JP2020514305A5 - - Google Patents
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- Publication number
- JP2020514305A5 JP2020514305A5 JP2019538173A JP2019538173A JP2020514305A5 JP 2020514305 A5 JP2020514305 A5 JP 2020514305A5 JP 2019538173 A JP2019538173 A JP 2019538173A JP 2019538173 A JP2019538173 A JP 2019538173A JP 2020514305 A5 JP2020514305 A5 JP 2020514305A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- composition according
- subject
- effective amount
- disease
- 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
- 238000000034 method Methods 0.000 claims description 59
- 239000008194 pharmaceutical composition Substances 0.000 claims description 57
- 208000001948 Farber Lipogranulomatosis Diseases 0.000 claims description 18
- 230000002378 acidificating effect Effects 0.000 claims description 14
- 102000004201 Ceramidases Human genes 0.000 claims description 10
- 108090000751 Ceramidases Proteins 0.000 claims description 10
- 239000013598 vector Substances 0.000 claims description 10
- YDNKGFDKKRUKPY-JHOUSYSJSA-N C16 ceramide Natural products CCCCCCCCCCCCCCCC(=O)N[C@@H](CO)[C@H](O)C=CCCCCCCCCCCCCC YDNKGFDKKRUKPY-JHOUSYSJSA-N 0.000 claims description 5
- CRJGESKKUOMBCT-VQTJNVASSA-N N-acetylsphinganine Chemical compound CCCCCCCCCCCCCCC[C@@H](O)[C@H](CO)NC(C)=O CRJGESKKUOMBCT-VQTJNVASSA-N 0.000 claims description 5
- 229940106189 ceramide Drugs 0.000 claims description 5
- ZVEQCJWYRWKARO-UHFFFAOYSA-N ceramide Natural products CCCCCCCCCCCCCCC(O)C(=O)NC(CO)C(O)C=CCCC=C(C)CCCCCCCCC ZVEQCJWYRWKARO-UHFFFAOYSA-N 0.000 claims description 5
- VVGIYYKRAMHVLU-UHFFFAOYSA-N newbouldiamide Natural products CCCCCCCCCCCCCCCCCCCC(O)C(O)C(O)C(CO)NC(=O)CCCCCCCCCCCCCCCCC VVGIYYKRAMHVLU-UHFFFAOYSA-N 0.000 claims description 5
- WWUZIQQURGPMPG-UHFFFAOYSA-N (-)-D-erythro-Sphingosine Natural products CCCCCCCCCCCCCC=CC(O)C(N)CO WWUZIQQURGPMPG-UHFFFAOYSA-N 0.000 claims description 4
- WWUZIQQURGPMPG-KRWOKUGFSA-N sphingosine Chemical compound CCCCCCCCCCCCC\C=C\[C@@H](O)[C@@H](N)CO WWUZIQQURGPMPG-KRWOKUGFSA-N 0.000 claims description 4
- 210000000952 spleen Anatomy 0.000 claims description 4
- 239000003636 conditioned culture medium Substances 0.000 claims description 3
- 241000699802 Cricetulus griseus Species 0.000 claims description 2
- 210000001672 ovary Anatomy 0.000 claims description 2
- 239000013612 plasmid Substances 0.000 claims description 2
- 239000013603 viral vector Substances 0.000 claims description 2
- 208000033149 Farber disease Diseases 0.000 claims 4
- 101000975753 Homo sapiens Acid ceramidase Proteins 0.000 claims 3
- 102000047408 human ASAH1 Human genes 0.000 claims 3
- 206010018691 Granuloma Diseases 0.000 claims 2
- 239000000243 solution Substances 0.000 claims 2
- 230000003442 weekly effect Effects 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 1
- 229940079593 drug Drugs 0.000 claims 1
- 239000007928 intraperitoneal injection Substances 0.000 claims 1
- 238000010253 intravenous injection Methods 0.000 claims 1
- 239000000203 mixture Substances 0.000 claims 1
- 206010049940 Lipogranuloma Diseases 0.000 description 2
- 238000001802 infusion Methods 0.000 description 2
- 238000007912 intraperitoneal administration Methods 0.000 description 2
- 238000001990 intravenous administration Methods 0.000 description 2
- 239000000126 substance Substances 0.000 description 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022181240A JP2023011938A (ja) | 2017-01-13 | 2022-11-11 | ファーバー病を治療するための組成物及び方法 |
| JP2024159770A JP2024169509A (ja) | 2017-01-13 | 2024-09-17 | ファーバー病を治療するための組成物及び方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762446166P | 2017-01-13 | 2017-01-13 | |
| US62/446,166 | 2017-01-13 | ||
| PCT/US2018/013509 WO2018132667A1 (en) | 2017-01-13 | 2018-01-12 | Compositions and methods for treating farber disease |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022181240A Division JP2023011938A (ja) | 2017-01-13 | 2022-11-11 | ファーバー病を治療するための組成物及び方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020514305A JP2020514305A (ja) | 2020-05-21 |
