RS66227B1 - Kompozicije i postupci za lečenje poremećaja mrežnjače - Google Patents

Kompozicije i postupci za lečenje poremećaja mrežnjače

Info

Publication number
RS66227B1
RS66227B1 RS20241186A RSP20241186A RS66227B1 RS 66227 B1 RS66227 B1 RS 66227B1 RS 20241186 A RS20241186 A RS 20241186A RS P20241186 A RSP20241186 A RS P20241186A RS 66227 B1 RS66227 B1 RS 66227B1
Authority
RS
Serbia
Prior art keywords
seq
sequence
aav
vector
tcu
Prior art date
Application number
RS20241186A
Other languages
English (en)
Serbian (sr)
Inventor
Robin Ali
Takaaki Matsuki
Alexander Smith
Anastasios Georgiadis
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 RS66227B1 publication Critical patent/RS66227B1/sr

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/22Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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

Landscapes

  • 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)
RS20241186A 2018-01-12 2019-01-14 Kompozicije i postupci za lečenje poremećaja mrežnjače RS66227B1 (sr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1800546.2A GB201800546D0 (en) 2018-01-12 2018-01-12 Treatment
EP19701018.4A EP3737423B1 (en) 2018-01-12 2019-01-14 Compositions and methods for treating retinal disorders
PCT/GB2019/050092 WO2019138250A1 (en) 2018-01-12 2019-01-14 Compositions and methods for treating retinal disorders

Publications (1)

Publication Number Publication Date
RS66227B1 true RS66227B1 (sr) 2024-12-31

Family

ID=61256183

Family Applications (1)

Application Number Title Priority Date Filing Date
RS20241186A RS66227B1 (sr) 2018-01-12 2019-01-14 Kompozicije i postupci za lečenje poremećaja mrežnjače

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
WO2019138250A1 (en) 2019-07-18
US20240067989A1 (en) 2024-02-29
BR112020014191A2 (pt) 2022-02-22
LT3737423T (lt) 2024-11-25
ES2988499T3 (es) 2024-11-20
PT3737423T (pt) 2024-10-03
SI3737423T1 (sl) 2025-03-31

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