JPWO2022020697A5 - - Google Patents
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- Publication number
- JPWO2022020697A5 JPWO2022020697A5 JP2023503171A JP2023503171A JPWO2022020697A5 JP WO2022020697 A5 JPWO2022020697 A5 JP WO2022020697A5 JP 2023503171 A JP2023503171 A JP 2023503171A JP 2023503171 A JP2023503171 A JP 2023503171A JP WO2022020697 A5 JPWO2022020697 A5 JP WO2022020697A5
- Authority
- JP
- Japan
- Prior art keywords
- expression cassette
- pharmaceutical composition
- vector
- slc13a5
- subject
- 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
- 239000008194 pharmaceutical composition Substances 0.000 claims 21
- 239000013598 vector Substances 0.000 claims 10
- 108700026244 Open Reading Frames Proteins 0.000 claims 7
- 108091033319 polynucleotide Proteins 0.000 claims 7
- 239000002157 polynucleotide Substances 0.000 claims 7
- 102000040430 polynucleotide Human genes 0.000 claims 7
- 101001093944 Homo sapiens Solute carrier family 13 member 5 Proteins 0.000 claims 5
- 102000045385 human SLC13A5 Human genes 0.000 claims 5
- 210000004027 cell Anatomy 0.000 claims 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 4
- 239000002773 nucleotide Substances 0.000 claims 4
- 125000003729 nucleotide group Chemical group 0.000 claims 4
- 230000008488 polyadenylation Effects 0.000 claims 4
- 108091006587 SLC13A5 Proteins 0.000 claims 3
- 101150118107 SLC13A5 gene Proteins 0.000 claims 3
- 102100035210 Solute carrier family 13 member 5 Human genes 0.000 claims 3
- 239000013607 AAV vector Substances 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 230000002159 abnormal effect Effects 0.000 claims 2
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 1
- 108010075600 citrate-binding transport protein Proteins 0.000 claims 1
- 230000007812 deficiency Effects 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- -1 expression cassette Substances 0.000 claims 1
- 210000005260 human cell Anatomy 0.000 claims 1
- 238000007913 intrathecal administration Methods 0.000 claims 1
- 238000007914 intraventricular administration Methods 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 210000000653 nervous system Anatomy 0.000 claims 1
- 208000024891 symptom Diseases 0.000 claims 1
- 239000013603 viral vector Substances 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063055795P | 2020-07-23 | 2020-07-23 | |
| US63/055,795 | 2020-07-23 | ||
| PCT/US2021/042930 WO2022020697A1 (en) | 2020-07-23 | 2021-07-23 | Optimized slc13a5 genes and expression cassettes and their use |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023536067A JP2023536067A (ja) | 2023-08-23 |
| JPWO2022020697A5 true JPWO2022020697A5 (https=) | 2024-07-09 |
| JP2023536067A5 JP2023536067A5 (https=) | 2024-07-09 |
Family
ID=79728993
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023503171A Pending JP2023536067A (ja) | 2020-07-23 | 2021-07-23 | 最適化されたｓｌｃ１３ａ５遺伝子および発現カセットならびにそれらの使用 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20230285595A1 (https=) |
| EP (2) | EP4185700B1 (https=) |
| JP (1) | JP2023536067A (https=) |
| CA (1) | CA3186207A1 (https=) |
| ES (1) | ES3057565T3 (https=) |
| WO (1) | WO2022020697A1 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20240342314A1 (en) * | 2021-09-30 | 2024-10-17 | The Board Of Regents Of The University Of Texas System | Slc13a5 gene therapy vectors and uses thereof |
Family Cites Families (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4501729A (en) | 1982-12-13 | 1985-02-26 | Research Corporation | Aerosolized amiloride treatment of retained pulmonary secretions |
| US5399346A (en) | 1989-06-14 | 1995-03-21 | The United States Of America As Represented By The Department Of Health And Human Services | Gene therapy |
| US5587308A (en) * | 1992-06-02 | 1996-12-24 | The United States Of America As Represented By The Department Of Health & Human Services | Modified adeno-associated virus vector capable of expression from a novel promoter |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US6040183A (en) | 1995-06-07 | 2000-03-21 | University Of North Carloina At Chapel Hill | Helper virus-free AAV production |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| CA2265460A1 (en) | 1996-09-11 | 1998-03-19 | The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services | Aav4 vector and uses thereof |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| EP1082413B1 (en) | 1998-05-28 | 2008-07-23 | THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES | Aav5 vector and uses thereof |
| AU774706B2 (en) | 1998-09-22 | 2004-07-08 | Johns Hopkins University, The | Methods for large-scale production of recombinant AAV vectors |
| CA2349838C (en) | 1998-11-05 | 2011-06-07 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| ES2340230T3 (es) | 1998-11-10 | 2010-05-31 | University Of North Carolina At Chapel Hill | Vectores viricos y sus procedimientos de preparacion y administracion. |
| NZ522840A (en) * | 2000-06-01 | 2004-12-24 | Univ North Carolina | A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together |
| US7201898B2 (en) | 2000-06-01 | 2007-04-10 | The University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| AU2003298675A1 (en) * | 2002-11-22 | 2004-06-18 | You-Jun Fei | NaCT AS A TARGET FOR LIFESPAN EXPANSION AND WEIGHT REDUCTION |
| AU2013243948A1 (en) * | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins associated with human disease |
| US9636370B2 (en) | 2012-09-28 | 2017-05-02 | The University Of North Carolina At Chapel Hill | AAV vectors targeted to oligodendrocytes |
| WO2016081811A1 (en) | 2014-11-21 | 2016-05-26 | The University Of North Carolina At Chapel Hill | Aav vectors targeted to the central nervous system |
| EP3443001B1 (en) * | 2016-04-11 | 2025-04-30 | Obsidian Therapeutics, Inc. | REGULATED BIOCIRCUIT SYSTEMS |
| RU2764919C2 (ru) * | 2016-06-13 | 2022-01-24 | Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл | Оптимизированные гены и экспрессионные кассеты cln1, и их применение |
| KR102709597B1 (ko) * | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| US20240342314A1 (en) * | 2021-09-30 | 2024-10-17 | The Board Of Regents Of The University Of Texas System | Slc13a5 gene therapy vectors and uses thereof |
-
2021
- 2021-07-23 EP EP21845857.8A patent/EP4185700B1/en active Active
- 2021-07-23 CA CA3186207A patent/CA3186207A1/en active Pending
- 2021-07-23 ES ES21845857T patent/ES3057565T3/es active Active
- 2021-07-23 US US18/006,278 patent/US20230285595A1/en active Pending
- 2021-07-23 EP EP25214109.8A patent/EP4700126A2/en active Pending
- 2021-07-23 WO PCT/US2021/042930 patent/WO2022020697A1/en not_active Ceased
- 2021-07-23 JP JP2023503171A patent/JP2023536067A/ja active Pending
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