CN107072982A - 治疗脂质贮积病的方法和组合物 - Google Patents

治疗脂质贮积病的方法和组合物 Download PDF

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Publication number
CN107072982A
CN107072982A CN201580032762.2A CN201580032762A CN107072982A CN 107072982 A CN107072982 A CN 107072982A CN 201580032762 A CN201580032762 A CN 201580032762A CN 107072982 A CN107072982 A CN 107072982A
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CN
China
Prior art keywords
cells
npc1
bryostatin
vimentin
pkc
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.)
Pending
Application number
CN201580032762.2A
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English (en)
Chinese (zh)
Inventor
扬尼斯·A·约安努
劳伦斯·阿尔特斯蒂尔
大卫·R·克罗克福德
萨萨帕纳·贡萨穆特
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.)
Icahn School of Medicine at Mount Sinai
TAO Synergies Inc
Original Assignee
Icahn School of Medicine at Mount Sinai
Neurotrope Bioscience Inc
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.)
Filing date
Publication date
Application filed by Icahn School of Medicine at Mount Sinai, Neurotrope Bioscience Inc filed Critical Icahn School of Medicine at Mount Sinai
Publication of CN107072982A publication Critical patent/CN107072982A/zh
Pending legal-status Critical Current

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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
    • A61K31/33—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
    • A61K31/185—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
    • 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
    • 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
    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients

Landscapes

  • Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Epidemiology (AREA)
  • Emergency Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • Obesity (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Heterocyclic Compounds That Contain Two Or More Ring Oxygen Atoms (AREA)
CN201580032762.2A 2014-04-18 2015-04-14 治疗脂质贮积病的方法和组合物 Pending CN107072982A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201461981473P 2014-04-18 2014-04-18
US61/981,473 2014-04-18
US201461987360P 2014-05-01 2014-05-01
US61/987,360 2014-05-01
PCT/US2015/025821 WO2015160851A1 (en) 2014-04-18 2015-04-14 Methods and compositions for treatment of lipid storage disorders

Publications (1)

Publication Number Publication Date
CN107072982A true CN107072982A (zh) 2017-08-18

Family

ID=54321042

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201580032762.2A Pending CN107072982A (zh) 2014-04-18 2015-04-14 治疗脂质贮积病的方法和组合物

Country Status (9)

Country Link
US (3) US9724328B2 (enExample)
EP (1) EP3131543A4 (enExample)
JP (1) JP2017511387A (enExample)
KR (1) KR20170031653A (enExample)
CN (1) CN107072982A (enExample)
CA (1) CA2946115A1 (enExample)
IL (1) IL248494A0 (enExample)
MX (1) MX2016013680A (enExample)
WO (1) WO2015160851A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112654351A (zh) * 2018-04-20 2021-04-13 乐巢拓普有限公司 稳定的多不饱和化合物及其用途

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11123324B2 (en) 2016-08-18 2021-09-21 Noster Inc. Immunomodulator

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009099563A2 (en) * 2008-02-05 2009-08-13 Blanchette Rockefeller Neurosciences Institute Combination of a nmda receptor channel blocker and a pkc activator for treatment of alzheimer's disease
WO2013071282A1 (en) * 2011-11-13 2013-05-16 Blanchette Rockefeller Neurosciences Institute Pkc activators and combinations thereof

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7256286B2 (en) 1999-11-30 2007-08-14 The Board Of Trustees Of The Leland Stanford Junior University Bryostatin analogues, synthetic methods and uses
US6624189B2 (en) 1999-11-30 2003-09-23 The Board Of Trustees Of The Leland Stanford Junior University Byrostatin analogues, synthetic methods and uses
WO2004004641A2 (en) 2002-07-02 2004-01-15 Blanchette Rockefeller Neurosciences Institute PKC ACTIVATION AS A MEANS FOR ENHANCING sAPPα SECRETION AND IMPROVING COGNITION USING BRYOSTATIN TYPE COMPOUNDS
US8497385B2 (en) 2007-08-31 2013-07-30 The Board Of Trustees Of The Leland Stanford Junior University Bryostatin analogues, synthetic methods and uses
EP2328572B1 (en) 2008-07-28 2018-06-13 Blanchette Rockefeller Neurosciences, Institute Pkc-activating compounds for the treatment of neurodegenerative diseases
US20120156180A1 (en) 2009-08-10 2012-06-21 The Board Of Trustees Of The University Of Illinois Compositions and methods for the treatment of krabbe and other neurodegenerative diseases
WO2015148975A1 (en) * 2014-03-27 2015-10-01 Alkon Daniel L Compositions and methods to treat niemann-pick disease

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009099563A2 (en) * 2008-02-05 2009-08-13 Blanchette Rockefeller Neurosciences Institute Combination of a nmda receptor channel blocker and a pkc activator for treatment of alzheimer's disease
WO2013071282A1 (en) * 2011-11-13 2013-05-16 Blanchette Rockefeller Neurosciences Institute Pkc activators and combinations thereof

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
FARSHAD TAMARI等: "PKC Activation in Niemann Pick C1 Cells Restores Subcellular Cholesterol Transport", 《PLOS ONE》 *
YVONNE LANGE等: "Effect of Protein Kinase C on Endoplasmic Reticulum Cholesterol", 《BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS》 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112654351A (zh) * 2018-04-20 2021-04-13 乐巢拓普有限公司 稳定的多不饱和化合物及其用途

Also Published As

Publication number Publication date
MX2016013680A (es) 2017-07-05
US20170172978A1 (en) 2017-06-22
CA2946115A1 (en) 2015-10-22
US20150297559A1 (en) 2015-10-22
EP3131543A1 (en) 2017-02-22
US9724328B2 (en) 2017-08-08
JP2017511387A (ja) 2017-04-20
IL248494A0 (en) 2016-12-29
US20190201377A1 (en) 2019-07-04
WO2015160851A1 (en) 2015-10-22
EP3131543A4 (en) 2017-12-20
KR20170031653A (ko) 2017-03-21

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