JP7088473B2 - ペルオキシソーム障害および白質ジストロフィーの処置のための組成物および方法 - Google Patents

ペルオキシソーム障害および白質ジストロフィーの処置のための組成物および方法 Download PDF

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JP7088473B2
JP7088473B2 JP2018521617A JP2018521617A JP7088473B2 JP 7088473 B2 JP7088473 B2 JP 7088473B2 JP 2018521617 A JP2018521617 A JP 2018521617A JP 2018521617 A JP2018521617 A JP 2018521617A JP 7088473 B2 JP7088473 B2 JP 7088473B2
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leukodystrophy
dendrimer
peroxisome
disease
cerebral
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JP2018531965A (ja
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スハタ カナン，
カナン ランガラマヌジャム，
ファン ザン，
セイエド アリ ファテミ，
ベラ ターク，
オズグル ゴック，
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ザ・ジョンズ・ホプキンス・ユニバーシティー
ケネディー クリーガー インスティテュート， インコーポレイテッド
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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
    • A61K31/045—Hydroxy compounds, e.g. alcohols; Salts thereof, e.g. alcoholates
    • A61K31/05—Phenols
    • 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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    • 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
    • A61K31/195—Carboxylic acids, e.g. valproic acid having an amino group
    • 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
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
    • A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
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    • A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
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    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
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    • A61K47/56—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
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    • A61K47/60—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
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    • A61P3/06—Antihyperlipidemics
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  • Diabetes (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Reproductive Health (AREA)
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  • Rheumatology (AREA)
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  • Gastroenterology & Hepatology (AREA)
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  • Hospice & Palliative Care (AREA)
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  • 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)
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JP2018521617A 2015-10-29 2016-10-31 ペルオキシソーム障害および白質ジストロフィーの処置のための組成物および方法 Active JP7088473B2 (ja)

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JP2022087580A JP2022105767A (ja) 2015-10-29 2022-05-30 ペルオキシソーム障害および白質ジストロフィーの処置のための組成物および方法

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US201562248163P 2015-10-29 2015-10-29
US62/248,163 2015-10-29
PCT/US2016/059697 WO2017075580A1 (en) 2015-10-29 2016-10-31 Compositions and methods for treatment of peroxisomal disorders and leukodystrophies

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US (2) US20170119899A1 (https=)
EP (1) EP3368088B1 (https=)
JP (2) JP7088473B2 (https=)
CN (1) CN108430514B (https=)
AU (1) AU2016343855B2 (https=)
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WO2018208707A1 (en) * 2017-05-08 2018-11-15 Neurovia, Inc. Methods and compositions for treating demyelinating diseases
EP3707193A1 (en) 2017-11-10 2020-09-16 The Johns Hopkins University Dendrimer delivery system and methods of use thereof
WO2019118951A2 (en) * 2017-12-15 2019-06-20 Duke University Compositions and methods of enhancing the homing and/or engraftment of hematopoietic cells in the central nervous system
US20220072019A1 (en) * 2018-10-19 2022-03-10 Disarm Therapeutics, Inc. Inhibitors of sarm1 in combination with nad+ or a nad+ precursor
WO2020142373A1 (en) * 2019-01-02 2020-07-09 Mayo Foundation For Medical Education And Research Methods and materials for treating leukodystrophies
JP7759322B2 (ja) 2019-12-04 2025-10-23 アシュバッタ セラピューティクス， インコーポレイテッド 眼に薬物送達するためのデンドリマー組成物および方法
IL297531A (en) * 2020-04-24 2022-12-01 Ashvattha Therapeutics Inc Dendrimer preparations and methods for treating severe acute respiratory distress syndrome
CA3189682A1 (en) * 2020-07-17 2022-01-20 Ashvattha Therapeutics, Inc. Dendrimer compositions and methods for drug delivery to injured kidney
EP4426847A4 (en) * 2021-11-04 2026-02-25 Childrens Hospital Philadelphia Compositions and methods for the treatment of metachromatic leukodystrophy and related disorders
US20260103728A1 (en) * 2022-10-12 2026-04-16 The Johns Hopkins University Method of treating lbsl by enhancing dars2 expression
CN116082153A (zh) * 2022-12-09 2023-05-09 长沙贝塔医药科技有限公司 一种丙烯酸酯类衍生物-13c的制备方法
EP4410284A1 (en) * 2023-02-02 2024-08-07 Apteeus Compound and method for the treatment of zellweger spectrum disorder
CN117643581A (zh) * 2023-11-29 2024-03-05 徐州医科大学 白藜芦醇在制备激活sirt1基因的药物中的应用
WO2025240286A1 (en) * 2024-05-14 2025-11-20 Washington State University Neuron targeted 2-deoxyglucose dendrimer for imaging and treatment of neurological diseases

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AU2016343855A1 (en) 2018-05-10
CA3003589A1 (en) 2017-05-04
CA3003589C (en) 2021-12-07
EP3368088B1 (en) 2023-06-07
JP2022105767A (ja) 2022-07-14
JP2018531965A (ja) 2018-11-01
CN108430514B (zh) 2023-11-03
WO2017075580A1 (en) 2017-05-04
US20170119899A1 (en) 2017-05-04
CN108430514A (zh) 2018-08-21
AU2016343855B2 (en) 2019-10-03
EP3368088A1 (en) 2018-09-05
US20220071923A1 (en) 2022-03-10

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