HRP20201225T1 - Terapije poboljšavanja gena kod nasljedne degeneracije mrežnice uzrokovanu mutacijama u genu prpf31 - Google Patents

Terapije poboljšavanja gena kod nasljedne degeneracije mrežnice uzrokovanu mutacijama u genu prpf31 Download PDF

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HRP20201225T1
HRP20201225T1 HRP20201225TT HRP20201225T HRP20201225T1 HR P20201225 T1 HRP20201225 T1 HR P20201225T1 HR P20201225T T HRP20201225T T HR P20201225TT HR P20201225 T HRP20201225 T HR P20201225T HR P20201225 T1 HRP20201225 T1 HR P20201225T1
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vector
prpf31
pharmaceutical composition
eye
mutations
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HRP20201225TT
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English (en)
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Eric A. Pierce
Michael H. FARKAS
Maria E. SOUSA
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Massachusetts Eye & Ear Infirmary
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • 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
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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
    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
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  • General Chemical & Material Sciences (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)

Claims (13)

1. Vektor Adeno-pridruženog virusa tipa 2 (AAV2) koji sadrži sekvencu koju kodira ljudski PRPF31, operativno povezan s promotorom koji pokreće ekspresiju u epitelnim stanicama retinalnog pigmenta (RPE), pri čemu sekvenca PRPF31 jest ili sadrži ili kodira isti protein kao i nts 1319-2818 od SEQ ID BR.:34.
2. Vektor iz tvrdnje 1, gdje je promotor CAG, CASI, RPE65 ili VMD2 promotor.
3. Vektor iz tvrdnje 1, u kojem je sekvenca PRPF31 optimizirana kodonom.
4. Vektor prema bilo kojoj od prethodnih tvrdnji, koji nadalje sadrži AAV kapsidni polipeptid koji ima aminokiselinsku sekvencu odabranu iz grupe koja se sastoji od SEQ ID BR.: 1, 3, 5, 7, 9, 11, 13, 15, 19, 20, 21, 22, 23, 24, 25 i 26.
5. Farmaceutski sastav koji sadrži vektor iz bilo koje tvrdnje od 1-4, formuliran za primjenu putem sub-retinalne injekcije.
6. Farmaceutski sastav koji se uglavnom sastoji od sustava za isporuku gena koji sadrži vektor bilo koje tvrdnje od 1 do 4 u otapalu.
7. Farmaceutski sastav koji se uglavnom sastoji od sustava za isporuku gena koji sadrži vektor bilo koje tvrdnje od 1 do 4 umetnut u matricu sa sporim otpuštanjem.
8. Farmaceutski sastav prema bilo kojoj od tvrdnji 6 i 7, u kojem sustav za isporuku gena nadalje sadrži pomagačkih virusa, proteina i / ili lipida.
9. Farmaceutski sastav koji sadrži jednu ili više rekombinantnih stanica koje sadrže vektor bilo koje tvrdnje od 1 do 4.
10. Vektor iz tvrdnji 1-4, za upotrebu u liječenju retinitisa pigmentose uzrokovanog mutacijama PRPF31 u oku ljudskog subjekta.
11. Vektor iz tvrdnji 1-4, za upotrebu kod povećanje ekspresije PRPF31 u oku ljudskog subjekta.
12. Farmaceutski sastav prema bilo kojoj od tvrdnji 5 do 9, namijenjen upotrebi u liječenju retinitis pigmentose uzrokovane mutacijama u PRPF31 u oku ljudskog subjekta.
13. Farmaceutski sastav prema bilo kojoj od tvrdnji 5 do 9, za upotrebu kod povećanje ekspresije PRPF31 u oku ljudskog subjekta.
HRP20201225TT 2015-03-06 2016-03-07 Terapije poboljšavanja gena kod nasljedne degeneracije mrežnice uzrokovanu mutacijama u genu prpf31 HRP20201225T1 (hr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201562129638P 2015-03-06 2015-03-06
US201562147307P 2015-04-14 2015-04-14
PCT/US2016/021226 WO2016144892A1 (en) 2015-03-06 2016-03-07 Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene
EP16762307.3A EP3265568B1 (en) 2015-03-06 2016-03-07 Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene

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HRP20201225T1 true HRP20201225T1 (hr) 2020-11-13

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US (3) US20180043034A1 (hr)
EP (2) EP3748007B1 (hr)
JP (3) JP6812368B2 (hr)
CY (1) CY1123158T1 (hr)
DK (1) DK3265568T3 (hr)
ES (1) ES2806054T3 (hr)
HR (1) HRP20201225T1 (hr)
HU (1) HUE051491T2 (hr)
LT (1) LT3265568T (hr)
PL (1) PL3265568T3 (hr)
PT (1) PT3265568T (hr)
RS (1) RS60668B1 (hr)
SI (1) SI3265568T1 (hr)
SM (1) SMT202000423T1 (hr)
WO (1) WO2016144892A1 (hr)

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JP2021059561A (ja) 2021-04-15
JP2023071867A (ja) 2023-05-23
HUE051491T2 (hu) 2021-03-01
EP3748007A1 (en) 2020-12-09
RS60668B1 (sr) 2020-09-30
JP2018511652A (ja) 2018-04-26
WO2016144892A1 (en) 2016-09-15
HK1247637A1 (en) 2018-09-28
EP3265568B1 (en) 2020-05-06
ES2806054T3 (es) 2021-02-16
DK3265568T3 (da) 2020-08-10
PT3265568T (pt) 2020-08-20
EP3748007B1 (en) 2024-06-26
JP7239550B2 (ja) 2023-03-14
JP6812368B2 (ja) 2021-01-13
CY1123158T1 (el) 2021-10-29
US20210069347A1 (en) 2021-03-11
EP3265568A4 (en) 2018-08-08
US20250195691A1 (en) 2025-06-19
PL3265568T3 (pl) 2020-11-30
EP3748007C0 (en) 2024-06-26
US20180043034A1 (en) 2018-02-15

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