EA201591626A1 - Искусственные факторы транскрипции для лечения заболеваний, вызываемых гаплонедостаточностью opa1 - Google Patents

Искусственные факторы транскрипции для лечения заболеваний, вызываемых гаплонедостаточностью opa1

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Publication number
EA201591626A1
EA201591626A1 EA201591626A EA201591626A EA201591626A1 EA 201591626 A1 EA201591626 A1 EA 201591626A1 EA 201591626 A EA201591626 A EA 201591626A EA 201591626 A EA201591626 A EA 201591626A EA 201591626 A1 EA201591626 A1 EA 201591626A1
Authority
EA
Eurasian Patent Office
Prior art keywords
opa1
artificial transcription
transcription factors
haplosettic
treatment
Prior art date
Application number
EA201591626A
Other languages
English (en)
Inventor
Альберт Нойтцнер
Йозеф Фламмер
Элис Хюкслей
Original Assignee
Алиофта Аг
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 Алиофта Аг filed Critical Алиофта Аг
Publication of EA201591626A1 publication Critical patent/EA201591626A1/ru

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/56—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
    • 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
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/06—Antiglaucoma agents or miotics
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/10—Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/71—Fusion polypeptide containing domain for protein-protein interaction containing domain for transcriptional activaation, e.g. VP16
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Toxicology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Immunology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)

Abstract

Настоящее изобретение относится к искусственному фактору транскрипции, содержащему многопалый белок типа "цинковых пальцев", специфически направленный на промотор ОРА1, соединенный с активирующим белковым доменом и последовательностью ядерной локализации. Искусственные факторы транскрипции, направленные на промотор ОРА1, являются подходящими для лечения заболеваний, ассоциированных с гаплонедостаточностью ОРА1, таких как аутосомно-доминантная атрофия зрительного нерва, синдромная аутосомно-доминантная атрофия зрительного нерва плюс и глаукома с нормальным давлением.
EA201591626A 2013-04-03 2014-04-02 Искусственные факторы транскрипции для лечения заболеваний, вызываемых гаплонедостаточностью opa1 EA201591626A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP13162189 2013-04-03
PCT/EP2014/056590 WO2014161881A1 (en) 2013-04-03 2014-04-02 Artificial transcription factors for the treatment of diseases caused by opa1 haploinsufficiency

Publications (1)

Publication Number Publication Date
EA201591626A1 true EA201591626A1 (ru) 2016-05-31

Family

ID=48044671

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201591626A EA201591626A1 (ru) 2013-04-03 2014-04-02 Искусственные факторы транскрипции для лечения заболеваний, вызываемых гаплонедостаточностью opa1

Country Status (16)

Country Link
US (1) US20160039893A1 (ru)
EP (1) EP2981550A1 (ru)
JP (1) JP2016515596A (ru)
KR (1) KR20160003691A (ru)
CN (1) CN105358568A (ru)
AR (1) AR095983A1 (ru)
AU (1) AU2014247131A1 (ru)
BR (1) BR112015025285A2 (ru)
CA (1) CA2908419A1 (ru)
EA (1) EA201591626A1 (ru)
MA (1) MA38543A1 (ru)
PH (1) PH12015502294A1 (ru)
SG (1) SG11201508061UA (ru)
TN (1) TN2015000436A1 (ru)
TW (1) TW201514200A (ru)
WO (1) WO2014161881A1 (ru)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3579858A4 (en) 2017-02-07 2020-12-23 The Regents of The University of California GENE THERAPY AGAINST HAPLOINSUFFICIENCY
CA3091799A1 (en) * 2018-02-27 2019-09-06 The Board Of Trustees Of The Leland Stanford Junior University Engineered immune cells as diagnostic probes of disease
CN110857440B (zh) * 2018-08-23 2021-02-19 武汉纽福斯生物科技有限公司 重组人ⅱ型线粒体动力蛋白样gtp酶基因序列及其应用
WO2023129940A1 (en) 2021-12-30 2023-07-06 Regel Therapeutics, Inc. Compositions for modulating expression of sodium voltage-gated channel alpha subunit 1 and uses thereof

Also Published As

Publication number Publication date
BR112015025285A2 (pt) 2017-10-10
EP2981550A1 (en) 2016-02-10
KR20160003691A (ko) 2016-01-11
SG11201508061UA (en) 2015-10-29
TN2015000436A1 (en) 2017-01-03
WO2014161881A1 (en) 2014-10-09
AR095983A1 (es) 2015-11-25
MA38543A1 (fr) 2017-02-28
CA2908419A1 (en) 2014-10-09
TW201514200A (zh) 2015-04-16
JP2016515596A (ja) 2016-05-30
CN105358568A (zh) 2016-02-24
AU2014247131A1 (en) 2015-10-22
PH12015502294A1 (en) 2016-02-15
US20160039893A1 (en) 2016-02-11

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