HRP20201831T1 - Optimizirani promoter rpe65 i kodirajuće sekvence - Google Patents

Optimizirani promoter rpe65 i kodirajuće sekvence Download PDF

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HRP20201831T1
HRP20201831T1 HRP20201831TT HRP20201831T HRP20201831T1 HR P20201831 T1 HRP20201831 T1 HR P20201831T1 HR P20201831T T HRP20201831T T HR P20201831TT HR P20201831 T HRP20201831 T HR P20201831T HR P20201831 T1 HRP20201831 T1 HR P20201831T1
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sequence
vector
rpe
seq
specific promoter
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HRP20201831TT
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Alexander Smith
Robin Ali
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Ucl Business Ltd
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    • 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs 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
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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
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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
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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
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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
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    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/18Carboxylic ester hydrolases (3.1.1)
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    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/01Carboxylic ester hydrolases (3.1.1)
    • C12Y301/01064Retinoid isomerohydrolase (3.1.1.64)
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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
    • C12N15/8509Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • C12N2015/8518Vectors 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
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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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    • 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

Claims (12)

1. Konstrukcija vektora koja obuhvaća promotor specifičan za retinalni pigmentni epitel (RPE) i operativno povezanu sekvencu polinukleotida, naznačena time, što promotor specifičan za RPE obuhvaća: (a) sekvencu susjednih nukleotida iz SEQ ID NO:1 koji dodjeljuju ekspresiju specifičnu za RPE na operativno povezanoj sekvenci polinukleotida, ili (b) sekvencu koja sadrži sekvencu najmanje 90 %-tno identičnu sekvenci iz (a) i koja zadržava djelovanje promotora specifičnog za RPE, i naznačena time, što operativno povezana sekvenca polinukleotida obuhvaća sekvencu SEQ ID NO: 4.
2. Konstrukcija vektora prema patentnom zahtjevu 1, naznačena time, što promotor specifičan za RPE obuhvaća: (a) najviše 1300 susjednih nukleotida iz SEQ ID NO:1, ili (b) sekvencu koja je najmanje 90 %-tno identična predmetnoj sekvenci od najviše 1300 susjednih nukleotida iz SEQ ID NO:1 i koja zadržava djelovanje promotora specifičnog za RPE.
3. Konstrukcija vektora prema patentnom zahtjevu 2, naznačena time, što promotor specifičan za RPE obuhvaća: (a) najviše 800 susjednih nukleotida iz SEQ ID NO:1, ili (b) sekvencu koja je najmanje 90 %-tno identična predmetnoj sekvenci od najviše 800 susjednih nukleotida iz SEQ ID NO: 1 i koja zadržava djelovanje promotora specifičnog za RPE, opcionalno naznačena time, što promotor specifičan za RPE obuhvaća: (a) sekvencu SEQ ID NO: 2 ili sekvencu nukleotida 12-761 iz SEQ ID NO: 2, ili (b) sekvencu koja je najmanje 90 %-tno identična predmetnoj sekvenci SEQ ID NO: 2, ili koja sadrži nukleotide 12-761 iz SEQ ID NO:2, i koja zadržava djelovanje promotora specifičnog za RPE.
4. Konstrukcija vektora prema bilo kojem od prethodnih patentnih zahtjeva, naznačena time, što je predmetna sekvenca iz (a) ili (b) duga najmanje 500 nukleotida.
5. Vektor koji obuhvaća konstrukciju vektora prema bilo kojem patentnom zahtjevu od 1 do 4.
6. Vektor prema patentnom zahtjevu 5, naznačen time, što je predmetni vektor virusni vektor.
7. Vektor prema patentnom zahtjevu 6, naznačen time, što je vektor vektor adenoasociranog virusa (AAV) ili obuhvaća genom AAV-a ili njegov derivat, opcionalno naznačen time, što derivat predstavlja kimerni derivat, rekombinirani derivat ili derivat modificiran kapsidom.
8. Vektor prema patentnom zahtjevu 7, naznačen time, što genom AAV-a potječe iz deriviranog serotipa ili izolata ili kladusa AAV-a, opcionalno naznačen time, što genom AAV-a potječe iz serotipa 2 AAV-a (AAV2), serotipa 4 AAV-a (AAV4), serotipa 5 AAV (AAV5) ili serotipa 8 AAV-a (AAV8) i/ili naznačen time, što je kapsid dobiven iz AAV5 ili AAV8, poželjno naznačen time, što je genom AAV-a dobiven iz AAV2, a kapsid je dobiven iz AAV5 ili AAV8.
9. Stanica domaćina koja sadrži vektor prema patentnom zahtjevu 5 ili proizvodi virusni vektor prema bilo kojem patentnom zahtjevu od 6 do 8, opcionalno naznačena time, što stanica domaćina jeste stanica HEK293 ili HEK293T.
10. Farmaceutski pripravak koji obuhvaća vektor prema bilo kojem patentnom zahtjevu od 5 do 8 i farmaceutski prihvatljivi nosač.
11. Vektor prema bilo kojem patentnom zahtjevu od 5 do 8 za primjenu u metodi sprječavanja ili liječenja retinalne distrofije.
12. Vektor za primjenu prema patentnom zahtjevu 11, naznačen time, što: (a) retinalna distrofija je Leberova kongenitanla amauroza (LCA), starosna makularna degeneracija (AMD), očni albinizam okulokutanog tipa 1 ili tipa Nettleship-Falls ili deficijencija MERTK-a; i/ili (b) liječenje ili prevencija postiže se primjenom vektora prema bilo kojem patentnom zahtjevu od 5 do 8 u pacijenta putem izravne retinalne, subretinalne ili intravitrealne injekcije, opcionalno naznačen time, što se vektor primjenjuje izravno u retinalni, subretinalni prostor ili intravitrealni prostor.
HRP20201831TT 2015-02-09 2020-11-17 Optimizirani promoter rpe65 i kodirajuće sekvence HRP20201831T1 (hr)

