CA2880880C - Cyclodextrin for the treatment of lysosomal storage diseases - Google Patents

Cyclodextrin for the treatment of lysosomal storage diseases Download PDF

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Publication number
CA2880880C
CA2880880C CA2880880A CA2880880A CA2880880C CA 2880880 C CA2880880 C CA 2880880C CA 2880880 A CA2880880 A CA 2880880A CA 2880880 A CA2880880 A CA 2880880A CA 2880880 C CA2880880 C CA 2880880C
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CA
Canada
Prior art keywords
cyclodextrin
disease
tocopherol
compound
cholesterol
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
CA2880880A
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English (en)
French (fr)
Other versions
CA2880880A1 (en
Inventor
John Mckew
Wei Zheng
Miao XU
Manju SWAROOP
Juan J. Marugan
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
United States Department of Health and Human Services
Original Assignee
United States Department of Health and Human Services
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.)
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Publication date
Application filed by United States Department of Health and Human Services filed Critical United States Department of Health and Human Services
Publication of CA2880880A1 publication Critical patent/CA2880880A1/en
Application granted granted Critical
Publication of CA2880880C publication Critical patent/CA2880880C/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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Classifications

    • 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
    • A61K31/35—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom
    • A61K31/352—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom condensed with carbocyclic rings, e.g. methantheline 
    • A61K31/353—3,4-Dihydrobenzopyrans, e.g. chroman, catechin
    • A61K31/355—Tocopherols, e.g. vitamin E
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/715—Polysaccharides, i.e. having more than five saccharide radicals attached to each other by glycosidic linkages; Derivatives thereof, e.g. ethers, esters
    • A61K31/716—Glucans
    • A61K31/724—Cyclodextrins
    • 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
    • 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
    • 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
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • 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
    • C—CHEMISTRY; METALLURGY
    • C08—ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08B—POLYSACCHARIDES; DERIVATIVES THEREOF
    • C08B37/00—Preparation of polysaccharides not provided for in groups C08B1/00 - C08B35/00; Derivatives thereof
    • C08B37/0006—Homoglycans, i.e. polysaccharides having a main chain consisting of one single sugar, e.g. colominic acid
    • C08B37/0009—Homoglycans, i.e. polysaccharides having a main chain consisting of one single sugar, e.g. colominic acid alpha-D-Glucans, e.g. polydextrose, alternan, glycogen; (alpha-1,4)(alpha-1,6)-D-Glucans; (alpha-1,3)(alpha-1,4)-D-Glucans, e.g. isolichenan or nigeran; (alpha-1,4)-D-Glucans; (alpha-1,3)-D-Glucans, e.g. pseudonigeran; Derivatives thereof
    • C08B37/0012—Cyclodextrin [CD], e.g. cycle with 6 units (alpha), with 7 units (beta) and with 8 units (gamma), large-ring cyclodextrin or cycloamylose with 9 units or more; Derivatives thereof
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/92—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving lipids, e.g. cholesterol, lipoproteins, or their receptors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Hematology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Diabetes (AREA)
  • Biochemistry (AREA)
  • Urology & Nephrology (AREA)
  • Immunology (AREA)
  • Endocrinology (AREA)
  • Obesity (AREA)
  • Biophysics (AREA)
  • Pathology (AREA)
  • Cell Biology (AREA)
  • Polymers & Plastics (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Food Science & Technology (AREA)
  • Physics & Mathematics (AREA)
  • Analytical Chemistry (AREA)
  • Materials Engineering (AREA)
  • General Physics & Mathematics (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Emergency Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CA2880880A 2012-08-03 2013-08-03 Cyclodextrin for the treatment of lysosomal storage diseases Active CA2880880C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261679668P 2012-08-03 2012-08-03
US61/679,668 2012-08-03
PCT/US2013/053527 WO2014022841A1 (en) 2012-08-03 2013-08-03 Cyclodextrin for the treatment of lysosomal storage diseases

