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

Info

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
Application number
PCT/US2009/037020
Other languages
French (fr)
Other versions
WO2009114729A2 (en
Inventor
Peter G. Schultz
Sassan Azarian
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

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)
  • 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)

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 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 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
WO2016088125A1 (en) 2014-12-03 2016-06-09 Dvashi Zeev Compositions and methods for treatment of retinal degenerative diseases
MA55947A (en) * 2016-08-11 2022-03-23 Intrabio Ltd PHARMACEUTICAL COMPOSITIONS AND USES DIRECTED TO LYSOSOMIAL 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
WO2021050102A1 (en) * 2019-09-09 2021-03-18 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Methods of restoring lysosomal function of retinal pigment epithelial cells by activation of tfeb
WO2021155195A1 (en) * 2020-01-29 2021-08-05 Georgetown University Compositions and methods for treating neurodegenerative, neurodevelopmental, myodegenerative, and lysosomal storage disorders
WO2023141606A2 (en) * 2022-01-20 2023-07-27 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

Similar Documents

Publication Publication Date Title
WO2009114729A3 (en) Compounds, compositions and methods for treating lysosomal storage diseases and disorders
WO2010127099A3 (en) Pharmaceutical compositions comprising epa and a cardiovascular agent and methods of using the same
WO2012024620A3 (en) Autotaxin inhibitors and uses thereof
MX337933B (en) Novel compositions for preventing and/or treating lysosomal storage disorders.
WO2010127152A3 (en) Compounds and compositions as microsomal prostaglandin e synthase-1 inhibitors
WO2012062925A3 (en) Compounds and methods for treating pain
MX350761B (en) Isoindolinone inhibitors of phosphatidylinositol 3-kinase.
WO2010033977A3 (en) Multiheteroaryl compounds as inhibitors of h-pgds and their use for treating prostaglandin d2 mediated diseases
MY163275A (en) Ibat inhibitors for the treatment of liver diseases
TN2010000122A1 (en) Compounds and compositions as modulators of gpr119 activity
MX2009009491A (en) Compounds and compositions as modulators of gpr119 activity.
WO2009129246A3 (en) Compositions and methods for preparing and using same
WO2010083239A3 (en) Therapeutic modulation of vaginal epithelium boundary lubrication
WO2007150046A3 (en) Methods and compositions for treating ophthalmic conditions via modulation of megalin activity
WO2012145651A3 (en) Compounds for the treatment of neuropsychiatric disorders
MX2012004078A (en) Compounds and compositions as modulators of gpr119 activity.
MX2011013032A (en) Inhibitors of phosphatidylinositol 3-kinase.
WO2010097788A3 (en) Visfatin therapeutic agents for the treatment of acne and other conditions
WO2012080497A3 (en) Use of lp-pla2 inhibitors in the treatment and prevention of eye diseases
WO2009085270A3 (en) Use of inhibitors of c0nnexin43 for treatment of fibrotic conditions
WO2010088450A3 (en) Arylamide derivatives useful in the treatment of diseases associated with serca activity
WO2011088160A3 (en) Novel cyp17 inhibitors
WO2010062506A3 (en) Decahydro-1h-indenoquinolinone and decahydro-3h-cyclopentaphenanthridinone cyp17 inhibitors
WO2011014520A3 (en) Compounds and compositions as modulators of gpr119 activity
WO2006078336A3 (en) Compositions and methods for the treatment and prevention of hyperproliferative diseases

Legal Events

Date Code Title Description
121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 09718685

Country of ref document: EP

Kind code of ref document: A2

NENP Non-entry into the national phase

Ref country code: DE

122 Ep: pct application non-entry in european phase

Ref document number: 09718685

Country of ref document: EP

Kind code of ref document: A2