KR20170083146A - 리소좀 질환들의 치료를 위한 방법들 및 조성물들 - Google Patents

리소좀 질환들의 치료를 위한 방법들 및 조성물들 Download PDF

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KR20170083146A
KR20170083146A KR1020177016827A KR20177016827A KR20170083146A KR 20170083146 A KR20170083146 A KR 20170083146A KR 1020177016827 A KR1020177016827 A KR 1020177016827A KR 20177016827 A KR20177016827 A KR 20177016827A KR 20170083146 A KR20170083146 A KR 20170083146A
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disease
tfeb
agent
cells
aspirin
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Korean (ko)
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칼리파다 파한
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러쉬 유니버시티 메디컬 센터
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/60—Salicylic acid; Derivatives thereof
    • A61K31/612—Salicylic acid; Derivatives thereof having the hydroxy group in position 2 esterified, e.g. salicylsulfuric acid
    • A61K31/616—Salicylic acid; Derivatives thereof having the hydroxy group in position 2 esterified, e.g. salicylsulfuric acid by carboxylic acids, e.g. acetylsalicylic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/045—Hydroxy compounds, e.g. alcohols; Salts thereof, e.g. alcoholates
    • A61K31/07—Retinol compounds, e.g. vitamin A
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/19—Carboxylic acids, e.g. valproic acid
    • A61K31/192—Carboxylic acids, e.g. valproic acid having aromatic groups, e.g. sulindac, 2-aryl-propionic acids, ethacrynic acid 
    • A—HUMAN NECESSITIES
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    • A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/19—Carboxylic acids, e.g. valproic acid
    • A61K31/20—Carboxylic acids, e.g. valproic acid having a carboxyl group bound to a chain of seven or more carbon atoms, e.g. stearic, palmitic, arachidic acids
    • A61K31/203—Retinoic acids ; Salts thereof
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/21—Esters, e.g. nitroglycerine, selenocyanates
    • A61K31/215—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids
    • A61K31/216—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids of acids having aromatic rings, e.g. benactizyne, clofibrate
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/21—Esters, e.g. nitroglycerine, selenocyanates
    • A61K31/215—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids
    • A61K31/22—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids of acyclic acids, e.g. pravastatin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/21—Esters, e.g. nitroglycerine, selenocyanates
    • A61K31/215—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids
    • A61K31/22—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids of acyclic acids, e.g. pravastatin
    • A61K31/222—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids of acyclic acids, e.g. pravastatin with compounds having aromatic groups, e.g. dipivefrine, ibopamine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/365—Lactones
    • A61K31/366—Lactones having six-membered rings, e.g. delta-lactones
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/40—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/40—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
    • A61K31/403—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil condensed with carbocyclic rings, e.g. carbazole
    • A61K31/404—Indoles, e.g. pindolol
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/47—Quinolines; Isoquinolines
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/02—Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2121/00—Preparations for use in therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00

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  • Health & Medical Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Emergency Medicine (AREA)
  • Psychology (AREA)
  • Hematology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Pain & Pain Management (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Urology & Nephrology (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
KR1020177016827A 2014-11-19 2015-11-16 리소좀 질환들의 치료를 위한 방법들 및 조성물들 Ceased KR20170083146A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201462081696P 2014-11-19 2014-11-19
US62/081,696 2014-11-19
PCT/US2015/060878 WO2016081365A1 (en) 2014-11-19 2015-11-16 Compositions and methods for treating lysosomal disorders

Publications (1)

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KR20170083146A true KR20170083146A (ko) 2017-07-17

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KR1020177016827A Ceased KR20170083146A (ko) 2014-11-19 2015-11-16 리소좀 질환들의 치료를 위한 방법들 및 조성물들

Country Status (9)

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US (3) US20170354666A1 (enExample)
EP (2) EP4026545A1 (enExample)
JP (4) JP2017536363A (enExample)
KR (1) KR20170083146A (enExample)
CN (1) CN107205976A (enExample)
AU (1) AU2015350223B2 (enExample)
CA (2) CA3176253A1 (enExample)
ES (1) ES2914085T3 (enExample)
WO (1) WO2016081365A1 (enExample)

