EA201791900A1 - Генная терапия для улучшения зрения - Google Patents

Генная терапия для улучшения зрения

Info

Publication number
EA201791900A1
EA201791900A1 EA201791900A EA201791900A EA201791900A1 EA 201791900 A1 EA201791900 A1 EA 201791900A1 EA 201791900 A EA201791900 A EA 201791900A EA 201791900 A EA201791900 A EA 201791900A EA 201791900 A1 EA201791900 A1 EA 201791900A1
Authority
EA
Eurasian Patent Office
Prior art keywords
photoreceptor
gene therapy
stick
reacts
improvement
Prior art date
Application number
EA201791900A
Other languages
English (en)
Inventor
Маттео Риззи
Робин Али
Александер Смит
Кодзи Нисигути
Original Assignee
Юсл Бизнес Плк
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 Юсл Бизнес Плк filed Critical Юсл Бизнес Плк
Publication of EA201791900A1 publication Critical patent/EA201791900A1/ru

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Classifications

    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • 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
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

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

Abstract

Изобретение относится к использованию векторов генной терапии для улучшения зрения путем введения в здоровые фоторецепторные клетки-палочки пациента, страдающего дисфункцией и/или деградацией фоторецепторов-колбочек, нуклеиновой кислоты, кодирующей генный продукт, который является светочувствительным и/или который изменяет эндогенную светочувствительную передачу сигналов в фоторецепторной клетке таким образом, что диапазон интенсивностей освещения, на который реагирует фоторецептор-палочка, увеличивается и/или увеличивается скорость, с которой фоторецептор-палочка реагирует на свет.
EA201791900A 2015-02-23 2016-02-19 Генная терапия для улучшения зрения EA201791900A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GB201503008A GB201503008D0 (en) 2015-02-23 2015-02-23 Treatment
PCT/GB2016/050419 WO2016135457A1 (en) 2015-02-23 2016-02-19 Gene therapy to improve vision

Publications (1)

Publication Number Publication Date
EA201791900A1 true EA201791900A1 (ru) 2018-02-28

Family

ID=52822045

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201791900A EA201791900A1 (ru) 2015-02-23 2016-02-19 Генная терапия для улучшения зрения

Country Status (29)

Country Link
US (2) US20180030477A1 (ru)
EP (1) EP3261679B1 (ru)
JP (1) JP6771474B2 (ru)
KR (1) KR102272466B1 (ru)
CN (1) CN107530449A (ru)
AU (2) AU2016225277B2 (ru)
BR (1) BR112017018062A2 (ru)
CA (1) CA2977464C (ru)
CY (1) CY1126098T1 (ru)
DK (1) DK3261679T3 (ru)
EA (1) EA201791900A1 (ru)
ES (1) ES2947308T3 (ru)
FI (1) FI3261679T3 (ru)
GB (1) GB201503008D0 (ru)
HK (2) HK1248595A1 (ru)
HR (1) HRP20230775T1 (ru)
HU (1) HUE062319T2 (ru)
IL (1) IL254066B (ru)
LT (1) LT3261679T (ru)
MX (1) MX2017010772A (ru)
MY (1) MY186444A (ru)
PH (1) PH12017501525A1 (ru)
PL (1) PL3261679T3 (ru)
PT (1) PT3261679T (ru)
RS (1) RS64372B1 (ru)
SG (2) SG11201706822XA (ru)
SI (1) SI3261679T1 (ru)
WO (1) WO2016135457A1 (ru)
ZA (1) ZA201706440B (ru)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20200032203A1 (en) * 2016-09-22 2020-01-30 Sorbonne Universite Optogenetically transformed photoreceptor precursor cells for the use in the treatment of retinal degenerative diseases

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2442670A1 (en) * 2001-04-13 2002-10-24 The Trustees Of The University Of Pennsylvania Method of treating or retarding the development of blindness
CN101484005A (zh) * 2006-05-04 2009-07-15 韦恩州立大学 通过向体内递送视紫红质核酸恢复视觉响应
AU2009237585C1 (en) * 2008-04-18 2013-09-26 Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute For Biomedical Research Novel therapeutical tools and methods for treating blindness
US20140099284A1 (en) * 2010-10-15 2014-04-10 Eos Neuroscience, Inc Modulation neural pathways
US8957028B2 (en) * 2010-11-13 2015-02-17 Massachusetts Institute Of Technology Red-shifted opsin molecules and uses thereof
WO2012167109A2 (en) * 2011-06-03 2012-12-06 Massachusetts Eye & Ear Infirmary Rpgrip1 gene therapy for leber congenital amaurosis
US20150038557A1 (en) * 2012-02-24 2015-02-05 Inserm (Institut National De La Sante Et De La Recherche Medicale) Methods and compositions for treatment of retinal degenerative diseases
US20160030765A1 (en) 2014-07-29 2016-02-04 Circuit Therapeutics, Inc. System and method for optogenetic therapy

Also Published As

Publication number Publication date
CA2977464C (en) 2022-04-05
FI3261679T3 (fi) 2023-06-28
KR20180012737A (ko) 2018-02-06
CN107530449A (zh) 2018-01-02
BR112017018062A2 (pt) 2018-04-10
NZ735735A (en) 2021-09-24
ZA201706440B (en) 2021-06-30
KR102272466B1 (ko) 2021-07-02
GB201503008D0 (en) 2015-04-08
HUE062319T2 (hu) 2023-10-28
SG11201706822XA (en) 2017-09-28
WO2016135457A1 (en) 2016-09-01
PH12017501525A1 (en) 2018-02-05
AU2022201553B2 (en) 2024-08-15
IL254066A0 (en) 2017-10-31
PL3261679T3 (pl) 2023-09-04
LT3261679T (lt) 2023-07-10
MX2017010772A (es) 2018-03-01
US20180030477A1 (en) 2018-02-01
AU2016225277B2 (en) 2021-12-09
ES2947308T3 (es) 2023-08-04
RS64372B1 (sr) 2023-08-31
IL254066B (en) 2021-10-31
PT3261679T (pt) 2023-07-04
SI3261679T1 (sl) 2023-09-29
MY186444A (en) 2021-07-22
SG10202005923WA (en) 2020-07-29
JP2018506982A (ja) 2018-03-15
DK3261679T3 (da) 2023-06-26
HK1248594A1 (zh) 2018-10-19
AU2022201553A1 (en) 2022-03-31
HK1248595A1 (zh) 2018-10-19
EP3261679B1 (en) 2023-05-03
CA2977464A1 (en) 2016-09-01
HRP20230775T1 (hr) 2023-10-27
JP6771474B2 (ja) 2020-10-21
US20200377907A1 (en) 2020-12-03
CY1126098T1 (el) 2023-11-15
EP3261679A1 (en) 2018-01-03
AU2016225277A1 (en) 2017-10-12

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