JPWO2022034130A5 - - Google Patents
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- Publication number
- JPWO2022034130A5 JPWO2022034130A5 JP2023509770A JP2023509770A JPWO2022034130A5 JP WO2022034130 A5 JPWO2022034130 A5 JP WO2022034130A5 JP 2023509770 A JP2023509770 A JP 2023509770A JP 2023509770 A JP2023509770 A JP 2023509770A JP WO2022034130 A5 JPWO2022034130 A5 JP WO2022034130A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleotide sequence
- seq
- identity
- nucleic acid
- vector
- 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
- 239000002773 nucleotide Substances 0.000 claims 52
- 125000003729 nucleotide group Chemical group 0.000 claims 52
- 102000019204 Progranulins Human genes 0.000 claims 21
- 108010012809 Progranulins Proteins 0.000 claims 21
- 108020004707 nucleic acids Proteins 0.000 claims 21
- 102000039446 nucleic acids Human genes 0.000 claims 21
- 150000007523 nucleic acids Chemical class 0.000 claims 21
- 239000012634 fragment Substances 0.000 claims 17
- 239000013598 vector Substances 0.000 claims 12
- 239000013603 viral vector Substances 0.000 claims 12
- 108010072388 Methyl-CpG-Binding Protein 2 Proteins 0.000 claims 10
- 102100039124 Methyl-CpG-binding protein 2 Human genes 0.000 claims 10
- 238000002347 injection Methods 0.000 claims 9
- 239000007924 injection Substances 0.000 claims 9
- 239000013607 AAV vector Substances 0.000 claims 8
- 101150083522 MECP2 gene Proteins 0.000 claims 4
- 210000001175 cerebrospinal fluid Anatomy 0.000 claims 4
- 230000007812 deficiency Effects 0.000 claims 4
- 201000010099 disease Diseases 0.000 claims 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 4
- 239000008194 pharmaceutical composition Substances 0.000 claims 4
- 101100076422 Mus musculus Mecp2 gene Proteins 0.000 claims 3
- 108010076504 Protein Sorting Signals Proteins 0.000 claims 3
- 108091093126 WHP Posttrascriptional Response Element Proteins 0.000 claims 3
- 210000004556 brain Anatomy 0.000 claims 3
- 230000008488 polyadenylation Effects 0.000 claims 3
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 2
- 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
- 241000649044 Adeno-associated virus 9 Species 0.000 claims 2
- 108700028369 Alleles Proteins 0.000 claims 2
- 201000011240 Frontotemporal dementia Diseases 0.000 claims 2
- 101150024624 GRN gene Proteins 0.000 claims 2
- 108091034057 RNA (poly(A)) Proteins 0.000 claims 2
- 241001492404 Woodchuck hepatitis virus Species 0.000 claims 2
- 125000003275 alpha amino acid group Chemical group 0.000 claims 2
- 210000004027 cell Anatomy 0.000 claims 2
- 238000000185 intracerebroventricular administration Methods 0.000 claims 2
- 230000004777 loss-of-function mutation Effects 0.000 claims 2
- 201000007605 neuronal ceroid lipofuscinosis 11 Diseases 0.000 claims 2
- 108091033319 polynucleotide Proteins 0.000 claims 2
- 102000040430 polynucleotide Human genes 0.000 claims 2
- 239000002157 polynucleotide Substances 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 102000004169 proteins and genes Human genes 0.000 claims 2
- 108020004705 Codon Proteins 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 101100076420 Homo sapiens MECP2 gene Proteins 0.000 claims 1
- 101001027324 Homo sapiens Progranulin Proteins 0.000 claims 1
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 1
- 210000001744 T-lymphocyte Anatomy 0.000 claims 1
- 210000001130 astrocyte Anatomy 0.000 claims 1
- 208000015114 central nervous system disease Diseases 0.000 claims 1
