IL287142B2 - Optimized RPE65 promoter and coding sequences - Google Patents

Optimized RPE65 promoter and coding sequences

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Publication number
IL287142B2
IL287142B2 IL287142A IL28714221A IL287142B2 IL 287142 B2 IL287142 B2 IL 287142B2 IL 287142 A IL287142 A IL 287142A IL 28714221 A IL28714221 A IL 28714221A IL 287142 B2 IL287142 B2 IL 287142B2
Authority
IL
Israel
Prior art keywords
sequence
rpe65
aav
seq
vector
Prior art date
Application number
IL287142A
Other languages
English (en)
Hebrew (he)
Other versions
IL287142B1 (en
IL287142A (en
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 IL287142A publication Critical patent/IL287142A/en
Publication of IL287142B1 publication Critical patent/IL287142B1/en
Publication of IL287142B2 publication Critical patent/IL287142B2/en

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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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/18—Carboxylic ester hydrolases (3.1.1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • 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
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • 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
    • 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
    • C12N15/861—Adenoviral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/01—Carboxylic ester hydrolases (3.1.1)
    • C12Y301/01064—Retinoid isomerohydrolase (3.1.1.64)
    • 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
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • C12N2015/8518—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic expressing industrially exogenous proteins, e.g. for pharmaceutical use, human insulin, blood factors, immunoglobulins, pseudoparticles
    • 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)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Urology & Nephrology (AREA)
  • Virology (AREA)
  • Vascular Medicine (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Ophthalmology & Optometry (AREA)
  • Dermatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Eye Examination Apparatus (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
IL287142A 2015-02-09 2016-02-08 Optimized RPE65 promoter and coding sequences IL287142B2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GBGB1502137.1A GB201502137D0 (en) 2015-02-09 2015-02-09 Treatment
PCT/GB2016/050289 WO2016128722A1 (en) 2015-02-09 2016-02-08 Optimized rpe65 promoter and coding sequences

Publications (3)

Publication Number Publication Date
IL287142A IL287142A (en) 2021-12-01
IL287142B1 IL287142B1 (en) 2023-07-01
IL287142B2 true IL287142B2 (en) 2023-11-01

Family

ID=52746363

Family Applications (2)

Application Number Title Priority Date Filing Date
IL287142A IL287142B2 (en) 2015-02-09 2016-02-08 Optimized RPE65 promoter and coding sequences
IL253915A IL253915B (en) 2015-02-09 2017-08-08 Optimized rpe65 promoter and coding sequences

Family Applications After (1)

Application Number Title Priority Date Filing Date
IL253915A IL253915B (en) 2015-02-09 2017-08-08 Optimized rpe65 promoter and coding sequences

Country Status (24)

Country Link
US (3) US10568973B2 (enExample)
EP (2) EP3769790A1 (enExample)
JP (1) JP6963503B2 (enExample)
CN (1) CN107429252B (enExample)
AU (1) AU2016217654B2 (enExample)
BR (1) BR112017017060A2 (enExample)
CA (1) CA2975850C (enExample)
CY (1) CY1123947T1 (enExample)
DK (1) DK3256169T3 (enExample)
ES (1) ES2830030T3 (enExample)
GB (1) GB201502137D0 (enExample)
HR (1) HRP20201831T1 (enExample)
HU (1) HUE052407T2 (enExample)
IL (2) IL287142B2 (enExample)
LT (1) LT3256169T (enExample)
MX (2) MX383316B (enExample)
PH (1) PH12017501430A1 (enExample)
PL (1) PL3256169T3 (enExample)
PT (1) PT3256169T (enExample)
RS (1) RS61079B1 (enExample)
SG (1) SG11201706520UA (enExample)
SI (1) SI3256169T1 (enExample)
SM (1) SMT202000655T1 (enExample)
WO (1) WO2016128722A1 (enExample)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201502137D0 (en) * 2015-02-09 2015-03-25 Ucl Business Plc Treatment
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
CA3022386A1 (en) * 2016-04-28 2017-11-02 Spark Therapeutics, Inc. Relative potency assay for viral vector encoding isomerohydrolases
US11591615B2 (en) * 2017-11-30 2023-02-28 Friedrich Miescher Institute For Biomedical Research SynPIII, a promoter for the specific expression of genes in retinal pigment epithelium
CN111118017B (zh) * 2018-11-01 2023-10-03 上海朗昇生物科技有限公司 治疗Leber先天性黑蒙症的载体及其应用
CN113795575A (zh) 2019-02-04 2021-12-14 自由行疗法有限公司 多核苷酸
WO2021055760A1 (en) * 2019-09-18 2021-03-25 Intergalactic Therapeutics, Inc. Synthetic dna vectors and methods of use
GB202002202D0 (en) 2020-02-18 2020-04-01 Ucl Business Plc Gene therapy
CN113952471A (zh) * 2020-07-21 2022-01-21 英斯培瑞有限公司 用于治疗眼部疾病的组合物和方法
JP2023535232A (ja) * 2020-07-21 2023-08-16 インスピラール リミテッド 眼疾患の処置のための組成物および方法
CN111826378B (zh) * 2020-08-05 2022-05-27 武汉纽福斯生物科技有限公司 一种编码人受体酪氨酸激酶Mer的核苷酸序列及其应用
CN112522292B (zh) * 2020-10-29 2023-05-02 南京启真基因工程有限公司 一种用于构建先天性黑蒙症克隆猪核供体细胞的CRISPR/Cas9系统及其应用
CN115997012B (zh) * 2021-09-06 2024-03-08 辉大(上海)生物科技有限公司 Rpe65相关眼睛疾病和障碍的治疗
EP4646212A1 (en) * 2023-01-05 2025-11-12 Opus Genetics, Inc. Gene therapy for ocular disease
CN116898991A (zh) * 2023-06-20 2023-10-20 浙江大学 一种可用于活体的可视化的神经活动状态评估方法及工具病毒载体
CN117018235A (zh) * 2023-06-20 2023-11-10 浙江大学 一种可用于活体的可视化神经环路的方法及工具病毒载体
WO2026150224A1 (en) 2024-12-30 2026-07-16 MeiraGTx Ocular UK Limited Modified adeno-associated virus (aav) viral capsid proteins and methods of use

