JP2020518266A5 - - Google Patents
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- Publication number
- JP2020518266A5 JP2020518266A5 JP2019560388A JP2019560388A JP2020518266A5 JP 2020518266 A5 JP2020518266 A5 JP 2020518266A5 JP 2019560388 A JP2019560388 A JP 2019560388A JP 2019560388 A JP2019560388 A JP 2019560388A JP 2020518266 A5 JP2020518266 A5 JP 2020518266A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- region
- strand sequence
- antisense strand
- sense 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
Links
- 230000000692 anti-sense effect Effects 0.000 claims 41
- 108091081021 Sense strand Proteins 0.000 claims 37
- 108090000623 proteins and genes Proteins 0.000 claims 36
- 239000002773 nucleotide Substances 0.000 claims 35
- 125000003729 nucleotide group Chemical group 0.000 claims 35
- 241000700605 Viruses Species 0.000 claims 26
- 108020004459 Small interfering RNA Proteins 0.000 claims 24
- 125000006850 spacer group Chemical group 0.000 claims 24
- 108020005029 5' Flanking Region Proteins 0.000 claims 20
- 108020005065 3' Flanking Region Proteins 0.000 claims 17
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 11
- 150000007523 nucleic acids Chemical group 0.000 claims 11
- 239000002245 particle Substances 0.000 claims 8
- 239000000203 mixture Substances 0.000 claims 7
- 210000004027 cell Anatomy 0.000 claims 6
- 238000000034 method Methods 0.000 claims 6
- 239000002062 molecular scaffold Substances 0.000 claims 6
- 230000003612 virological effect Effects 0.000 claims 5
- 241000702421 Dependoparvovirus Species 0.000 claims 4
- 230000000295 complement effect Effects 0.000 claims 4
- 108020004999 messenger RNA Proteins 0.000 claims 4
- 239000008194 pharmaceutical composition Substances 0.000 claims 3
- FDKWRPBBCBCIGA-REOHCLBHSA-N (2r)-2-azaniumyl-3-$l^{1}-selanylpropanoate Chemical compound [Se]C[C@H](N)C(O)=O FDKWRPBBCBCIGA-REOHCLBHSA-N 0.000 claims 2
- FDKWRPBBCBCIGA-UWTATZPHSA-N D-Selenocysteine Natural products [Se]C[C@@H](N)C(O)=O FDKWRPBBCBCIGA-UWTATZPHSA-N 0.000 claims 2
- 108091034120 Epstein–Barr virus-encoded small RNA Proteins 0.000 claims 2
- 108010017842 Telomerase Proteins 0.000 claims 2
- 201000010099 disease Diseases 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- ZKZBPNGNEQAJSX-UHFFFAOYSA-N selenocysteine Natural products [SeH]CC(N)C(O)=O ZKZBPNGNEQAJSX-UHFFFAOYSA-N 0.000 claims 2
- 229940055619 selenocysteine Drugs 0.000 claims 2
- 235000016491 selenocysteine Nutrition 0.000 claims 2
- 241000701161 unidentified adenovirus Species 0.000 claims 2
- 210000001130 astrocyte Anatomy 0.000 claims 1
- 210000000234 capsid Anatomy 0.000 claims 1
- 210000003169 central nervous system Anatomy 0.000 claims 1
- 208000015114 central nervous system disease Diseases 0.000 claims 1
- 210000003618 cortical neuron Anatomy 0.000 claims 1
- 230000000694 effects Effects 0.000 claims 1
- 210000004962 mammalian cell Anatomy 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 210000002161 motor neuron Anatomy 0.000 claims 1
- 210000002569 neuron Anatomy 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 108091033319 polynucleotide Proteins 0.000 claims 1
- 102000040430 polynucleotide Human genes 0.000 claims 1
- 239000002157 polynucleotide Substances 0.000 claims 1
- 230000003362 replicative effect Effects 0.000 claims 1
- 230000008685 targeting Effects 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023075746A JP7768928B2 (ja) | 2017-05-05 | 2023-05-01 | 調節性ポリヌクレオチド |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762501787P | 2017-05-05 | 2017-05-05 | |
| US62/501,787 | 2017-05-05 | ||
| US201762507923P | 2017-05-18 | 2017-05-18 | |
| US62/507,923 | 2017-05-18 | ||
| US201762520093P | 2017-06-15 | 2017-06-15 | |
| US62/520,093 | 2017-06-15 | ||
