HRP20240871T1 - Postupci liječenja cln2 bolesti kod pedijatrijskih subjekata - Google Patents
Postupci liječenja cln2 bolesti kod pedijatrijskih subjekata Download PDFInfo
- Publication number
- HRP20240871T1 HRP20240871T1 HRP20240871TT HRP20240871T HRP20240871T1 HR P20240871 T1 HRP20240871 T1 HR P20240871T1 HR P20240871T T HRP20240871T T HR P20240871TT HR P20240871 T HRP20240871 T HR P20240871T HR P20240871 T1 HRP20240871 T1 HR P20240871T1
- Authority
- HR
- Croatia
- Prior art keywords
- subject
- use according
- rhtpp1
- preparation
- administered
- Prior art date
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Classifications
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- 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 or 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)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Organic Chemistry (AREA)
- Ophthalmology & Optometry (AREA)
- Dermatology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- General Chemical & Material Sciences (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Wood Science & Technology (AREA)
- Genetics & Genomics (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Psychology (AREA)
- Inorganic Chemistry (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 |
|---|---|
| HRP20240871T1 true HRP20240871T1 (hr) | 2024-10-11 |
Family
ID=72470612
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20240871TT HRP20240871T1 (hr) | 2019-08-29 | 2020-08-31 | Postupci liječenja cln2 bolesti kod pedijatrijskih subjekata |
| HRP20220370AA HRP20220370A1 (hr) | 2019-08-29 | 2020-08-31 | Postupci liječenja cln2 bolesti kod pedijatrijskih subjekata |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20220370AA HRP20220370A1 (hr) | 2019-08-29 | 2020-08-31 | Postupci liječenja cln2 bolesti kod pedijatrijskih subjekata |
Country Status (25)
| Country | Link |
|---|---|
| US (1) | US20220273775A1 (https=) |
| EP (2) | EP4021575B1 (https=) |
| JP (2) | JP2022546042A (https=) |
| KR (1) | KR20220054375A (https=) |
| CN (1) | CN114828960A (https=) |
| AR (1) | AR119871A1 (https=) |
| AU (1) | AU2020336984B2 (https=) |
| BR (1) | BR112022003598A2 (https=) |
| CA (1) | CA3152521A1 (https=) |
| CL (1) | CL2022000496A1 (https=) |
| DK (1) | DK4021575T3 (https=) |
| ES (1) | ES2987156T3 (https=) |
| FI (1) | FI4021575T3 (https=) |
| HR (2) | HRP20240871T1 (https=) |
| HU (1) | HUE067135T2 (https=) |
| IL (1) | IL290763A (https=) |
| LT (1) | LT4021575T (https=) |
| MX (1) | MX2022002451A (https=) |
| PL (1) | PL4021575T3 (https=) |
| PT (1) | PT4021575T (https=) |
| RS (1) | RS65763B1 (https=) |
| SI (1) | SI4021575T1 (https=) |
| SM (1) | SMT202400315T1 (https=) |
| TW (1) | TW202122106A (https=) |
| WO (1) | WO2021042020A1 (https=) |
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 |
| LT2588130T (lt) * | 2010-06-25 | 2016-12-12 | Shire Human Genetic Therapies, Inc. | Terapinės priemonės pristatymas cns |
| US20140377244A1 (en) | 2011-12-23 | 2014-12-25 | 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 |
