KR102368920B1 - 안 질환 치료용 올리고뉴클레오타이드 - Google Patents

안 질환 치료용 올리고뉴클레오타이드 Download PDF

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KR102368920B1
KR102368920B1 KR1020187031243A KR20187031243A KR102368920B1 KR 102368920 B1 KR102368920 B1 KR 102368920B1 KR 1020187031243 A KR1020187031243 A KR 1020187031243A KR 20187031243 A KR20187031243 A KR 20187031243A KR 102368920 B1 KR102368920 B1 KR 102368920B1
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aon
ush2a
mrna
seq
skipping
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KR20180134931A (ko
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헤스터 카타리나 반 디에펜
희 람 찬
쟌네 주하 투루넨
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프로큐알 테라퓨틱스 Ⅱ 비.브이.
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
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    • C12N15/09—Recombinant DNA-technology
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Bioinformatics & Cheminformatics (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
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  • 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)
KR1020187031243A 2016-04-25 2017-04-25 안 질환 치료용 올리고뉴클레오타이드 Expired - Fee Related KR102368920B1 (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB1607141.7 2016-04-25
GB201607141 2016-04-25
PCT/EP2017/059830 WO2017186739A1 (en) 2016-04-25 2017-04-25 Oligonucleotides to treat eye disease

Publications (2)

Publication Number Publication Date
KR20180134931A KR20180134931A (ko) 2018-12-19
KR102368920B1 true KR102368920B1 (ko) 2022-02-28

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KR1020187031243A Expired - Fee Related KR102368920B1 (ko) 2016-04-25 2017-04-25 안 질환 치료용 올리고뉴클레오타이드

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

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JP7043082B2 (ja) 2016-04-25 2022-03-29 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 眼疾患を処置するオリゴヌクレオチド
WO2017220751A1 (en) 2016-06-22 2017-12-28 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
WO2019123430A1 (en) 2017-12-21 2019-06-27 Casebia Therapeutics Llp 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
US12398393B2 (en) 2019-01-28 2025-08-26 Proqr Therapeutics Ii B.V. RNA-editing oligonucleotides for the treatment of usher syndrome
MX2021009750A (es) * 2019-02-12 2021-09-08 Univ Degli Studi Di Trento Moleculas del acido ribonucleico guia de cas12a y usos de las mismas.
US20220177894A1 (en) 2019-04-02 2022-06-09 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for immunotherapy
EP3956449B1 (en) 2019-04-15 2026-02-25 EdiGene Therapeutics (Beijing) Inc. Methods and compositions for editing rnas
CA3136172A1 (en) 2019-04-18 2020-10-22 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
EP3997229A4 (en) 2019-07-12 2024-07-03 Peking University TARGETED RNA EDITING BY HARNESSING ENDOGENOUS ADAR USING MODIFIED RNA
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
CA3158528A1 (en) 2019-12-23 2021-07-01 Jim SWILDENS 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
BR112022012921A2 (pt) * 2019-12-30 2022-09-06 Edigene Therapeutics Beijing Inc Métodos para editar de forma alvejada ácido ribonucleico e para tratar a síndrome de usher tipo ii, ácido ribonucleico de recrutamento da adenosina desaminase, construto ou vetor de entrega, célula, e, uso do ácido ribonucleico de recrutamento da adenosina desaminase ou do construto ou vetor de entrega
CA3166720A1 (en) 2020-03-04 2021-09-10 Jim SWILDENS Antisense oligonucleotides for use in the treatment of usher syndrome
EP4301856A2 (en) 2021-03-05 2024-01-10 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.
EP4479535A1 (en) 2022-02-14 2024-12-25 ProQR Therapeutics II B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
EP4599060A1 (en) * 2022-10-06 2025-08-13 Stichting Radboud universitair medisch centrum Antisense oligonucleotides for treatment of usher 2a. exons 30-31
EP4599061A1 (en) * 2022-10-06 2025-08-13 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
EP4619522A1 (en) * 2022-11-15 2025-09-24 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

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US20170059052A1 (en) 2015-08-25 2017-03-02 Mueller International, Llc Valve seat stiffener

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IL262199A (en) 2018-12-31
US10617707B2 (en) 2020-04-14
JP7043082B2 (ja) 2022-03-29
CN109072239A (zh) 2018-12-21
WO2017186739A1 (en) 2017-11-02
JP2019515688A (ja) 2019-06-13
IL262199B (en) 2022-03-01
US20210401871A1 (en) 2021-12-30
ES2801823T3 (es) 2021-01-14
US20190381089A1 (en) 2019-12-19
US11123360B2 (en) 2021-09-21
EA201892431A1 (ru) 2019-04-30
EP3448999B1 (en) 2020-05-13
DK3448999T3 (da) 2020-06-08
PL3448999T3 (pl) 2020-11-30
AU2017257292A1 (en) 2018-12-06
KR20180134931A (ko) 2018-12-19
MX2018013003A (es) 2019-01-28
CA3021899A1 (en) 2017-11-02
EP3448999A1 (en) 2019-03-06
ZA201806628B (en) 2019-07-31
US20200237802A1 (en) 2020-07-30

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