PL3448999T3 - Oligonukleotydy do leczenia choroby oka - Google Patents

Oligonukleotydy do leczenia choroby oka

Info

Publication number
PL3448999T3
PL3448999T3 PL17721984T PL17721984T PL3448999T3 PL 3448999 T3 PL3448999 T3 PL 3448999T3 PL 17721984 T PL17721984 T PL 17721984T PL 17721984 T PL17721984 T PL 17721984T PL 3448999 T3 PL3448999 T3 PL 3448999T3
Authority
PL
Poland
Prior art keywords
oligonucleotides
eye disease
treat eye
treat
disease
Prior art date
Application number
PL17721984T
Other languages
English (en)
Polish (pl)
Inventor
Hester Catharina VAN DIEPEN
Hee Lam Chan
Janne Juha TURUNEN
Original Assignee
Proqr Therapeutics Ii B.V.
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 Proqr Therapeutics Ii B.V. filed Critical Proqr Therapeutics Ii B.V.
Publication of PL3448999T3 publication Critical patent/PL3448999T3/pl

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0048Eye, e.g. artificial tears
    • 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
    • CCHEMISTRY; METALLURGY
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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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/333Modified A
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521Methyl
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
PL17721984T 2016-04-25 2017-04-25 Oligonukleotydy do leczenia choroby oka PL3448999T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB201607141 2016-04-25
EP17721984.7A EP3448999B1 (en) 2016-04-25 2017-04-25 Oligonucleotides to treat eye disease
PCT/EP2017/059830 WO2017186739A1 (en) 2016-04-25 2017-04-25 Oligonucleotides to treat eye disease

Publications (1)

Publication Number Publication Date
PL3448999T3 true PL3448999T3 (pl) 2020-11-30

Family

ID=58672573

Family Applications (1)

Application Number Title Priority Date Filing Date
PL17721984T PL3448999T3 (pl) 2016-04-25 2017-04-25 Oligonukleotydy do leczenia choroby oka

Country Status (15)

Country Link
US (3) US10617707B2 (OSRAM)
EP (1) EP3448999B1 (OSRAM)
JP (1) JP7043082B2 (OSRAM)
KR (1) KR102368920B1 (OSRAM)
CN (1) CN109072239A (OSRAM)
AU (1) AU2017257292A1 (OSRAM)
CA (1) CA3021899A1 (OSRAM)
DK (1) DK3448999T3 (OSRAM)
EA (1) EA201892431A1 (OSRAM)
ES (1) ES2801823T3 (OSRAM)
IL (1) IL262199B (OSRAM)
MX (1) MX2018013003A (OSRAM)
PL (1) PL3448999T3 (OSRAM)
WO (1) WO2017186739A1 (OSRAM)
ZA (1) ZA201806628B (OSRAM)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3448999B1 (en) 2016-04-25 2020-05-13 ProQR Therapeutics II B.V. Oligonucleotides to treat eye disease
EP3475424A1 (en) 2016-06-22 2019-05-01 ProQR Therapeutics II B.V. Single-stranded rna-editing oligonucleotides
CN110352244B (zh) 2016-09-01 2023-03-21 ProQR治疗上市公司Ⅱ 化学修饰的编辑rna的单链寡核苷酸
GB201706009D0 (en) 2017-04-13 2017-05-31 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of stargardt disease
AU2018393050A1 (en) 2017-12-21 2020-06-18 Bayer Healthcare Llc Materials and methods for treatment of Usher Syndrome Type 2A
CA3084633A1 (en) 2017-12-21 2019-06-27 Crispr Therapeutics Ag Materials and methods for treatment of usher syndrome type 2a and/or non-syndromic autosomal recessive retinitis pigmentosa (arrp)
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
JP2022523302A (ja) 2019-01-28 2022-04-22 プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ アッシャー症候群の処置のためのrna編集オリゴヌクレオチド
EA202192233A1 (ru) * 2019-02-12 2021-11-11 Университа' Дельи Студи Ди Тренто МОЛЕКУЛЫ ГИДОВОЙ РНК Cas12a И ИХ ПРИМЕНЕНИЕ
EP3947678A1 (en) 2019-04-02 2022-02-09 ProQR Therapeutics II B.V. Antisense oligonucleotides for immunotherapy
CR20210572A (es) 2019-04-15 2022-04-07 Univ Beijing Métodos y composiciones para editar ácido ribonucleicos (arn)
EP3956447A1 (en) 2019-04-18 2022-02-23 ProQR Therapeutics II B.V. Antisense oligonucleotides for the treatment of usher syndrome
WO2020254249A1 (en) 2019-06-21 2020-12-24 Proqr Therapeutics Ii B.V. Delivery of nucleic acids for the treatment of auditory disorders
MX2022000497A (es) 2019-07-12 2022-02-03 Univ Beijing Edicion de acido ribonucleico (arn) dirigido aprovechando la adenosina desaminasa que actua sobre acido ribonucleico endogeno (adar) utilizando acidos ribonucleicos (arn) modificados geneticamente.
WO2021018750A1 (en) 2019-07-26 2021-02-04 Proqr Therapeutics Ii B.V. Ophthalmic compositions comprising viscosifying polymers and nucleic acids
US20220290154A1 (en) * 2019-08-08 2022-09-15 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides rescue aberrant splicing of ABCA4
AU2020414395A1 (en) 2019-12-23 2022-07-21 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for nucleotide deamination in the treatment of Stargardt disease
CN113122577A (zh) * 2019-12-30 2021-07-16 博雅辑因(北京)生物科技有限公司 一种治疗Usher综合征的方法和其组合物
US20230067480A1 (en) * 2019-12-30 2023-03-02 Edigene Therapeutics (Beijing) Inc. Method for treating usher syndrome and composition thereof
US20230134677A1 (en) 2020-03-04 2023-05-04 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for use in the treatment of usher syndrome
WO2022184888A2 (en) 2021-03-05 2022-09-09 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for use in the treatment of corneal dystrophies
TR2022001648A2 (tr) * 2022-02-09 2022-02-21 T C Ueskuedar Ueniversitesi Ush2a kaynaklı retinitis pigmentosa hastalığının genetik tedavisi için ekzon 13 atlama işlevi gören kseno nükleik asit antisens-oligonükleotit (xna-aso) dizileri.
US20250154504A1 (en) 2022-02-14 2025-05-15 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
AU2023357354A1 (en) * 2022-10-06 2025-04-10 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for treatment of usher 2a. exons 30-31
AU2023357356A1 (en) * 2022-10-06 2025-04-10 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for treatment of usher 2a. exon 68
EP4603585A1 (en) * 2022-10-11 2025-08-20 Guangzhou Reforgene Medicine Co., Ltd. Snrna nucleic acid molecule and application thereof
WO2024105063A1 (en) * 2022-11-15 2024-05-23 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for treatment of usher 2a. exons 39-40
WO2025007937A1 (zh) * 2023-07-04 2025-01-09 广州瑞风生物科技有限公司 靶向USH2A pre-mRNA假外显子PE40的snRNA及其应用

