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WO2012037547A3 - Methods and compositions for inhibiting autophagy for the treatment of fibrosis - Google Patents

Methods and compositions for inhibiting autophagy for the treatment of fibrosis Download PDF

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Publication number
WO2012037547A3
WO2012037547A3 PCT/US2011/052128 US2011052128W WO2012037547A3 WO 2012037547 A3 WO2012037547 A3 WO 2012037547A3 US 2011052128 W US2011052128 W US 2011052128W WO 2012037547 A3 WO2012037547 A3 WO 2012037547A3
Authority
WO
WIPO (PCT)
Prior art keywords
fibrosis
treatment
methods
compositions
inhibiting autophagy
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/US2011/052128
Other languages
French (fr)
Other versions
WO2012037547A2 (en
Inventor
Scott L. Friedman
Zahra Ghiassi-Nejad
Virginia Hernandez-Gea
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
Original Assignee
Mount Sinai School of Medicine
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 Mount Sinai School of Medicine filed Critical Mount Sinai School of Medicine
Priority to EP11826075.1A priority Critical patent/EP2616082A2/en
Publication of WO2012037547A2 publication Critical patent/WO2012037547A2/en
Publication of WO2012037547A3 publication Critical patent/WO2012037547A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/335Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/365Lactones
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/335Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/365Lactones
    • A61K31/366Lactones having six-membered rings, e.g. delta-lactones
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/47Quinolines; Isoquinolines
    • A61K31/47064-Aminoquinolines; 8-Aminoquinolines, e.g. chloroquine, primaquine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic 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
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A61K31/52Purines, e.g. adenine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Molecular Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Pulmonology (AREA)
  • Urology & Nephrology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Plant Substances (AREA)

Abstract

Provided are compositions and methods for the treatment of fibrosis. More particularly, autophagy inhibitors and methods of their use are provided for the treatment of fibrosis of liver, lung, kidney, and other tissues, and for the inhibition of stellate cell activation.
PCT/US2011/052128 2010-09-17 2011-09-19 Methods and compositions for inhibiting autophagy for the treatment of fibrosis Ceased WO2012037547A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
EP11826075.1A EP2616082A2 (en) 2010-09-17 2011-09-19 Methods and compositions for inhibiting autophagy for the treatment of fibrosis

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US38407610P 2010-09-17 2010-09-17
US61/384,076 2010-09-17

Publications (2)

Publication Number Publication Date
WO2012037547A2 WO2012037547A2 (en) 2012-03-22
WO2012037547A3 true WO2012037547A3 (en) 2012-06-28

Family

ID=45832287

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2011/052128 Ceased WO2012037547A2 (en) 2010-09-17 2011-09-19 Methods and compositions for inhibiting autophagy for the treatment of fibrosis

Country Status (2)

Country Link
EP (1) EP2616082A2 (en)
WO (1) WO2012037547A2 (en)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE102013110608A1 (en) * 2013-09-26 2015-03-26 Florian Lang Substance for inhibiting tissue calcification, tissue fibrosis and age-associated diseases
CN103948601A (en) * 2014-05-06 2014-07-30 山东大学 Application of 3-methyladenine (3-MA) in preparing medicament for treating atherosclerosis and related diseases thereof
GB201512609D0 (en) 2015-07-17 2015-08-26 Royal Veterinary College The Methods
US10618939B2 (en) * 2016-06-29 2020-04-14 The Hong Kong Polytechnic University Hydrocarbon-stapled polypeptides for enhancement of endosome-lysosomal degradation
EP3620164A1 (en) * 2018-09-05 2020-03-11 Genoscience Pharma SAS Substituted 2,4 diamino-quinoline as new medicament for fibrosis, autophagy and cathepsins b (ctsb), l (ctsl) and d (ctsd) related diseases
CN115151561A (en) * 2019-09-25 2022-10-04 前沿药品公司 Targeted autophagy conjugates and methods
CN113134004A (en) * 2021-03-29 2021-07-20 中国医学科学院医学生物学研究所 Application of 3-methyladenine in preparation of drug for preventing acute pneumonia induced by pseudomonas aeruginosa
CN115960884B (en) * 2022-03-19 2024-03-12 三峡大学 Screening method for nucleic acid aptamer APT-Tan targeting activated hepatic stellate cells

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030203867A1 (en) * 2000-06-30 2003-10-30 Greenberg Arnold H. NIP3 family of proteins
WO2004035782A2 (en) * 2002-10-17 2004-04-29 Artemis Pharmaceuticals Gmbh Sirna mediated gene silencing in transgenic animals
US20040087016A1 (en) * 2000-05-12 2004-05-06 University Of Utah Research Foundation Compositions and methods for cell dedifferentiation and tissue regeneration
US20050009053A1 (en) * 2003-04-25 2005-01-13 Sebastian Boecker Fragmentation-based methods and systems for de novo sequencing
US20080194504A1 (en) * 2004-02-06 2008-08-14 Advanced Bionutrition Corporation Rna-Mediated Interference to Control Disease in Terrestrial and Aquaculture Animals
WO2009065090A2 (en) * 2007-11-14 2009-05-22 University Of Hawaii Pharmaceutical compositions for the treatment of conditions responsive to proteasome inhibition
US20100190749A1 (en) * 2008-11-03 2010-07-29 Pingda Ren Benzoxazole kinase inhibitors and methods of use

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040087016A1 (en) * 2000-05-12 2004-05-06 University Of Utah Research Foundation Compositions and methods for cell dedifferentiation and tissue regeneration
US20030203867A1 (en) * 2000-06-30 2003-10-30 Greenberg Arnold H. NIP3 family of proteins
WO2004035782A2 (en) * 2002-10-17 2004-04-29 Artemis Pharmaceuticals Gmbh Sirna mediated gene silencing in transgenic animals
US20050009053A1 (en) * 2003-04-25 2005-01-13 Sebastian Boecker Fragmentation-based methods and systems for de novo sequencing
US20080194504A1 (en) * 2004-02-06 2008-08-14 Advanced Bionutrition Corporation Rna-Mediated Interference to Control Disease in Terrestrial and Aquaculture Animals
WO2009065090A2 (en) * 2007-11-14 2009-05-22 University Of Hawaii Pharmaceutical compositions for the treatment of conditions responsive to proteasome inhibition
US20100190749A1 (en) * 2008-11-03 2010-07-29 Pingda Ren Benzoxazole kinase inhibitors and methods of use

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
DATABASE GENBANK 7 April 2005 (2005-04-07), retrieved from http://www.ncbi.nlm.nih.gov/nuccore/BV482162 accession no. V482162 *
DATABASE GENBANK retrieved from http://www.ncbi.nlm.nih.gov/nucgss/FH102951 accession no. H102951 *
LUCIANI ET AL.: "Defective CFTR induces aggresome formation and lung inflammation in cystic fibrosis through ROS-mediated autophagy inhibition.", NATURE CELL BIOLOGY, vol. 12, no. 9, September 2010 (2010-09-01), pages 863 - 875 *

Also Published As

Publication number Publication date
EP2616082A2 (en) 2013-07-24
WO2012037547A2 (en) 2012-03-22

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