ES2988499T3 - Composiciones y métodos para tratar trastornos de la retina - Google Patents

Composiciones y métodos para tratar trastornos de la retina Download PDF

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Publication number
ES2988499T3
ES2988499T3 ES19701018T ES19701018T ES2988499T3 ES 2988499 T3 ES2988499 T3 ES 2988499T3 ES 19701018 T ES19701018 T ES 19701018T ES 19701018 T ES19701018 T ES 19701018T ES 2988499 T3 ES2988499 T3 ES 2988499T3
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seq
sequence
vector
aav
tcu
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Spanish (es)
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Robin Ali
Takaaki Matsuki
Alexander Smith
Anastasios Georgiadis
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UCL Business Ltd
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UCL Business Ltd
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • A61K35/761Adenovirus
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/12Viral antigens
    • A61K39/235Adenoviridae
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0048Eye, e.g. artificial tears
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • C12N15/861Adenoviral vectors
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14132Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2800/00Nucleic acids vectors
    • C12N2800/22Vectors comprising a coding region that has been codon optimised for expression in a respective host
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector 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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Virology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Mycology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
ES19701018T 2018-01-12 2019-01-14 Composiciones y métodos para tratar trastornos de la retina Active ES2988499T3 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GBGB1800546.2A GB201800546D0 (en) 2018-01-12 2018-01-12 Treatment
PCT/GB2019/050092 WO2019138250A1 (en) 2018-01-12 2019-01-14 Compositions and methods for treating retinal disorders

Publications (1)

Publication Number Publication Date
ES2988499T3 true ES2988499T3 (es) 2024-11-20

Family

ID=61256183

Family Applications (1)

Application Number Title Priority Date Filing Date
ES19701018T Active ES2988499T3 (es) 2018-01-12 2019-01-14 Composiciones y métodos para tratar trastornos de la retina

Country Status (26)

Country Link
US (2) US11680276B2 (https=)
EP (1) EP3737423B1 (https=)
JP (1) JP7289306B2 (https=)
KR (1) KR102786552B1 (https=)
CN (1) CN111936172A (https=)
AU (1) AU2019206314B2 (https=)
BR (1) BR112020014191A2 (https=)
CA (1) CA3088079A1 (https=)
DK (1) DK3737423T3 (https=)
EA (1) EA202091655A1 (https=)
ES (1) ES2988499T3 (https=)
FI (1) FI3737423T3 (https=)
GB (1) GB201800546D0 (https=)
HR (1) HRP20241483T1 (https=)
HU (1) HUE068987T2 (https=)
IL (1) IL275985A (https=)
LT (1) LT3737423T (https=)
MX (1) MX2020007366A (https=)
PH (1) PH12020551071A1 (https=)
PL (1) PL3737423T3 (https=)
PT (1) PT3737423T (https=)
RS (1) RS66227B1 (https=)
SG (1) SG11202006618PA (https=)
SI (1) SI3737423T1 (https=)
SM (1) SMT202400410T1 (https=)
WO (1) WO2019138250A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20230058556A (ko) 2013-03-15 2023-05-03 아스텔라스 인스티튜트 포 리제너러티브 메디슨 만능 줄기 세포로부터 제조된 광수용기 및 광수용기 전구체
US11241460B2 (en) 2013-03-15 2022-02-08 Astellas Institute For Regenerative Medicine Photoreceptors and photoreceptor progenitors produced from pluripotent stem cells
GB2584664B (en) * 2019-06-10 2023-05-24 Newcells Biotech Ltd Improved retinal organoids and methods of making the same
CN113025616B (zh) * 2021-04-21 2023-05-02 中国热带农业科学院橡胶研究所 一种橡胶树泛素基因启动子proHbUBI2及其克隆与应用
CN118076629A (zh) * 2021-05-20 2024-05-24 阿特玛治疗公司 Kcnv2变体及其用途
WO2023039175A2 (en) * 2021-09-09 2023-03-16 Applied Genetic Technologies Corporation Predictors of achromatopsia treatment efficacy

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20090018031A1 (en) 2006-12-07 2009-01-15 Switchgear Genomics Transcriptional regulatory elements of biological pathways tools, and methods
WO2011034947A2 (en) * 2009-09-15 2011-03-24 University Of Washington Reagents and methods for modulating cone photoreceptor activity
EP3591055A1 (en) * 2011-01-07 2020-01-08 Applied Genetic Technologies Corporation Promoters derived from the 5' nontranslated region (5'-ntr) of the human cyclic nucleotide gated beta 3 (cngb3) gene, expression cassettes and vectors comprising said promoters, for use treating achromatopsia and other diseases
KR102537394B1 (ko) * 2014-03-17 2023-05-30 애드베룸 바이오테크놀로지스, 인코포레이티드 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법
MY187898A (en) * 2015-03-02 2021-10-27 Adverum Biotechnologies Inc Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
WO2017144080A1 (en) 2016-02-23 2017-08-31 Eyeserv Gmbh Gene therapy for the treatment of a disease of retinal cone cells

Also Published As

Publication number Publication date
US12467066B2 (en) 2025-11-11
EP3737423B1 (en) 2024-09-18
JP7289306B2 (ja) 2023-06-09
PH12020551071A1 (en) 2021-09-06
CN111936172A (zh) 2020-11-13
JP2021510301A (ja) 2021-04-22
KR20200141435A (ko) 2020-12-18
EP3737423A1 (en) 2020-11-18
MX2020007366A (es) 2020-09-03
DK3737423T3 (da) 2024-10-21
AU2019206314B2 (en) 2025-02-13
IL275985A (en) 2020-08-31
KR102786552B1 (ko) 2025-03-26
SG11202006618PA (en) 2020-08-28
FI3737423T3 (fi) 2024-11-02
CA3088079A1 (en) 2019-07-18
AU2019206314A1 (en) 2020-08-13
HRP20241483T1 (hr) 2025-01-03
GB201800546D0 (en) 2018-02-28
NZ766316A (en) 2024-05-31
PL3737423T3 (pl) 2025-01-27
SMT202400410T1 (it) 2025-01-14
EA202091655A1 (ru) 2020-11-20
HUE068987T2 (hu) 2025-02-28
US11680276B2 (en) 2023-06-20
US20200392536A1 (en) 2020-12-17
RS66227B1 (sr) 2024-12-31
WO2019138250A1 (en) 2019-07-18
US20240067989A1 (en) 2024-02-29
BR112020014191A2 (pt) 2022-02-22
LT3737423T (lt) 2024-11-25
PT3737423T (pt) 2024-10-03
SI3737423T1 (sl) 2025-03-31

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