CA2977464C - Gene therapy to improve vision - Google Patents

Gene therapy to improve vision Download PDF

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Publication number
CA2977464C
CA2977464C CA2977464A CA2977464A CA2977464C CA 2977464 C CA2977464 C CA 2977464C CA 2977464 A CA2977464 A CA 2977464A CA 2977464 A CA2977464 A CA 2977464A CA 2977464 C CA2977464 C CA 2977464C
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CA
Canada
Prior art keywords
aav
rod
use according
light
photoreceptor
Prior art date
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Active
Application number
CA2977464A
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English (en)
French (fr)
Other versions
CA2977464A1 (en
Inventor
Matteo Rizzi
Robin Ali
Alexander Smith
Koji Nishiguchi
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
UCL Business Ltd
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
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Publication of CA2977464A1 publication Critical patent/CA2977464A1/en
Application granted granted Critical
Publication of CA2977464C publication Critical patent/CA2977464C/en
Active legal-status Critical Current
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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)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (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)
CA2977464A 2015-02-23 2016-02-19 Gene therapy to improve vision Active CA2977464C (en)

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 (2)

Publication Number Publication Date
CA2977464A1 CA2977464A1 (en) 2016-09-01
CA2977464C true CA2977464C (en) 2022-04-05

Family

ID=52822045

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2977464A Active CA2977464C (en) 2015-02-23 2016-02-19 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) SG10202005923WA (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
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
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
EP3437473A1 (en) * 2006-05-04 2019-02-06 Wayne State University Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids
KR20100136550A (ko) * 2008-04-18 2010-12-28 노파르티스 포르슝스티프퉁 쯔바이크니덜라쑹 프리드리히 미셔 인스티튜트 포 바이오메디칼 리서치 실명을 치료하기 위한 신규의 치료 도구 및 방법
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
JP2017527419A (ja) 2014-07-29 2017-09-21 サーキット セラピューティクス， インコーポレイテッド 光遺伝学療法のためのシステムおよび方法

Also Published As

Publication number Publication date
IL254066A0 (en) 2017-10-31
NZ735735A (en) 2021-09-24
EP3261679A1 (en) 2018-01-03
MX2017010772A (es) 2018-03-01
US20180030477A1 (en) 2018-02-01
JP2018506982A (ja) 2018-03-15
CN107530449A (zh) 2018-01-02
HK1248594A1 (zh) 2018-10-19
HRP20230775T1 (hr) 2023-10-27
AU2022201553A1 (en) 2022-03-31
PL3261679T3 (pl) 2023-09-04
ZA201706440B (en) 2021-06-30
RS64372B1 (sr) 2023-08-31
CY1126098T1 (el) 2023-11-15
HK1248595A1 (en) 2018-10-19
AU2016225277B2 (en) 2021-12-09
PT3261679T (pt) 2023-07-04
US20250290096A1 (en) 2025-09-18
IL254066B (en) 2021-10-31
GB201503008D0 (en) 2015-04-08
BR112017018062A2 (pt) 2018-04-10
WO2016135457A1 (en) 2016-09-01
MY186444A (en) 2021-07-22
SG10202005923WA (en) 2020-07-29
ES2947308T3 (es) 2023-08-04
LT3261679T (lt) 2023-07-10
AU2022201553B2 (en) 2024-08-15
PH12017501525A1 (en) 2018-02-05
KR102272466B1 (ko) 2021-07-02
AU2016225277A1 (en) 2017-10-12
US20200377907A1 (en) 2020-12-03
KR20180012737A (ko) 2018-02-06
DK3261679T3 (da) 2023-06-26
SG11201706822XA (en) 2017-09-28
FI3261679T3 (fi) 2023-06-28
SMT202300200T1 (it) 2023-09-06
EP3261679B1 (en) 2023-05-03
HUE062319T2 (hu) 2023-10-28
CA2977464A1 (en) 2016-09-01
PH12017501525B1 (en) 2023-08-30
SI3261679T1 (sl) 2023-09-29
EA201791900A1 (ru) 2018-02-28
JP6771474B2 (ja) 2020-10-21

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Effective date: 20210218