JPWO2020219726A5 - - Google Patents
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- Publication number
- JPWO2020219726A5 JPWO2020219726A5 JP2021562980A JP2021562980A JPWO2020219726A5 JP WO2020219726 A5 JPWO2020219726 A5 JP WO2020219726A5 JP 2021562980 A JP2021562980 A JP 2021562980A JP 2021562980 A JP2021562980 A JP 2021562980A JP WO2020219726 A5 JPWO2020219726 A5 JP WO2020219726A5
- Authority
- JP
- Japan
- Prior art keywords
- fusion protein
- nucleic acid
- seq
- acid construct
- recombinant virus
- 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.)
- Granted
Links
- 108020001507 fusion proteins Proteins 0.000 claims 28
- 102000037865 fusion proteins Human genes 0.000 claims 28
- 108020004707 nucleic acids Proteins 0.000 claims 16
- 102000039446 nucleic acids Human genes 0.000 claims 16
- 150000007523 nucleic acids Chemical class 0.000 claims 16
- 241000700605 Viruses Species 0.000 claims 15
- 239000008194 pharmaceutical composition Substances 0.000 claims 11
- 101150014718 C9orf72 gene Proteins 0.000 claims 10
- 230000035897 transcription Effects 0.000 claims 10
- 238000013518 transcription Methods 0.000 claims 10
- 102000043334 C9orf72 Human genes 0.000 claims 9
- 108700030955 C9orf72 Proteins 0.000 claims 9
- 108700028369 Alleles Proteins 0.000 claims 8
- 101710185494 Zinc finger protein Proteins 0.000 claims 8
- 102100023597 Zinc finger protein 816 Human genes 0.000 claims 8
- 210000005260 human cell Anatomy 0.000 claims 7
- 210000004027 cell Anatomy 0.000 claims 6
- 230000000692 anti-sense effect Effects 0.000 claims 5
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 claims 4
- 102100025169 Max-binding protein MNT Human genes 0.000 claims 3
- 238000000034 method Methods 0.000 claims 3
- 108091006107 transcriptional repressors Proteins 0.000 claims 3
- 230000003612 virological effect Effects 0.000 claims 3
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 108091026890 Coding region Proteins 0.000 claims 2
- 241000702421 Dependoparvovirus Species 0.000 claims 2
- 108700024394 Exon Proteins 0.000 claims 2
- 201000011240 Frontotemporal dementia Diseases 0.000 claims 2
- 108700029229 Transcriptional Regulatory Elements Proteins 0.000 claims 2
- HCHKCACWOHOZIP-UHFFFAOYSA-N Zinc Chemical compound [Zn] HCHKCACWOHOZIP-UHFFFAOYSA-N 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 210000004263 induced pluripotent stem cell Anatomy 0.000 claims 2
- 108020004999 messenger RNA Proteins 0.000 claims 2
- 210000002569 neuron Anatomy 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 230000000754 repressing effect Effects 0.000 claims 2
- 239000011701 zinc Substances 0.000 claims 2
- 229910052725 zinc Inorganic materials 0.000 claims 2
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 1
- 101100383812 Homo sapiens C9orf72 gene Proteins 0.000 claims 1
- 101000821100 Homo sapiens Synapsin-1 Proteins 0.000 claims 1
- 101000818735 Homo sapiens Zinc finger protein 10 Proteins 0.000 claims 1
- 108091023040 Transcription factor Proteins 0.000 claims 1
- 102000040945 Transcription factor Human genes 0.000 claims 1
- 125000003275 alpha amino acid group Chemical group 0.000 claims 1
- 210000004556 brain Anatomy 0.000 claims 1
- 210000004958 brain cell Anatomy 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 210000001671 embryonic stem cell Anatomy 0.000 claims 1
- 230000004927 fusion Effects 0.000 claims 1
- 102000056115 human SYN1 Human genes 0.000 claims 1
- 102000044778 human ZNF10 Human genes 0.000 claims 1
- 230000001939 inductive effect Effects 0.000 claims 1
- 230000002401 inhibitory effect Effects 0.000 claims 1
- 238000000185 intracerebroventricular administration Methods 0.000 claims 1
- 238000007917 intracranial administration Methods 0.000 claims 1
- 238000007913 intrathecal administration Methods 0.000 claims 1
- 238000001990 intravenous administration Methods 0.000 claims 1
- 230000035772 mutation Effects 0.000 claims 1
- 210000000461 neuroepithelial cell Anatomy 0.000 claims 1
- 210000004498 neuroglial cell Anatomy 0.000 claims 1
- 210000001778 pluripotent stem cell Anatomy 0.000 claims 1
- 108090000765 processed proteins & peptides Proteins 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 230000006798 recombination Effects 0.000 claims 1
- 238000005215 recombination Methods 0.000 claims 1
- 210000000278 spinal cord Anatomy 0.000 claims 1
- 210000000130 stem cell Anatomy 0.000 claims 1