| JP2020514305A5 true JP2020514305A5 (enExample) | 2021-02-12 |
| JP7576293B2 JP7576293B2 (ja) | 2024-10-31 |
Family
ID=62839661
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019538173A Active JP7576293B2 (ja) | 2017-01-13 | 2018-01-12 | ファーバー病を治療するための組成物及び方法 |
| JP2022181240A Pending JP2023011938A (ja) | 2017-01-13 | 2022-11-11 | ファーバー病を治療するための組成物及び方法 |
| JP2024159770A Pending JP2024169509A (ja) | 2017-01-13 | 2024-09-17 | ファーバー病を治療するための組成物及び方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022181240A Pending JP2023011938A (ja) | 2017-01-13 | 2022-11-11 | ファーバー病を治療するための組成物及び方法 |
| JP2024159770A Pending JP2024169509A (ja) | 2017-01-13 | 2024-09-17 | ファーバー病を治療するための組成物及び方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (4) | US20190343936A1 (enExample) |
| EP (2) | EP4295903A3 (enExample) |
| JP (3) | JP7576293B2 (enExample) |
| AU (2) | AU2018207564A1 (enExample) |
| CA (1) | CA3049771A1 (enExample) |
| JO (1) | JOP20190164B1 (enExample) |
| MX (1) | MX2019008038A (enExample) |
| WO (1) | WO2018132667A1 (enExample) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2018207564A1 (en) * | 2017-01-13 | 2019-08-08 | Icahn School Of Medicine At Mount Sinai | Compositions and methods for treating farber disease |
| US11597917B2 (en) * | 2017-07-06 | 2023-03-07 | The Medical College Of Wisconsin, Inc. | In vitro and in vivo enrichment strategy targeting lymphocytes derived from vector transduced HSCs for therapy of disorders |
| BR112020015003A2 (pt) | 2018-02-02 | 2020-12-29 | Enzyvant Therapeutics Gmbh | Métodos para o tratamento da doença de farber |
| EP4285921B1 (en) | 2018-04-27 | 2025-10-08 | The Medical College of Wisconsin, Inc. | Use of lentivector-transduced t-rapa cells for amelioration of lysosomal storage disorders |
| WO2020152532A1 (en) | 2019-01-23 | 2020-07-30 | Enzyvant Therapeutics Gmbh | Method of ameliorating a pro-inflammatory immunophenotype in farber disease subjects by repeated administration of a recombinant human acid ceramidase |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| LT2854910T (lt) * | 2012-06-01 | 2020-05-25 | Icahn School Of Medicine At Mount Sinai | Ceramido lygiai infekcijos gydymo ir prevencijos metu |
| HUE046113T2 (hu) * | 2013-03-14 | 2020-02-28 | Icahn School Med Mount Sinai | Terápiás sav ceramidáz készítmények és azok elõállítási és felhasználási eljárásai |
| AU2018207564A1 (en) * | 2017-01-13 | 2019-08-08 | Icahn School Of Medicine At Mount Sinai | Compositions and methods for treating farber disease |
| WO2019060837A1 (en) * | 2017-09-25 | 2019-03-28 | Enzyvant Farber Gmbh | PROCESS FOR PRODUCING RECOMBINANT HUMAN ACID CERAMIDASE |
| BR112020015003A2 (pt) * | 2018-02-02 | 2020-12-29 | Enzyvant Therapeutics Gmbh | Métodos para o tratamento da doença de farber |
-
2018
- 2018-01-12 AU AU2018207564A patent/AU2018207564A1/en not_active Abandoned
- 2018-01-12 JP JP2019538173A patent/JP7576293B2/ja active Active
- 2018-01-12 EP EP23184825.0A patent/EP4295903A3/en active Pending
- 2018-01-12 WO PCT/US2018/013509 patent/WO2018132667A1/en not_active Ceased
- 2018-01-12 MX MX2019008038A patent/MX2019008038A/es unknown
- 2018-01-12 JO JOP/2019/0164A patent/JOP20190164B1/ar active
- 2018-01-12 US US16/477,710 patent/US20190343936A1/en not_active Abandoned
- 2018-01-12 CA CA3049771A patent/CA3049771A1/en active Pending
- 2018-01-12 EP EP18739237.8A patent/EP3568154B1/en active Active
-
2022
- 2022-05-31 US US17/828,131 patent/US20220313800A1/en not_active Abandoned
- 2022-11-11 JP JP2022181240A patent/JP2023011938A/ja active Pending
-
2023
- 2023-11-13 US US18/507,791 patent/US20240075113A1/en not_active Abandoned
-
2024
- 2024-06-26 US US18/755,376 patent/US20250170225A1/en active Pending
- 2024-09-17 JP JP2024159770A patent/JP2024169509A/ja active Pending
-
2025
- 2025-01-14 AU AU2025200254A patent/AU2025200254A1/en active Pending
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