DK2624832T3 - Hdac-inhibitorer til behandling af charcot-marie-tooths sygdom - Google Patents

Hdac-inhibitorer til behandling af charcot-marie-tooths sygdom Download PDF

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Publication number
DK2624832T3
DK2624832T3 DK11767988.6T DK11767988T DK2624832T3 DK 2624832 T3 DK2624832 T3 DK 2624832T3 DK 11767988 T DK11767988 T DK 11767988T DK 2624832 T3 DK2624832 T3 DK 2624832T3
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Denmark
Prior art keywords
mice
hspb1
disease
mutant
axonal
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DK11767988.6T
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English (en)
Inventor
Den Bosch Ludo Van
Wim Robberecht
Outryve D'ydewalle Constantin Van
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Vib Vzw
Life Sciences Res Partners Vzw
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    • 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
    • A61K31/4353Heterocyclic 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 ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/437Heterocyclic 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 ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
    • A01K67/0275Genetically modified vertebrates, e.g. transgenic
    • 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
    • A61K31/41Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
    • A61K31/42Oxazoles
    • A61K31/422Oxazoles not condensed and containing further heterocyclic rings
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Environmental Sciences (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Animal Husbandry (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)

Claims (5)

1. Selektiv HDAC6-inhibitor til anvendelse til behandling af Charcot-Marie-Tooths sygdom.
2. Inhibitor ifølge krav 1 til anvendelse til behandling af CMT-sygdommen ifølge krav 1, hvor Charcot-Marie-Tooths sygdom er aksonal CMT eller distal hereditær motorisk neuropati.
3. Inhibitor ifølge krav 1 til anvendelse til behandling af Charcot-Marie-Tooths sygdom ifølge krav 1, hvor CMT-sygdommen er kendetegnet ved mindst én mutation i HSPBl-genet eller - proteinet.
4. Inhibitor ifølge krav 1 til anvendelse til behandling af CMT-sygdommen ifølge krav 3, hvor mutationen i HSPBl-genet eller -proteinet er en mutation i alfa-crystallindomænet af HSPB1, især i rest S135, eller i den C-terminale hale, især i rest P182.
5. Inhibitor ifølge krav 1 til anvendelse til behandling af CMT-sygdommen ifølge krav 1, hvor den selektive HDAC6-inhibitor er valgt blandt tubacin og tubastatin A.
DK11767988.6T 2010-10-08 2011-10-06 Hdac-inhibitorer til behandling af charcot-marie-tooths sygdom DK2624832T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US40479610P 2010-10-08 2010-10-08
PCT/EP2011/067438 WO2012045804A1 (en) 2010-10-08 2011-10-06 Hdac inhibitors to treat charcot-marie-tooth disease

Publications (1)

Publication Number Publication Date
DK2624832T3 true DK2624832T3 (da) 2018-01-08

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ID=44789459

Family Applications (1)

Application Number Title Priority Date Filing Date
DK11767988.6T DK2624832T3 (da) 2010-10-08 2011-10-06 Hdac-inhibitorer til behandling af charcot-marie-tooths sygdom

Country Status (7)

Country Link
US (3) US20130227717A1 (da)
EP (1) EP2624832B1 (da)
AU (1) AU2011311531B2 (da)
CA (1) CA2812855C (da)
DK (1) DK2624832T3 (da)
ES (1) ES2647368T3 (da)
WO (1) WO2012045804A1 (da)