Family Cites Families (10)

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Publication number Priority date Publication date Assignee Title
CA2386239A1 (en) 1999-10-04 2001-04-12 University Of Medicine And Dentistry Of New Jersey Methods for identifying rna binding compounds
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
US20100196889A1 (en) 2006-11-13 2010-08-05 Bankaitis-Davis Danute M Gene Expression Profiling for Identification, Monitoring and Treatment of Colorectal Cancer
US8093000B2 (en) 2008-05-09 2012-01-10 The Regents Of The University Of California Methods for predicting and treating tumors resistant to drug, immunotherapy, and radiation
US20110070582A1 (en) 2008-11-03 2011-03-24 Source Precision Medicine, Inc. d/b/d Source MDX Gene Expression Profiling for Predicting the Response to Immunotherapy and/or the Survivability of Melanoma Subjects
EP2425814B1 (en) 2010-09-03 2013-06-19 Novagali Pharma S.A. A water-in-oil type emulsion for treating a disease of the eye
US10131910B2 (en) * 2014-07-10 2018-11-20 Stichting Katholieke Universiteit Antisense oligonucleotides for the treatment of Usher syndrome type 2
US9927034B2 (en) * 2015-08-25 2018-03-27 Mueller International, Llc Valve seat stiffener
WO2017060317A1 (en) 2015-10-05 2017-04-13 Proqr Therapeutics Ii B.V. Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion
EP3448999B1 (en) 2016-04-25 2020-05-13 ProQR Therapeutics II B.V. Oligonucleotides to treat eye disease

Also Published As

Publication number Publication date
JP2019515688A (ja) 2019-06-13
US10617707B2 (en) 2020-04-14
US20210401871A1 (en) 2021-12-30
CN109072239A (zh) 2018-12-21
JP7043082B2 (ja) 2022-03-29
ZA201806628B (en) 2019-07-31
IL262199B (en) 2022-03-01
IL262199A (en) 2018-12-31
EP3448999B1 (en) 2020-05-13
DK3448999T3 (da) 2020-06-08
US20200237802A1 (en) 2020-07-30
CA3021899A1 (en) 2017-11-02
EA201892431A1 (ru) 2019-04-30
EP3448999A1 (en) 2019-03-06
KR20180134931A (ko) 2018-12-19
WO2017186739A1 (en) 2017-11-02
US11123360B2 (en) 2021-09-21
KR102368920B1 (ko) 2022-02-28
US20190381089A1 (en) 2019-12-19
AU2017257292A1 (en) 2018-12-06
MX2018013003A (es) 2019-01-28
ES2801823T3 (es) 2021-01-14

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