Publications (2)

Publication Number Publication Date
CA2880880A1 CA2880880A1 (en) 2014-02-06
CA2880880C true CA2880880C (en) 2021-01-26

Family

ID=49170855

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2880880A Active CA2880880C (en) 2012-08-03 2013-08-03 Cyclodextrin for the treatment of lysosomal storage diseases

Country Status (7)

Country Link
US (2) US20150216895A1 (enExample)
EP (1) EP2879714B1 (enExample)
JP (3) JP2015524444A (enExample)
CN (2) CN107865968B (enExample)
AU (2) AU2013296170A1 (enExample)
CA (1) CA2880880C (enExample)
WO (1) WO2014022841A1 (enExample)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2880880C (en) 2012-08-03 2021-01-26 John Mckew Cyclodextrin for the treatment of lysosomal storage diseases
WO2015083736A1 (ja) 2013-12-05 2015-06-11 国立大学法人熊本大学 コレステロール蓄積疾患治療薬、およびそのスクリーニング方法
KR20170012555A (ko) 2014-06-12 2017-02-02 더 유니버시티 오브 노트르 담 듀락 신경 질환 및 대뇌 상해의 치료를 위한 조성물 및 방법
US10239830B2 (en) * 2015-02-11 2019-03-26 Icahn School Of Medicine At Mount Sinai Benzenesulfonamide upregulators of NPC1 for Neimann-Pick disease and other lysosomal storage disorders
AU2016276774A1 (en) 2015-06-10 2018-01-18 Vtesse, Inc. Hydroxypropyl beta-cyclodextrin compositions and methods
EP3405501B1 (en) * 2016-01-21 2023-11-15 Aten Porus Lifesciences Cyclodextrin based polymers, methods, compositions and applications thereof
IL298681A (en) 2016-08-02 2023-01-01 Univ Virginia Commonwealth Preparations
CN109982718A (zh) 2016-09-19 2019-07-05 阿托恩波罗斯生命科学 基于环糊精的聚合物、方法、组合物及其应用
JPWO2018199287A1 (ja) * 2017-04-28 2020-03-12 国立大学法人京都大学 クリスタリン網膜症の処置および／または予防方法
WO2019067145A1 (en) * 2017-08-28 2019-04-04 Asdera Llc USE OF CYCLODEXTRINS IN DISEASES AND DISORDERS INVOLVING PHOSPHOLIPID DISRUPTION
JP2020535235A (ja) * 2017-09-28 2020-12-03 アスデラ エルエルシー リン脂質の調節異常が関与する疾患および障害におけるシクロデキストリンの使用
EP3873604B1 (en) 2018-10-29 2024-08-21 Cyclo Therapeutics, Inc. Methods for treating alzheimer's disease with hp-[beta]-cyclodextrin
AU2020204925B2 (en) 2019-01-03 2025-11-20 Cyclarity Therapeutics, Inc. Cyclodextrin dimers, compositions thereof, and uses thereof
JP7264452B2 (ja) * 2019-04-15 2023-04-25 塩水港精糖株式会社 ライソゾーム病の治療または予防のための医薬組成物
US12226431B2 (en) 2021-09-11 2025-02-18 Porosome Therapeutics, Inc. Aerosol and topical administration of a formulation containing cyclodextrin, quercetin and zinc, in combination or separately, to mitigate infection by enveloped viruses
JP7551339B2 (ja) * 2020-06-04 2024-09-17 株式会社三共 遊技機
JP7551340B2 (ja) * 2020-06-04 2024-09-17 株式会社三共 遊技機
WO2022051631A1 (en) * 2020-09-03 2022-03-10 University Of Notre Dame Du Lac Therapy to stimulate hippocampal neural progenitors and adult neurogenesis
US11446325B2 (en) * 2020-09-30 2022-09-20 Cyclolab Cyclodextrin Research And Development Laboratory Ltd. Cyclodextrin derivatives in the treatment or prevention of lysosomal neurodegenerative diseases
JP2025506676A (ja) 2022-02-18 2025-03-13 ベレン セラピューティクス ピー．ビー．シー． ヒドロキシプロピル－ベータ－シクロデキストリンの組成物及びその精製方法
WO2025090588A1 (en) 2023-10-24 2025-05-01 Minerva Neurosciences, Inc. Roluperidone for the treatment of lysosomal storage disorders and their symptoms