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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
EP16703850.4A EP3256169B1 (en) 2015-02-09 2016-02-08 Optimized rpe65 promoter and coding sequences

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BR (1) BR112017017060A2 (hr)
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HR (1) HRP20201831T1 (hr)
HU (1) HUE052407T2 (hr)
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GB201502137D0 (en) * 2015-02-09 2015-03-25 Ucl Business Plc Treatment
EP3448371A4 (en) * 2016-04-28 2019-09-11 Spark Therapeutics, Inc. TESTING THE RELATIVE EFFECTIVENESS OF ISOMERHYDROLASES FOR THE CODING OF VIRAL VECTORS
RU2020121408A (ru) 2017-11-30 2021-12-30 Фридрих Мишер Инститьют Фор Байомедикал Рисерч ПРОМОТОР SynPIII ДЛЯ СПЕЦИФИЧЕСКОЙ ЭКСПРЕССИИ ГЕНОВ В ПИГМЕНТНОМ ЭПИТЕЛИИ СЕТЧАТКИ
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
GB202002202D0 (en) 2020-02-18 2020-04-01 Ucl Business Plc Gene therapy
AU2021314400A1 (en) * 2020-07-21 2023-03-23 Frontera Therapeutics, Inc. Compositions and methods for the treatment of ocular diseases
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
AU2022340595A1 (en) * 2021-09-06 2024-04-18 Huidagene Therapeutics (Singapore) Pte. Ltd. Treatment of rpe65-associated eye diseases and disorders

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US7740836B2 (en) 2006-05-03 2010-06-22 Fondazione Telethon Methods and compositions for recovering or improving visual function
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HUE044089T2 (hu) 2011-07-18 2019-09-30 Inst Res Biomedicine Neutralizáló anti-influenza A antitestek és alkalmazásaik
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MX2017010292A (es) 2018-03-23
IL287142A (en) 2021-12-01
HK1248525A1 (zh) 2018-10-19
US10568973B2 (en) 2020-02-25
IL287142B1 (en) 2023-07-01
EP3256169A1 (en) 2017-12-20
RS61079B1 (sr) 2020-12-31
WO2016128722A1 (en) 2016-08-18
MX2021006762A (es) 2021-07-15
US20200179535A1 (en) 2020-06-11
SG11201706520UA (en) 2017-09-28
US20180021458A1 (en) 2018-01-25
CN107429252B (zh) 2022-03-29
SI3256169T1 (sl) 2021-01-29
AU2016217654B2 (en) 2021-12-09
IL253915A0 (en) 2017-10-31
DK3256169T3 (da) 2020-11-23
BR112017017060A2 (pt) 2018-04-10
PL3256169T3 (pl) 2021-05-04
EP3256169B1 (en) 2020-09-02
AU2016217654A1 (en) 2017-09-28
HUE052407T2 (hu) 2021-04-28
PH12017501430A1 (en) 2018-01-15
PT3256169T (pt) 2020-11-04
IL253915B (en) 2021-10-31
CA2975850A1 (en) 2016-08-18
EP3769790A1 (en) 2021-01-27
CY1123947T1 (el) 2022-03-24
LT3256169T (lt) 2021-01-11
CN107429252A (zh) 2017-12-01
GB201502137D0 (en) 2015-03-25
ES2830030T3 (es) 2021-06-02
JP2018510620A (ja) 2018-04-19
JP6963503B2 (ja) 2021-11-10

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