JP6771474B2 - 視覚改善のための遺伝子治療 - Google Patents

視覚改善のための遺伝子治療 Download PDF

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Publication number
JP6771474B2
JP6771474B2 JP2017544639A JP2017544639A JP6771474B2 JP 6771474 B2 JP6771474 B2 JP 6771474B2 JP 2017544639 A JP2017544639 A JP 2017544639A JP 2017544639 A JP2017544639 A JP 2017544639A JP 6771474 B2 JP6771474 B2 JP 6771474B2
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rod
aav
photoreceptor
vector
pharmaceutical composition
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Japanese (ja)
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JP2018506982A5 (enExample
JP2018506982A (ja
Inventor
リッツィ，マッテオ
アリ，ロビン
スミス，アレキサンダー
ニシグチ，コージ
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UCL Business Ltd
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UCL Biomedica PLC
UCL Business Ltd
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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)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Ophthalmology & Optometry (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)
JP2017544639A 2015-02-23 2016-02-19 視覚改善のための遺伝子治療 Active JP6771474B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB201503008A GB201503008D0 (en) 2015-02-23 2015-02-23 Treatment
GB1503008.3 2015-02-23
PCT/GB2016/050419 WO2016135457A1 (en) 2015-02-23 2016-02-19 Gene therapy to improve vision

Publications (3)

Publication Number Publication Date
JP2018506982A JP2018506982A (ja) 2018-03-15
JP2018506982A5 JP2018506982A5 (enExample) 2019-04-04
JP6771474B2 true JP6771474B2 (ja) 2020-10-21

Family

ID=52822045

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2017544639A Active JP6771474B2 (ja) 2015-02-23 2016-02-19 視覚改善のための遺伝子治療

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
EP3516046B1 (en) * 2016-09-22 2022-07-13 Sorbonne Université Optogenetically transformed photoreceptor precursor cells for the use in the treatment of retinal degenerative diseases
JP2024524600A (ja) * 2021-07-06 2024-07-05 インスティテュート オブ モレキュラー アンド クリニカル オフサルモロジー バーゼル 桿体光受容体における遺伝子の特異的発現のためのプロモーター

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
EP3437473A1 (en) * 2006-05-04 2019-02-06 Wayne State University Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids
PT2271369E (pt) * 2008-04-18 2014-07-10 Novartis Forschungsstiftung Novas ferramentas terapêuticas e métodos para tratar a cegueira
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
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
US20170182191A1 (en) * 2014-07-29 2017-06-29 Circuit Therapeutics, Inc. System and method for optogenetic therapy

Also Published As

Publication number Publication date
PH12017501525B1 (en) 2023-08-30
AU2016225277A1 (en) 2017-10-12
PT3261679T (pt) 2023-07-04
HRP20230775T1 (hr) 2023-10-27
EP3261679A1 (en) 2018-01-03
GB201503008D0 (en) 2015-04-08
FI3261679T3 (fi) 2023-06-28
WO2016135457A1 (en) 2016-09-01
HUE062319T2 (hu) 2023-10-28
ZA201706440B (en) 2021-06-30
EA201791900A1 (ru) 2018-02-28
DK3261679T3 (da) 2023-06-26
CA2977464A1 (en) 2016-09-01
LT3261679T (lt) 2023-07-10
AU2022201553A1 (en) 2022-03-31
KR102272466B1 (ko) 2021-07-02
SI3261679T1 (sl) 2023-09-29
JP2018506982A (ja) 2018-03-15
MX2017010772A (es) 2018-03-01
US20250290096A1 (en) 2025-09-18
PH12017501525A1 (en) 2018-02-05
ES2947308T3 (es) 2023-08-04
US20200377907A1 (en) 2020-12-03
CN107530449A (zh) 2018-01-02
CA2977464C (en) 2022-04-05
BR112017018062A2 (pt) 2018-04-10
PL3261679T3 (pl) 2023-09-04
US20180030477A1 (en) 2018-02-01
RS64372B1 (sr) 2023-08-31
NZ735735A (en) 2021-09-24
HK1248594A1 (zh) 2018-10-19
IL254066B (en) 2021-10-31
HK1248595A1 (en) 2018-10-19
AU2022201553B2 (en) 2024-08-15
IL254066A0 (en) 2017-10-31
CY1126098T1 (el) 2023-11-15
AU2016225277B2 (en) 2021-12-09
EP3261679B1 (en) 2023-05-03
SMT202300200T1 (it) 2023-09-06
SG11201706822XA (en) 2017-09-28
MY186444A (en) 2021-07-22
KR20180012737A (ko) 2018-02-06
SG10202005923WA (en) 2020-07-29

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