DK3445451T3 - Sammensætninger og fremgangsmåder til behandling af lysosomale lagringssygdomme og sygdomme kendetegnet ved lysosomal dysfunktion - Google Patents
Sammensætninger og fremgangsmåder til behandling af lysosomale lagringssygdomme og sygdomme kendetegnet ved lysosomal dysfunktion Download PDFInfo
- Publication number
- DK3445451T3 DK3445451T3 DK17786739.7T DK17786739T DK3445451T3 DK 3445451 T3 DK3445451 T3 DK 3445451T3 DK 17786739 T DK17786739 T DK 17786739T DK 3445451 T3 DK3445451 T3 DK 3445451T3
- Authority
- DK
- Denmark
- Prior art keywords
- lysosomal
- diseases
- compositions
- treatment
- methods
- Prior art date
Links
- 208000015439 Lysosomal storage disease Diseases 0.000 title 2
- 208000033868 Lysosomal disease Diseases 0.000 title 1
- 230000004064 dysfunction Effects 0.000 title 1
- 230000002132 lysosomal effect Effects 0.000 title 1
- 238000000034 method Methods 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7016—Disaccharides, e.g. lactose, lactulose
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/4353—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
- A61K31/4375—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a six-membered ring having nitrogen as a ring heteroatom, e.g. quinolizines, naphthyridines, berberine, vincamine
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
- A61K31/445—Non condensed piperidines, e.g. piperocaine
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Organic Chemistry (AREA)
- Physiology (AREA)
- Nutrition Science (AREA)
- Diabetes (AREA)
- Hospice & Palliative Care (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Psychiatry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662325535P | 2016-04-21 | 2016-04-21 | |
| US201762475295P | 2017-03-23 | 2017-03-23 | |
| PCT/US2017/028904 WO2017185010A1 (en) | 2016-04-21 | 2017-04-21 | Compositions and methods for the treatment of lysosomal storage disorders and disorders characterized by lysosomal dysfunction |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK3445451T3 true DK3445451T3 (da) | 2021-11-08 |
Family
ID=60089338
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK17786739.7T DK3445451T3 (da) | 2016-04-21 | 2017-04-21 | Sammensætninger og fremgangsmåder til behandling af lysosomale lagringssygdomme og sygdomme kendetegnet ved lysosomal dysfunktion |
Country Status (9)
| Country | Link |
|---|---|
| US (3) | US10512656B2 (da) |
| EP (1) | EP3445451B1 (da) |
| JP (1) | JP7302871B2 (da) |
| CN (1) | CN109789316B (da) |
| AU (1) | AU2017252563B2 (da) |
| DK (1) | DK3445451T3 (da) |
| MX (1) | MX388562B (da) |
| NZ (1) | NZ748599A (da) |
| WO (1) | WO2017185010A1 (da) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6837835B2 (ja) | 2013-05-07 | 2021-03-03 | バイオ・ブラスト・ファーマ・リミテッドＢｉｏ Ｂｌａｓｔ Ｐｈａｒｍａ Ｌｔｄ． | トレハロースの非経口投与によるタンパク質凝集ミオパシーおよび神経変性疾患の治療 |
| AU2016342027B2 (en) | 2015-10-23 | 2021-05-13 | Navitor Pharmaceuticals, Inc. | Modulators of Sestrin-GATOR2 interaction and uses thereof |
