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HUE067135T2 - Eljárások CLN2 betegség kezelésére gyermekekben - Google Patents

Eljárások CLN2 betegség kezelésére gyermekekben

Info

Publication number
HUE067135T2
HUE067135T2 HUE20771408A HUE20771408A HUE067135T2 HU E067135 T2 HUE067135 T2 HU E067135T2 HU E20771408 A HUE20771408 A HU E20771408A HU E20771408 A HUE20771408 A HU E20771408A HU E067135 T2 HUE067135 T2 HU E067135T2
Authority
HU
Hungary
Prior art keywords
methods
pediatric subjects
cln2 disease
treating cln2
treating
Prior art date
Application number
HUE20771408A
Other languages
English (en)
Inventor
David Jacoby
Joshua Henshaw
Original Assignee
Biomarin Pharm Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Biomarin Pharm Inc filed Critical Biomarin Pharm Inc
Publication of HUE067135T2 publication Critical patent/HUE067135T2/hu

Links

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)
HUE20771408A 2019-08-29 2020-08-31 Eljárások CLN2 betegség kezelésére gyermekekben HUE067135T2 (hu)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201962893535P 2019-08-29 2019-08-29

Publications (1)

Publication Number Publication Date
HUE067135T2 true HUE067135T2 (hu) 2024-10-28

Family

ID=72470612

Family Applications (1)

Application Number Title Priority Date Filing Date
HUE20771408A HUE067135T2 (hu) 2019-08-29 2020-08-31 Eljárások CLN2 betegség kezelésére gyermekekben

Country Status (25)

Country Link
US (1) US20220273775A1 (hu)
EP (2) EP4438120A3 (hu)
JP (1) JP2022546042A (hu)
KR (1) KR20220054375A (hu)
CN (1) CN114828960A (hu)
AR (1) AR119871A1 (hu)
AU (1) AU2020336984A1 (hu)
BR (1) BR112022003598A2 (hu)
CA (1) CA3152521A1 (hu)
CL (1) CL2022000496A1 (hu)
DK (1) DK4021575T3 (hu)
ES (1) ES2987156T3 (hu)
FI (1) FI4021575T3 (hu)
HR (2) HRP20220370A1 (hu)
HU (1) HUE067135T2 (hu)
IL (1) IL290763A (hu)
LT (1) LT4021575T (hu)
MX (1) MX2022002451A (hu)
PL (1) PL4021575T3 (hu)
PT (1) PT4021575T (hu)
RS (1) RS65763B1 (hu)
SI (1) SI4021575T1 (hu)
SM (1) SMT202400315T1 (hu)
TW (1) TW202122106A (hu)
WO (1) WO2021042020A1 (hu)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
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)

* Cited by examiner, † Cited by third party
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

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
SI4021575T1 (sl) 2024-10-30
JP2022546042A (ja) 2022-11-02
BR112022003598A2 (pt) 2022-05-24
WO2021042020A1 (en) 2021-03-04
IL290763A (en) 2022-04-01

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