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WO2009114729A3 - Compounds, compositions and methods for treating lysosomal storage diseases and disorders - Google Patents

Compounds, compositions and methods for treating lysosomal storage diseases and disorders Download PDF

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Publication number
WO2009114729A3
WO2009114729A3 PCT/US2009/037020 US2009037020W WO2009114729A3 WO 2009114729 A3 WO2009114729 A3 WO 2009114729A3 US 2009037020 W US2009037020 W US 2009037020W WO 2009114729 A3 WO2009114729 A3 WO 2009114729A3
Authority
WO
WIPO (PCT)
Prior art keywords
disorders
storage diseases
compositions
compounds
methods
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.)
Ceased
Application number
PCT/US2009/037020
Other languages
French (fr)
Other versions
WO2009114729A2 (en
Inventor
Peter G. Schultz
Sassan Azarian
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.)
IRM LLC
Original Assignee
IRM LLC
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 IRM LLC filed Critical IRM LLC
Publication of WO2009114729A2 publication Critical patent/WO2009114729A2/en
Publication of WO2009114729A3 publication Critical patent/WO2009114729A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

Links

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
    • A61K31/13—Amines
    • 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
    • A61K31/34—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having five-membered rings with one oxygen as the only ring hetero atom, e.g. isosorbide
    • A61K31/343—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having five-membered rings with one oxygen as the only ring hetero atom, e.g. isosorbide condensed with a carbocyclic ring, e.g. coumaran, bufuralol, befunolol, clobenfurol, amiodarone
    • 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
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • A61K31/451—Non condensed piperidines, e.g. piperocaine having a carbocyclic group directly attached to the heterocyclic ring, e.g. glutethimide, meperidine, loperamide, phencyclidine, piminodine
    • 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
    • A61K31/4706—4-Aminoquinolines; 8-Aminoquinolines, e.g. chloroquine, primaquine
    • 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
    • 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
    • A61K31/54—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one sulfur as the ring hetero atoms, e.g. sulthiame
    • A61K31/5415—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one sulfur as the ring hetero atoms, e.g. sulthiame ortho- or peri-condensed with carbocyclic ring systems, e.g. phenothiazine, chlorpromazine, piroxicam
    • 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
    • A61K31/55—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Provided herein are lysosomotropic agents, composition and methods used to treat lysosomal-storage diseases, including ophthalmic conditions associated with the overproduction of waste products that accumulate during the course of phototransduction.
PCT/US2009/037020 2008-03-14 2009-03-12 Compounds, compositions and methods for treating lysosomal storage diseases and disorders Ceased WO2009114729A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US3660308P 2008-03-14 2008-03-14
US61/036,603 2008-03-14

Publications (2)

Publication Number Publication Date
WO2009114729A2 WO2009114729A2 (en) 2009-09-17
WO2009114729A3 true WO2009114729A3 (en) 2009-11-05

Family

ID=40674147

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2009/037020 Ceased WO2009114729A2 (en) 2008-03-14 2009-03-12 Compounds, compositions and methods for treating lysosomal storage diseases and disorders

Country Status (1)

Country Link
WO (1) WO2009114729A2 (en)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011116123A1 (en) * 2010-03-19 2011-09-22 Irm Llc Tafamidis for the treatment of ophthalmic diseases
FR2970965A1 (en) * 2011-01-31 2012-08-03 Centre Nat Rech Scient ANTI-ANGIOGENIC COMPOUNDS, PHARMACEUTICAL COMPOSITIONS COMPRISING SAME, AND USE THEREOF
IL234638A0 (en) * 2014-09-14 2014-12-02 Yeda Res & Dev Nmda receptor antagonists for treating gaucher disease
ES2971592T3 (en) * 2014-12-03 2024-06-06 Mor Research Applic Ltd Compositions for the treatment of degenerative retinal diseases
RS61442B1 (en) 2016-08-11 2021-03-31 Intrabio Ltd Pharmaceutical compositions and uses directed to lysosomal storage disorders
WO2019173482A1 (en) * 2018-03-06 2019-09-12 Sanford Burnham Prebys Medical Discovery Institute 4-aminoquinoline compounds for the treatment of angiogenesis
JP7131803B2 (en) * 2018-05-15 2022-09-06 学校法人大阪医科薬科大学 Preventive or therapeutic agent for diseases caused by Gb3 accumulation
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 activation of tfeb
CA3166527A1 (en) * 2020-01-29 2021-08-05 Charbel MOUSSA Compositions and methods for treating neurodegenerative, neurodevelopmental, myodegenerative, and lysosomal storage disorders
US20250144101A1 (en) * 2022-01-20 2025-05-08 The Johns Hopkins University Lysosomal dysfunction in neurological and psychiatric disorders

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006116716A2 (en) * 2005-04-27 2006-11-02 University Of Florida Materials and methods for enhanced degradation of mutant proteins associated with human disease
WO2007014327A2 (en) * 2005-07-27 2007-02-01 University Of Florida Research Foundation, Inc. Small compounds that correct protein misfolding and uses thereof
WO2007022076A2 (en) * 2005-08-13 2007-02-22 The Uab Research Foundation Prevention of neurodegeneration by macrolide antibiotics

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006116716A2 (en) * 2005-04-27 2006-11-02 University Of Florida Materials and methods for enhanced degradation of mutant proteins associated with human disease
WO2007014327A2 (en) * 2005-07-27 2007-02-01 University Of Florida Research Foundation, Inc. Small compounds that correct protein misfolding and uses thereof
WO2007022076A2 (en) * 2005-08-13 2007-02-22 The Uab Research Foundation Prevention of neurodegeneration by macrolide antibiotics

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
A. NIEMANN ET AL: "The lysosomotropic agent monodansylcadaverine also acts as a solvent polarity probe", JOURNAL OF HISTOCHEMISRTY AND CYTOCHEMISTRY, vol. 48, no. 2, February 2000 (2000-02-01), pages 251 - 258, XP002531220 *
SHAIKH N A ET AL: "AMIODARONE - AN INHIBITOR OF PHOSPHOLIPASE ACTIVITY: A COMPARATIVE STUDY OF THE INHIBITORY EFFECTS OF AMIODARONE, CHLOROQUINE AND CHLORPROMAZINE", MOLECULAR AND CELLULAR BIOCHEMISTRY, NORWELL, MA, US, vol. 76, 1 January 1987 (1987-01-01), pages 163 - 172, XP002935442, ISSN: 0300-8177 *

Also Published As

Publication number Publication date
WO2009114729A2 (en) 2009-09-17

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