| KR102738860B1 (ko) * | 2015-10-23 | 2024-12-04 | 유니버시티 오브 아이오와 리써치 파운데이션 | 리소좀 축적 질환 (lsd)을 갖는 포유동물의 치료 방법에 사용하기 위한 aav 입자를 포함하는 약제학적 조성물 |
| BR112021009370A2 (pt) * | 2018-11-14 | 2021-08-17 | Regenxbio Inc. | método de tratamento da doença de batten cln2, composição farmacêutica e kit |
-
2020
- 2020-08-31 PT PT207714080T patent/PT4021575T/pt unknown
- 2020-08-31 SM SM20240315T patent/SMT202400315T1/it unknown
- 2020-08-31 PL PL20771408.0T patent/PL4021575T3/pl unknown
- 2020-08-31 HU HUE20771408A patent/HUE067135T2/hu unknown
- 2020-08-31 AU AU2020336984A patent/AU2020336984B2/en active Active
- 2020-08-31 MX MX2022002451A patent/MX2022002451A/es unknown
- 2020-08-31 SI SI202030455T patent/SI4021575T1/sl unknown
- 2020-08-31 HR HRP20240871TT patent/HRP20240871T1/hr unknown
- 2020-08-31 FI FIEP20771408.0T patent/FI4021575T3/fi active
- 2020-08-31 EP EP20771408.0A patent/EP4021575B1/en active Active
- 2020-08-31 AR ARP200102442A patent/AR119871A1/es 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 BR BR112022003598A patent/BR112022003598A2/pt unknown
- 2020-08-31 TW TW109129724A patent/TW202122106A/zh unknown
- 2020-08-31 CN CN202080068971.3A patent/CN114828960A/zh active Pending
- 2020-08-31 EP EP24177169.0A patent/EP4438120A3/en active Pending
- 2020-08-31 JP JP2022513165A patent/JP2022546042A/ja active Pending
- 2020-08-31 US US17/638,029 patent/US20220273775A1/en active Pending
- 2020-08-31 KR KR1020227010387A patent/KR20220054375A/ko active Pending
- 2020-08-31 HR HRP20220370AA patent/HRP20220370A1/hr not_active Application Discontinuation
- 2020-08-31 ES ES20771408T patent/ES2987156T3/es active Active
- 2020-08-31 CA CA3152521A patent/CA3152521A1/en active Pending
- 2020-08-31 WO PCT/US2020/048704 patent/WO2021042020A1/en not_active Ceased
- 2020-08-31 RS RS20240792A patent/RS65763B1/sr unknown
-
2022
- 2022-02-21 IL IL290763A patent/IL290763A/en unknown
- 2022-02-28 CL CL2022000496A patent/CL2022000496A1/es unknown
-
2025
- 2025-08-12 JP JP2025134120A patent/JP2025165414A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| WO2021042020A1 (en) | 2021-03-04 |
| RS65763B1 (sr) | 2024-08-30 |
| SI4021575T1 (sl) | 2024-10-30 |
| PT4021575T (pt) | 2024-07-30 |
| PL4021575T3 (pl) | 2024-09-23 |
| EP4438120A3 (en) | 2024-12-11 |
| BR112022003598A2 (pt) | 2022-05-24 |
| EP4021575B1 (en) | 2024-05-22 |
| HRP20220370A1 (hr) | 2022-09-16 |
| TW202122106A (zh) | 2021-06-16 |
| ES2987156T3 (es) | 2024-11-14 |
| EP4438120A2 (en) | 2024-10-02 |
| LT4021575T (lt) | 2024-08-12 |
| IL290763A (en) | 2022-04-01 |
| AU2020336984A1 (en) | 2022-03-10 |
| AR119871A1 (es) | 2022-01-19 |
| CL2022000496A1 (es) | 2022-10-21 |
| MX2022002451A (es) | 2022-06-02 |
| JP2022546042A (ja) | 2022-11-02 |
| CA3152521A1 (en) | 2021-03-04 |
| FI4021575T3 (fi) | 2024-07-24 |
| HUE067135T2 (hu) | 2024-10-28 |
| SMT202400315T1 (it) | 2024-09-16 |
| DK4021575T3 (da) | 2024-08-05 |
| CN114828960A (zh) | 2022-07-29 |
| EP4021575A1 (en) | 2022-07-06 |
| KR20220054375A (ko) | 2022-05-02 |
| JP2025165414A (ja) | 2025-11-04 |
| AU2020336984B2 (en) | 2026-04-23 |
| US20220273775A1 (en) | 2022-09-01 |
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