| NZ748599A (en) | 2016-04-21 | 2022-08-26 | Baylor College Medicine | Compositions and methods for the treatment of lysosomal storage disorders and disorders characterized by lysosomal dysfunction |
| TWI776886B (zh) | 2017-04-26 | 2022-09-11 | 美商奈維特製藥公司 | Sestrin-gator2交互作用之調節劑及其用途 |
| JP7644002B2 (ja) | 2018-10-24 | 2025-03-11 | ナビター ファーマシューティカルズ， インコーポレイテッド | 多形化合物およびその使用 |
| JP7712673B2 (ja) | 2018-12-05 | 2025-07-24 | ワシントン・ユニバーシティ | 神経の疾患を検出し、予防し、回復に向かわせ、治療する方法 |
| US20220211813A1 (en) * | 2019-04-12 | 2022-07-07 | Loma Linda University | Methods for treatment of niemann-pick disease type c |
| US12612380B2 (en) | 2019-05-10 | 2026-04-28 | Alectos Therapeutics 1Nc | Non-lysosomal glucosylceramidase inhibitors and uses thereof |
| MX2022002652A (es) * | 2019-09-09 | 2022-04-06 | Univ Pittsburgh Commonwealth Sys Higher Education | Metodos para restaurar la funcion lisosomal de celulas epiteliales de pigmento retiniano mediante activacion de tfeb. |
| JP2022550570A (ja) * | 2019-10-01 | 2022-12-02 | シーロス セラピューティクス， インコーポレイテッド | トレハロース製剤およびその使用 |
| EP4051259A4 (en) | 2019-11-01 | 2023-11-22 | Navitor Pharmaceuticals, Inc. | METHOD OF TREATMENT USING A MMOC1 MODULATOR |
| US12528773B2 (en) | 2020-05-07 | 2026-01-20 | Alectos Therapeutics Inc. | Non-lysosomal glucosylceramidase inhibitors and uses thereof |
| EP3944858A1 (en) | 2020-07-30 | 2022-02-02 | Beyond Batten Disease Foundation | Combination product containing trehalose and miglustat for treating lysosomal diseases |
| US12019081B2 (en) | 2020-11-06 | 2024-06-25 | University Of Kentucky Research Foundation | Method for detection and quantification of CLN3 protein |
| KR102580852B1 (ko) * | 2021-04-13 | 2023-09-20 | 강원대학교 산학협력단 | 발리다마이신 a를 포함하는 뇌전증 완화 및 치료용 조성물 |
| EP4465990A4 (en) * | 2022-01-20 | 2026-04-08 | Univ Johns Hopkins | LYSOSOMAL DYSFUNCTION IN NEUROLOGICAL AND PSYCHIATRIC DISORDERS |
| CN120187429A (zh) * | 2022-04-22 | 2025-06-20 | 安斯泰来工程小分子美国股份有限公司 | 转录因子eb激活因子及其用途 |
| CN116819066A (zh) * | 2023-06-30 | 2023-09-29 | 复旦大学附属中山医院 | 肝癌微环境检测试剂盒 |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0693558B1 (en) * | 1994-07-19 | 2002-12-04 | Kabushiki Kaisha Hayashibara Seibutsu Kagaku Kenkyujo | Trehalose and its production and use |
| AU2006247065B8 (en) | 2005-05-17 | 2012-07-12 | Amicus Therapeutics, Inc. | A method for the treatment of Pompe disease using 1-deoxynojirimycin derivatives |
| GB0712494D0 (en) | 2007-06-27 | 2007-08-08 | Isis Innovation | Substrate reduction therapy |
| EP2475376B1 (en) * | 2009-06-17 | 2016-03-30 | BioMarin Pharmaceutical Inc. | Formulations for lysosomal enzymes |
| US20130312784A1 (en) | 2012-05-24 | 2013-11-28 | For Your Ease Only, Inc. | Cosmetic organizer |
| WO2014089449A1 (en) * | 2012-12-07 | 2014-06-12 | Rush University Nedical Center | Composition and method for treating neuronal ceroid lipofuscinosis |
| WO2014110270A1 (en) | 2013-01-09 | 2014-07-17 | Amicus Therapeutics, Inc. | Stable parenteral dnj compositions |
