HRP20171905B1 - Formulacije tpp1 i postupci liječenja bolesti cln2 - Google Patents

Formulacije tpp1 i postupci liječenja bolesti cln2 Download PDF

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Publication number
HRP20171905B1
HRP20171905B1 HRP20171905AA HRP20171905A HRP20171905B1 HR P20171905 B1 HRP20171905 B1 HR P20171905B1 HR P20171905A A HRP20171905A A HR P20171905AA HR P20171905 A HRP20171905 A HR P20171905A HR P20171905 B1 HRP20171905 B1 HR P20171905B1
Authority
HR
Croatia
Prior art keywords
concentration
rhtpp1
disease
cln2
patients
Prior art date
Application number
HRP20171905AA
Other languages
English (en)
Croatian (hr)
Inventor
Thomas W. Lester
Saeed Moshashaee
Augustus O. Okhamafe
Charles A. O'neill
Original Assignee
Biomarin Pharmaceutical 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 Pharmaceutical Inc. filed Critical Biomarin Pharmaceutical Inc.
Publication of HRP20171905A2 publication Critical patent/HRP20171905A2/hr
Publication of HRP20171905B1 publication Critical patent/HRP20171905B1/hr

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Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/48Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/4813Exopeptidases (3.4.11. to 3.4.19)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/48Hydrolases (3) acting on peptide bonds (3.4)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/02Inorganic compounds
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00Preparations for testing in vivo
    • A61K49/06Nuclear magnetic resonance [NMR] contrast preparations; Magnetic resonance imaging [MRI] contrast preparations
    • A61K49/08Nuclear magnetic resonance [NMR] contrast preparations; Magnetic resonance imaging [MRI] contrast preparations characterised by the carrier
    • A61K49/10Organic compounds
    • A61K49/14Peptides, e.g. proteins
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0048Eye, e.g. artificial tears
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0085Brain, e.g. brain implants; Spinal cord
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/08Solutions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61MDEVICES 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
    • A61M5/00Devices for bringing media into the body in a subcutaneous, intra-vascular or intramuscular way; Accessories therefor, e.g. filling or cleaning devices, arm-rests
    • A61M5/14Infusion devices, e.g. infusing by gravity; Blood infusion; Accessories therefor
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/485Exopeptidases (3.4.11-3.4.19)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/14Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
    • C12Y304/14009Tripeptidyl-peptidase I (3.4.14.9)
    • YGENERAL 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
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02ATECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

  • 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)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Ophthalmology & Optometry (AREA)
  • Molecular Biology (AREA)
  • Inorganic Chemistry (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Vascular Medicine (AREA)
  • Anesthesiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Hematology (AREA)
HRP20171905AA 2015-05-08 2016-05-05 Formulacije tpp1 i postupci liječenja bolesti cln2 HRP20171905B1 (hr)

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 (2)

Publication Number Publication Date
HRP20171905A2 HRP20171905A2 (hr) 2018-05-18
HRP20171905B1 true HRP20171905B1 (hr) 2023-03-17

Family

ID=57222195

Family Applications (2)

Application Number Title Priority Date Filing Date
HRP20240050TT HRP20240050T1 (hr) 2015-05-08 2016-05-05 Formulacije tpp1 i postupci liječenja bolesti cln2
HRP20171905AA HRP20171905B1 (hr) 2015-05-08 2016-05-05 Formulacije tpp1 i postupci liječenja bolesti cln2

Family Applications Before (1)

Application Number Title Priority Date Filing Date
HRP20240050TT HRP20240050T1 (hr) 2015-05-08 2016-05-05 Formulacije tpp1 i postupci liječenja bolesti cln2

Country Status (25)

Country Link
US (3) US10279015B2 (enExample)
EP (2) EP4327873A3 (enExample)
JP (3) JP6931330B2 (enExample)
KR (1) KR102375051B1 (enExample)
CN (2) CN107847615B (enExample)
AU (1) AU2016262434B2 (enExample)
CA (1) CA2984448A1 (enExample)
CL (1) CL2017002825A1 (enExample)
DK (1) DK3294345T3 (enExample)
ES (1) ES2968375T3 (enExample)
FI (1) FI3294345T3 (enExample)
HR (2) HRP20240050T1 (enExample)
HU (1) HUE065649T2 (enExample)
IL (3) IL255307B (enExample)
LT (1) LT3294345T (enExample)
MX (1) MX2017014204A (enExample)
PL (1) PL3294345T3 (enExample)
PT (1) PT3294345T (enExample)
RS (1) RS65101B1 (enExample)
RU (1) RU2733464C2 (enExample)
SI (1) SI3294345T1 (enExample)
SM (1) SMT202400026T1 (enExample)
TW (1) TWI752907B (enExample)
WO (1) WO2016182862A1 (enExample)
ZA (1) ZA201707373B (enExample)

Families Citing this family (14)

