102000003802 alpha-Synuclein Human genes 0.000 claims 2
- 108090000185 alpha-Synuclein Proteins 0.000 claims 2
- 230000002401 inhibitory effect Effects 0.000 claims 2
- 238000000034 method Methods 0.000 claims 2
- 229920001184 polypeptide Polymers 0.000 claims 2
- 108090000765 processed proteins & peptides Proteins 0.000 claims 2
- 102000004196 processed proteins & peptides Human genes 0.000 claims 2
- 239000004055 small Interfering RNA Substances 0.000 claims 2
- 230000003612 virological effect Effects 0.000 claims 2
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 1
- 102100026882 Alpha-synuclein Human genes 0.000 claims 1
- 206010012289 Dementia Diseases 0.000 claims 1
- 102100021579 Enhancer of filamentation 1 Human genes 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
- 101000898310 Homo sapiens Enhancer of filamentation 1 Proteins 0.000 claims 1
- 241000283973 Oryctolagus cuniculus 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
- 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
- 108010006025 bovine growth hormone Proteins 0.000 claims 1
- 230000000295 complement effect Effects 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
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 239000003623 enhancer Substances 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 230000035772 mutation Effects 0.000 claims 1
- 230000004770 neurodegeneration Effects 0.000 claims 1
- 208000015122 neurodegenerative disease Diseases 0.000 claims 1
- 239000013608 rAAV vector Substances 0.000 claims 1
- 239000013646 rAAV2 vector Substances 0.000 claims 1
- 230000001177 retroviral effect Effects 0.000 claims 1
- 230000000392 somatic 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
- 238000011144 upstream manufacturing Methods 0.000 claims 1
- 210000002845 virion Anatomy 0.000 claims 1
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 |
| JPWO2020028816A5 true JPWO2020028816A5 (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 (1) | EP3830265A1 (enExample) |
| JP (2) | JP2021534814A (enExample) |
| KR (1) | KR20210062627A (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) | IL280581A (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 (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3684932A1 (en) | 2017-09-22 | 2020-07-29 | Genzyme Corporation | Variant rnai |
| PH12021550254A1 (en) | 2018-08-03 | 2023-02-06 | Genzyme Corp | Variant rnai against alpha-synuclein |
| CN115777021A (zh) * | 2020-03-18 | 2023-03-10 | 马萨诸塞大学 | 用于snca调节的寡核苷酸 |
| 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 |
| EP4359525A1 (en) * | 2021-06-21 | 2024-05-01 | uniQure biopharma B.V. | Gene constructs for silencing alpha-synuclein and uses thereof |
| AU2023248463A1 (en) * | 2022-04-06 | 2024-11-28 | Genzyme Corporation | Targeted gene therapy for dm-1 myotonic dystrophy |
| 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 |
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 |
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | 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 |
| DE60039766D1 (de) | 1999-08-09 | 2008-09-18 | Targeted Genetics Corp | Terologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen |
| ES2256265T3 (es) | 2000-06-01 | 2006-07-16 | University Of North Carolina At Chapel Hill | Vectores de parvovirus duplicados. |
| 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 |
| CA2915124C (en) | 2001-11-13 | 2018-08-14 | The Trustees Of The University Of Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| 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 |
| EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid proteins |
| PT2164967E (pt) | 2007-05-31 | 2015-10-27 | Univ Iowa Res Found | Redução da toxicidade não dirigida do arn de interferência |
| GB0816778D0 (en) | 2008-09-12 | 2008-10-22 | Isis Innovation | Gene silencing |
| EP3514232A1 (en) | 2010-04-23 | 2019-07-24 | 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 |
| AU2011329777B2 (en) | 2010-11-17 | 2016-06-09 | Ionis Pharmaceuticals, Inc. | Modulation of alpha synuclein expression |
| JP6224459B2 (ja) | 2011-02-17 | 2017-11-01 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 組織特異性を改変し、ａａｖ９媒介遺伝子導入を改善するための組成物および方法 |
| BR112014021104B1 (pt) | 2012-02-29 | 2023-03-28 | Sangamo Biosciences, Inc | Proteína de fusão de ocorrência não natural compreendendo um domínio de ligação de dna de dedo de zinco manipulado que se liga a um gene htt, seu uso, método in vitro de modificação da expressão de um gene htt em uma célula, e método de geração de um sistema modelo para o estudo da doença de huntington |
| EP4600255A3 (en) | 2014-05-02 | 2025-10-22 | Genzyme Corporation | Aav vectors for retinal and cns gene therapy |
| US10450563B2 (en) | 2015-02-10 | 2019-10-22 | Genzyme Corporation | Variant RNAi |
| PH12021550254A1 (en) | 2018-08-03 | 2023-02-06 | Genzyme Corp | Variant rnai against alpha-synuclein |
-
2019
- 2019-08-02 PH PH1/2021/550254A patent/PH12021550254A1/en unknown
- 2019-08-02 TW TW113117876A patent/TW202509215A/zh unknown
- 2019-08-02 BR BR112021001980-4A patent/BR112021001980A2/pt unknown
- 2019-08-02 US US17/265,489 patent/US12365895B2/en active Active
- 2019-08-02 SG SG11202101072UA patent/SG11202101072UA/en unknown
- 2019-08-02 TW TW108127564A patent/TW202022116A/zh unknown
- 2019-08-02 MX MX2021001395A patent/MX2021001395A/es unknown
- 2019-08-02 AR ARP190102204A patent/AR114540A1/es unknown
- 2019-08-02 CN CN201980063972.6A patent/CN112805382A/zh active Pending
- 2019-08-02 KR KR1020217006255A patent/KR20210062627A/ko active Pending
- 2019-08-02 EP EP19752626.2A patent/EP3830265A1/en active Pending
- 2019-08-02 AU AU2019314529A patent/AU2019314529B2/en active Active
- 2019-08-02 WO PCT/US2019/044924 patent/WO2020028816A1/en not_active Ceased
- 2019-08-02 CA CA3108526A patent/CA3108526A1/en active Pending
- 2019-08-02 JP JP2021529252A patent/JP2021534814A/ja active Pending
-
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