| JP6837835B2 (ja) | 2013-05-07 | 2021-03-03 | バイオ・ブラスト・ファーマ・リミテッドＢｉｏ Ｂｌａｓｔ Ｐｈａｒｍａ Ｌｔｄ． | トレハロースの非経口投与によるタンパク質凝集ミオパシーおよび神経変性疾患の治療 |
| US9084720B2 (en) | 2013-05-07 | 2015-07-21 | BioBlast Pharma Ltd. | Compositions and methods for treating oculopharyngeal muscular dystrophy |
| JP6130224B2 (ja) * | 2013-05-27 | 2017-05-17 | 公益財団法人微生物化学研究会 | 新規化合物レンツトレハロース、その製造方法、及びその用途、並びに、新規微生物 |
| US20160136123A1 (en) * | 2013-06-14 | 2016-05-19 | Vojo P. Deretic | Treatment of autophagy-related disorders |
| CA2933554A1 (en) | 2013-12-12 | 2015-06-18 | The Brigham And Women's Hospital, Inc. | Treating neurodegenerative disease |
| WO2015097088A1 (en) * | 2013-12-23 | 2015-07-02 | Bcn Peptides, S.A. | Bicalutamide analogs or (s)-bi calutam ide as exocytosis activating compounds for use in the treatment of a lysosomal storage disorder or glycogenosis |
| CA3012502A1 (en) | 2016-02-08 | 2017-08-17 | Junaxo, Inc. | Use of trehalose for treatment of neurological diseases |
| NZ748599A (en) | 2016-04-21 | 2022-08-26 | Baylor College Medicine | Compositions and methods for the treatment of lysosomal storage disorders and disorders characterized by lysosomal dysfunction |
| EP3318277A1 (en) | 2016-11-04 | 2018-05-09 | Institut du Cerveau et de la Moelle Epiniere-ICM | Inhibitors of glucosylceramide synthase for the treatment of motor neuron diseases |
-
2017
- 2017-04-21 NZ NZ748599A patent/NZ748599A/en unknown
- 2017-04-21 EP EP17786739.7A patent/EP3445451B1/en active Active
- 2017-04-21 CN CN201780038419.8A patent/CN109789316B/zh active Active
- 2017-04-21 JP JP2019531851A patent/JP7302871B2/ja active Active
- 2017-04-21 MX MX2018012902A patent/MX388562B/es unknown
- 2017-04-21 US US15/494,136 patent/US10512656B2/en active Active
- 2017-04-21 DK DK17786739.7T patent/DK3445451T3/da active
- 2017-04-21 WO PCT/US2017/028904 patent/WO2017185010A1/en not_active Ceased
- 2017-04-21 AU AU2017252563A patent/AU2017252563B2/en active Active
-
2019
- 2019-04-04 US US16/375,521 patent/US20200000832A1/en not_active Abandoned
- 2019-04-04 US US16/375,445 patent/US11083741B2/en active Active
Also Published As
| Publication number | Publication date |
|---|---|
| BR112018071684A2 (pt) | 2019-02-19 |
| CA3021846A1 (en) | 2017-10-26 |
| WO2017185010A1 (en) | 2017-10-26 |
| MX2018012902A (es) | 2019-06-06 |
| JP2021517552A (ja) | 2021-07-26 |
| EP3445451B1 (en) | 2021-08-18 |
| NZ748599A (en) | 2022-08-26 |
| EP3445451A1 (en) | 2019-02-27 |
| US11083741B2 (en) | 2021-08-10 |
| AU2017252563B2 (en) | 2022-07-21 |
| US20200000832A1 (en) | 2020-01-02 |
| US20190321383A1 (en) | 2019-10-24 |
| CN109789316A (zh) | 2019-05-21 |
| AU2017252563A1 (en) | 2018-12-06 |
| CN109789316B (zh) | 2022-09-27 |
| JP7302871B2 (ja) | 2023-07-04 |
| MX388562B (es) | 2025-03-20 |
| US10512656B2 (en) | 2019-12-24 |
| EP3445451A4 (en) | 2019-12-18 |
| US20170304339A1 (en) | 2017-10-26 |
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