JP6550388B2 - 神経変性疾患の治療 - Google Patents

神経変性疾患の治療 Download PDF

Info

Publication number
JP6550388B2
JP6550388B2 JP2016539206A JP2016539206A JP6550388B2 JP 6550388 B2 JP6550388 B2 JP 6550388B2 JP 2016539206 A JP2016539206 A JP 2016539206A JP 2016539206 A JP2016539206 A JP 2016539206A JP 6550388 B2 JP6550388 B2 JP 6550388B2
Authority
JP
Japan
Prior art keywords
laccer
b4galt6
subject
level
astrocytes
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.)
Expired - Fee Related
Application number
JP2016539206A
Other languages
English (en)
Japanese (ja)
Other versions
JP2017502278A5 (enExample
JP2017502278A (ja
Inventor
ジェイ. キンタナ,フランシスコ
ジェイ. キンタナ,フランシスコ
マヨ,リオル
ウェイナー,ハワード
ハルセ,レザ
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.)
The Brigham and Women's Hospital Inc
Original Assignee
The Brigham and Women's Hospital 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 The Brigham and Women's Hospital Inc filed Critical The Brigham and Women's Hospital Inc
Publication of JP2017502278A publication Critical patent/JP2017502278A/ja
Publication of JP2017502278A5 publication Critical patent/JP2017502278A5/ja
Application granted granted Critical
Publication of JP6550388B2 publication Critical patent/JP6550388B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/53—Immunoassay; Biospecific binding assay; Materials therefor
    • G01N33/564—Immunoassay; Biospecific binding assay; Materials therefor for pre-existing immune complex or autoimmune disease, i.e. systemic lupus erythematosus, rheumatoid arthritis, multiple sclerosis, rheumatoid factors or complement components C1-C9
    • 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
    • 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
    • A61K31/535—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 oxygen as the ring hetero atoms, e.g. 1,2-oxazines
    • A61K31/5375—1,4-Oxazines, e.g. morpholine
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/715—Polysaccharides, i.e. having more than five saccharide radicals attached to each other by glycosidic linkages; Derivatives thereof, e.g. ethers, esters
    • A61K31/716—Glucans
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • C12N15/1137—Non-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 against enzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2400/00—Assays, e.g. immunoassays or enzyme assays, involving carbohydrates
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2405/00—Assays, e.g. immunoassays or enzyme assays, involving lipids
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/28—Neurological disorders
    • G01N2800/285—Demyelinating diseases; Multipel sclerosis
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/50—Determining the risk of developing a disease
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/52—Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
  • Urology & Nephrology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Rehabilitation Therapy (AREA)
  • General Physics & Mathematics (AREA)
  • Analytical Chemistry (AREA)
  • Food Science & Technology (AREA)
  • Pathology (AREA)
  • Cell Biology (AREA)
  • Rheumatology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Neurosurgery (AREA)
JP2016539206A 2013-12-12 2014-12-12 神経変性疾患の治療 Expired - Fee Related JP6550388B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201361963738P 2013-12-12 2013-12-12
US61/963,738 2013-12-12
US201462049813P 2014-09-12 2014-09-12
US62/049,813 2014-09-12
PCT/US2014/070099 WO2015089443A2 (en) 2013-12-12 2014-12-12 Treating neurodegenerative disease

Publications (3)

Publication Number Publication Date
JP2017502278A JP2017502278A (ja) 2017-01-19
JP2017502278A5 JP2017502278A5 (enExample) 2018-02-01
JP6550388B2 true JP6550388B2 (ja) 2019-07-24

Family

ID=53371971

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2016539206A Expired - Fee Related JP6550388B2 (ja) 2013-12-12 2014-12-12 神経変性疾患の治療

Country Status (7)

Country Link
US (1) US9927437B2 (enExample)
EP (1) EP3080602B1 (enExample)
JP (1) JP6550388B2 (enExample)
KR (1) KR20160089528A (enExample)
CN (1) CN106030302B (enExample)
CA (1) CA2933554A1 (enExample)
WO (1) WO2015089443A2 (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10512656B2 (en) 2016-04-21 2019-12-24 Baylor College Of Medicine Compositions and methods for the treatment of lysosomal storage disorders and disorders characterized by lysosomal dysfunction
JP7084689B2 (ja) * 2016-12-08 2022-06-15 公益財団法人東京都医学総合研究所 脳組織の異常を伴う非ヒト動物の作製方法およびその利用
WO2019030645A1 (en) * 2017-08-08 2019-02-14 Kashiv Pharma Llc Pharmaceutical composition comprising eliglustat
JP2021515037A (ja) 2018-02-26 2021-06-17 アントルクス,インコーポレーテッド 寛容原性リポソーム及びその使用方法
WO2019169317A1 (en) * 2018-03-02 2019-09-06 Stueve Olaf Methods and compositions for treating natalizumab-associated progressive multifocal encephalopathy
EP3833398A4 (en) * 2018-09-21 2021-12-22 University of Connecticut COMPOSITIONS AND METHODS OF RESTORING PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN'S SYNDROME IN HUMANS
CN114717232A (zh) * 2020-12-22 2022-07-08 未来智人再生医学研究院(广州)有限公司 表达irf-1靶向抑制因子的多能干细胞及其衍生物与应用
CN116794308B (zh) * 2023-07-20 2026-05-26 中国药科大学 β-1,4-半乳糖基转移酶1及其抑制剂在制备治疗急慢性肝病药物中的应用