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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
EP3607320A4 (en) 2017-04-03 2021-04-21 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
US11478603B2 (en) 2017-12-31 2022-10-25 Neuroenhancement Lab, LLC Method and apparatus for neuroenhancement to enhance emotional response
US12280219B2 (en) 2017-12-31 2025-04-22 NeuroLight, Inc. 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
CN113382683A (zh) 2018-09-14 2021-09-10 纽罗因恒思蒙特实验有限责任公司 改善睡眠的系统和方法
US11786694B2 (en) 2019-05-24 2023-10-17 NeuroLight, Inc. Device, method, and app for facilitating sleep
US20220381784A1 (en) * 2019-06-07 2022-12-01 The Regents Of The University Of California Systems comprising substrates and methods of using the same for detection of pancreatic cancers
SMT202400315T1 (it) * 2019-08-29 2024-09-16 Biomarin Pharm Inc Metodi per il trattamento della malattia cln2 in soggetti pediatrici
IT201900023142A1 (it) * 2019-12-05 2021-06-05 Sifi Spa Composizione topica oculare contenente metaboliti della fermentazione di lactobacillus
US20230060797A1 (en) * 2021-09-02 2023-03-02 Jcr Pharmaceuticals Co., Ltd. Therapeutic compound for neuronal ceroid lipofuscinosis
CN117731312A (zh) 2022-09-13 2024-03-22 上海联影医疗科技股份有限公司 一种医学图像的扫描方法和系统

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US20100210604A1 (en) * 2007-06-13 2010-08-19 Meythaler Jay M 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
US20110166074A1 (en) * 2006-10-18 2011-07-07 Cornell Research Foundation, Inc. Cln2 treatment of alzheimer's disease
US20120148558A1 (en) * 2003-08-29 2012-06-14 Biomarin Pharmaceutical Inc. Delivery of Therapeutic Compounds to the Brain and Other Tissues
WO2014085621A1 (en) * 2012-11-27 2014-06-05 Biomarin Pharmaceutical Inc. Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof
WO2014089449A1 (en) * 2012-12-07 2014-06-12 Rush University Nedical Center Composition and method for treating neuronal ceroid lipofuscinosis
WO2015013148A2 (en) * 2013-07-26 2015-01-29 University Of Iowa Research Foundation Methods and compositions for treating brain diseases

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US20110166074A1 (en) * 2006-10-18 2011-07-07 Cornell Research Foundation, Inc. Cln2 treatment of alzheimer's disease
US20100210604A1 (en) * 2007-06-13 2010-08-19 Meythaler Jay M Zwitterion solution for low-volume therapeutic delivery
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US20120308544A1 (en) * 2009-12-14 2012-12-06 Robert Steinfeld Substances and Methods for the Treatment of Lysosmal Storage Diseases
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WO2014089449A1 (en) * 2012-12-07 2014-06-12 Rush University Nedical Center Composition and method for treating neuronal ceroid lipofuscinosis
WO2015013148A2 (en) * 2013-07-26 2015-01-29 University Of Iowa Research Foundation Methods and compositions for treating brain diseases

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Also Published As

Publication number Publication date
IL299024B2 (en) 2025-12-01
US10279015B2 (en) 2019-05-07
EP4327873A2 (en) 2024-02-28
SI3294345T1 (sl) 2024-03-29
US10758598B2 (en) 2020-09-01
RS65101B1 (sr) 2024-02-29
MX2017014204A (es) 2018-07-06
CN114225018A (zh) 2022-03-25
KR102375051B1 (ko) 2022-03-15
JP2024026739A (ja) 2024-02-28
SMT202400026T1 (it) 2024-03-13
TW201704469A (zh) 2017-02-01
US20200353059A1 (en) 2020-11-12
IL299024B1 (en) 2025-08-01
AU2016262434A1 (en) 2017-11-16
CN107847615A (zh) 2018-03-27
IL273913B2 (en) 2023-06-01
RU2733464C2 (ru) 2020-10-01
JP6931330B2 (ja) 2021-09-01
EP3294345A4 (en) 2019-01-16
BR112017023805A2 (pt) 2018-07-31
TWI752907B (zh) 2022-01-21
JP2021175760A (ja) 2021-11-04
AU2016262434B2 (en) 2022-06-02
CN107847615B (zh) 2022-01-25
PT3294345T (pt) 2024-01-18
EP4327873A3 (en) 2024-05-01
WO2016182862A1 (en) 2016-11-17
CL2017002825A1 (es) 2018-06-22
HK1250479A1 (en) 2018-12-21
US11229687B2 (en) 2022-01-25
RU2017142727A3 (enExample) 2019-12-11
DK3294345T3 (da) 2024-01-22
PL3294345T3 (pl) 2024-07-01
ZA201707373B (en) 2019-02-27
HUE065649T2 (hu) 2024-06-28
JP7421520B2 (ja) 2024-01-24
FI3294345T3 (fi) 2024-01-15
US20160324942A1 (en) 2016-11-10
IL273913A (en) 2020-05-31
KR20180004158A (ko) 2018-01-10
CA2984448A1 (en) 2016-11-17
RU2017142727A (ru) 2019-06-10
EP3294345A1 (en) 2018-03-21
EP3294345B1 (en) 2023-10-18
HRP20240050T1 (hr) 2024-03-29
HRP20171905A2 (hr) 2018-05-18
LT3294345T (lt) 2024-02-12
US20190216905A1 (en) 2019-07-18
IL255307A0 (en) 2017-12-31
ES2968375T3 (es) 2024-05-09
JP2018521964A (ja) 2018-08-09
IL255307B (en) 2021-03-25
IL299024A (en) 2023-02-01

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