JPWO2021050991A5 - - Google Patents
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- Publication number
- JPWO2021050991A5 JPWO2021050991A5 JP2022516687A JP2022516687A JPWO2021050991A5 JP WO2021050991 A5 JPWO2021050991 A5 JP WO2021050991A5 JP 2022516687 A JP2022516687 A JP 2022516687A JP 2022516687 A JP2022516687 A JP 2022516687A JP WO2021050991 A5 JPWO2021050991 A5 JP WO2021050991A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- fxn
- human
- acid construct
- acid sequence
- 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
- 150000007523 nucleic acids Chemical class 0.000 claims description 123
- 102000003869 Frataxin Human genes 0.000 claims description 71
- 108090000217 Frataxin Proteins 0.000 claims description 71
- 108020004707 nucleic acids Proteins 0.000 claims description 69
- 102000039446 nucleic acids Human genes 0.000 claims description 69
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 47
- 108020003589 5' Untranslated Regions Proteins 0.000 claims description 28
- 102000009572 RNA Polymerase II Human genes 0.000 claims description 26
- 108010009460 RNA Polymerase II Proteins 0.000 claims description 26
- 239000013598 vector Substances 0.000 claims description 24
- 239000013607 AAV vector Substances 0.000 claims description 23
- 238000000034 method Methods 0.000 claims description 15
- 210000005260 human cell Anatomy 0.000 claims description 14
- 239000002245 particle Substances 0.000 claims description 12
- 239000000203 mixture Substances 0.000 claims description 11
- 208000024412 Friedreich ataxia Diseases 0.000 claims description 8
- 239000013603 viral vector Substances 0.000 claims description 8
- ZKHQWZAMYRWXGA-KQYNXXCUSA-J ATP(4-) Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](COP([O-])(=O)OP([O-])(=O)OP([O-])([O-])=O)[C@@H](O)[C@H]1O ZKHQWZAMYRWXGA-KQYNXXCUSA-J 0.000 claims description 6
- ZKHQWZAMYRWXGA-UHFFFAOYSA-N Adenosine triphosphate Natural products C1=NC=2C(N)=NC=NC=2N1C1OC(COP(O)(=O)OP(O)(=O)OP(O)(O)=O)C(O)C1O ZKHQWZAMYRWXGA-UHFFFAOYSA-N 0.000 claims description 6
- 108010014064 CCCTC-Binding Factor Proteins 0.000 claims description 6
- 102100021393 Transcriptional repressor CTCFL Human genes 0.000 claims description 6
- 210000004413 cardiac myocyte Anatomy 0.000 claims description 6
- 210000004027 cell Anatomy 0.000 claims description 6
- 210000002569 neuron Anatomy 0.000 claims description 6
- 239000008194 pharmaceutical composition Substances 0.000 claims description 4
- 210000002363 skeletal muscle cell Anatomy 0.000 claims description 4
- 210000000329 smooth muscle myocyte Anatomy 0.000 claims description 4
- 238000011144 upstream manufacturing Methods 0.000 claims description 4
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims description 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 2
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims description 2
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 2
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 2
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 2
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims description 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 2
- 108020004705 Codon Proteins 0.000 claims description 2
- 241000702421 Dependoparvovirus Species 0.000 claims description 2
- 102100036912 Desmin Human genes 0.000 claims description 2
- 108010044052 Desmin Proteins 0.000 claims description 2
- 108091026898 Leader sequence (mRNA) Proteins 0.000 claims description 2
- 210000001130 astrocyte Anatomy 0.000 claims description 2
- 210000005045 desmin Anatomy 0.000 claims description 2
- 210000003494 hepatocyte Anatomy 0.000 claims description 2
- 102000043827 human Smooth muscle Human genes 0.000 claims description 2