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CA3176253A1 (en) * 2014-11-19 2016-05-26 Rush University Medical Center Use of cinnamic acid or sodium benzoate for treating lysosomal disorders
CN109563024B (zh) 2016-06-13 2023-06-06 心悦生医股份有限公司 苯甲酸锂的共晶及其用途
CN109563018A (zh) * 2016-06-13 2019-04-02 心悦生医股份有限公司 苯甲酸钠的共晶及其用途
US11020366B2 (en) * 2016-12-29 2021-06-01 Rush University Medical Center Locomotor activity and increase in longevity of late infantile neuronal ceriod lipofuscinosis subjects by Gemfibrozil
JP6991246B2 (ja) * 2017-02-08 2022-02-03 ノバルティス アーゲー Ｆｇｆ２１模倣抗体及びその使用
WO2019152391A1 (en) * 2018-01-30 2019-08-08 Rush University Medical Center Sequential staining for multiplex analyses of tissues and cells
JP7366408B2 (ja) * 2018-02-06 2023-10-23 国立大学法人京都大学 ライソゾーム病の予防及び治療剤
US20210186937A1 (en) * 2018-05-22 2021-06-24 Duke University Compositions and methods for treating neurodegenerative disorders with rifaximin
EP3876932A4 (en) * 2018-11-05 2022-07-20 Rush University Medical Center Nasal delivery of low-dose aspirin for the treatment of neurodegenerative and lysosomal storage diseases
MX2021008152A (es) * 2019-01-03 2021-10-13 Univ Pittsburgh Commonwealth Sys Higher Education Metodos y materiales para aumentar los niveles del polipeptido del factor de transcripcion eb.
EA202192199A1 (ru) * 2019-02-25 2021-12-17 Раш Юниверсити Медикал Сентер Композиции, содержащие коричную кислоту, и способы их применения
CN113811359A (zh) * 2019-03-22 2021-12-17 拉什大学医学中心 用于溶酶体贮积症的经鼻基因递送和口服肉桂酸、油酰胺或吉非贝齐的组合
CA3153963A1 (en) * 2019-09-09 2021-03-18 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Methods of restoring lysosomal function of retinal pigment epithelial cells by activation of tfeb
WO2021067243A1 (en) * 2019-10-01 2021-04-08 Seelos Therapeutics, Inc. Trehalose formulations and uses thereof
WO2022159086A1 (en) * 2021-01-20 2022-07-28 Rush University Medical Center Improved treatment for globoid cell leukodsytrophy or krabbe disease
EP4447952A4 (en) * 2021-12-16 2025-11-19 Us Gov Veterans Affairs Benzoic acid salts for the treatment of injuries and disorders of the nervous system
US12042476B2 (en) * 2022-02-04 2024-07-23 Mcmaster University Methods for the treatment of lysosomal storage diseases

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CA2311356C (en) 1998-01-28 2004-07-13 Warner-Lambert Company Method for treating alzheimer's disease
US20010028895A1 (en) 2000-02-04 2001-10-11 Bisgaier Charles L. Methods of treating alzheimer's disease
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GB0718824D0 (en) * 2007-09-26 2007-11-07 Univ Ramot Methods of treating lysosomal storage disorders
US20120114670A1 (en) * 2007-10-02 2012-05-10 University Of Rochester Methods and compositions related to synergistic responses to oncogenic mutations
US9388413B2 (en) * 2008-10-08 2016-07-12 Trustees Of Dartmouth College Method for selectively inhibiting ACAT1 in the treatment of neurodegenerative diseases
US9388414B2 (en) * 2008-10-08 2016-07-12 Trustees Of Dartmouth College Method for selectively inhibiting ACAT1 in the treatment of neurodegenerative diseases
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JP2014511391A (ja) * 2011-03-07 2014-05-15 フォンダッツィオーネ・テレソン Ｔｆｅｂリン酸化阻害剤およびその使用
US9845327B2 (en) * 2011-06-22 2017-12-19 The General Hospital Corporation Treatment of proteinopathies
PL399467A1 (pl) * 2012-06-08 2013-12-09 3G Therapeutics Inc. Zastosowanie genisteiny do obnizania poziomu zwiazków organicznych spichrzanych w komórkach w leczeniu i/lub zapobieganiu lizosomalnych chorób spichrzeniowych (LChS)
US9750712B2 (en) 2012-12-07 2017-09-05 Rush University Medical Center Composition and method for treating neuronal ceroid lipofuscinosis
CA3176253A1 (en) * 2014-11-19 2016-05-26 Rush University Medical Center Use of cinnamic acid or sodium benzoate for treating lysosomal disorders

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AU2015350223B2 (en) 2021-04-01
CA2967066A1 (en) 2016-05-26
US12023345B2 (en) 2024-07-02
CN107205976A (zh) 2017-09-26
EP4026545A1 (en) 2022-07-13
US20250134911A1 (en) 2025-05-01
JP7752958B2 (ja) 2025-10-14
JP2022003060A (ja) 2022-01-11
WO2016081365A1 (en) 2016-05-26
US20170354666A1 (en) 2017-12-14
ES2914085T3 (es) 2022-06-07
JP2020100621A (ja) 2020-07-02
JP2021107404A (ja) 2021-07-29
JP2017536363A (ja) 2017-12-07
EP3220906B1 (en) 2022-03-02
CA3176253A1 (en) 2016-05-26
EP3220906A4 (en) 2018-04-25
AU2015350223A1 (en) 2017-05-11
EP3220906A1 (en) 2017-09-27
US20230037062A1 (en) 2023-02-02

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