| PCT/US2018/031108 WO2018204797A1 (en) | 2017-05-05 | 2018-05-04 | Modulatory polynucleotides |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023075746A Division JP7768928B2 (ja) | 2017-05-05 | 2023-05-01 | 調節性ポリヌクレオチド |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020518266A JP2020518266A (ja) | 2020-06-25 |
| JP2020518266A5 true JP2020518266A5 (https=) | 2021-07-26 |
Family
ID=64016722
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019560388A Pending JP2020518266A (ja) | 2017-05-05 | 2018-05-04 | 調節性ポリヌクレオチド |
| JP2023075746A Active JP7768928B2 (ja) | 2017-05-05 | 2023-05-01 | 調節性ポリヌクレオチド |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023075746A Active JP7768928B2 (ja) | 2017-05-05 | 2023-05-01 | 調節性ポリヌクレオチド |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US20200270635A1 (https=) |
| EP (1) | EP3619310A4 (https=) |
| JP (2) | JP2020518266A (https=) |
| CN (2) | CN118910166A (https=) |
| AU (1) | AU2018260998B2 (https=) |
| CA (1) | CA3061365A1 (https=) |
| SG (1) | SG11201909777YA (https=) |
| TW (1) | TWI897844B (https=) |
| WO (1) | WO2018204797A1 (https=) |
Families Citing this family (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9585971B2 (en) | 2013-09-13 | 2017-03-07 | California Institute Of Technology | Recombinant AAV capsid protein |
| CN119876138A (zh) | 2014-11-14 | 2025-04-25 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| CA3193811A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| KR102423442B1 (ko) | 2015-12-11 | 2022-07-20 | 캘리포니아 인스티튜트 오브 테크놀로지 | 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드 |
| CA3024449A1 (en) | 2016-05-18 | 2017-11-23 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| CA3024448C (en) | 2016-05-18 | 2025-09-09 | Voyager Therapeutics, Inc. | MODULATING POLYNUCLEOTIDES |
| JP2019530737A (ja) | 2016-08-23 | 2019-10-24 | アコーオス インコーポレイテッド | ヒト対象において非加齢性聴力障害を治療するための組成物および方法 |
| EP3618839A4 (en) | 2017-05-05 | 2021-06-09 | Voyager Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| TW202413649A (zh) | 2017-10-16 | 2024-04-01 | 美商航海家醫療公司 | 肌萎縮性脊髓側索硬化症(als)之治療 |
| US12060567B2 (en) | 2018-06-13 | 2024-08-13 | Voyager Therapeutics, Inc. | Engineered untranslated regions (UTR) for AAV production |
| US20210254103A1 (en) * | 2018-07-02 | 2021-08-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord |
| AU2019310459A1 (en) | 2018-07-24 | 2021-02-18 | Voyager Therapeutics, Inc. | Systems and methods for producing gene therapy formulations |
| WO2020072849A1 (en) | 2018-10-04 | 2020-04-09 | Voyager Therapeutics, Inc. | Methods for measuring the titer and potency of viral vector particles |
| WO2020072844A1 (en) | 2018-10-05 | 2020-04-09 | Voyager Therapeutics, Inc. | Engineered nucleic acid constructs encoding aav production proteins |
| CA3116701A1 (en) | 2018-10-15 | 2020-04-23 | Voyager Therapeutics, Inc. | Expression vectors for large-scale production of raav in the baculovirus/sf9 system |
| JP2022522995A (ja) | 2019-01-18 | 2022-04-21 | ボイジャー セラピューティクス インコーポレイテッド | Ａａｖ粒子を生成するための方法及びシステム |
| WO2020223280A1 (en) * | 2019-04-29 | 2020-11-05 | Voyager Therapeutics, Inc. | Aav variants with enhanced tropism |
| EP3962536A1 (en) | 2019-04-29 | 2022-03-09 | Voyager Therapeutics, Inc. | Systems and methods for producing baculoviral infected insect cells (biics) in bioreactors |
| WO2021030125A1 (en) | 2019-08-09 | 2021-02-18 | Voyager Therapeutics, Inc. | Cell culture medium for use in producing gene therapy products in bioreactors |
| TW202122582A (zh) | 2019-08-26 | 2021-06-16 | 美商航海家醫療公司 | 病毒蛋白之控制表現 |
| JP7807380B2 (ja) | 2020-02-21 | 2026-01-27 | アコーオス インコーポレイテッド | ヒト対象において非加齢関連聴力障害を処置するための組成物および方法 |
| CN111450083A (zh) * | 2020-04-28 | 2020-07-28 | 天津大学 | 一种肝癌靶向治疗纳米颗粒的合成方法 |
| GB202010981D0 (en) * | 2020-07-16 | 2020-09-02 | Ucl Business Ltd | Gene therapy for neuromuscular and neuromotor disorders |
| CN116234917A (zh) | 2020-07-27 | 2023-06-06 | 安杰瑞姆生物科学公司 | Dna分子组合物及其制备方法和使用方法 |