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4727064A (en) 1984-04-25 1988-02-23 The United States Of America As Represented By The Department Of Health And Human Services Pharmaceutical preparations containing cyclodextrin derivatives
US6407079B1 (en) 1985-07-03 2002-06-18 Janssen Pharmaceutica N.V. Pharmaceutical compositions containing drugs which are instable or sparingly soluble in water and methods for their preparation
US5376645A (en) 1990-01-23 1994-12-27 University Of Kansas Derivatives of cyclodextrins exhibiting enhanced aqueous solubility and the use thereof
US5262404A (en) 1990-02-15 1993-11-16 The Trustees Of The University Of Pennsylvania/Childrens Hospital Corporation Cyclodextrin polymers and cyclodextrins immobilized on a solid surface
EP0803243B1 (en) 1996-04-24 2003-03-19 Pfizer Inc. Cyclodextrins and phenolic compounds in dental products
FR2767834B1 (fr) 1997-08-29 1999-12-03 Inst Francais Du Petrole Mono et di-derives de cyclodextrines, leurs synthese et purification et leur utilisation en support
AU2001259725A1 (en) 2000-05-15 2001-11-26 The Procter And Gamble Company Compositions comprising cyclodextrin derivatives
US7442372B2 (en) * 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
WO2005112637A1 (en) * 2004-05-14 2005-12-01 Decode Chemistry, Inc. Formulations for non-parenteral use including hydrophobic cyclodextrins
EP1655034A1 (en) 2004-10-10 2006-05-10 Université de Liège Use of cyclodextrin for treatment and prevention of bronchial inflammatory diseases.
US20090258001A1 (en) * 2006-06-06 2009-10-15 Paul Ponath Administration of anti-CD3 antibodies in the treatment of autoimmune diseases
US20120077774A1 (en) * 2009-05-28 2012-03-29 Cornell University Compositions and their use for removing cholesterol
US9044451B2 (en) * 2010-07-19 2015-06-02 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Use of delta tocopherol for the treatment of lysosomal storage disorders
WO2012100142A2 (en) * 2011-01-20 2012-07-26 Cornell University Treatments for retinal disorders
CA2880880C (en) 2012-08-03 2021-01-26 John Mckew Cyclodextrin for the treatment of lysosomal storage diseases
FR3014694B1 (fr) 2013-12-13 2016-11-11 Roquette Freres Compositions a base de methyl-cyclodextrines pour le traitement et/ou la prevention de maladies par augmentation du taux de cholesterol-hdl

Also Published As

Publication number Publication date
HK1210960A1 (en) 2016-05-13
EP2879714B1 (en) 2021-04-14
AU2018202964A1 (en) 2018-05-17
CN107865968A (zh) 2018-04-03
WO2014022841A1 (en) 2014-02-06
AU2013296170A1 (en) 2015-03-12
CA2880880A1 (en) 2014-02-06
CN104684585A (zh) 2015-06-03
JP2015524444A (ja) 2015-08-24
JP2018162277A (ja) 2018-10-18
JP2020097620A (ja) 2020-06-25
US20180110798A1 (en) 2018-04-26
US11020422B2 (en) 2021-06-01
JP6858286B2 (ja) 2021-04-14
EP2879714A1 (en) 2015-06-10
CN107865968B (zh) 2021-03-05
AU2018202964B2 (en) 2019-08-22
US20150216895A1 (en) 2015-08-06

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