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5700909A (en) * 1993-07-30 1997-12-23 The Regents Of The University Of California Prosaposin and cytokine-derived peptides
EP1825853A2 (en) * 1998-07-27 2007-08-29 Johns Hopkins University Methods for treating conditions modulated by lactosyceramide
US20070270350A1 (en) * 2003-12-23 2007-11-22 Musc Foundation For Research Development Methods and Compositions for the Prevention and Treatment of Inflammatory Diseases or Conditions
FR2869915B1 (fr) * 2004-05-07 2006-08-18 Merck Sante Soc Par Actions Si Gm3 synthase, comme cible therapeutique dans les complications microvasculaires du diabete
WO2005123055A2 (en) * 2004-06-14 2005-12-29 Musc Foundation For Research Development Methods for treating inflammatory disorders
US7572592B2 (en) * 2005-01-31 2009-08-11 Glycominds Ltd Method for diagnosing multiple sclerosis
US8263576B2 (en) * 2006-08-15 2012-09-11 Mayo Foundation For Medical Education And Research Non-natural sphingolipid analogs and uses thereof
EP2353004B1 (en) 2008-11-12 2018-01-03 Yeda Research and Development Co. Ltd. Diagnosis of multiple sclerosis
US20120128665A1 (en) * 2010-11-18 2012-05-24 Synta Pharmaceuticals Corp. Preselection of subjects for therapeutic treatment based on hypoxic status
AU2011349256B2 (en) * 2010-12-20 2017-01-05 Scidec Therapeutics, Inc. Methods for treating neurological conditions and compositions and materials therefor
CN102994554A (zh) * 2011-09-15 2013-03-27 花王株式会社 神经酰胺和/或葡萄糖神经酰胺产生促进剂的制造方法
CN103059160B (zh) * 2011-10-20 2015-12-02 中国科学院上海药物研究所 β-葡聚糖GFPBW1及其制备方法和用途

Also Published As

Publication number Publication date
US20160313324A1 (en) 2016-10-27
CN106030302B (zh) 2019-09-24
KR20160089528A (ko) 2016-07-27
EP3080602A4 (en) 2017-07-05
WO2015089443A3 (en) 2015-11-12
US9927437B2 (en) 2018-03-27
EP3080602A2 (en) 2016-10-19
CA2933554A1 (en) 2015-06-18
EP3080602B1 (en) 2018-11-28
JP2017502278A (ja) 2017-01-19
WO2015089443A2 (en) 2015-06-18
CN106030302A (zh) 2016-10-12

Similar Documents

Publication Publication Date Title
US9927437B2 (en) Treating neurodegenerative disease
Mayo et al. Regulation of astrocyte activation by glycolipids drives chronic CNS inflammation
Radandish et al. The role of distinct subsets of macrophages in the pathogenesis of MS and the impact of different therapeutic agents on these populations
US9248128B2 (en) Method for enhancing remyelination using GLI1 inhibitors
Smith et al. The impact of regional astrocyte interferon-γ signaling during chronic autoimmunity: a novel role for the immunoproteasome
Cacabelos et al. Neuroimmune crosstalk in CNS disorders: the histamine connection
CA3127452A1 (en) In vitro human blood brain barrier
De Vito et al. MiR‐142‐3p regulates synaptopathy‐driven disease progression in multiple sclerosis
Ulrich et al. Machine learning approach identifies new pathways associated with demyelination in a viral model of multiple sclerosis
Zhan et al. A DEAD‐box RNA helicase Ddx54 protein in oligodendrocytes is indispensable for myelination in the central nervous system
Mayo et al. B4GALT6 regulates astrocyte activation during CNS inflammation
US11208475B1 (en) Methods and compositions for treating inflammatory or autoimmune diseases or conditions using serotonin receptor activators
Hollingsworth et al. Colostrum extracellular vesicles are neuroprotective in models of Parkinson's disease
Liu et al. Atsttrin reduces lipopolysaccharide-induced neuroinflammation by inhibiting the nuclear factor kappa B signaling pathway
US9139879B2 (en) TAM receptors and TAM receptor ligands in detection and modulation of neuropathological disease
Zhang et al. Microglial repopulation alleviates surgery‐induced neuroinflammation and cognitive impairment in a ZEB1‐dependent manner
US11946046B2 (en) Staufen1 regulating agents and associated methods
US20130309238A1 (en) Signal transduction pathway modulation
Dejbakht et al. Multiple Sclerosis: New Insights into Molecular Pathogenesis and Novel Platforms for Disease Treatment
KR20240159829A (ko) 면역계를 조정하기 위한 조성물 및 방법
HK1230277A1 (en) Treating neurodegenerative disease
HK1230277B (en) Treating neurodegenerative disease
CN102370981B (zh) 防治神经退行性疾病的试剂和方法
EP4076429A1 (en) Compositions and methods for treating neuromuscular disorders
Chen Profiling the Molecular Signatures of Astrocytes in Synucleinopathy and Therapeutic Targeting in Aging

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20171212

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20171212

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20180921

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20181030

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20190130

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20190618

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20190701

R150 Certificate of patent or registration of utility model

Ref document number: 6550388

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

LAPS Cancellation because of no payment of annual fees