DK4021575T3 - Fremgangsmåder til behandling af cln2-sygdom hos børn - Google Patents

Fremgangsmåder til behandling af cln2-sygdom hos børn Download PDF

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Publication number
DK4021575T3
DK4021575T3 DK20771408.0T DK20771408T DK4021575T3 DK 4021575 T3 DK4021575 T3 DK 4021575T3 DK 20771408 T DK20771408 T DK 20771408T DK 4021575 T3 DK4021575 T3 DK 4021575T3
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DK
Denmark
Prior art keywords
children
approaches
treatment
cln2 disease
cln2
Prior art date
Application number
DK20771408.0T
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
Application granted granted Critical
Publication of DK4021575T3 publication Critical patent/DK4021575T3/da

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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)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (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)
  • Hospice & Palliative Care (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Genetics & Genomics (AREA)
  • General Engineering & Computer Science (AREA)
  • Psychology (AREA)
  • Biochemistry (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)
DK20771408.0T 2019-08-29 2020-08-31 Fremgangsmåder til behandling af cln2-sygdom hos børn DK4021575T3 (da)

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
DK4021575T3 true DK4021575T3 (da) 2024-08-05

Family

ID=72470612

Family Applications (1)

Application Number Title Priority Date Filing Date
DK20771408.0T DK4021575T3 (da) 2019-08-29 2020-08-31 Fremgangsmåder til behandling af cln2-sygdom hos børn

Country Status (25)

Country Link
US (1) US20220273775A1 (da)
EP (2) EP4021575B1 (da)
JP (2) JP2022546042A (da)
KR (1) KR20220054375A (da)
CN (1) CN114828960A (da)
AR (1) AR119871A1 (da)
AU (1) AU2020336984B2 (da)
BR (1) BR112022003598A2 (da)
CA (1) CA3152521A1 (da)
CL (1) CL2022000496A1 (da)
DK (1) DK4021575T3 (da)
ES (1) ES2987156T3 (da)
FI (1) FI4021575T3 (da)
HR (2) HRP20220370A1 (da)
HU (1) HUE067135T2 (da)
IL (1) IL290763A (da)
LT (1) LT4021575T (da)
MX (1) MX2022002451A (da)
PL (1) PL4021575T3 (da)
PT (1) PT4021575T (da)
RS (1) RS65763B1 (da)
SI (1) SI4021575T1 (da)
SM (1) SMT202400315T1 (da)
TW (1) TW202122106A (da)
WO (1) WO2021042020A1 (da)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12303693B2 (en) * 2021-04-02 2025-05-20 Neuropace, Inc. Systems and methods for controlling operation of an implanted neurostimulation system based on a mapping of episode durations and seizure probability biomarkers

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
EP2588130B1 (en) * 2010-06-25 2016-08-17 Shire Human Genetic Therapies, Inc. Cns delivery of therapeutic agents
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
SG11201803218PA (en) * 2015-10-23 2018-05-30 Univ Iowa Res Found Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection
IL327339A (en) * 2018-11-14 2026-05-01 Regenxbio Inc Gene therapy for neural ceroid lipofuscinosis

Also Published As

Publication number Publication date
CN114828960A (zh) 2022-07-29
LT4021575T (lt) 2024-08-12
SI4021575T1 (sl) 2024-10-30
CA3152521A1 (en) 2021-03-04
PT4021575T (pt) 2024-07-30
EP4021575A1 (en) 2022-07-06
RS65763B1 (sr) 2024-08-30
FI4021575T3 (fi) 2024-07-24
JP2025165414A (ja) 2025-11-04
HRP20240871T1 (hr) 2024-10-11
TW202122106A (zh) 2021-06-16
BR112022003598A2 (pt) 2022-05-24
AU2020336984A1 (en) 2022-03-10
US20220273775A1 (en) 2022-09-01
ES2987156T3 (es) 2024-11-14
KR20220054375A (ko) 2022-05-02
AR119871A1 (es) 2022-01-19
MX2022002451A (es) 2022-06-02
WO2021042020A1 (en) 2021-03-04
PL4021575T3 (pl) 2024-09-23
EP4438120A3 (en) 2024-12-11
CL2022000496A1 (es) 2022-10-21
AU2020336984B2 (en) 2026-04-23
JP2022546042A (ja) 2022-11-02
EP4438120A2 (en) 2024-10-02
SMT202400315T1 (it) 2024-09-16
EP4021575B1 (en) 2024-05-22
HUE067135T2 (hu) 2024-10-28
IL290763A (en) 2022-04-01
HRP20220370A1 (hr) 2022-09-16

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