JPWO2022016055A5 - - Google Patents
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- Publication number
- JPWO2022016055A5 JPWO2022016055A5 JP2023503074A JP2023503074A JPWO2022016055A5 JP WO2022016055 A5 JPWO2022016055 A5 JP WO2022016055A5 JP 2023503074 A JP2023503074 A JP 2023503074A JP 2023503074 A JP2023503074 A JP 2023503074A JP WO2022016055 A5 JPWO2022016055 A5 JP WO2022016055A5
- Authority
- JP
- Japan
- Prior art keywords
- human
- aav
- fmrp
- vector
- composition
- 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
- 101000828537 Homo sapiens Synaptic functional regulator FMR1 Proteins 0.000 claims 28
- 102000045409 human FMR1 Human genes 0.000 claims 28
- 239000013607 AAV vector Substances 0.000 claims 23
- 239000013598 vector Substances 0.000 claims 20
- 239000000203 mixture Substances 0.000 claims 19
- 239000002773 nucleotide Substances 0.000 claims 19
- 125000003729 nucleotide group Chemical group 0.000 claims 19
- 230000011664 signaling Effects 0.000 claims 16
- 102000007338 Fragile X Mental Retardation Protein Human genes 0.000 claims 10
- 108010032606 Fragile X Mental Retardation Protein Proteins 0.000 claims 10
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 8
- 101000899111 Homo sapiens Hemoglobin subunit beta Proteins 0.000 claims 8
- 108700011259 MicroRNAs Proteins 0.000 claims 6
- 230000001105 regulatory effect Effects 0.000 claims 6
- 241000702421 Dependoparvovirus Species 0.000 claims 5
- 239000002245 particle Substances 0.000 claims 5
- 230000003612 virological effect Effects 0.000 claims 5
- 102000011755 Phosphoglycerate Kinase Human genes 0.000 claims 4
- 101001099217 Thermotoga maritima (strain ATCC 43589 / DSM 3109 / JCM 10099 / NBRC 100826 / MSB8) Triosephosphate isomerase Proteins 0.000 claims 4
- 102000004169 proteins and genes Human genes 0.000 claims 4
- 108090000623 proteins and genes Proteins 0.000 claims 4
- 208000001914 Fragile X syndrome Diseases 0.000 claims 3
- 108091007780 MiR-122 Proteins 0.000 claims 3
- 102100026808 Mitochondrial import inner membrane translocase subunit Tim8 A Human genes 0.000 claims 3
- 102000001708 Protein Isoforms Human genes 0.000 claims 3
- 108010029485 Protein Isoforms Proteins 0.000 claims 3
- 210000005013 brain tissue Anatomy 0.000 claims 3
- 238000002347 injection Methods 0.000 claims 3
- 239000007924 injection Substances 0.000 claims 3
- 108091051828 miR-122 stem-loop Proteins 0.000 claims 3
- 108091071651 miR-208 stem-loop Proteins 0.000 claims 3
- 108091084446 miR-208a stem-loop Proteins 0.000 claims 3
- 108091062547 miR-208a-1 stem-loop Proteins 0.000 claims 3
- 108091055375 miR-208a-2 stem-loop Proteins 0.000 claims 3
- 108091047268 miR-208b stem-loop Proteins 0.000 claims 3
- 108091048941 miR-499a stem-loop Proteins 0.000 claims 3
- 239000002679 microRNA Substances 0.000 claims 3
- 241001492404 Woodchuck hepatitis virus Species 0.000 claims 2
- 230000001124 posttranscriptional effect Effects 0.000 claims 2
- 238000011144 upstream manufacturing Methods 0.000 claims 2
- 125000000539 amino acid group Chemical group 0.000 claims 1
- 210000004556 brain Anatomy 0.000 claims 1
- 210000000234 capsid Anatomy 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 239000012634 fragment Substances 0.000 claims 1
- 208000015181 infectious disease Diseases 0.000 claims 1
- 238000010253 intravenous injection Methods 0.000 claims 1
- 238000007914 intraventricular administration Methods 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063053461P | 2020-07-17 | 2020-07-17 | |
| US63/053,461 | 2020-07-17 | ||
| PCT/US2021/041975 WO2022016055A1 (en) | 2020-07-17 | 2021-07-16 | Methods and compositions for treatment of fragile x syndrome |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023534293A JP2023534293A (ja) | 2023-08-08 |
| JPWO2022016055A5 true JPWO2022016055A5 (https=) | 2024-07-24 |
| JP2023534293A5 JP2023534293A5 (https=) | 2024-07-24 |
Family
ID=79554278
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023503074A Pending JP2023534293A (ja) | 2020-07-17 | 2021-07-16 | 脆弱ｘ症候群の治療のための方法及び組成物 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20230295654A1 (https=) |
| EP (1) | EP4181877A4 (https=) |
| JP (1) | JP2023534293A (https=) |
| KR (1) | KR20230084465A (https=) |
| CN (1) | CN117136077A (https=) |
| AU (1) | AU2021308666A1 (https=) |
| CA (1) | CA3189657A1 (https=) |
| IL (1) | IL299889A (https=) |
| WO (1) | WO2022016055A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20250195497A1 (en) * | 2022-03-22 | 2025-06-19 | Kyoto University | Therapeutic or prophylactic medicine for fragile x syndrome |
| GB202206336D0 (en) * | 2022-04-29 | 2022-06-15 | Univ Edinburgh | Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders |
| AU2024313758A1 (en) | 2023-06-23 | 2026-01-08 | Uniqure Biopharma B.V. | Novel fragile x constructs |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2012056440A1 (en) * | 2010-10-28 | 2012-05-03 | Nanodoc Ltd. | COMPOSITIONS AND METHODS FOR ACTIVATING EXPRESSION BY A SPECIFIC ENDOGENOUS miRNA |
| CA3019315A1 (en) * | 2015-04-23 | 2016-10-27 | University Of Massachusetts | Modulation of aav vector transgene expression |
| GB2574243A (en) * | 2018-05-31 | 2019-12-04 | The Govening Council Of The Univ Of Toronto | Adeno-associated viral vector-mediated gene therapy for treating fragile X-associated disorders |
| US12421524B2 (en) * | 2018-09-24 | 2025-09-23 | Merck Sharp & Dohme Llc | Expression vectors for eukaryotic expression systems |
-
2021
- 2021-07-16 WO PCT/US2021/041975 patent/WO2022016055A1/en not_active Ceased
- 2021-07-16 JP JP2023503074A patent/JP2023534293A/ja active Pending
- 2021-07-16 CA CA3189657A patent/CA3189657A1/en active Pending
- 2021-07-16 EP EP21843256.5A patent/EP4181877A4/en active Pending
- 2021-07-16 CN CN202180059850.7A patent/CN117136077A/zh active Pending
- 2021-07-16 IL IL299889A patent/IL299889A/en unknown
- 2021-07-16 AU AU2021308666A patent/AU2021308666A1/en active Pending
- 2021-07-16 KR KR1020237005196A patent/KR20230084465A/ko active Pending
- 2021-07-16 US US18/005,757 patent/US20230295654A1/en active Pending
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