- 239000003085 diluting agent Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 238000007913 intrathecal administration Methods 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 210000002569 neuron Anatomy 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 239000013612 plasmid Substances 0.000 claims 1
- 230000001124 posttranscriptional effect Effects 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063064431P | 2020-08-12 | 2020-08-12 | |
| US63/064,431 | 2020-08-12 | ||
| PCT/EP2021/072365 WO2022034130A1 (en) | 2020-08-12 | 2021-08-11 | Gene therapy using nucleic acid constructs comprising methyl cpg binding protein 2 (mecp2) promoter sequences |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023537980A JP2023537980A (ja) | 2023-09-06 |
| JPWO2022034130A5 true JPWO2022034130A5 (es) | 2024-07-25 |
Family
ID=77655525
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023509770A Pending JP2023537980A (ja) | 2020-08-12 | 2021-08-11 | メチルｃｐｇ結合タンパク質２（ｍｅｃｐ２）プロモータ配列を含む核酸構築体を用いた遺伝子治療 |
Country Status (18)
| Country | Link |
|---|---|
| US (1) | US20230295657A1 (es) |
| EP (1) | EP4196171A1 (es) |
| JP (1) | JP2023537980A (es) |
| KR (1) | KR20230044506A (es) |
| CN (1) | CN116113441A (es) |
| AR (1) | AR123206A1 (es) |
| AU (1) | AU2021325717A1 (es) |
| BR (1) | BR112023002374A2 (es) |
| CA (1) | CA3188748A1 (es) |
| CL (1) | CL2023000419A1 (es) |
| CO (1) | CO2023000444A2 (es) |
| EC (1) | ECSP23016688A (es) |
| IL (1) | IL300294A (es) |
| MX (1) | MX2023001701A (es) |
| PE (1) | PE20230914A1 (es) |
| TW (1) | TW202221018A (es) |
| WO (1) | WO2022034130A1 (es) |
| ZA (1) | ZA202300378B (es) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB202216168D0 (en) | 2022-10-31 | 2022-12-14 | UCB Biopharma SRL | Route of administration |
| WO2024163823A1 (en) * | 2023-02-02 | 2024-08-08 | Shape Therapeutics Inc. | Tissue-specific enhancers for regulating transcription |
| WO2024189094A1 (en) * | 2023-03-14 | 2024-09-19 | UCB Biopharma SRL | Gene therapy |
Family Cites Families (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| AU2015364636B9 (en) * | 2014-12-16 | 2021-12-02 | Board Of Regents Of The University Of Nebraska | Gene therapy for Juvenile Batten Disease |
| WO2017151884A1 (en) * | 2016-03-02 | 2017-09-08 | The Children's Hospital Of Philadelphia | Therapy for frontotemporal dementia |
-
2021
- 2021-08-11 MX MX2023001701A patent/MX2023001701A/es unknown
- 2021-08-11 CA CA3188748A patent/CA3188748A1/en active Pending
- 2021-08-11 EP EP21766127.1A patent/EP4196171A1/en active Pending
- 2021-08-11 AR ARP210102238A patent/AR123206A1/es unknown
- 2021-08-11 JP JP2023509770A patent/JP2023537980A/ja active Pending
- 2021-08-11 BR BR112023002374A patent/BR112023002374A2/pt unknown
- 2021-08-11 PE PE2023000142A patent/PE20230914A1/es unknown
- 2021-08-11 KR KR1020237007287A patent/KR20230044506A/ko active Search and Examination
- 2021-08-11 IL IL300294A patent/IL300294A/en unknown
- 2021-08-11 TW TW110129603A patent/TW202221018A/zh unknown
- 2021-08-11 CN CN202180056525.5A patent/CN116113441A/zh active Pending
- 2021-08-11 US US18/021,005 patent/US20230295657A1/en active Pending
- 2021-08-11 AU AU2021325717A patent/AU2021325717A1/en active Pending
- 2021-08-11 WO PCT/EP2021/072365 patent/WO2022034130A1/en active Application Filing
-
2023
- 2023-01-09 ZA ZA2023/00378A patent/ZA202300378B/en unknown
- 2023-01-16 CO CONC2023/0000444A patent/CO2023000444A2/es unknown
- 2023-02-10 CL CL2023000419A patent/CL2023000419A1/es unknown
- 2023-03-08 EC ECSENADI202316688A patent/ECSP23016688A/es unknown
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