HK1248595A1 - 改善視覺的基因療法 - Google Patents

改善視覺的基因療法

Info

Publication number
HK1248595A1
HK1248595A1 HK18108490.8A HK18108490A HK1248595A1 HK 1248595 A1 HK1248595 A1 HK 1248595A1 HK 18108490 A HK18108490 A HK 18108490A HK 1248595 A1 HK1248595 A1 HK 1248595A1
Authority
HK
Hong Kong
Prior art keywords
gene therapy
improve vision
vision
improve
therapy
Prior art date
Application number
HK18108490.8A
Other languages
English (en)
Inventor
Matteo Rizzi
Robin Ali
Alexander Smith
Koji Nishiguchi
Original Assignee
Ucl Business Ltd
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 Ucl Business Ltd filed Critical Ucl Business Ltd
Publication of HK1248595A1 publication Critical patent/HK1248595A1/zh

Links

Classifications

    • 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
    • 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
    • A61K48/0075—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
    • 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
    • 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)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Toxicology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
HK18108490.8A 2015-02-23 2018-07-03 改善視覺的基因療法 HK1248595A1 (zh)

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
HK1248595A1 true HK1248595A1 (zh) 2018-10-19

Family

ID=52822045

Family Applications (2)

Application Number Title Priority Date Filing Date
HK18108485.5A HK1248594A1 (zh) 2015-02-23 2018-07-03 改善視覺的基因療法
HK18108490.8A HK1248595A1 (zh) 2015-02-23 2018-07-03 改善視覺的基因療法

Family Applications Before (1)

Application Number Title Priority Date Filing Date
HK18108485.5A HK1248594A1 (zh) 2015-02-23 2018-07-03 改善視覺的基因療法

Country Status (29)

Country Link
US (2) US20180030477A1 (zh)
EP (1) EP3261679B1 (zh)
JP (1) JP6771474B2 (zh)
KR (1) KR102272466B1 (zh)
CN (1) CN107530449A (zh)
AU (2) AU2016225277B2 (zh)
BR (1) BR112017018062A2 (zh)
CA (1) CA2977464C (zh)
CY (1) CY1126098T1 (zh)
DK (1) DK3261679T3 (zh)
EA (1) EA201791900A1 (zh)
ES (1) ES2947308T3 (zh)
FI (1) FI3261679T3 (zh)
GB (1) GB201503008D0 (zh)
HK (2) HK1248594A1 (zh)
HR (1) HRP20230775T1 (zh)
HU (1) HUE062319T2 (zh)
IL (1) IL254066B (zh)
LT (1) LT3261679T (zh)
MX (1) MX2017010772A (zh)
MY (1) MY186444A (zh)
PH (1) PH12017501525A1 (zh)
PL (1) PL3261679T3 (zh)
PT (1) PT3261679T (zh)
RS (1) RS64372B1 (zh)
SG (2) SG10202005923WA (zh)
SI (1) SI3261679T1 (zh)
WO (1) WO2016135457A1 (zh)
ZA (1) ZA201706440B (zh)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018055131A1 (en) * 2016-09-22 2018-03-29 Universite Pierre Et Marie Curie (Paris 6) 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
JP2009536219A (ja) * 2006-05-04 2009-10-08 ウェイン・ステート・ユニバーシティー ロドプシン拡散のインビボ送達による視覚応答の回復
WO2009127705A1 (en) * 2008-04-18 2009-10-22 Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute For Biomedical Research Novel therapeutical tools and methods for treating blindness
GB2498325B (en) * 2010-10-15 2018-05-02 Eos Neuroscience Inc Engineered human GRM6 enhancer sequence for use in eye therapies
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
LT3261679T (lt) 2023-07-10
AU2016225277B2 (en) 2021-12-09
US20200377907A1 (en) 2020-12-03
CA2977464A1 (en) 2016-09-01
JP6771474B2 (ja) 2020-10-21
GB201503008D0 (en) 2015-04-08
FI3261679T3 (fi) 2023-06-28
EA201791900A1 (ru) 2018-02-28
CY1126098T1 (el) 2023-11-15
CA2977464C (en) 2022-04-05
EP3261679A1 (en) 2018-01-03
IL254066B (en) 2021-10-31
AU2022201553A1 (en) 2022-03-31
EP3261679B1 (en) 2023-05-03
MY186444A (en) 2021-07-22
US20180030477A1 (en) 2018-02-01
SG10202005923WA (en) 2020-07-29
JP2018506982A (ja) 2018-03-15
HK1248594A1 (zh) 2018-10-19
IL254066A0 (en) 2017-10-31
KR20180012737A (ko) 2018-02-06
HRP20230775T1 (hr) 2023-10-27
PL3261679T3 (pl) 2023-09-04
ES2947308T3 (es) 2023-08-04
RS64372B1 (sr) 2023-08-31
DK3261679T3 (da) 2023-06-26
HUE062319T2 (hu) 2023-10-28
MX2017010772A (es) 2018-03-01
PH12017501525A1 (en) 2018-02-05
SG11201706822XA (en) 2017-09-28
PT3261679T (pt) 2023-07-04
BR112017018062A2 (pt) 2018-04-10
CN107530449A (zh) 2018-01-02
ZA201706440B (en) 2021-06-30
WO2016135457A1 (en) 2016-09-01
SI3261679T1 (sl) 2023-09-29
AU2016225277A1 (en) 2017-10-12
KR102272466B1 (ko) 2021-07-02
NZ735735A (en) 2021-09-24

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