CL2017002825A1 - Formulaciones de tpp1 y métodos para tratar la enfermedad de cln2 - Google Patents

Formulaciones de tpp1 y métodos para tratar la enfermedad de cln2

Info

Publication number
CL2017002825A1
CL2017002825A1 CL2017002825A CL2017002825A CL2017002825A1 CL 2017002825 A1 CL2017002825 A1 CL 2017002825A1 CL 2017002825 A CL2017002825 A CL 2017002825A CL 2017002825 A CL2017002825 A CL 2017002825A CL 2017002825 A1 CL2017002825 A1 CL 2017002825A1
Authority
CL
Chile
Prior art keywords
formulations
methods
cln2
treat
tpp1
Prior art date
Application number
CL2017002825A
Other languages
English (en)
Spanish (es)
Inventor
Thomas W Lester
Saeed Moshashaee
Augustus O Okhamafe
Charles A O'neill
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 CL2017002825A1 publication Critical patent/CL2017002825A1/es

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)
    • 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
    • A61K9/0085—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)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Ophthalmology & Optometry (AREA)
  • Inorganic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Vascular Medicine (AREA)
  • Hematology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Anesthesiology (AREA)
CL2017002825A 2015-05-08 2017-11-08 Formulaciones de tpp1 y métodos para tratar la enfermedad de cln2 CL2017002825A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562158789P 2015-05-08 2015-05-08
US201662300171P 2016-02-26 2016-02-26

Publications (1)

Publication Number Publication Date
CL2017002825A1 true CL2017002825A1 (es) 2018-06-22

Family

ID=57222195

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2017002825A CL2017002825A1 (es) 2015-05-08 2017-11-08 Formulaciones de tpp1 y métodos para tratar la enfermedad de cln2

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) 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)

* 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
US11723579B2 (en) 2017-09-19 2023-08-15 Neuroenhancement Lab, LLC Method and apparatus for neuroenhancement
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
US11478603B2 (en) 2017-12-31 2022-10-25 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
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
SI4021575T1 (sl) * 2019-08-29 2024-10-30 Biomarin Pharmaceutical Inc. Metode za zdravljenje bolezni CLN2 pri prediatričnih subjektih
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
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
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
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
WO2013096899A2 (en) 2011-12-23 2013-06-27 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
RU2680581C2 (ru) * 2012-11-27 2019-02-22 Байомарин Фармасьютикал Инк. Направленные терапевтические химерные белки лизосомальных ферментов и их применение
US9750712B2 (en) * 2012-12-07 2017-09-05 Rush University Medical Center Composition and method for treating neuronal ceroid lipofuscinosis
MX373332B (es) 2013-07-26 2020-05-21 Univ Iowa Res Found UNA PARTÍCULA rAAV Y UN AGENTE DE INMUNOSUPRESIÓN PARA USARSE EN EL TRATAMIENTO DE ENFERMEDADES.
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
JP2024026739A (ja) 2024-02-28
MX2017014204A (es) 2018-07-06
HK1250479A1 (en) 2018-12-21
ZA201707373B (en) 2019-02-27
HRP20240050T1 (hr) 2024-03-29
EP3294345B1 (en) 2023-10-18
HRP20171905B1 (hr) 2023-03-17
CN114225018A (zh) 2022-03-25
IL299024B2 (en) 2025-12-01
CA2984448A1 (en) 2016-11-17
AU2016262434B2 (en) 2022-06-02
PT3294345T (pt) 2024-01-18
LT3294345T (lt) 2024-02-12
JP2018521964A (ja) 2018-08-09
US10758598B2 (en) 2020-09-01
ES2968375T3 (es) 2024-05-09
US10279015B2 (en) 2019-05-07
TW201704469A (zh) 2017-02-01
US11229687B2 (en) 2022-01-25
HRP20171905A2 (hr) 2018-05-18
SMT202400026T1 (it) 2024-03-13
PL3294345T3 (pl) 2024-07-01
RU2733464C2 (ru) 2020-10-01
CN107847615B (zh) 2022-01-25
DK3294345T3 (da) 2024-01-22
US20160324942A1 (en) 2016-11-10
EP4327873A2 (en) 2024-02-28
JP2021175760A (ja) 2021-11-04
US20190216905A1 (en) 2019-07-18
EP3294345A4 (en) 2019-01-16
IL299024A (en) 2023-02-01
US20200353059A1 (en) 2020-11-12
IL299024B1 (en) 2025-08-01
RS65101B1 (sr) 2024-02-29
SI3294345T1 (sl) 2024-03-29
FI3294345T3 (fi) 2024-01-15
WO2016182862A1 (en) 2016-11-17
JP7421520B2 (ja) 2024-01-24
IL273913B2 (en) 2023-06-01
TWI752907B (zh) 2022-01-21
JP6931330B2 (ja) 2021-09-01
EP4327873A3 (en) 2024-05-01
IL255307A0 (en) 2017-12-31
EP3294345A1 (en) 2018-03-21
IL273913A (en) 2020-05-31
KR20180004158A (ko) 2018-01-10
RU2017142727A (ru) 2019-06-10
CN107847615A (zh) 2018-03-27
IL255307B (en) 2021-03-25
RU2017142727A3 (enExample) 2019-12-11
KR102375051B1 (ko) 2022-03-15
BR112017023805A2 (pt) 2018-07-31
AU2016262434A1 (en) 2017-11-16
HUE065649T2 (hu) 2024-06-28

