AU2016225277B2 - Gene therapy to improve vision - Google Patents

Gene therapy to improve vision Download PDF

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Publication number
AU2016225277B2
AU2016225277B2 AU2016225277A AU2016225277A AU2016225277B2 AU 2016225277 B2 AU2016225277 B2 AU 2016225277B2 AU 2016225277 A AU2016225277 A AU 2016225277A AU 2016225277 A AU2016225277 A AU 2016225277A AU 2016225277 B2 AU2016225277 B2 AU 2016225277B2
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AU
Australia
Prior art keywords
rod
light
photoreceptor
nucleic acid
aav
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AU2016225277A
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English (en)
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AU2016225277A1 (en
Inventor
Robin Ali
Koji Nishiguchi
Matteo Rizzi
Alexander Smith
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UCL Business Ltd
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UCL Business Ltd
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Publication of AU2016225277B2 publication Critical patent/AU2016225277B2/en
Priority to AU2022201553A priority Critical patent/AU2022201553B2/en
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    • 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
    • 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
    • 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)
AU2016225277A 2015-02-23 2016-02-19 Gene therapy to improve vision Active AU2016225277B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2022201553A AU2022201553B2 (en) 2015-02-23 2022-03-07 Gene therapy to improve vision

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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2022201553A Division AU2022201553B2 (en) 2015-02-23 2022-03-07 Gene therapy to improve vision

Publications (2)

Publication Number Publication Date
AU2016225277A1 AU2016225277A1 (en) 2017-10-12
AU2016225277B2 true AU2016225277B2 (en) 2021-12-09

Family

ID=52822045

Family Applications (2)

Application Number Title Priority Date Filing Date
AU2016225277A Active AU2016225277B2 (en) 2015-02-23 2016-02-19 Gene therapy to improve vision
AU2022201553A Active AU2022201553B2 (en) 2015-02-23 2022-03-07 Gene therapy to improve vision

Family Applications After (1)

Application Number Title Priority Date Filing Date
AU2022201553A Active AU2022201553B2 (en) 2015-02-23 2022-03-07 Gene therapy to improve vision

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

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
I Sandoval et al, "AVV-directed overexpression of R9AP and RGS9-1 in Rat Retina", Investigative ophthalmology&visual science *

Also Published As

Publication number Publication date
JP2018506982A (ja) 2018-03-15
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
HK1248594A1 (zh) 2018-10-19
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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