JOP20190164B1 - تركيبات وطرق لعلاج داء فاربر - Google Patents
تركيبات وطرق لعلاج داء فاربرInfo
- Publication number
- JOP20190164B1 JOP20190164B1 JOP/2019/0164A JOP20190164A JOP20190164B1 JO P20190164 B1 JOP20190164 B1 JO P20190164B1 JO P20190164 A JOP20190164 A JO P20190164A JO P20190164 B1 JOP20190164 B1 JO P20190164B1
- Authority
- JO
- Jordan
- Prior art keywords
- compositions
- methods
- farber disease
- treating farber
- treating
- Prior art date
Links
- 208000001948 Farber Lipogranulomatosis Diseases 0.000 title abstract 2
- 208000033149 Farber disease Diseases 0.000 title abstract 2
- 239000000203 mixture Substances 0.000 title abstract 2
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/50—Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/52—Genes encoding for enzymes or proenzymes
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
- C12N9/80—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5) acting on amide bonds in linear amides (3.5.1)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/01—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in linear amides (3.5.1)
- C12Y305/01023—Ceramidase (3.5.1.23)
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Virology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Enzymes And Modification Thereof (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762446166P | 2017-01-13 | 2017-01-13 | |
| PCT/US2018/013509 WO2018132667A1 (en) | 2017-01-13 | 2018-01-12 | Compositions and methods for treating farber disease |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JOP20190164A1 JOP20190164A1 (ar) | 2019-07-02 |
| JOP20190164B1 true JOP20190164B1 (ar) | 2023-09-17 |
Family
ID=62839661
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JOP/2019/0164A JOP20190164B1 (ar) | 2017-01-13 | 2018-01-12 | تركيبات وطرق لعلاج داء فاربر |
Country Status (8)
| Country | Link |
|---|---|
| US (4) | US20190343936A1 (enExample) |
| EP (2) | EP3568154B1 (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 |
|---|---|---|---|---|
| JP7576293B2 (ja) * | 2017-01-13 | 2024-10-31 | アイカーン スクール オブ メディシン アット マウント サイナイ | ファーバー病を治療するための組成物及び方法 |
| WO2019009979A1 (en) * | 2017-07-06 | 2019-01-10 | The Medical College Of Wisconsin, Inc. | NEW IN VITRO AND IN VIVO ENRICHMENT STRATEGY TARGETING CSH-DERIVED LYMPHOCYTES TRANSDUCED BY A VECTOR FOR DISEASE THERAPY |
| US20190240298A1 (en) * | 2018-02-02 | 2019-08-08 | Enzyvant Farber Gmbh | Methods for treating farber disease |
| WO2019210301A1 (en) | 2018-04-27 | 2019-10-31 | 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 |
|---|---|---|---|---|
| EP3679923B1 (en) * | 2012-06-01 | 2021-08-04 | Icahn School of Medicine at Mount Sinai | Ceramide levels in the treatment and prevention of infections |
| EP3659619A1 (en) * | 2013-03-14 | 2020-06-03 | Icahn School of Medicine at Mount Sinai | Therapeutic acid ceramidase compositions and methods of making and using them |
| JP7576293B2 (ja) * | 2017-01-13 | 2024-10-31 | アイカーン スクール オブ メディシン アット マウント サイナイ | ファーバー病を治療するための組成物及び方法 |
| WO2019060837A1 (en) * | 2017-09-25 | 2019-03-28 | Enzyvant Farber Gmbh | PROCESS FOR PRODUCING RECOMBINANT HUMAN ACID CERAMIDASE |
| US20190240298A1 (en) * | 2018-02-02 | 2019-08-08 | Enzyvant Farber Gmbh | Methods for treating farber disease |
-
2018
- 2018-01-12 JP JP2019538173A patent/JP7576293B2/ja active Active
- 2018-01-12 US US16/477,710 patent/US20190343936A1/en not_active Abandoned
- 2018-01-12 AU AU2018207564A patent/AU2018207564A1/en not_active Abandoned
- 2018-01-12 WO PCT/US2018/013509 patent/WO2018132667A1/en not_active Ceased
- 2018-01-12 JO JOP/2019/0164A patent/JOP20190164B1/ar active
- 2018-01-12 MX MX2019008038A patent/MX2019008038A/es unknown
- 2018-01-12 EP EP18739237.8A patent/EP3568154B1/en active Active
- 2018-01-12 EP EP23184825.0A patent/EP4295903A3/en active Pending
- 2018-01-12 CA CA3049771A patent/CA3049771A1/en active Pending
-
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
Also Published As
| Publication number | Publication date |
|---|---|
| EP3568154A1 (en) | 2019-11-20 |
| US20250170225A1 (en) | 2025-05-29 |
| JP2020514305A (ja) | 2020-05-21 |
| JP7576293B2 (ja) | 2024-10-31 |
| WO2018132667A1 (en) | 2018-07-19 |
| CA3049771A1 (en) | 2018-07-19 |
| JP2024169509A (ja) | 2024-12-05 |
| EP4295903A3 (en) | 2024-03-27 |
| EP3568154A4 (en) | 2020-11-18 |
| JP2023011938A (ja) | 2023-01-24 |
| EP4295903A2 (en) | 2023-12-27 |
| JOP20190164A1 (ar) | 2019-07-02 |
| AU2018207564A1 (en) | 2019-08-08 |
| MX2019008038A (es) | 2020-02-05 |
| US20240075113A1 (en) | 2024-03-07 |
| AU2025200254A1 (en) | 2025-01-30 |
| US20190343936A1 (en) | 2019-11-14 |
| US20220313800A1 (en) | 2022-10-06 |
| EP3568154B1 (en) | 2023-07-12 |
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