KR102786552B1 - 망막 질환 치료를 위한 조성물 및 방법 - Google Patents

망막 질환 치료를 위한 조성물 및 방법 Download PDF

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KR102786552B1
KR102786552B1 KR1020207023349A KR20207023349A KR102786552B1 KR 102786552 B1 KR102786552 B1 KR 102786552B1 KR 1020207023349 A KR1020207023349 A KR 1020207023349A KR 20207023349 A KR20207023349 A KR 20207023349A KR 102786552 B1 KR102786552 B1 KR 102786552B1
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sequence
seq
aav
vector
polynucleotide molecule
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KR20200141435A (ko
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로빈 알리
타카아키 마츠키
알렉산더 스미스
아나스타시오스 조르지아디스
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유씨엘 비즈니스 리미티드
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • 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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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    • 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
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    • 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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  • 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)
KR1020207023349A 2018-01-12 2019-01-14 망막 질환 치료를 위한 조성물 및 방법 Active KR102786552B1 (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB1800546.2 2018-01-12
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 (2)

Publication Number Publication Date
KR20200141435A KR20200141435A (ko) 2020-12-18
KR102786552B1 true KR102786552B1 (ko) 2025-03-26

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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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011034947A2 (en) 2009-09-15 2011-03-24 University Of Washington Reagents and methods for modulating cone photoreceptor activity
WO2015142941A1 (en) 2014-03-17 2015-09-24 Avalanche Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells

Family Cites Families (4)

* 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
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
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

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011034947A2 (en) 2009-09-15 2011-03-24 University Of Washington Reagents and methods for modulating cone photoreceptor activity
WO2015142941A1 (en) 2014-03-17 2015-09-24 Avalanche Biotechnologies, Inc. Compositions and methods for enhanced gene expression in 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
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
ES2988499T3 (es) 2024-11-20
PT3737423T (pt) 2024-10-03
SI3737423T1 (sl) 2025-03-31

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