SI4021575T1 - Metode za zdravljenje bolezni CLN2 pri prediatričnih subjektih - Google Patents
Metode za zdravljenje bolezni CLN2 pri prediatričnih subjektihInfo
- Publication number
- SI4021575T1 SI4021575T1 SI202030455T SI202030455T SI4021575T1 SI 4021575 T1 SI4021575 T1 SI 4021575T1 SI 202030455 T SI202030455 T SI 202030455T SI 202030455 T SI202030455 T SI 202030455T SI 4021575 T1 SI4021575 T1 SI 4021575T1
- Authority
- SI
- Slovenia
- Prior art keywords
- methods
- pediatric subjects
- cln2 disease
- treating cln2
- treating
- Prior art date
Links
- 201000010099 disease Diseases 0.000 title 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title 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)
- A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
- A61K38/4813—Exopeptidases (3.4.11. to 3.4.19)
-
- 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
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/02—Inorganic compounds
-
- 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
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- 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
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
- A61K9/0024—Solid, semi-solid or solidifying implants, which are implanted or injected in body tissue
-
- 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
- A61K9/0048—Eye, e.g. artificial tears
-
- 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
- A61K9/0048—Eye, e.g. artificial tears
- A61K9/0051—Ocular inserts, ocular implants
-
- 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
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
-
- 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
- A61K9/06—Ointments; Bases therefor; Other semi-solid forms, e.g. creams, sticks, gels
-
- 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
- A61K9/08—Solutions
-
- 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
- A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
- A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
- C12Y304/14009—Tripeptidyl-peptidase I (3.4.14.9)
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Organic Chemistry (AREA)
- Dermatology (AREA)
- Ophthalmology & Optometry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- General Chemical & Material Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Inorganic Chemistry (AREA)
- Psychology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Zoology (AREA)
- Genetics & Genomics (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962893535P | 2019-08-29 | 2019-08-29 | |
| EP20771408.0A EP4021575B1 (en) | 2019-08-29 | 2020-08-31 | Methods for treating cln2 disease in pediatric subjects |
| PCT/US2020/048704 WO2021042020A1 (en) | 2019-08-29 | 2020-08-31 | Methods for treating cln2 disease in pediatric subjects |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SI4021575T1 true SI4021575T1 (sl) | 2024-10-30 |
Family
ID=72470612
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SI202030455T SI4021575T1 (sl) | 2019-08-29 | 2020-08-31 | Metode za zdravljenje bolezni CLN2 pri prediatričnih subjektih |
Country Status (25)
| Country | Link |
|---|---|
| US (1) | US20220273775A1 (sl) |
| EP (2) | EP4438120A3 (sl) |
| JP (1) | JP2022546042A (sl) |
| KR (1) | KR20220054375A (sl) |
| CN (1) | CN114828960A (sl) |
| AR (1) | AR119871A1 (sl) |
| AU (1) | AU2020336984A1 (sl) |
| BR (1) | BR112022003598A2 (sl) |
| CA (1) | CA3152521A1 (sl) |
| CL (1) | CL2022000496A1 (sl) |
| DK (1) | DK4021575T3 (sl) |
| ES (1) | ES2987156T3 (sl) |
| FI (1) | FI4021575T3 (sl) |
| HR (2) | HRP20220370A1 (sl) |
| HU (1) | HUE067135T2 (sl) |
| IL (1) | IL290763A (sl) |
| LT (1) | LT4021575T (sl) |
| MX (1) | MX2022002451A (sl) |
| PL (1) | PL4021575T3 (sl) |
| PT (1) | PT4021575T (sl) |
| RS (1) | RS65763B1 (sl) |
| SI (1) | SI4021575T1 (sl) |
| SM (1) | SMT202400315T1 (sl) |
| TW (1) | TW202122106A (sl) |
