IL299024B2 - Tpp1 formulations and methods for treating cln2 disease - Google Patents

Tpp1 formulations and methods for treating cln2 disease

Info

Publication number
IL299024B2
IL299024B2 IL299024A IL29902422A IL299024B2 IL 299024 B2 IL299024 B2 IL 299024B2 IL 299024 A IL299024 A IL 299024A IL 29902422 A IL29902422 A IL 29902422A IL 299024 B2 IL299024 B2 IL 299024B2
Authority
IL
Israel
Prior art keywords
concentration
rhtpp1
dose
formulation
composition
Prior art date
Application number
IL299024A
Other languages
English (en)
Hebrew (he)
Other versions
IL299024A (en
IL299024B1 (en
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 IL299024A publication Critical patent/IL299024A/en
Publication of IL299024B1 publication Critical patent/IL299024B1/en
Publication of IL299024B2 publication Critical patent/IL299024B2/en

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)
    • 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
    • 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
    • A61K9/0085—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)
    • 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
    • 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

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)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (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)
  • Microbiology (AREA)
  • Inorganic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Vascular Medicine (AREA)
  • Anesthesiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Hematology (AREA)
IL299024A 2015-05-08 2016-05-05 Tpp1 formulations and methods for treating cln2 disease IL299024B2 (en)

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 (3)

Publication Number Publication Date
IL299024A IL299024A (en) 2023-02-01
IL299024B1 IL299024B1 (en) 2025-08-01
IL299024B2 true IL299024B2 (en) 2025-12-01

Family

ID=57222195

Family Applications (3)

Application Number Title Priority Date Filing Date
IL299024A IL299024B2 (en) 2015-05-08 2016-05-05 Tpp1 formulations and methods for treating cln2 disease
IL255307A IL255307B (en) 2015-05-08 2017-10-29 Tpp1 formulations and uses thereon in cln2 disease
IL273913A IL273913B2 (en) 2015-05-08 2020-04-12 Tripeptidyl peptidase-1 preparations and methods for treating neuronal ceroid liposuction disease

Family Applications After (2)

Application Number Title Priority Date Filing Date
IL255307A IL255307B (en) 2015-05-08 2017-10-29 Tpp1 formulations and uses thereon in cln2 disease
IL273913A IL273913B2 (en) 2015-05-08 2020-04-12 Tripeptidyl peptidase-1 preparations and methods for treating neuronal ceroid liposuction disease

Country Status (25)

Country Link
US (3) US10279015B2 (enExample)
EP (2) EP3294345B1 (enExample)
JP (3) JP6931330B2 (enExample)
KR (1) KR102375051B1 (enExample)
CN (2) CN114225018A (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) HRP20171905B1 (enExample)
HU (1) HUE065649T2 (enExample)
IL (3) IL299024B2 (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)

* 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
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
US12280219B2 (en) 2017-12-31 2025-04-22 NeuroLight, Inc. Method and apparatus for neuroenhancement to enhance emotional response
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
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
HRP20240871T1 (hr) * 2019-08-29 2024-10-11 Biomarin Pharmaceutical Inc. Postupci liječenja cln2 bolesti kod pedijatrijskih subjekata
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

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20120148558A1 (en) * 2003-08-29 2012-06-14 Biomarin Pharmaceutical Inc. Delivery of Therapeutic Compounds to the Brain and Other Tissues
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

Family Cites Families (14)

* 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
MXPA03010341A (es) * 2001-05-18 2004-03-10 Solvay Pharm Gmbh Uso de compuestos con actividad inhibitoria de epn/mp combinada en la preparacion de medicamentos.
AU2002362436A1 (en) * 2001-10-03 2003-04-14 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
WO2008049058A2 (en) 2006-10-18 2008-04-24 Cornell Research Foundation, Inc. Cln2 treatment of alzheimer's disease
CA2721135A1 (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
EP2723359A4 (en) * 2011-06-24 2015-03-11 Amylin Pharmaceuticals Llc METHOD FOR THE TREATMENT OF DIABETES WITH THE HELP OF DELAYED RELEASED FORMULATIONS FROM GLP-1 RECEPTOR AGONISTS
US20150000936A1 (en) * 2011-12-13 2015-01-01 Schlumberger Technology Corporation Energization of an element with a thermally expandable material
US20140377244A1 (en) 2011-12-23 2014-12-25 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
KR102262882B1 (ko) * 2012-11-27 2021-06-10 바이오마린 파머수티컬 인크. 표적화된 치료적 리소좀 효소 융합 단백질들 및 그들의 용도들
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

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20120148558A1 (en) * 2003-08-29 2012-06-14 Biomarin Pharmaceutical Inc. Delivery of Therapeutic Compounds to the Brain and Other Tissues
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

Also Published As

Publication number Publication date
IL299024A (en) 2023-02-01
US20160324942A1 (en) 2016-11-10
RS65101B1 (sr) 2024-02-29
MX2017014204A (es) 2018-07-06
WO2016182862A1 (en) 2016-11-17
IL299024B1 (en) 2025-08-01
EP4327873A3 (en) 2024-05-01
US11229687B2 (en) 2022-01-25
PL3294345T3 (pl) 2024-07-01
RU2017142727A3 (enExample) 2019-12-11
JP6931330B2 (ja) 2021-09-01
CL2017002825A1 (es) 2018-06-22
RU2017142727A (ru) 2019-06-10
IL273913A (en) 2020-05-31
IL255307B (en) 2021-03-25
EP3294345A1 (en) 2018-03-21
PT3294345T (pt) 2024-01-18
US20190216905A1 (en) 2019-07-18
EP3294345A4 (en) 2019-01-16
JP2018521964A (ja) 2018-08-09
EP4327873A2 (en) 2024-02-28
SMT202400026T1 (it) 2024-03-13
AU2016262434B2 (en) 2022-06-02
SI3294345T1 (sl) 2024-03-29
CA2984448A1 (en) 2016-11-17
HRP20171905B1 (hr) 2023-03-17
TWI752907B (zh) 2022-01-21
KR102375051B1 (ko) 2022-03-15
ZA201707373B (en) 2019-02-27
US10758598B2 (en) 2020-09-01
CN107847615A (zh) 2018-03-27
JP2024026739A (ja) 2024-02-28
BR112017023805A2 (pt) 2018-07-31
LT3294345T (lt) 2024-02-12
FI3294345T3 (fi) 2024-01-15
US20200353059A1 (en) 2020-11-12
KR20180004158A (ko) 2018-01-10
HK1250479A1 (en) 2018-12-21
ES2968375T3 (es) 2024-05-09
HUE065649T2 (hu) 2024-06-28
RU2733464C2 (ru) 2020-10-01
US10279015B2 (en) 2019-05-07
DK3294345T3 (da) 2024-01-22
JP2021175760A (ja) 2021-11-04
JP7421520B2 (ja) 2024-01-24
AU2016262434A1 (en) 2017-11-16
CN107847615B (zh) 2022-01-25
HRP20240050T1 (hr) 2024-03-29
HRP20171905A2 (hr) 2018-05-18
IL255307A0 (en) 2017-12-31
IL273913B2 (en) 2023-06-01
CN114225018A (zh) 2022-03-25
EP3294345B1 (en) 2023-10-18
TW201704469A (zh) 2017-02-01

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