| WO2022032153A1 (en) | 2020-08-06 | 2022-02-10 | Voyager Therapeutics, Inc. | Cell culture medium for use in producing gene therapy products in bioreactors |
| WO2022072324A1 (en) * | 2020-09-29 | 2022-04-07 | NeuExcell Therapeutics Inc. | Isl1 and lhx3 vector |
| US20240269322A1 (en) * | 2020-10-21 | 2024-08-15 | Chan Zuckerberg Biohub, Inc. | Adeno-associated virus virions and methods of use thereof |
| US20240141377A1 (en) | 2021-03-03 | 2024-05-02 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| US20240141378A1 (en) | 2021-03-03 | 2024-05-02 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| MX2024004217A (es) | 2021-10-08 | 2024-06-26 | Dyno Therapeutics Inc | Variantes de cápside y métodos de uso de estas. |
| US20260085328A1 (en) | 2022-09-08 | 2026-03-26 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| KR20250129743A (ko) | 2022-12-29 | 2025-08-29 | 보이저 테라퓨틱스, 인크. | Mapt를 조절하기 위한 조성물 및 방법 |
| WO2024226761A2 (en) | 2023-04-26 | 2024-10-31 | Voyager Therapeutics, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis |
| WO2024226790A1 (en) * | 2023-04-26 | 2024-10-31 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| WO2025122644A1 (en) | 2023-12-05 | 2025-06-12 | Voyager Therapeutics, Inc. | Compositions and methods for regulating mapt |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2187626C (en) * | 1994-04-13 | 2009-11-03 | Michael G. Kaplitt | Aav-mediated delivery of dna to cells of the nervous system |
| US20080318210A1 (en) * | 2003-08-27 | 2008-12-25 | Rosetta Genomics | Bioinformatically detectable group of novel regulatory viral and viral associated oligonucleotides and uses thereof |
| JP2006523103A (ja) * | 2003-02-21 | 2006-10-12 | ザ ペン ステイト リサーチ ファウンデーション | Ｒｎａ干渉組成物および方法 |
| WO2004101787A1 (ja) * | 2003-05-14 | 2004-11-25 | Japan Science And Technology Agency | ハンチンチン遺伝子の発現抑制 |
| US20050064489A1 (en) * | 2003-09-24 | 2005-03-24 | Zhang Fang Liang | Engineered U6 and H1 promoters |
| WO2006055727A2 (en) * | 2004-11-18 | 2006-05-26 | The Board Of Trustees Of The University Of Illinois | MULTICISTRONIC siRNA CONSTRUCTS TO INHIBIT TUMORS |
| EP2152874A2 (en) * | 2007-04-26 | 2010-02-17 | University of Iowa Research Foundation | Rna interference suppression of neurodegenerative diseases and methods of use thereof |
| US9493774B2 (en) * | 2009-01-05 | 2016-11-15 | Rxi Pharmaceuticals Corporation | Inhibition of PCSK9 through RNAi |
| JP2012528588A (ja) * | 2009-06-05 | 2012-11-15 | ソル，ダイ‐ウ | 単一またはマルチ標的遺伝子を抑制するマルチ−シストロンｓｈＲＮＡ発現カセット |
| EP4410805A3 (en) * | 2014-03-18 | 2024-11-27 | University of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| CN119876138A (zh) * | 2014-11-14 | 2025-04-25 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| CA3193811A1 (en) * | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| EP3448875A4 (en) * | 2016-04-29 | 2020-04-08 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
-
2018
- 2018-05-04 JP JP2019560388A patent/JP2020518266A/ja active Pending
- 2018-05-04 CA CA3061365A patent/CA3061365A1/en active Pending
- 2018-05-04 CN CN202411018854.2A patent/CN118910166A/zh active Pending
- 2018-05-04 EP EP18795140.5A patent/EP3619310A4/en active Pending
- 2018-05-04 TW TW107115348A patent/TWI897844B/zh active
- 2018-05-04 US US16/611,046 patent/US20200270635A1/en not_active Abandoned
- 2018-05-04 AU AU2018260998A patent/AU2018260998B2/en active Active
- 2018-05-04 CN CN201880042534.7A patent/CN110914427B/zh active Active
- 2018-05-04 WO PCT/US2018/031108 patent/WO2018204797A1/en not_active Ceased
- 2018-05-04 SG SG11201909777Y patent/SG11201909777YA/en unknown
-
2021
- 2021-12-23 US US17/561,252 patent/US20220333131A1/en active Pending
-
2023
- 2023-05-01 JP JP2023075746A patent/JP7768928B2/ja active Active
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