| WO (1) | WO2021042020A1 (sl) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20220323019A1 (en) * | 2021-04-02 | 2022-10-13 | Neuropace, Inc. | Systems and methods for obtaining a clinical response estimate biomarker using machine-learned models trained on implanted neurostimulator data |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6302685B1 (en) | 1997-09-16 | 2001-10-16 | University Of Medicine And Dentistry Of New Jersey | Human lysosomal protein and methods of its use |
| US20020006400A1 (en) * | 2000-05-11 | 2002-01-17 | Peter Lobel | Recombinant human CLN2 protein and methods of its production and use |
| US7442372B2 (en) | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| EP3875107A1 (en) * | 2010-06-25 | 2021-09-08 | Shire Human Genetic Therapies, Inc. | Pharmaceutical formulation comprising a replacement enzyme for a lysosomal enzyme for use in treating lysosomal storage disease intrathecally |
| WO2013096899A2 (en) | 2011-12-23 | 2013-06-27 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| TWI752907B (zh) * | 2015-05-08 | 2022-01-21 | 美商拜奧馬林製藥公司 | 用於治療cln2疾病之tpp1調配物及方法 |
| US20180236105A1 (en) * | 2015-10-23 | 2018-08-23 | University Of Iowa Research Foundation | Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection |
| IL315701A (en) * | 2018-11-14 | 2024-11-01 | Regenxbio Inc | Gene therapy for neuronal ceroid lipofuscinosis |
-
2020
- 2020-08-31 CA CA3152521A patent/CA3152521A1/en active Pending
- 2020-08-31 SI SI202030455T patent/SI4021575T1/sl unknown
- 2020-08-31 EP EP24177169.0A patent/EP4438120A3/en active Pending
- 2020-08-31 CN CN202080068971.3A patent/CN114828960A/zh active Pending
- 2020-08-31 PL PL20771408.0T patent/PL4021575T3/pl unknown
- 2020-08-31 RS RS20240792A patent/RS65763B1/sr unknown
- 2020-08-31 JP JP2022513165A patent/JP2022546042A/ja active Pending
- 2020-08-31 SM SM20240315T patent/SMT202400315T1/it unknown
- 2020-08-31 HR HRP20220370AA patent/HRP20220370A1/hr not_active Application Discontinuation
- 2020-08-31 EP EP20771408.0A patent/EP4021575B1/en active Active
- 2020-08-31 WO PCT/US2020/048704 patent/WO2021042020A1/en active Application Filing
- 2020-08-31 KR KR1020227010387A patent/KR20220054375A/ko active Pending
- 2020-08-31 MX MX2022002451A patent/MX2022002451A/es unknown
- 2020-08-31 HR HRP20240871TT patent/HRP20240871T1/hr unknown
- 2020-08-31 LT LTEPPCT/US2020/048704T patent/LT4021575T/lt unknown
- 2020-08-31 DK DK20771408.0T patent/DK4021575T3/da active
- 2020-08-31 HU HUE20771408A patent/HUE067135T2/hu unknown
- 2020-08-31 FI FIEP20771408.0T patent/FI4021575T3/fi active
- 2020-08-31 AR ARP200102442A patent/AR119871A1/es unknown
- 2020-08-31 US US17/638,029 patent/US20220273775A1/en not_active Abandoned
- 2020-08-31 BR BR112022003598A patent/BR112022003598A2/pt unknown
- 2020-08-31 TW TW109129724A patent/TW202122106A/zh unknown
- 2020-08-31 ES ES20771408T patent/ES2987156T3/es active Active
- 2020-08-31 PT PT207714080T patent/PT4021575T/pt unknown
- 2020-08-31 AU AU2020336984A patent/AU2020336984A1/en active Pending
-
2022
- 2022-02-21 IL IL290763A patent/IL290763A/en unknown
- 2022-02-28 CL CL2022000496A patent/CL2022000496A1/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| CL2022000496A1 (es) | 2022-10-21 |
| HRP20240871T1 (hr) | 2024-10-11 |
| US20220273775A1 (en) | 2022-09-01 |
| PL4021575T3 (pl) | 2024-09-23 |
| AU2020336984A1 (en) | 2022-03-10 |
| HRP20220370A1 (hr) | 2022-09-16 |
| EP4438120A2 (en) | 2024-10-02 |
| RS65763B1 (sr) | 2024-08-30 |
| ES2987156T3 (es) | 2024-11-14 |
| TW202122106A (zh) | 2021-06-16 |
| PT4021575T (pt) | 2024-07-30 |
| EP4021575A1 (en) | 2022-07-06 |
| EP4438120A3 (en) | 2024-12-11 |
| SMT202400315T1 (it) | 2024-09-16 |
| CN114828960A (zh) | 2022-07-29 |
| DK4021575T3 (da) | 2024-08-05 |
| KR20220054375A (ko) | 2022-05-02 |
| MX2022002451A (es) | 2022-06-02 |
| CA3152521A1 (en) | 2021-03-04 |
| FI4021575T3 (fi) | 2024-07-24 |
| LT4021575T (lt) | 2024-08-12 |
| AR119871A1 (es) | 2022-01-19 |
| EP4021575B1 (en) | 2024-05-22 |
| JP2022546042A (ja) | 2022-11-02 |
| BR112022003598A2 (pt) | 2022-05-24 |
| WO2021042020A1 (en) | 2021-03-04 |
| IL290763A (en) | 2022-04-01 |
| HUE067135T2 (hu) | 2024-10-28 |
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