AU2016343855B2 - Compositions and methods for treatment of peroxisomal disorders and leukodystrophies - Google Patents

Compositions and methods for treatment of peroxisomal disorders and leukodystrophies Download PDF

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AU2016343855B2
AU2016343855B2 AU2016343855A AU2016343855A AU2016343855B2 AU 2016343855 B2 AU2016343855 B2 AU 2016343855B2 AU 2016343855 A AU2016343855 A AU 2016343855A AU 2016343855 A AU2016343855 A AU 2016343855A AU 2016343855 B2 AU2016343855 B2 AU 2016343855B2
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agent
dendrimer
dendrimers
therapeutic agent
leukodystrophy
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AU2016343855A1 (en
Inventor
Seyed Ali FATEMI
Ozgul GOK
Sujatha Kannan
Kannan Rangaramanujam
Bela TURK
Fan Zhang
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Johns Hopkins University
Kennedy Krieger Institute Inc
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Johns Hopkins University
Kennedy Krieger Institute Inc
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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
    • 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
    • A61K31/195—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
    • 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
    • A61K47/50—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
    • 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
    • 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
    • A61K47/59—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
    • A61K47/595—Polyamides, e.g. nylon
    • 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
    • A61K47/50—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
    • 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
    • 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
    • A61K47/59—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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
    • A61K49/0004—Screening or testing of compounds for diagnosis of disorders, assessment of conditions, e.g. renal clearance, gastric emptying, testing for diabetes, allergy, rheuma, pancreas functions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
    • A61K49/001—Preparation for luminescence or biological staining
    • A61K49/0013—Luminescence
    • A61K49/0017—Fluorescence in vivo
    • A61K49/0019—Fluorescence in vivo characterised by the fluorescent group, e.g. oligomeric, polymeric or dendritic molecules
    • A61K49/0021—Fluorescence in vivo characterised by the fluorescent group, e.g. oligomeric, polymeric or dendritic molecules the fluorescent group being a small organic molecule
    • A61K49/0032—Methine dyes, e.g. cyanine dyes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
    • A61K49/001—Preparation for luminescence or biological staining
    • A61K49/0013—Luminescence
    • A61K49/0017—Fluorescence in vivo
    • A61K49/005—Fluorescence in vivo characterised by the carrier molecule carrying the fluorescent agent
    • A61K49/0054—Macromolecular compounds, i.e. oligomers, polymers, dendrimers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P15/00—Drugs for genital or sexual disorders; Contraceptives
    • A61P15/08—Drugs for genital or sexual disorders; Contraceptives for gonadal disorders or for enhancing fertility, e.g. inducers of ovulation or of spermatogenesis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/18—Antioxidants, e.g. antiradicals
    • 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
    • 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
    • A61P39/00—General protective or antinoxious agents
    • A61P39/06—Free radical scavengers or antioxidants
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Public Health (AREA)
  • Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Endocrinology (AREA)
  • Diabetes (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biochemistry (AREA)
  • Reproductive Health (AREA)
  • Rheumatology (AREA)
  • Pathology (AREA)
  • Urology & Nephrology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Pregnancy & Childbirth (AREA)
  • Dermatology (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)
  • Medicinal Preparation (AREA)
AU2016343855A 2015-10-29 2016-10-31 Compositions and methods for treatment of peroxisomal disorders and leukodystrophies Active AU2016343855B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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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AU2016343855A1 AU2016343855A1 (en) 2018-05-10
AU2016343855B2 true AU2016343855B2 (en) 2019-10-03

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US (2) US20170119899A1 (enExample)
EP (1) EP3368088B1 (enExample)
JP (2) JP7088473B2 (enExample)
CN (1) CN108430514B (enExample)
AU (1) AU2016343855B2 (enExample)
CA (1) CA3003589C (enExample)
WO (1) WO2017075580A1 (enExample)

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JP6342575B2 (ja) * 2014-08-13 2018-06-13 ザ・ジョンズ・ホプキンス・ユニバーシティー 脳腫瘍への選択的デンドリマー送達
EP3615086A4 (en) 2017-04-27 2021-01-06 The Johns Hopkins University COMPOSITIONS OF DENDRIMERS FOR USE IN ANGIOGRAPHY
WO2018208707A1 (en) * 2017-05-08 2018-11-15 Neurovia, Inc. Methods and compositions for treating demyelinating diseases
CN111615528A (zh) 2017-11-10 2020-09-01 约翰霍普金斯大学 树枝状聚合物递送系统和其使用方法
US11679104B2 (en) 2017-12-15 2023-06-20 Duke University Compositions and methods of enhancing the homing and/or engraftment of hematopoietic cells in the central nervous system
EP3866811A4 (en) * 2018-10-19 2022-11-02 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
CN116075322A (zh) * 2020-04-24 2023-05-05 阿什瓦塔治疗股份有限公司 用于治疗重度急性呼吸窘迫综合征的树枝状大分子组合物和方法
PH12023550133A1 (en) * 2020-07-17 2024-06-24 Ashvattha Therapeutics Inc Dendrimer compositions and methods for drug delivery to injured kidney
WO2023081750A1 (en) * 2021-11-04 2023-05-11 The Children's Hospital Of Philadelphia Compositions and methods for treating metachromatic leukodystrophy disease and related disorders
WO2024081687A2 (en) * 2022-10-12 2024-04-18 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

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WO2009046446A2 (en) * 2007-10-05 2009-04-09 Wayne State University Dendrimers for sustained release of compounds
US20120003155A1 (en) * 2009-06-15 2012-01-05 National Institutes Of Health Dendrimer based nanodevices for therapeutic and imaging purposes
WO2014197909A1 (en) * 2013-06-07 2014-12-11 The Administrators Of The Tulane Educational Fund Analogs of pituitary adenylate cyclase-activating polypeptide (pacap) and methods for their use
WO2015027068A1 (en) * 2013-08-21 2015-02-26 Concordia University Dendrimer-resveratrol complex

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JP2016517883A (ja) * 2013-05-03 2016-06-20 オレゴン ヘルス アンド サイエンス ユニバーシティー Ｘ連鎖副腎白質ジストロフィーの処置におけるソベチロムの使用
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US20080031848A1 (en) * 2006-05-22 2008-02-07 Elan Pharmaceuticals, Inc. Preparation of Polymer Conjugates of Therapeutic, Agricultural, and Food Additive Compounds
WO2009046446A2 (en) * 2007-10-05 2009-04-09 Wayne State University Dendrimers for sustained release of compounds
US20120003155A1 (en) * 2009-06-15 2012-01-05 National Institutes Of Health Dendrimer based nanodevices for therapeutic and imaging purposes
WO2014197909A1 (en) * 2013-06-07 2014-12-11 The Administrators Of The Tulane Educational Fund Analogs of pituitary adenylate cyclase-activating polypeptide (pacap) and methods for their use
WO2015027068A1 (en) * 2013-08-21 2015-02-26 Concordia University Dendrimer-resveratrol complex

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CA3003589A1 (en) 2017-05-04
US20220071923A1 (en) 2022-03-10
WO2017075580A1 (en) 2017-05-04
EP3368088C0 (en) 2023-06-07
CA3003589C (en) 2021-12-07
AU2016343855A1 (en) 2018-05-10
JP2022105767A (ja) 2022-07-14
US20170119899A1 (en) 2017-05-04
JP2018531965A (ja) 2018-11-01
JP7088473B2 (ja) 2022-06-21
CN108430514B (zh) 2023-11-03
EP3368088B1 (en) 2023-06-07
EP3368088A1 (en) 2018-09-05
CN108430514A (zh) 2018-08-21

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