MA38543A1 - Facteurs de transcription artificiels pour le traitement de maladies provoquées par une haplo-insuffisance d'opa1 - Google Patents

Facteurs de transcription artificiels pour le traitement de maladies provoquées par une haplo-insuffisance d'opa1

Info

Publication number
MA38543A1
MA38543A1 MA38543A MA38543A MA38543A1 MA 38543 A1 MA38543 A1 MA 38543A1 MA 38543 A MA38543 A MA 38543A MA 38543 A MA38543 A MA 38543A MA 38543 A1 MA38543 A1 MA 38543A1
Authority
MA
Morocco
Prior art keywords
opa1
haploinsufficiency
treatment
transcription factors
diseases caused
Prior art date
Application number
MA38543A
Other languages
English (en)
Inventor
Josef Flammer
Albert Neutzner
Alice Huxley
Original Assignee
Aliophtha Ag
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 Aliophtha Ag filed Critical Aliophtha Ag
Publication of MA38543A1 publication Critical patent/MA38543A1/fr

Links

Classifications

    • 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)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

L'invention concerne un facteur de transcription artificiel comprenant une protéine à doigt de zinc polydactyle ciblant spécifiquement le promoteur d'
MA38543A 2013-04-03 2015-10-21 Facteurs de transcription artificiels pour le traitement de maladies provoquées par une haplo-insuffisance d'opa1 MA38543A1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP13162189 2013-04-03
PCT/EP2014/056590 WO2014161881A1 (fr) 2013-04-03 2014-04-02 Facteurs de transcription artificiels pour le traitement de maladies provoquées par une haplo-insuffisance d'opa1

Publications (1)

Publication Number Publication Date
MA38543A1 true MA38543A1 (fr) 2017-02-28

Family

ID=48044671

Family Applications (1)

Application Number Title Priority Date Filing Date
MA38543A MA38543A1 (fr) 2013-04-03 2015-10-21 Facteurs de transcription artificiels pour le traitement de maladies provoquées par une haplo-insuffisance d'opa1

Country Status (16)

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

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11730828B2 (en) 2017-02-07 2023-08-22 The Regents Of The University Of California Gene therapy for haploinsufficiency
CA3091799A1 (fr) * 2018-02-27 2019-09-06 The Board Of Trustees Of The Leland Stanford Junior University Cellules immunitaires modifiees en tant que sondes de diagnostic de maladie
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
CA3244955A1 (fr) 2021-12-30 2023-07-06 Regel Therapeutics, Inc. Compositions pour la modulation de l’expression de la sous-unité alpha 1 du canal sodique à tension et leurs utilisations
KR20260040173A (ko) 2023-05-26 2026-03-24 레겔 테라퓨틱스, 인코포레이티드 나트륨 전압 개폐 채널 알파 서브유닛 2의 발현을 조정하기 위한 조성물 및 이의 용도

Also Published As

Publication number Publication date
CA2908419A1 (fr) 2014-10-09
KR20160003691A (ko) 2016-01-11
AU2014247131A1 (en) 2015-10-22
EP2981550A1 (fr) 2016-02-10
EA201591626A1 (ru) 2016-05-31
CN105358568A (zh) 2016-02-24
WO2014161881A1 (fr) 2014-10-09
PH12015502294A1 (en) 2016-02-15
BR112015025285A2 (pt) 2017-10-10
TW201514200A (zh) 2015-04-16
JP2016515596A (ja) 2016-05-30
TN2015000436A1 (en) 2017-01-03
SG11201508061UA (en) 2015-10-29
AR095983A1 (es) 2015-11-25
US20160039893A1 (en) 2016-02-11

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