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) | CN110914427B (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 |
|---|---|---|---|---|
| ES2739288T3 (es) | 2013-09-13 | 2020-01-30 | California Inst Of Techn | Recuperación selectiva |
| WO2016077687A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| KR20230145206A (ko) | 2014-11-14 | 2023-10-17 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| KR102423442B1 (ko) | 2015-12-11 | 2022-07-20 | 캘리포니아 인스티튜트 오브 테크놀로지 | 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드 |
| KR20240056729A (ko) | 2016-05-18 | 2024-04-30 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| KR102427379B1 (ko) | 2016-05-18 | 2022-08-02 | 보이저 테라퓨틱스, 인크. | 헌팅톤 질환을 치료하기 위한 조성물 및 방법 |
| EP3510161A4 (en) | 2016-08-23 | 2020-04-22 | Akouos, Inc. | COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON |
| JP2020518258A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法 |
| 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 |
| WO2019241486A1 (en) | 2018-06-13 | 2019-12-19 | Voyager Therapeutics, Inc. | Engineered 5' untranslated regions (5' 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 |
| CN112770812A (zh) | 2018-07-24 | 2021-05-07 | 沃雅戈治疗公司 | 产生基因治疗制剂的系统和方法 |
| 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 |
| CN113166781A (zh) | 2018-10-15 | 2021-07-23 | 沃雅戈治疗公司 | 在杆状病毒/Sf9系统中大规模生产rAAV的表达载体 |
| SG11202107645RA (en) | 2019-01-18 | 2021-08-30 | Voyager Therapeutics Inc | Methods and systems for producing aav particles |
| 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 |
| EP4010465A1 (en) | 2019-08-09 | 2022-06-15 | Voyager Therapeutics, Inc. | Cell culture medium for use in producing gene therapy products in bioreactors |
| EP4022070A1 (en) | 2019-08-26 | 2022-07-06 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| KR20220157944A (ko) | 2020-02-21 | 2022-11-29 | 아카우오스, 인크. | 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법 |
| 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 |
| JP2023535632A (ja) | 2020-07-27 | 2023-08-18 | アンジャリウム バイオサイエンシズ エージー | Ｄｎａ分子の組成物、その作製方法、及びその使用方法 |
| WO2022032153A1 (en) | 2020-08-06 | 2022-02-10 | Voyager Therapeutics, Inc. | Cell culture medium for use in producing gene therapy products in bioreactors |
| US20220098616A1 (en) * | 2020-09-29 | 2022-03-31 | NeuExcell Therapeutics Inc. | ISL1 and LHX3 VECTOR |
| WO2022087104A1 (en) * | 2020-10-21 | 2022-04-28 | Chan Zuckerberg Biohub, Inc. | Adeno-associated virus virions and methods of use thereof |
| WO2022187473A2 (en) | 2021-03-03 | 2022-09-09 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| WO2022187548A1 (en) | 2021-03-03 | 2022-09-09 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| KR20240095539A (ko) | 2021-10-08 | 2024-06-25 | 디노 테라퓨틱스, 인코포레이티드 | 캡시드 변이체 및 이의 사용 방법 |
| 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 |
| WO2024226790A1 (en) * | 2023-04-26 | 2024-10-31 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| WO2024226761A2 (en) | 2023-04-26 | 2024-10-31 | Voyager Therapeutics, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis |
| 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 |
|---|---|---|---|---|
| ATE386131T1 (de) * | 1994-04-13 | 2008-03-15 | Univ Rockefeller | Aav-vermittelte überbringung von dna in zellen des nervensystems |
| US20080318210A1 (en) * | 2003-08-27 | 2008-12-25 | Rosetta Genomics | Bioinformatically detectable group of novel regulatory viral and viral associated oligonucleotides and uses thereof |
| WO2005017127A2 (en) * | 2003-02-21 | 2005-02-24 | The Penn State Research Foundation | Rna interference compositions and methods |
| US7589189B2 (en) * | 2003-05-14 | 2009-09-15 | Japan Science And Technology Agency | Inhibition of the expression of huntingtin gene |
| US20050064489A1 (en) * | 2003-09-24 | 2005-03-24 | Zhang Fang Liang | Engineered U6 and H1 promoters |
| ES2556272T3 (es) * | 2004-11-18 | 2016-01-14 | The Board Of Trustees Of The University Of Illinois | Constructos de ARNsi multicistrónico para inhibir tumores |
| EP2152874A2 (en) * | 2007-04-26 | 2010-02-17 | University of Iowa Research Foundation | Rna interference suppression of neurodegenerative diseases and methods of use thereof |
| WO2010078536A1 (en) * | 2009-01-05 | 2010-07-08 | 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 |
| CA2942515C (en) * | 2014-03-18 | 2025-12-09 | University Of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| KR20230145206A (ko) * | 2014-11-14 | 2023-10-17 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| WO2016077687A1 (en) * | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| US20190224339A1 (en) * | 2016-04-29 | 2019-07-25 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
-
2018
- 2018-05-04 EP EP18795140.5A patent/EP3619310A4/en active Pending
- 2018-05-04 CA CA3061365A patent/CA3061365A1/en active Pending
- 2018-05-04 WO PCT/US2018/031108 patent/WO2018204797A1/en not_active Ceased
- 2018-05-04 CN CN201880042534.7A patent/CN110914427B/zh active Active
- 2018-05-04 AU AU2018260998A patent/AU2018260998B2/en active Active
- 2018-05-04 JP JP2019560388A patent/JP2020518266A/ja active Pending
- 2018-05-04 TW TW107115348A patent/TWI897844B/zh active
- 2018-05-04 SG SG11201909777Y patent/SG11201909777YA/en unknown
- 2018-05-04 US US16/611,046 patent/US20200270635A1/en not_active Abandoned
- 2018-05-04 CN CN202411018854.2A patent/CN118910166A/zh active Pending
-
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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