CN107847615B - 用于治疗cln2疾病的tpp1调配物及方法 - Google Patents

用于治疗cln2疾病的tpp1调配物及方法 Download PDF

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CN107847615B
CN107847615B CN201680025984.6A CN201680025984A CN107847615B CN 107847615 B CN107847615 B CN 107847615B CN 201680025984 A CN201680025984 A CN 201680025984A CN 107847615 B CN107847615 B CN 107847615B
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rhtpp1
concentration
disease
cln2
formulation
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CN107847615A (zh
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T·W·莱斯特
S·莫沙希
A·O·奥克哈马菲
C·A·奥尼尔
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Biomarin Pharmaceutical Inc
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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
    • 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
    • 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
    • A61K9/08—Solutions
    • 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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  • 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)
CN201680025984.6A 2015-05-08 2016-05-05 用于治疗cln2疾病的tpp1调配物及方法 Active CN107847615B (zh)

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US201562158789P 2015-05-08 2015-05-08
US62/158,789 2015-05-08
US201662300171P 2016-02-26 2016-02-26
US62/300,171 2016-02-26
PCT/US2016/031074 WO2016182862A1 (en) 2015-05-08 2016-05-05 Tpp1 formulations and methods for treating cln2 disease

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Families Citing this family (14)

* 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
WO2018187385A1 (en) 2017-04-03 2018-10-11 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
WO2019133997A1 (en) 2017-12-31 2019-07-04 Neuroenhancement Lab, LLC System and method 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
WO2020056418A1 (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
PT4021575T (pt) * 2019-08-29 2024-07-30 Biomarin Pharm Inc Métodos para o tratamento da doença cln2 em sujeitos pediátricos
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

Family Cites Families (17)

* 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
JP2004536063A (ja) * 2001-05-18 2004-12-02 ゾルファイ　ファーマスーティカルズ　ゲゼルシャフト　ミット　ベシュレンクテル　ハフツング 医薬の製造における組み合わせたｎｅｐ／ｍｐ−抑制活性を有する化合物の使用
US20050130117A1 (en) * 2001-10-03 2005-06-16 Davis Cong L. 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
WO2008049058A2 (en) 2006-10-18 2008-04-24 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
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
JP2014520159A (ja) * 2011-06-24 2014-08-21 アミリン・ファーマシューティカルズ，リミテッド・ライアビリティ・カンパニー Ｇｌｐ−１受容体アゴニストの徐放性製剤による糖尿病の治療方法
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
DK3115372T3 (da) * 2012-11-27 2019-06-11 Biomarin Pharm Inc Målrettede terapeutiske lysosomale enzymfusionsproteiner og anvendelser deraf
US9750712B2 (en) 2012-12-07 2017-09-05 Rush University Medical Center Composition and method for treating neuronal ceroid lipofuscinosis
DK3024497T3 (da) * 2013-07-26 2021-04-12 Univ Iowa Res Found Fremgangsmåder og præparater til behandling af hjernesygdomme
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

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
"Intrathecal tripeptidyl-peptidase 1 reduces lysosomal storage in a canine model of late infantile neuronal ceroid lipofuscinosis";Brian R. Vuillemenot等;《Molecular Genetics and Metabolism》;20110630(第104期);摘要，第326页右栏 *

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RU2733464C2 (ru) 2020-10-01
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ZA201707373B (en) 2019-02-27
JP7421520B2 (ja) 2024-01-24
IL299024B1 (en) 2025-08-01
EP3294345B1 (en) 2023-10-18
EP4327873A3 (en) 2024-05-01
BR112017023805A2 (pt) 2018-07-31
HK1250479A1 (en) 2018-12-21
AU2016262434B2 (en) 2022-06-02
SMT202400026T1 (it) 2024-03-13
IL273913B2 (en) 2023-06-01
EP3294345A4 (en) 2019-01-16
US20190216905A1 (en) 2019-07-18
US20200353059A1 (en) 2020-11-12
FI3294345T3 (fi) 2024-01-15
JP6931330B2 (ja) 2021-09-01
DK3294345T3 (da) 2024-01-22
CN114225018A (zh) 2022-03-25
IL273913A (en) 2020-05-31
IL299024A (en) 2023-02-01
IL255307B (en) 2021-03-25
WO2016182862A1 (en) 2016-11-17
HRP20171905B1 (hr) 2023-03-17
US11229687B2 (en) 2022-01-25
US10758598B2 (en) 2020-09-01
KR20180004158A (ko) 2018-01-10
KR102375051B1 (ko) 2022-03-15
HRP20240050T1 (hr) 2024-03-29
PL3294345T3 (pl) 2024-07-01
ES2968375T3 (es) 2024-05-09
MX2017014204A (es) 2018-07-06
US20160324942A1 (en) 2016-11-10
HUE065649T2 (hu) 2024-06-28
EP4327873A2 (en) 2024-02-28
RS65101B1 (sr) 2024-02-29
JP2024026739A (ja) 2024-02-28
EP3294345A1 (en) 2018-03-21
AU2016262434A1 (en) 2017-11-16
TW201704469A (zh) 2017-02-01
HRP20171905A2 (hr) 2018-05-18
IL299024B2 (en) 2025-12-01
IL255307A0 (en) 2017-12-31
CL2017002825A1 (es) 2018-06-22
PT3294345T (pt) 2024-01-18
CA2984448A1 (en) 2016-11-17
SI3294345T1 (sl) 2024-03-29
JP2021175760A (ja) 2021-11-04
JP2018521964A (ja) 2018-08-09
TWI752907B (zh) 2022-01-21
CN107847615A (zh) 2018-03-27
US10279015B2 (en) 2019-05-07
RU2017142727A3 (enExample) 2019-12-11

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