DK3294345T3 - Tpp1-formuleringer og fremgangsmåder til behandling af cln2-sygdom - Google Patents

Tpp1-formuleringer og fremgangsmåder til behandling af cln2-sygdom Download PDF

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Publication number
DK3294345T3
DK3294345T3 DK16793229.2T DK16793229T DK3294345T3 DK 3294345 T3 DK3294345 T3 DK 3294345T3 DK 16793229 T DK16793229 T DK 16793229T DK 3294345 T3 DK3294345 T3 DK 3294345T3
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DK
Denmark
Prior art keywords
tpp1
formulations
treatment
methods
cln2 disease
Prior art date
Application number
DK16793229.2T
Other languages
English (en)
Inventor
Saeed Moshashaee
Augustus O Okhamafe
Charles A O'neill
Thomas W Lester
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
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Application filed by Biomarin Pharm Inc filed Critical Biomarin Pharm Inc
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Publication of DK3294345T3 publication Critical patent/DK3294345T3/da

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    • 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
    • A61K9/0012—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
    • 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
    • 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)
    • 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
    • A61K49/00—Preparations for testing in vivo
    • A61K49/06—Nuclear magnetic resonance [NMR] contrast preparations; Magnetic resonance imaging [MRI] contrast preparations
    • A61K49/08—Nuclear magnetic resonance [NMR] contrast preparations; Magnetic resonance imaging [MRI] contrast preparations characterised by the carrier
    • A61K49/10—Organic compounds
    • A61K49/14—Peptides, e.g. proteins
    • 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
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M5/00—Devices for bringing media into the body in a subcutaneous, intra-vascular or intramuscular way; Accessories therefor, e.g. filling or cleaning devices, arm-rests
    • A61M5/14—Infusion devices, e.g. infusing by gravity; Blood infusion; Accessories therefor
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/485—Exopeptidases (3.4.11-3.4.19)
    • 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)
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Ophthalmology & Optometry (AREA)
  • Molecular Biology (AREA)
  • Inorganic Chemistry (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Vascular Medicine (AREA)
  • Anesthesiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Hematology (AREA)
DK16793229.2T 2015-05-08 2016-05-05 Tpp1-formuleringer og fremgangsmåder til behandling af cln2-sygdom DK3294345T3 (da)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562158789P 2015-05-08 2015-05-08
US201662300171P 2016-02-26 2016-02-26
PCT/US2016/031074 WO2016182862A1 (en) 2015-05-08 2016-05-05 Tpp1 formulations and methods for treating cln2 disease

Publications (1)

Publication Number Publication Date
DK3294345T3 true DK3294345T3 (da) 2024-01-22

Family

ID=57222195

Family Applications (1)

Application Number Title Priority Date Filing Date
DK16793229.2T DK3294345T3 (da) 2015-05-08 2016-05-05 Tpp1-formuleringer og fremgangsmåder til behandling af cln2-sygdom

Country Status (24)

Country Link
US (3) US10279015B2 (da)
EP (2) EP3294345B1 (da)
JP (3) JP6931330B2 (da)
KR (1) KR102375051B1 (da)
CN (2) CN114225018A (da)
AU (1) AU2016262434B2 (da)
BR (1) BR112017023805A2 (da)
CA (1) CA2984448A1 (da)
CL (1) CL2017002825A1 (da)
DK (1) DK3294345T3 (da)
ES (1) ES2968375T3 (da)
FI (1) FI3294345T3 (da)
HK (1) HK1250479A1 (da)
HR (2) HRP20240050T1 (da)
IL (3) IL299024A (da)
LT (1) LT3294345T (da)
MX (1) MX2017014204A (da)
PT (1) PT3294345T (da)
RS (1) RS65101B1 (da)
RU (1) RU2733464C2 (da)
SI (1) SI3294345T1 (da)
TW (1) TWI752907B (da)
WO (1) WO2016182862A1 (da)
ZA (1) ZA201707373B (da)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US11726091B2 (en) 2017-04-03 2023-08-15 The Regents Of The University Of California Compositions and methods of diagnosing pancreatic cancer
WO2019060298A1 (en) 2017-09-19 2019-03-28 Neuroenhancement Lab, LLC METHOD AND APPARATUS FOR NEURO-ACTIVATION
US11717686B2 (en) 2017-12-04 2023-08-08 Neuroenhancement Lab, LLC Method and apparatus for neuroenhancement to facilitate learning and performance
US11318277B2 (en) 2017-12-31 2022-05-03 Neuroenhancement Lab, LLC Method and apparatus for neuroenhancement to enhance emotional response
US11364361B2 (en) 2018-04-20 2022-06-21 Neuroenhancement Lab, LLC System and method for inducing sleep by transplanting mental states
CA3112564A1 (en) 2018-09-14 2020-03-19 Neuroenhancement Lab, LLC System and method of improving sleep
US11786694B2 (en) 2019-05-24 2023-10-17 NeuroLight, Inc. Device, method, and app for facilitating sleep
WO2020247952A2 (en) * 2019-06-07 2020-12-10 The Regents Of The University Of California Systems comprising substrates and methods of using the same for detection of pancreatic cancers
JP2022546042A (ja) * 2019-08-29 2022-11-02 バイオマリン ファーマシューティカル インコーポレイテッド 小児対象のｃｌｎ２疾患を治療するための方法
US20230060797A1 (en) * 2021-09-02 2023-03-02 Jcr Pharmaceuticals Co., Ltd. Therapeutic compound for neuronal ceroid lipofuscinosis