Similar Documents

Publication Publication Date Title
CL2017002825A1 (es) Formulaciones de tpp1 y métodos para tratar la enfermedad de cln2
MX2019007586A (es) Composiciones que comprenden tacrolimus para el tratamiento de enfermedades inflamatorias intraoculares.
MX381639B (es) Composicion oftalmica para el tratamiento de la enfermedad del ojo seco.
CO2017004525A2 (es) Dihidropirrolopiridinas inhibidoras de ror-gamma
IL262702B (en) Use of topical formulations of cannabinoids in the treatment of epidermolysis bullosa and related connective tissue disorders
BR112018000691A2 (pt) inibidores peptídicos do receptor de interleucina-23 e seu uso para tratar doenças inflamatórias
MX2020007060A (es) Compuestos, composiciones y metodos para tratar enfermedades que involucren tejidos con enfermedades acidas o hipoxicas.
BR112019006463A2 (pt) composição oral de canabinoides extraídos e métodos de uso da mesma
MX378579B (es) Uso de pridopidina para mejorar la funcion cognitiva y para tratamiento de enfermedad de alzheimer.
MX390120B (es) Moduladores de la somatostatina y usos de los mismos.
MX2016010328A (es) Tratamiento con halogeno de ataque cardiaco y lesion isquemica.
BR112018005905A2 (pt) ?composto, composição farmacêutica, métodos para evitar ou tratar uma doença ou condição, lesões de órgão, hepatotoxicidade e fígado gorduroso, e, uso de um composto?
CY1120642T1 (el) Φαρμακευτικη συνθεση της ιβουπροφαιμνης και της τραμαδολης για οφθαλμολογικη χρηση
CO2018012180A2 (es) Métodos de tratamiento para enfermedades colestásicas y fibróticas
EA201692402A1 (ru) Лекарственные формы для местного применения и их использование
MX385229B (es) Composiciones farmaceuticas que comprenden un antagonista de integrina alfa4 para uso en el tratamiento de condiciones inflamatorias oculares.
CL2021000129A1 (es) Apirasas solubilizadas, métodos y usos
BR112017013674A2 (pt) métodos e composições para tratamento de doenças cerebrais.
BR112017008805A2 (pt) tratamento de córnea usando laminina
CL2019000625A1 (es) Combinación de agonistas de fxr.
MX2020009532A (es) Oligonucleotidos modificados para uso en el tratamiento de tauopatias.
MX2022013283A (es) Composiciones para limpieza de colon y tratamiento de trastornos gastrointestinales.
BR112018073355A2 (pt) gordura e seus usos médicos
AR112291A1 (es) Composición para su uso en la prevención y/o el tratamiento de la mucositis orogastrointestinal inducida por un tratamiento oncológico
MX2018002298A (es) Metodos de uso de interleucina 10 para el tratamiento de enfermedades y trastornos.