HK1248594A1 - 改善视觉的基因疗法 - Google Patents

改善视觉的基因疗法 Download PDF

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Publication number
HK1248594A1
HK1248594A1 HK18108485.5A HK18108485A HK1248594A1 HK 1248594 A1 HK1248594 A1 HK 1248594A1 HK 18108485 A HK18108485 A HK 18108485A HK 1248594 A1 HK1248594 A1 HK 1248594A1
Authority
HK
Hong Kong
Prior art keywords
vector
rod
light
aav
use according
Prior art date
Application number
HK18108485.5A
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English (en)
Chinese (zh)
Inventor
M‧瑞兹
M‧瑞茲
R‧阿里
A‧史密斯
K‧西口
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 HK1248594A1 publication Critical patent/HK1248594A1/zh

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    • 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

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  • 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)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Ophthalmology & Optometry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Toxicology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
HK18108485.5A 2015-02-23 2016-02-19 改善视觉的基因疗法 HK1248594A1 (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB1503008.3 2015-02-23
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
HK1248594A1 true HK1248594A1 (zh) 2018-10-19

Family

ID=52822045

Family Applications (1)

Application Number Title Priority Date Filing Date
HK18108485.5A HK1248594A1 (zh) 2015-02-23 2016-02-19 改善视觉的基因疗法

Country Status (29)

Country Link
US (3) US20180030477A1 (enExample)
EP (1) EP3261679B1 (enExample)
JP (1) JP6771474B2 (enExample)
KR (1) KR102272466B1 (enExample)
CN (1) CN107530449A (enExample)
AU (2) AU2016225277B2 (enExample)
CA (1) CA2977464C (enExample)
CY (1) CY1126098T1 (enExample)
DK (1) DK3261679T3 (enExample)
EA (1) EA201791900A1 (enExample)
ES (1) ES2947308T3 (enExample)
FI (1) FI3261679T3 (enExample)
GB (1) GB201503008D0 (enExample)
HK (1) HK1248594A1 (enExample)
HR (1) HRP20230775T1 (enExample)
HU (1) HUE062319T2 (enExample)
IL (1) IL254066B (enExample)
LT (1) LT3261679T (enExample)
MX (1) MX2017010772A (enExample)
MY (1) MY186444A (enExample)
PH (1) PH12017501525B1 (enExample)
PL (1) PL3261679T3 (enExample)
PT (1) PT3261679T (enExample)
RS (1) RS64372B1 (enExample)
SG (2) SG11201706822XA (enExample)
SI (1) SI3261679T1 (enExample)
SM (1) SMT202300200T1 (enExample)
WO (1) WO2016135457A1 (enExample)
ZA (1) ZA201706440B (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3037436A1 (en) * 2016-09-22 2018-03-29 Sorbonne Universite Optogenetically transformed photoreceptor precursor cells for the use in the treatment of retinal degenerative diseases
WO2023280388A1 (en) * 2021-07-06 2023-01-12 Institute Of Molecular And Clinical Ophthalmology Basel Promoter for specific expression of genes in rod photoreceptors

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
CA2685900A1 (en) * 2006-05-04 2007-11-15 Wayne State University Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids
MX2010011467A (es) * 2008-04-18 2011-02-15 Novartis Forschungsstiftung Novedosas herramientas y metodos terapeuticos para tratar la ceguera.
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
WO2013124477A1 (en) * 2012-02-24 2013-08-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and compositions for treatment of retinal degenerative diseases
AU2015296502A1 (en) * 2014-07-29 2017-02-23 Circuit Therapeutics, Inc. System and method for optogenetic therapy

Also Published As

Publication number Publication date
JP2018506982A (ja) 2018-03-15
AU2016225277B2 (en) 2021-12-09
SMT202300200T1 (it) 2023-09-06
CY1126098T1 (el) 2023-11-15
US20250290096A1 (en) 2025-09-18
GB201503008D0 (en) 2015-04-08
SI3261679T1 (sl) 2023-09-29
MX2017010772A (es) 2018-03-01
CA2977464C (en) 2022-04-05
EA201791900A1 (ru) 2018-02-28
WO2016135457A1 (en) 2016-09-01
HUE062319T2 (hu) 2023-10-28
FI3261679T3 (fi) 2023-06-28
ZA201706440B (en) 2021-06-30
US20200377907A1 (en) 2020-12-03
BR112017018062A2 (pt) 2018-04-10
PH12017501525B1 (en) 2023-08-30
SG10202005923WA (en) 2020-07-29
KR102272466B1 (ko) 2021-07-02
IL254066A0 (en) 2017-10-31
AU2022201553A1 (en) 2022-03-31
AU2016225277A1 (en) 2017-10-12
RS64372B1 (sr) 2023-08-31
HRP20230775T1 (hr) 2023-10-27
US20180030477A1 (en) 2018-02-01
SG11201706822XA (en) 2017-09-28
LT3261679T (lt) 2023-07-10
JP6771474B2 (ja) 2020-10-21
IL254066B (en) 2021-10-31
PL3261679T3 (pl) 2023-09-04
DK3261679T3 (da) 2023-06-26
HK1248595A1 (en) 2018-10-19
EP3261679B1 (en) 2023-05-03
AU2022201553B2 (en) 2024-08-15
CN107530449A (zh) 2018-01-02
PT3261679T (pt) 2023-07-04
MY186444A (en) 2021-07-22
PH12017501525A1 (en) 2018-02-05
CA2977464A1 (en) 2016-09-01
ES2947308T3 (es) 2023-08-04
EP3261679A1 (en) 2018-01-03
NZ735735A (en) 2021-09-24
KR20180012737A (ko) 2018-02-06

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