CN109072239A - 治疗眼病的寡核苷酸 - Google Patents

治疗眼病的寡核苷酸 Download PDF

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Publication number
CN109072239A
CN109072239A CN201780025611.3A CN201780025611A CN109072239A CN 109072239 A CN109072239 A CN 109072239A CN 201780025611 A CN201780025611 A CN 201780025611A CN 109072239 A CN109072239 A CN 109072239A
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aon
ush2a
seq
present
exon
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Chinese (zh)
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H·C·范迪鹏
H·L·陈
J·J·图鲁宁
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ProQR Therapeutics II BV
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • 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)
CN201780025611.3A 2016-04-25 2017-04-25 治疗眼病的寡核苷酸 Pending CN109072239A (zh)

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 (1)

Publication Number Publication Date
CN109072239A true CN109072239A (zh) 2018-12-21

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EP (1) EP3448999B1 (enExample)
JP (1) JP7043082B2 (enExample)
KR (1) KR102368920B1 (enExample)
CN (1) CN109072239A (enExample)
AU (1) AU2017257292A1 (enExample)
CA (1) CA3021899A1 (enExample)
DK (1) DK3448999T3 (enExample)
EA (1) EA201892431A1 (enExample)
ES (1) ES2801823T3 (enExample)
IL (1) IL262199B (enExample)
MX (1) MX2018013003A (enExample)
PL (1) PL3448999T3 (enExample)
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Cited By (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021136404A1 (zh) * 2019-12-30 2021-07-08 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
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
CN113614231A (zh) * 2019-02-12 2021-11-05 特伦托大学 CAS12a向导RNA分子及其用途
US11661596B2 (en) 2019-07-12 2023-05-30 Peking University Targeted RNA editing by leveraging endogenous ADAR using engineered RNAs
US11702658B2 (en) 2019-04-15 2023-07-18 Edigene Therapeutics (Beijing) Inc. Methods and compositions for editing RNAs
WO2024078345A1 (zh) * 2022-10-11 2024-04-18 广州瑞风生物科技有限公司 snRNA核酸分子及其应用
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

Families Citing this family (21)

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ES2801823T3 (es) 2016-04-25 2021-01-14 Proqr Therapeutics Ii Bv Oligonucleótidos para tratar una enfermedad ocular
CN109477103A (zh) 2016-06-22 2019-03-15 ProQR治疗上市公司Ⅱ 单链rna-编辑寡核苷酸
NZ751483A (en) 2016-09-01 2022-07-01 Proqr Therapeutics Ii Bv Chemically modified single-stranded rna-editing oligonucleotides
GB201706009D0 (en) 2017-04-13 2017-05-31 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of stargardt disease
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)
AU2018393050A1 (en) 2017-12-21 2020-06-18 Bayer Healthcare Llc Materials and methods for treatment of Usher Syndrome Type 2A
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
CA3124493A1 (en) 2019-01-28 2020-08-06 Proqr Therapeutics Ii B.V. Rna-editing oligonucleotides for the treatment of usher syndrome
US20220177894A1 (en) 2019-04-02 2022-06-09 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for immunotherapy
AU2020259856A1 (en) 2019-04-18 2021-11-18 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
US20220265695A1 (en) 2019-07-26 2022-08-25 Proqr Therapeutics Ii B.V. Opthalmic compositions comprising viscosifying polymers and nucleic acids
EP4010475A1 (en) * 2019-08-08 2022-06-15 UCL Business Ltd Antisense oligonucleotides rescue aberrant splicing of abca4
WO2021130313A1 (en) 2019-12-23 2021-07-01 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for nucleotide deamination in the treatment of stargardt disease
WO2021175904A1 (en) 2020-03-04 2021-09-10 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.
EP4479535A1 (en) 2022-02-14 2024-12-25 ProQR Therapeutics II B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
EP4599061A1 (en) * 2022-10-06 2025-08-13 Stichting Radboud universitair medisch centrum Antisense oligonucleotides for treatment of usher 2a. exon 68
WO2024074668A1 (en) * 2022-10-06 2024-04-11 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for treatment of usher 2a. exons 30-31
WO2024105063A1 (en) * 2022-11-15 2024-05-23 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for treatment of usher 2a. exons 39-40

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Cited By (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113614231A (zh) * 2019-02-12 2021-11-05 特伦托大学 CAS12a向导RNA分子及其用途
US11702658B2 (en) 2019-04-15 2023-07-18 Edigene Therapeutics (Beijing) Inc. Methods and compositions for editing RNAs
US11661596B2 (en) 2019-07-12 2023-05-30 Peking University Targeted RNA editing by leveraging endogenous ADAR using engineered RNAs
WO2021136404A1 (zh) * 2019-12-30 2021-07-08 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
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
WO2024078345A1 (zh) * 2022-10-11 2024-04-18 广州瑞风生物科技有限公司 snRNA核酸分子及其应用
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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KR102368920B1 (ko) 2022-02-28
US20200237802A1 (en) 2020-07-30
CA3021899A1 (en) 2017-11-02
US20190381089A1 (en) 2019-12-19
EA201892431A1 (ru) 2019-04-30
JP2019515688A (ja) 2019-06-13
ES2801823T3 (es) 2021-01-14
DK3448999T3 (da) 2020-06-08
AU2017257292A1 (en) 2018-12-06
IL262199B (en) 2022-03-01
MX2018013003A (es) 2019-01-28
IL262199A (en) 2018-12-31
KR20180134931A (ko) 2018-12-19
US11123360B2 (en) 2021-09-21
JP7043082B2 (ja) 2022-03-29
US20210401871A1 (en) 2021-12-30
EP3448999B1 (en) 2020-05-13
EP3448999A1 (en) 2019-03-06
US10617707B2 (en) 2020-04-14
PL3448999T3 (pl) 2020-11-30
WO2017186739A1 (en) 2017-11-02
ZA201806628B (en) 2019-07-31

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Application publication date: 20181221