HRP20192141T1 - Genska terapija za retinitis pigmentosa - Google Patents

Genska terapija za retinitis pigmentosa Download PDF

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Publication number
HRP20192141T1
HRP20192141T1 HRP20192141TT HRP20192141T HRP20192141T1 HR P20192141 T1 HRP20192141 T1 HR P20192141T1 HR P20192141T T HRP20192141T T HR P20192141TT HR P20192141 T HRP20192141 T HR P20192141T HR P20192141 T1 HRP20192141 T1 HR P20192141T1
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Croatia
Prior art keywords
nucleic acid
seq
acid encoding
raav
mir
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HRP20192141TT
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English (en)
Croatian (hr)
Inventor
Catherine O'riordan
Matthew ADAMOWICZ
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Genzyme Corporation
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=52829343&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=HRP20192141(T1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genzyme Corporation filed Critical Genzyme Corporation
Publication of HRP20192141T1 publication Critical patent/HRP20192141T1/hr

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted 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
    • 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
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Ophthalmology & Optometry (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Dermatology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
HRP20192141TT 2014-03-21 2015-03-20 Genska terapija za retinitis pigmentosa HRP20192141T1 (hr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461969027P 2014-03-21 2014-03-21
PCT/US2015/021896 WO2015143418A2 (en) 2014-03-21 2015-03-20 Gene therapy for retinitis pigmentosa
EP15716232.2A EP3119437B1 (en) 2014-03-21 2015-03-20 Gene therapy for retinitis pigmentosa

Publications (1)

Publication Number Publication Date
HRP20192141T1 true HRP20192141T1 (hr) 2020-02-21

Family

ID=52829343

Family Applications (2)

Application Number Title Priority Date Filing Date
HRP20231077TT HRP20231077T1 (hr) 2014-03-21 2015-03-20 Genska terapija za pigmentni retinitis
HRP20192141TT HRP20192141T1 (hr) 2014-03-21 2015-03-20 Genska terapija za retinitis pigmentosa

Family Applications Before (1)

Application Number Title Priority Date Filing Date
HRP20231077TT HRP20231077T1 (hr) 2014-03-21 2015-03-20 Genska terapija za pigmentni retinitis

Country Status (36)

Country Link
US (3) US10383953B2 (OSRAM)
EP (3) EP4345165A3 (OSRAM)
JP (3) JP6669664B2 (OSRAM)
KR (2) KR20230006039A (OSRAM)
CN (2) CN106456660B (OSRAM)
AR (1) AR099837A1 (OSRAM)
AU (3) AU2015230942B2 (OSRAM)
CA (2) CA3254798A1 (OSRAM)
CL (1) CL2016002333A1 (OSRAM)
CR (1) CR20160480A (OSRAM)
DK (2) DK3119437T3 (OSRAM)
DO (1) DOP2016000237A (OSRAM)
EA (1) EA201691891A1 (OSRAM)
EC (1) ECSP16083000A (OSRAM)
ES (2) ES2760263T3 (OSRAM)
FI (1) FI3628334T3 (OSRAM)
HR (2) HRP20231077T1 (OSRAM)
HU (2) HUE063460T2 (OSRAM)
IL (3) IL247543B (OSRAM)
LT (2) LT3628334T (OSRAM)
MA (1) MA39390B2 (OSRAM)
MX (2) MX376190B (OSRAM)
MY (2) MY203654A (OSRAM)
NZ (1) NZ724622A (OSRAM)
PE (1) PE20161252A1 (OSRAM)
PH (1) PH12016501684B1 (OSRAM)
PL (2) PL3119437T3 (OSRAM)
PT (2) PT3628334T (OSRAM)
RS (2) RS64611B1 (OSRAM)
SG (3) SG11201607005UA (OSRAM)
SI (2) SI3119437T1 (OSRAM)
TW (2) TWI780401B (OSRAM)
UA (1) UA120050C2 (OSRAM)
UY (1) UY36044A (OSRAM)
WO (1) WO2015143418A2 (OSRAM)
ZA (1) ZA201605924B (OSRAM)

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FI3628334T3 (fi) 2023-09-15
HRP20231077T1 (hr) 2023-12-22
KR20160127832A (ko) 2016-11-04
EP3119437B1 (en) 2019-09-04
ECSP16083000A (es) 2017-02-24
US12201698B2 (en) 2025-01-21
MX2016012201A (es) 2017-01-19
IL284741B2 (en) 2023-02-01
LT3628334T (lt) 2023-09-25
NZ724622A (en) 2022-05-27
WO2015143418A3 (en) 2015-11-19
PT3119437T (pt) 2019-12-12
JP2017509632A (ja) 2017-04-06
RS59634B1 (sr) 2020-01-31
JP6669664B2 (ja) 2020-03-18
CA2943185A1 (en) 2015-09-24
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IL284741B (en) 2022-10-01
PH12016501684A1 (en) 2016-10-03
NZ762841A (en) 2023-11-24
UA120050C2 (uk) 2019-09-25
SI3628334T1 (sl) 2023-11-30
SG10201808218YA (en) 2018-10-30
EP3119437A2 (en) 2017-01-25
KR20230006039A (ko) 2023-01-10
US20220054657A1 (en) 2022-02-24
CN106456660A (zh) 2017-02-22
PL3628334T3 (pl) 2023-12-18
WO2015143418A2 (en) 2015-09-24
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AU2015230942A1 (en) 2016-10-13
CA2943185C (en) 2025-09-16
JP2020059737A (ja) 2020-04-16
LT3119437T (lt) 2019-12-27
SI3119437T1 (sl) 2020-01-31
EA201691891A1 (ru) 2017-01-30
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IL284741A (en) 2021-08-31
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MX376190B (es) 2025-03-07
TWI780401B (zh) 2022-10-11
MA39390A1 (fr) 2017-12-29
AU2021200988B2 (en) 2024-08-01
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JP2022084810A (ja) 2022-06-07
US11103598B2 (en) 2021-08-31
US20170173183A1 (en) 2017-06-22
AU2015230942B2 (en) 2020-11-19
MA39390B2 (fr) 2022-04-29
TW201622752A (zh) 2016-07-01
BR112016021017A2 (pt) 2017-10-03
CA3254798A1 (en) 2025-12-01
AU2021200988A1 (en) 2021-03-11
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HUE046454T2 (hu) 2020-03-30
JP7048563B2 (ja) 2022-04-05
SG11201607005UA (en) 2016-09-29
CL2016002333A1 (es) 2017-05-26
JP7534348B2 (ja) 2024-08-14
TW202021627A (zh) 2020-06-16
IL275918B (en) 2021-07-29
AR099837A1 (es) 2016-08-24
ZA201605924B (en) 2017-09-27
AU2024227776A1 (en) 2024-11-28
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PT3628334T (pt) 2023-09-26
DOP2016000237A (es) 2016-10-16
HUE063460T2 (hu) 2024-01-28
CR20160480A (es) 2016-12-14
US10383953B2 (en) 2019-08-20
IL275918A (en) 2020-08-31
PH12016501684B1 (en) 2023-03-17
DK3628334T3 (da) 2023-10-02
DK3628334T5 (da) 2024-09-02
CN106456660B (zh) 2022-05-31
MY190726A (en) 2022-05-12
PL3119437T3 (pl) 2020-04-30
CN115252823A (zh) 2022-11-01
EP4345165A3 (en) 2024-09-25
RS64611B1 (sr) 2023-10-31
PE20161252A1 (es) 2016-11-30
SG10201912968WA (en) 2020-02-27
US20200046851A1 (en) 2020-02-13

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