- 108700038605 human Smooth muscle Proteins 0.000 claims description 2
- 210000004185 liver Anatomy 0.000 claims description 2
- 210000005229 liver cell Anatomy 0.000 claims description 2
- 210000000274 microglia Anatomy 0.000 claims description 2
- 210000004248 oligodendroglia Anatomy 0.000 claims description 2
- 239000013612 plasmid Substances 0.000 claims description 2
- 210000002027 skeletal muscle Anatomy 0.000 claims description 2
- 210000000130 stem cell Anatomy 0.000 claims description 2
- 101001052793 Homo sapiens GDP-L-fucose synthase Proteins 0.000 description 1
- 102000050085 human TSTA3 Human genes 0.000 description 1
- 239000013589 supplement Substances 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962899953P | 2019-09-13 | 2019-09-13 | |
| US62/899,953 | 2019-09-13 | ||
| PCT/US2020/050551 WO2021050991A1 (en) | 2019-09-13 | 2020-09-11 | Compositions and methods for treatment of friedreich's ataxia |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022548270A JP2022548270A (ja) | 2022-11-17 |
| JPWO2021050991A5 true JPWO2021050991A5 (https=) | 2023-07-25 |
| JP2022548270A5 JP2022548270A5 (https=) | 2023-07-25 |
Family
ID=74865919
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022516687A Pending JP2022548270A (ja) | 2019-09-13 | 2020-09-11 | フリードライヒ運動失調症の治療のための組成物及び方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20220211737A1 (https=) |
| EP (1) | EP4028027A4 (https=) |
| JP (1) | JP2022548270A (https=) |
| AU (1) | AU2020346914A1 (https=) |
| CA (1) | CA3151073A1 (https=) |
| WO (1) | WO2021050991A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4426845A4 (en) * | 2021-11-01 | 2025-10-01 | Univ Florida | AAV-MEDIATED THERAPIES FOR VISION LOSS ASSOCIATED WITH FRIEDREICH ATAXIA |
| WO2024097772A1 (en) * | 2022-11-01 | 2024-05-10 | Lacerta Therapeutics, Inc. | Compositions and methods for treatment of friedreich's ataxia |
| WO2025157942A2 (en) * | 2024-01-23 | 2025-07-31 | Icm (Institut Du Cerveau Et De La Moelle Épinière) | Encapsulated engineered cells for treating a disease or condition affecting the nervous system |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3245221A4 (en) * | 2015-01-16 | 2018-06-13 | Voyager Therapeutics, Inc. | Central nervous system targeting polynucleotides |
| WO2016172659A1 (en) * | 2015-04-24 | 2016-10-27 | University Of Florida Research Foundation, Inc. | Aav vector for treatment of friedreich's ataxia |
| WO2017075338A2 (en) * | 2015-10-29 | 2017-05-04 | Voyager Therapeutics, Inc. | Delivery of central nervous system targeting polynucleotides |
| RU2021102893A (ru) * | 2015-11-05 | 2021-03-03 | Бамбу Терапьютикс, Инк. | Модифицированные гены атаксии фридрейха и векторы для генной терапии |
| CN110139933B (zh) * | 2016-11-09 | 2024-03-08 | 英特瑞克斯顿股份有限公司 | 共济蛋白表达构建体 |
| EP3641834B1 (en) * | 2017-06-19 | 2023-10-04 | Translate Bio, Inc. | Messenger rna therapy for the treatment of friedreich's ataxia |
| WO2019000093A1 (en) * | 2017-06-29 | 2019-01-03 | UNIVERSITé LAVAL | PLATINUM TALE AND USE THEREOF TO INCREASE THE EXPRESSION OF FRATAXIN |
| WO2019076973A1 (en) * | 2017-10-17 | 2019-04-25 | Fundació Institut D'investigació En Ciències De La Salut Germans Trias I Pujol | VECTORS FOR THE TREATMENT OF FRIEDREICH ATAXIA |
-
2020
- 2020-09-11 JP JP2022516687A patent/JP2022548270A/ja active Pending
- 2020-09-11 AU AU2020346914A patent/AU2020346914A1/en active Pending
- 2020-09-11 US US17/434,318 patent/US20220211737A1/en not_active Abandoned
- 2020-09-11 CA CA3151073A patent/CA3151073A1/en active Pending
- 2020-09-11 WO PCT/US2020/050551 patent/WO2021050991A1/en not_active Ceased
- 2020-09-11 EP EP20863569.8A patent/EP4028027A4/en active Pending