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
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)
- Animal Behavior & Ethology (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Chemical & Material Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Nutrition Science (AREA)
- Physiology (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (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 (10)
| Country | Link |
|---|---|
| US (3) | US10512656B2 (da) |
| EP (1) | EP3445451B1 (da) |
| JP (1) | JP7302871B2 (da) |
| CN (1) | CN109789316B (da) |
| AU (1) | AU2017252563B2 (da) |
| CA (1) | CA3021846A1 (da) |
| DK (1) | DK3445451T3 (da) |
| MX (1) | MX2018012902A (da) |
| NZ (1) | NZ748599A (da) |
| WO (1) | WO2017185010A1 (da) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3445451B1 (en) | 2016-04-21 | 2021-08-18 | Baylor College of Medicine | Compositions and methods for the treatment of lysosomal storage disorders and disorders characterized by lysosomal dysfunction |
| US11345654B2 (en) * | 2018-10-24 | 2022-05-31 | Navitor Pharmaceuticals, Inc. | Polymorphic compounds and uses thereof |
| US20220211813A1 (en) * | 2019-04-12 | 2022-07-07 | Loma Linda University | Methods for treatment of niemann-pick disease type c |
| KR20220062577A (ko) * | 2019-09-09 | 2022-05-17 | 유니버시티 오브 피츠버그-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션 | Tfeb의 활성화에 의해 망막 색소 상피 세포의 리소좀 기능을 회복시키는 방법 |
| AU2020357800A1 (en) * | 2019-10-01 | 2022-04-14 | Seelos Therapeutics, Inc. | Trehalose formulations 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 |
| WO2023141606A2 (en) * | 2022-01-20 | 2023-07-27 | The Johns Hopkins University | Lysosomal dysfunction in neurological and psychiatric disorders |
| TW202400160A (zh) * | 2022-04-22 | 2024-01-01 | 美商米突倍基公司 | 轉錄因子eb活化子及其用途 |
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 |
| EP3782655A1 (en) | 2005-05-17 | 2021-02-24 | Amicus Therapeutics, Inc. | A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives |
| GB0712494D0 (en) | 2007-06-27 | 2007-08-08 | Isis Innovation | Substrate reduction therapy |
| ES2569514T3 (es) * | 2009-06-17 | 2016-05-11 | Biomarin Pharmaceutical Inc. | Formulaciones para enzimas lisosómicas |
| US20130312784A1 (en) | 2012-05-24 | 2013-11-28 | For Your Ease Only, Inc. | Cosmetic organizer |
| US9750712B2 (en) * | 2012-12-07 | 2017-09-05 | Rush University Medical Center | Composition and method for treating neuronal ceroid lipofuscinosis |
| WO2014110270A1 (en) | 2013-01-09 | 2014-07-17 | Amicus Therapeutics, Inc. | Stable parenteral dnj compositions |
| US9084720B2 (en) | 2013-05-07 | 2015-07-21 | BioBlast Pharma Ltd. | Compositions and methods for treating oculopharyngeal muscular dystrophy |
| EP2994145B1 (en) | 2013-05-07 | 2023-01-04 | Bio Blast Pharma Ltd. | Treatment of oculopharyngeal muscular dystrophy by intravenous administration of trehalose |
| 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 |
| WO2014200705A1 (en) * | 2013-06-14 | 2014-12-18 | Stc.Unm | Treatment of autophagy-related disorders |
| KR20160089528A (ko) | 2013-12-12 | 2016-07-27 | 더 브리검 앤드 우먼즈 하스피털, 인크. | 신경퇴행성 질환의 치료 |
| PL3086784T3 (pl) * | 2013-12-23 | 2019-09-30 | Bcn Peptides, S.A. | Analogi bikalutamidu lub (S)-bikalutamidu jako związki aktywujące egzocytozę do stosowania w leczeniu lizosomalnych chorób spichrzeniowych lub glikogenozy |
| US10736909B2 (en) | 2016-02-08 | 2020-08-11 | Junaxo, Inc. | Use of trehalose for treatment of neurological diseases |
| EP3445451B1 (en) | 2016-04-21 | 2021-08-18 | Baylor College of 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 EP EP17786739.7A patent/EP3445451B1/en active Active
- 2017-04-21 NZ NZ748599A patent/NZ748599A/en unknown
- 2017-04-21 AU AU2017252563A patent/AU2017252563B2/en active Active
- 2017-04-21 DK DK17786739.7T patent/DK3445451T3/da active
