LT4021575T - Cln2 ligos gydymo būdai pediatriniams pacientams - Google Patents
Cln2 ligos gydymo būdai pediatriniams pacientamsInfo
- Publication number
- LT4021575T LT4021575T LTEPPCT/US2020/048704T LTUS2020048704T LT4021575T LT 4021575 T LT4021575 T LT 4021575T LT US2020048704 T LTUS2020048704 T LT US2020048704T LT 4021575 T LT4021575 T LT 4021575T
- Authority
- LT
- Lithuania
- 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)
- Veterinary Medicine (AREA)
- Public Health (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)
- General Chemical & Material Sciences (AREA)
- Gastroenterology & Hepatology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Zoology (AREA)
- Genetics & Genomics (AREA)
- Inorganic Chemistry (AREA)
- Wood Science & Technology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Psychology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (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 (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962893535P | 2019-08-29 | 2019-08-29 | |
| 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 |
|---|---|
| LT4021575T true LT4021575T (lt) | 2024-08-12 |
Family
ID=72470612
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| LTEPPCT/US2020/048704T LT4021575T (lt) | 2019-08-29 | 2020-08-31 | Cln2 ligos gydymo būdai pediatriniams pacientams |
Country Status (19)
| Country | Link |
|---|---|
| US (1) | US20220273775A1 (lt) |
| EP (1) | EP4021575B1 (lt) |
| JP (1) | JP2022546042A (lt) |
| KR (1) | KR20220054375A (lt) |
| CN (1) | CN114828960A (lt) |
| AR (1) | AR119871A1 (lt) |
| AU (1) | AU2020336984A1 (lt) |
| BR (1) | BR112022003598A2 (lt) |
| CA (1) | CA3152521A1 (lt) |
| CL (1) | CL2022000496A1 (lt) |
| DK (1) | DK4021575T3 (lt) |
| FI (1) | FI4021575T3 (lt) |
| HR (1) | HRP20220370A1 (lt) |
| IL (1) | IL290763A (lt) |
| LT (1) | LT4021575T (lt) |
| MX (1) | MX2022002451A (lt) |
| PT (1) | PT4021575T (lt) |
| TW (1) | TW202122106A (lt) |
| WO (1) | WO2021042020A1 (lt) |
Family Cites Families (7)
| 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 |
| 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 |
| KR20180067684A (ko) * | 2015-10-23 | 2018-06-20 | 유니버시티 오브 아이오와 리써치 파운데이션 | 인지 보호를 제공하면서 질환 개시 및 진행을 지연시키기 위해 유전자 요법을 이용하는 신경퇴행성 질환의 치료 방법 |
| MX2021005517A (es) * | 2018-11-14 | 2021-06-18 | Regenxbio Inc | Terapia genica para lipofuscinosis neuronal ceroidea. |
-
2020
- 2020-08-31 KR KR1020227010387A patent/KR20220054375A/ko unknown
- 2020-08-31 BR BR112022003598A patent/BR112022003598A2/pt unknown
- 2020-08-31 AR ARP200102442A patent/AR119871A1/es unknown
- 2020-08-31 DK DK20771408.0T patent/DK4021575T3/da active
- 2020-08-31 MX MX2022002451A patent/MX2022002451A/es unknown
- 2020-08-31 JP JP2022513165A patent/JP2022546042A/ja active Pending
- 2020-08-31 LT LTEPPCT/US2020/048704T patent/LT4021575T/lt unknown
- 2020-08-31 TW TW109129724A patent/TW202122106A/zh unknown
- 2020-08-31 CA CA3152521A patent/CA3152521A1/en active Pending
- 2020-08-31 PT PT207714080T patent/PT4021575T/pt unknown
- 2020-08-31 FI FIEP20771408.0T patent/FI4021575T3/fi active
- 2020-08-31 CN CN202080068971.3A patent/CN114828960A/zh active Pending
- 2020-08-31 EP EP20771408.0A patent/EP4021575B1/en active Active
- 2020-08-31 AU AU2020336984A patent/AU2020336984A1/en active Pending
- 2020-08-31 HR HRP20220370AA patent/HRP20220370A1/hr unknown
- 2020-08-31 US US17/638,029 patent/US20220273775A1/en active Pending
- 2020-08-31 WO PCT/US2020/048704 patent/WO2021042020A1/en active Application Filing
-
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 |
|---|---|
| AR119871A1 (es) | 2022-01-19 |
| DK4021575T3 (da) | 2024-08-05 |
| CN114828960A (zh) | 2022-07-29 |
| CA3152521A1 (en) | 2021-03-04 |
| EP4021575B1 (en) | 2024-05-22 |
| EP4021575A1 (en) | 2022-07-06 |
| AU2020336984A1 (en) | 2022-03-10 |
| KR20220054375A (ko) | 2022-05-02 |
| IL290763A (en) | 2022-04-01 |
| US20220273775A1 (en) | 2022-09-01 |
| TW202122106A (zh) | 2021-06-16 |
| BR112022003598A2 (pt) | 2022-05-24 |
| FI4021575T3 (fi) | 2024-07-24 |
| HRP20220370A1 (hr) | 2022-09-16 |
| WO2021042020A1 (en) | 2021-03-04 |
| CL2022000496A1 (es) | 2022-10-21 |
| MX2022002451A (es) | 2022-06-02 |
| PT4021575T (pt) | 2024-07-30 |
| JP2022546042A (ja) | 2022-11-02 |
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