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WO2009105690A2 (en) * 2008-02-21 2009-08-27 Targeted Genetics Corporation Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea
WO2013173129A2 (en) * 2012-05-15 2013-11-21 Avalanche Australia Pty Ltd. Treatment of amd using aav sflt-1

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JP4819683B2 (ja) * 2004-08-06 2011-11-24 一般財団法人化学及血清療法研究所 酵母プロモーター
US7740836B2 (en) 2006-05-03 2010-06-22 Fondazione Telethon Methods and compositions for recovering or improving visual function
HUE044089T2 (hu) * 2011-07-18 2019-09-30 Inst Res Biomedicine Neutralizáló anti-influenza A antitestek és alkalmazásaik
US9163259B2 (en) 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
GB201502137D0 (en) * 2015-02-09 2015-03-25 Ucl Business Plc Treatment
GB202002073D0 (en) * 2020-02-14 2020-04-01 Ucl Business Ltd Horizontal cells
GB202002202D0 (en) * 2020-02-18 2020-04-01 Ucl Business Plc Gene therapy

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009105690A2 (en) * 2008-02-21 2009-08-27 Targeted Genetics Corporation Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea
WO2013173129A2 (en) * 2012-05-15 2013-11-21 Avalanche Australia Pty Ltd. Treatment of amd using aav sflt-1

Non-Patent Citations (2)

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BAINBRIDGE, JAMES WB, ET AL., EFFECT OF GENE THERAPY ON VISUAL FUNCTION IN LEBER'S CONGENITALAMAUROSIS., 31 December 2008 (2008-12-31) *
DATABASE GENESEQ (ONLINE): TREATMENT-RELATED PROMOTER SEQUENCE, SEQ ID NO: 30., ", 16 January 2014 (2014-01-16) *

Also Published As

Publication number Publication date
JP2018510620A (ja) 2018-04-19
AU2016217654B2 (en) 2021-12-09
IL253915B (en) 2021-10-31
CA2975850A1 (en) 2016-08-18
CY1123947T1 (el) 2022-03-24
US20200179535A1 (en) 2020-06-11
US20180021458A1 (en) 2018-01-25
MX2021006762A (es) 2021-07-15
HK1248525A1 (zh) 2018-10-19
IL287142B1 (en) 2023-07-01
PT3256169T (pt) 2020-11-04
JP6963503B2 (ja) 2021-11-10
EP3256169A1 (en) 2017-12-20
SG11201706520UA (en) 2017-09-28
WO2016128722A1 (en) 2016-08-18
GB201502137D0 (en) 2015-03-25
NZ735329A (en) 2024-08-30
PL3256169T3 (pl) 2021-05-04
RS61079B1 (sr) 2020-12-31
US10568973B2 (en) 2020-02-25
DK3256169T3 (da) 2020-11-23
ES2830030T3 (es) 2021-06-02
AU2016217654A1 (en) 2017-09-28
EP3769790A1 (en) 2021-01-27
PH12017501430A1 (en) 2018-01-15
IL287142A (en) 2021-12-01
US20240293580A1 (en) 2024-09-05
SMT202000655T1 (it) 2021-01-05
BR112017017060A2 (pt) 2018-04-10
EP3256169B1 (en) 2020-09-02
MX2017010292A (es) 2018-03-23
CA2975850C (en) 2024-06-18
CN107429252A (zh) 2017-12-01
CN107429252B (zh) 2022-03-29
HRP20201831T1 (hr) 2021-01-08
HUE052407T2 (hu) 2021-04-28
IL253915A0 (en) 2017-10-31
SI3256169T1 (sl) 2021-01-29
MX383316B (es) 2025-03-13
LT3256169T (lt) 2021-01-11
US11969478B2 (en) 2024-04-30

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