- 239000013603 viral vector Substances 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025002919A JP2025069161A (ja) | 2019-04-23 | 2025-01-08 | ９番染色体オープンリーディングフレーム７２遺伝子発現のモジュレーターおよびその使用 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962837523P | 2019-04-23 | 2019-04-23 | |
| US62/837,523 | 2019-04-23 | ||
| US202062964844P | 2020-01-23 | 2020-01-23 | |
| US62/964,844 | 2020-01-23 | ||
| PCT/US2020/029590 WO2020219726A1 (en) | 2019-04-23 | 2020-04-23 | Modulators of chromosome 9 open reading frame 72 gene expression and uses thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025002919A Division JP2025069161A (ja) | 2019-04-23 | 2025-01-08 | ９番染色体オープンリーディングフレーム７２遺伝子発現のモジュレーターおよびその使用 |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022529500A JP2022529500A (ja) | 2022-06-22 |
| JP2022529500A5 JP2022529500A5 (https=) | 2023-04-28 |
| JPWO2020219726A5 true JPWO2020219726A5 (https=) | 2023-04-28 |
| JP7618581B2 JP7618581B2 (ja) | 2025-01-21 |
Family
ID=70680681
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021562980A Active JP7618581B2 (ja) | 2019-04-23 | 2020-04-23 | ９番染色体オープンリーディングフレーム７２遺伝子発現のモジュレーターおよびその使用 |
| JP2025002919A Pending JP2025069161A (ja) | 2019-04-23 | 2025-01-08 | ９番染色体オープンリーディングフレーム７２遺伝子発現のモジュレーターおよびその使用 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025002919A Pending JP2025069161A (ja) | 2019-04-23 | 2025-01-08 | ９番染色体オープンリーディングフレーム７２遺伝子発現のモジュレーターおよびその使用 |
Country Status (16)
| Country | Link |
|---|---|
| US (3) | US12139517B2 (https=) |
| EP (1) | EP3958871A1 (https=) |
| JP (2) | JP7618581B2 (https=) |
| KR (1) | KR20220003561A (https=) |
| CN (2) | CN113766921B (https=) |
| AU (1) | AU2020262281B2 (https=) |
| BR (1) | BR112021020868A2 (https=) |
| CA (1) | CA3137368A1 (https=) |
| CO (1) | CO2021015653A2 (https=) |
| IL (1) | IL287384A (https=) |
| MX (1) | MX2021012836A (https=) |
| PE (1) | PE20212332A1 (https=) |
| PH (1) | PH12021552554A1 (https=) |
| SG (1) | SG11202111443SA (https=) |
| TW (2) | TW202521561A (https=) |
| WO (1) | WO2020219726A1 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3120799A1 (en) | 2018-12-20 | 2020-06-25 | Regeneron Pharmaceuticals, Inc. | Nuclease-mediated repeat expansion |
| MX2021012836A (es) * | 2019-04-23 | 2021-12-10 | Sangamo Therapeutics Inc | Moduladores de la expresion del gen del marco de lectura abierto 72 del cromosoma 9 y usos de los mismos. |
| GB202010075D0 (en) * | 2020-07-01 | 2020-08-12 | Imp College Innovations Ltd | Therapeutic nucleic acids, peptides and uses |
| JP2024534523A (ja) | 2021-09-21 | 2024-09-20 | スクライブ・セラピューティクス・インコーポレイテッド | 操作されたｃａｓｘリプレッサー系 |
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| DE69942334D1 (de) | 1998-03-02 | 2010-06-17 | Massachusetts Inst Technology | Poly-zinkfinger-proteine mit verbesserten linkern |
| DE69929600T2 (de) | 1998-05-27 | 2006-09-07 | Avigen Inc., Alameda | Konvektion-erhöhte verabreichung aadc-kodierende aav vektoren |
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| MX2021012836A (es) * | 2019-04-23 | 2021-12-10 | Sangamo Therapeutics Inc | Moduladores de la expresion del gen del marco de lectura abierto 72 del cromosoma 9 y usos de los mismos. |
-
2020
- 2020-04-23 MX MX2021012836A patent/MX2021012836A/es unknown
- 2020-04-23 CN CN202080031118.4A patent/CN113766921B/zh active Active
- 2020-04-23 BR BR112021020868A patent/BR112021020868A2/pt unknown
- 2020-04-23 CN CN202511030848.3A patent/CN120960394A/zh active Pending
- 2020-04-23 PH PH1/2021/552554A patent/PH12021552554A1/en unknown
- 2020-04-23 KR KR1020217037624A patent/KR20220003561A/ko active Pending
- 2020-04-23 PE PE2021001757A patent/PE20212332A1/es unknown
- 2020-04-23 EP EP20725373.3A patent/EP3958871A1/en active Pending
- 2020-04-23 TW TW113139769A patent/TW202521561A/zh unknown
- 2020-04-23 JP JP2021562980A patent/JP7618581B2/ja active Active
- 2020-04-23 CA CA3137368A patent/CA3137368A1/en active Pending
- 2020-04-23 SG SG11202111443SA patent/SG11202111443SA/en unknown
- 2020-04-23 WO PCT/US2020/029590 patent/WO2020219726A1/en not_active Ceased
- 2020-04-23 AU AU2020262281A patent/AU2020262281B2/en active Active
- 2020-04-23 TW TW109113701A patent/TWI862584B/zh active
- 2020-04-23 US US16/856,774 patent/US12139517B2/en active Active
-
2021
- 2021-10-19 IL IL287384A patent/IL287384A/en unknown
- 2021-11-23 CO CONC2021/0015653A patent/CO2021015653A2/es unknown
-
2024
- 2024-11-09 US US18/942,681 patent/US20250066433A1/en active Pending
- 2024-11-11 US US18/943,130 patent/US20250066434A1/en active Pending
-
2025
- 2025-01-08 JP JP2025002919A patent/JP2025069161A/ja active Pending
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