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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
PL367093A1 (en) * 2001-05-18 2005-02-21 Solvay Pharmaceuticals Gmbh Use of compounds with combined nep/mp-inhibitory activity in the preparation of medicaments
WO2003029277A2 (en) * 2001-10-03 2003-04-10 Rigel Pharmaceuticals, Inc. Modulators of lymphocyte activation and migration
CA2388659A1 (en) * 2002-05-31 2003-11-30 Mcgill University Phospholipase a2 expression and activity and use thereof for diagnosis, prognostication, prevention and treatment of neural inflammatory and demyelinating disease
US7442372B2 (en) * 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
US20110166074A1 (en) 2006-10-18 2011-07-07 Cornell Research Foundation, Inc. Cln2 treatment of alzheimer's disease
WO2008157308A2 (en) * 2007-06-13 2008-12-24 Wayne State University Board Of Governors A zwitterion solution for low-volume therapeutic delivery
EP2333074A1 (en) 2009-12-14 2011-06-15 Robert Steinfeld Substances and methods for the treatment of lysosmal storage diseases
WO2012015910A2 (en) * 2010-07-28 2012-02-02 Allon Therapeutics Inc. Use of adnf polypeptides for treating neurodegenerative diseases
WO2012177929A2 (en) * 2011-06-24 2012-12-27 Amylin Pharmaceuticals, Inc. Methods for treating diabetes with extended release formulations of glp-1 receptor agonists
US20150000936A1 (en) * 2011-12-13 2015-01-01 Schlumberger Technology Corporation Energization of an element with a thermally expandable material
EP2793922B1 (en) 2011-12-23 2019-10-30 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
AU2013352184B2 (en) * 2012-11-27 2018-05-31 Biomarin Pharmaceutical Inc. Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof
US9750712B2 (en) * 2012-12-07 2017-09-05 Rush University Medical Center Composition and method for treating neuronal ceroid lipofuscinosis
RU2018128780A (ru) * 2013-07-26 2018-12-05 Юниверсити Оф Айова Рисерч Фаундейшн Способы и композиции для лечения болезней мозга
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

Also Published As

Publication number Publication date
PT3294345T (pt) 2024-01-18
JP7421520B2 (ja) 2024-01-24
IL299024A (en) 2023-02-01
SI3294345T1 (sl) 2024-03-29
RU2017142727A (ru) 2019-06-10
EP4327873A2 (en) 2024-02-28
US11229687B2 (en) 2022-01-25
HK1250479A1 (zh) 2018-12-21
US20160324942A1 (en) 2016-11-10
JP2024026739A (ja) 2024-02-28
LT3294345T (lt) 2024-02-12
KR102375051B1 (ko) 2022-03-15
BR112017023805A2 (pt) 2018-07-31
US20190216905A1 (en) 2019-07-18
CA2984448A1 (en) 2016-11-17
IL255307B (en) 2021-03-25
HRP20171905B1 (hr) 2023-03-17
RS65101B1 (sr) 2024-02-29
CN114225018A (zh) 2022-03-25
FI3294345T3 (fi) 2024-01-15
IL255307A0 (en) 2017-12-31
IL273913B2 (en) 2023-06-01
AU2016262434B2 (en) 2022-06-02
RU2017142727A3 (da) 2019-12-11
HRP20171905A2 (hr) 2018-05-18
ZA201707373B (en) 2019-02-27
WO2016182862A1 (en) 2016-11-17
EP4327873A3 (en) 2024-05-01
US10279015B2 (en) 2019-05-07
ES2968375T3 (es) 2024-05-09
TWI752907B (zh) 2022-01-21
TW201704469A (zh) 2017-02-01
US10758598B2 (en) 2020-09-01
HRP20240050T1 (hr) 2024-03-29
IL273913A (en) 2020-05-31
EP3294345B1 (en) 2023-10-18
JP6931330B2 (ja) 2021-09-01
EP3294345A1 (en) 2018-03-21
JP2021175760A (ja) 2021-11-04
US20200353059A1 (en) 2020-11-12
AU2016262434A1 (en) 2017-11-16
JP2018521964A (ja) 2018-08-09
EP3294345A4 (en) 2019-01-16
CN107847615A (zh) 2018-03-27
KR20180004158A (ko) 2018-01-10
RU2733464C2 (ru) 2020-10-01
CN107847615B (zh) 2022-01-25
CL2017002825A1 (es) 2018-06-22
MX2017014204A (es) 2018-07-06

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