Families Citing this family (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2011311531B2 (en) 2010-10-08 2014-11-20 Life Sciences Research Partners Vzw HDAC inhibitors to treat Charcot-Marie-Tooth disease
WO2014072714A1 (en) 2012-11-07 2014-05-15 Karus Therapeutics Ltd Novel histone deacetylase inhibitors and their use in therapy
WO2014163380A1 (ko) * 2013-04-02 2014-10-09 사회복지법인 삼성생명공익재단 샤르코-마리-투스 질환 치료제의 스크리닝 방법 및 이에 이용되는 자가분화운동신경세포
WO2014163381A1 (ko) * 2013-04-02 2014-10-09 사회복지법인 삼성생명공익재단 Hsp27 변이(s135f) 매개의 샤르코-마리-투스 질환동물모델
CN105283544B (zh) * 2013-04-02 2019-10-18 三星生命公益财团 作为hsp27突变型(s135f)携带者的cmt病的动物模型
KR20140120834A (ko) * 2013-04-02 2014-10-14 주식회사 종근당 샤르코-마리-투스 질환 치료제의 스크리닝 방법 및 이에 이용되는 자가 분화 운동신경세포
AU2014264370B2 (en) 2013-05-10 2017-12-14 Karus Therapeutics Ltd Novel histone deacetylase inhibitors
GB201419264D0 (en) 2014-10-29 2014-12-10 Karus Therapeutics Ltd Compounds
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US20160158231A1 (en) * 2014-12-05 2016-06-09 Vib Vzw Pyrimidine hydroxy amide compounds for treating peripheral neuropathy
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WO2016126721A1 (en) 2015-02-02 2016-08-11 Forma Therapeutics, Inc. 3-aryl-bicyclic [4,5,0] hydroxamic acids as hdac inhibitors
US10272084B2 (en) 2015-06-01 2019-04-30 Regenacy Pharmaceuticals, Llc Histone deacetylase 6 selective inhibitors for the treatment of cisplatin-induced peripheral neuropathy
US10555935B2 (en) 2016-06-17 2020-02-11 Forma Therapeutics, Inc. 2-spiro-5- and 6-hydroxamic acid indanes as HDAC inhibitors
WO2018125799A2 (en) 2016-12-29 2018-07-05 Viamet Pharmaceuticals (Bermuda), Ltd. Metalloenzyme inhibitor compounds
AU2017387033B2 (en) 2016-12-29 2024-02-01 Ji Xing Pharmaceuticals Hong Kong Limited Metalloenzyme inhibitor compounds
WO2018165520A1 (en) 2017-03-10 2018-09-13 Vps-3, Inc. Metalloenzyme inhibitor compounds
WO2020018647A1 (en) * 2018-07-17 2020-01-23 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Methods of treating pacs1 and pacs2 syndromes
WO2020081737A1 (en) 2018-10-16 2020-04-23 Massachusetts Institute Of Technology Compositions and methods for inducing intestinal stem cell regeneration
CA3156060A1 (en) 2019-09-27 2021-04-01 Takeda Pharmaceutical Company Limited 2-ISOINDOL-1,3,4-OXADIAZOLE DERIVATIVES USEFUL AS HDAC6 INHIBITORS
EP4326263A1 (en) 2021-04-23 2024-02-28 Tenaya Therapeutics, Inc. Hdac6 inhibitors for use in the treatment of dilated cardiomyopathy
TW202308619A (zh) 2021-05-04 2023-03-01 美商特納亞治療股份有限公司 用於治療代謝疾病和hfpef的hdac6抑制劑
WO2022251871A1 (en) * 2021-05-27 2022-12-01 The Brigham And Women's Hospital, Inc. Small molecule inhibitors of formation of neutrophil-derived extracellular traps (netosis) and uses thereof
WO2023288110A1 (en) * 2021-07-15 2023-01-19 The United States Government As Represented By The Department Of Veterans Affairs Romidepsin as a therapeutic agent for nerve-injury induced neuropathic pain and spasticity

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Publication number Publication date
CA2812855A1 (en) 2012-04-12
AU2011311531A1 (en) 2013-04-11
US9238028B2 (en) 2016-01-19
CA2812855C (en) 2017-12-19
AU2011311531B2 (en) 2014-11-20
US20140329849A1 (en) 2014-11-06
US20130227717A1 (en) 2013-08-29
US20160000766A1 (en) 2016-01-07
WO2012045804A1 (en) 2012-04-12
EP2624832A1 (en) 2013-08-14
ES2647368T3 (es) 2017-12-21
EP2624832B1 (en) 2017-09-27

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