- 2017-04-21 JP JP2019531851A patent/JP7302871B2/ja active Active
- 2017-04-21 MX MX2018012902A patent/MX2018012902A/es unknown
- 2017-04-21 WO PCT/US2017/028904 patent/WO2017185010A1/en active Application Filing
- 2017-04-21 CA CA3021846A patent/CA3021846A1/en active Pending
- 2017-04-21 US US15/494,136 patent/US10512656B2/en active Active
- 2017-04-21 CN CN201780038419.8A patent/CN109789316B/zh active Active
-
2019
- 2019-04-04 US US16/375,445 patent/US11083741B2/en active Active
- 2019-04-04 US US16/375,521 patent/US20200000832A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| JP7302871B2 (ja) | 2023-07-04 |
| NZ748599A (en) | 2022-08-26 |
| CN109789316B (zh) | 2022-09-27 |
| US11083741B2 (en) | 2021-08-10 |
| US10512656B2 (en) | 2019-12-24 |
| US20200000832A1 (en) | 2020-01-02 |
| AU2017252563A1 (en) | 2018-12-06 |
| WO2017185010A1 (en) | 2017-10-26 |
| EP3445451A4 (en) | 2019-12-18 |
| US20170304339A1 (en) | 2017-10-26 |
| US20190321383A1 (en) | 2019-10-24 |
| EP3445451B1 (en) | 2021-08-18 |
| CN109789316A (zh) | 2019-05-21 |
| MX2018012902A (es) | 2019-06-06 |
| CA3021846A1 (en) | 2017-10-26 |
| EP3445451A1 (en) | 2019-02-27 |
| BR112018071684A2 (pt) | 2019-02-19 |
| AU2017252563B2 (en) | 2022-07-21 |
| JP2021517552A (ja) | 2021-07-26 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| DK3445451T3 (da) | Sammensætninger og fremgangsmåder til behandling af lysosomale lagringssygdomme og sygdomme kendetegnet ved lysosomal dysfunktion | |
| DK3102673T3 (da) | Fremgangsmåder og sammensætninger til behandling af beta-talassæmi | |
| DK3510157T3 (da) | Pd-1-homing-endonucleasevarianter, sammensætninger og fremgangsmåder til anvendelse | |
| DK3326641T3 (da) | Rna-holdig sammensætning til behandling af tumorsygdomme | |
| DK3185876T3 (da) | Sammensætninger og fremgangsmåder til behandling af neurologiske lidelser | |
| DK3119797T3 (da) | Raav-baserede sammensætninger og fremgangsmåder til behandling af amyotrofisk lateralsklerose | |
| DK3445388T3 (da) | Materialer og fremgangsmåder til behandling af hæmoglobinopatier | |
| DK3889145T3 (da) | 8-cyano-5-piperidino-quinoliner som TLR7/8-antagonister og anvendelser deraf til behandling af immunsygdomme | |
| DK3206497T3 (da) | Sammensætninger og fremgangsmåder til behandling af meibomsk kirtel-dysfunktion | |
| DK3212226T3 (da) | Sammensætninger og anvendelsesfremgangsmåder til behandling af metaboliske lidelser | |
| DK3600270T3 (da) | Forbindelser og sammensætninger til behandling af hæmatologiske lidelser | |
| DK3492632T3 (da) | Metoder til behandling af lithiumholdige materialer | |
| DK3240612T3 (da) | Fremgangsmåder til behandling af nethindesygdomme | |
| DK3481846T3 (da) | 11-substituerede 24-hydroxysteroler til anvendelse til behandling af nmda-relaterede tilstande | |
| DK3277270T3 (da) | Sammensætninger og fremgangsmåder til behandling af anæmi | |
| DK3393655T3 (da) | Fenfluramin-sammensætninger og fremgangsmåder til fremstilling af samme | |
| DK3189074T3 (da) | Sammensætninger og metoder til behandling og forebyggelse af inflammation | |
| DK3209696T3 (da) | Fremgangsmåder og sammensætninger til diagnosticering og behandling af glioblastom | |
| DK3081263T3 (da) | Sammensætninger og redskaber til behandling af glaukom | |
| DK3524255T3 (da) | Sammensætning til behandling af acne | |
| DK3157534T3 (da) | Acetylcholinesterasehæmmere til behandling af dermatologiske tilstande | |
| DK3142664T3 (da) | Sammensætninger og fremgangsmåder til behandling og diagnosticering af øjensygdomme | |
| DK3110446T3 (da) | Fremgangsmåder og sammensætninger til behandling af Siglec-8-associerede sygdomme | |
| DK3430143T3 (da) | Inhibitorer af SRSF1 til behandlingen af neurodegenerative sygdomme | |
| DK3200748T3 (da) | Sammensætninger og fremgangsmåder til behandling og profylakse af infektioner på operationssted |