ES2914085T3 - Composiciones y métodos para el tratamiento de trastornos lisosomales - Google Patents

Composiciones y métodos para el tratamiento de trastornos lisosomales Download PDF

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Publication number
ES2914085T3
ES2914085T3 ES15861122T ES15861122T ES2914085T3 ES 2914085 T3 ES2914085 T3 ES 2914085T3 ES 15861122 T ES15861122 T ES 15861122T ES 15861122 T ES15861122 T ES 15861122T ES 2914085 T3 ES2914085 T3 ES 2914085T3
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tfeb
disease
ppara
composition
lysosomal
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Spanish (es)
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Kalipada Pahan
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Rush University Medical Center
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Rush University Medical Center
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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
    • A61K31/33—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
    • A61K31/045—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
    • 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
    • A61K31/20—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
    • 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
    • A61K31/216—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
    • 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)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Emergency Medicine (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (AREA)
  • Diabetes (AREA)
  • Psychiatry (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Urology & Nephrology (AREA)
  • Pain & Pain Management (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (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)
ES15861122T 2014-11-19 2015-11-16 Composiciones y métodos para el tratamiento de trastornos lisosomales Active ES2914085T3 (es)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
ES2914085T3 true ES2914085T3 (es) 2022-06-07

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ES15861122T Active ES2914085T3 (es) 2014-11-19 2015-11-16 Composiciones y métodos para el tratamiento de trastornos lisosomales

Country Status (9)

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

Families Citing this family (19)

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SG11201509030TA (en) 2013-05-07 2015-11-27 Bio Blast Pharma Ltd Treatment of protein aggregation myopathic and neurodegenerative diseases by parenteral administration of trehalose
WO2016081365A1 (en) * 2014-11-19 2016-05-26 Rush University Medical Center Compositions and methods for treating lysosomal disorders
RU2765625C2 (ru) * 2016-06-13 2022-02-01 Сайньюрекс Интернэшнл (Тайвань) Корп. Сокристаллы бензоата лития и их применения
ES2936094T3 (es) * 2016-06-13 2023-03-14 Syneurx Int Taiwan Corp Cocristales de benzoato de sodio y usos de los mismos
BR112019010210A2 (pt) * 2016-12-29 2019-09-03 Rush University Medical Center melhora na atividade locomotora e aumento da longevidade de indivíduos com lipofuscinose ceroide neuronal infantil tardia por gemfibrozil
US10899844B2 (en) * 2017-02-08 2021-01-26 Novartis Ag FGF21 mimetic antibodies and uses thereof
JP2021511799A (ja) * 2018-01-30 2021-05-13 ラッシュ ユニヴァーシティ メディカル センター 組織および細胞の多重分析のための連続染色
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
EP3796939A4 (en) * 2018-05-22 2022-03-23 Duke University COMPOSITIONS AND METHODS FOR THE TREATMENT OF NEURODEGENERATIVE DISEASES WITH RIFAXIMIN
US12397004B2 (en) 2018-11-05 2025-08-26 Rush University Medical Center Nasal delivery of low-dose aspirin for the treatment of neurodegenerative and lysosomal storage diseases
AU2020204717B2 (en) 2019-01-03 2025-01-16 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Methods and materials for increasing transcription factor EB polypeptide levels
US12478598B2 (en) 2019-02-25 2025-11-25 Rush University Medical Center Compositions including cinnamic acid and methods of use thereof
KR20210143848A (ko) * 2019-03-22 2021-11-29 러쉬 유니버시티 메디컬 센터 리소좀 축적 장애를 위한 비강 유전자 전달 및 경구 신남산, 올레아미드 또는 젬피브로질의 병용
EP4028025A4 (en) * 2019-09-09 2023-07-19 University of Pittsburgh - of the Commonwealth System of Higher Education METHODS OF RESTORING LYSOSOMAL FUNCTION OF RETINAL PIGMENT EPITHELIAL CELLS BY TFEB ACTIVATION
CA3150309A1 (en) * 2019-10-01 2021-04-08 Seelos Therapeutics, Inc. Trehalose formulations and uses thereof
CN116723870A (zh) * 2021-01-20 2023-09-08 拉什大学医学中心 球形细胞脑白质营养不良症或克拉伯病的改良治疗
JP2025503457A (ja) * 2021-12-16 2025-02-04 ザ ユナイテッド ステイツ ガバメント アズ リプレゼンティド バイ ザ デパートメント オブ ヴェテランズ アフェアーズ 神経系損傷及び障害の治療のための安息香酸塩
US20250144101A1 (en) * 2022-01-20 2025-05-08 The Johns Hopkins University Lysosomal dysfunction in neurological and psychiatric disorders
US12042476B2 (en) * 2022-02-04 2024-07-23 Mcmaster University Methods for the treatment of lysosomal storage diseases

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PL1732605T3 (pl) * 2004-03-29 2019-09-30 Wyeth Llc Multiwitaminowe i mineralne suplementy odżywcze
US20060135612A1 (en) * 2004-12-17 2006-06-22 U.S. Department Of Veterans Affairs Method of ameliorating or abrogating the effects of a neurodegenerative disorder, such as amyotrophic lateral sclerosis (ALS), by using a HDAC inhibiting agent
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WO2014089449A1 (en) * 2012-12-07 2014-06-12 Rush University Nedical Center Composition and method for treating neuronal ceroid lipofuscinosis
WO2016081365A1 (en) * 2014-11-19 2016-05-26 Rush University Medical Center Compositions and methods for treating lysosomal disorders

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EP3220906B1 (en) 2022-03-02
US20230037062A1 (en) 2023-02-02
JP2022003060A (ja) 2022-01-11
JP7752958B2 (ja) 2025-10-14
WO2016081365A1 (en) 2016-05-26
AU2015350223A1 (en) 2017-05-11
EP3220906A4 (en) 2018-04-25
KR20170083146A (ko) 2017-07-17
US20250134911A1 (en) 2025-05-01
US20170354666A1 (en) 2017-12-14
CA2967066A1 (en) 2016-05-26
EP3220906A1 (en) 2017-09-27
EP4026545A1 (en) 2022-07-13
JP2020100621A (ja) 2020-07-02
JP2021107404A (ja) 2021-07-29
US12023345B2 (en) 2024-07-02
CA2967066C (en) 2026-01-13
JP2017536363A (ja) 2017-12-07
CN107205976A (zh) 2017-09-26
CA3176253A1 (en) 2016-05-26
AU2015350223B2 (en) 2021-04-01

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