JP2019515688A5 - - Google Patents

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JP2019515688A5
JP2019515688A5 JP2019506546A JP2019506546A JP2019515688A5 JP 2019515688 A5 JP2019515688 A5 JP 2019515688A5 JP 2019506546 A JP2019506546 A JP 2019506546A JP 2019506546 A JP2019506546 A JP 2019506546A JP 2019515688 A5 JP2019515688 A5 JP 2019515688A5
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JP
Japan
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aon
ush2a
mrna
skipping
vector
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JP2019506546A
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English (en)
Japanese (ja)
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JP2019515688A (ja
JP7043082B2 (ja
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Priority claimed from PCT/EP2017/059830 external-priority patent/WO2017186739A1/en
Publication of JP2019515688A publication Critical patent/JP2019515688A/ja
Publication of JP2019515688A5 publication Critical patent/JP2019515688A5/ja
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Publication of JP7043082B2 publication Critical patent/JP7043082B2/ja
Expired - Fee Related legal-status Critical Current
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JP2019506546A 2016-04-25 2017-04-25 眼疾患を処置するオリゴヌクレオチド Expired - Fee Related JP7043082B2 (ja)

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 (3)

Publication Number Publication Date
JP2019515688A JP2019515688A (ja) 2019-06-13
JP2019515688A5 true JP2019515688A5 (cg-RX-API-DMAC10.html) 2020-06-11
JP7043082B2 JP7043082B2 (ja) 2022-03-29

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Family Applications (1)

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JP2019506546A Expired - Fee Related JP7043082B2 (ja) 2016-04-25 2017-04-25 眼疾患を処置するオリゴヌクレオチド

Country Status (15)

Country Link
US (3) US10617707B2 (cg-RX-API-DMAC10.html)
EP (1) EP3448999B1 (cg-RX-API-DMAC10.html)
JP (1) JP7043082B2 (cg-RX-API-DMAC10.html)
KR (1) KR102368920B1 (cg-RX-API-DMAC10.html)
CN (1) CN109072239A (cg-RX-API-DMAC10.html)
AU (1) AU2017257292A1 (cg-RX-API-DMAC10.html)
CA (1) CA3021899A1 (cg-RX-API-DMAC10.html)
DK (1) DK3448999T3 (cg-RX-API-DMAC10.html)
EA (1) EA201892431A1 (cg-RX-API-DMAC10.html)
ES (1) ES2801823T3 (cg-RX-API-DMAC10.html)
IL (1) IL262199B (cg-RX-API-DMAC10.html)
MX (1) MX2018013003A (cg-RX-API-DMAC10.html)
PL (1) PL3448999T3 (cg-RX-API-DMAC10.html)
WO (1) WO2017186739A1 (cg-RX-API-DMAC10.html)
ZA (1) ZA201806628B (cg-RX-API-DMAC10.html)

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AU2015367378A1 (en) 2014-12-17 2017-06-15 Proqr Therapeutics Ii B.V. Targeted RNA editing
MX2018013003A (es) 2016-04-25 2019-01-28 Proqr Therapeutics Ii Bv Oligonucleotidos para tratar una enfermedad ocular.
EP3475424A1 (en) 2016-06-22 2019-05-01 ProQR Therapeutics II B.V. Single-stranded rna-editing oligonucleotides
WO2018041973A1 (en) 2016-09-01 2018-03-08 Proqr Therapeutics Ii B.V. 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
EP3918067A1 (en) 2019-01-28 2021-12-08 ProQR Therapeutics II B.V. Rna-editing oligonucleotides for the treatment of usher syndrome
US20220145305A1 (en) * 2019-02-12 2022-05-12 Universita' Degli Studi Di Trento CAS12a GUIDE RNA MOLECULES AND USES THEREOF
US20220177894A1 (en) 2019-04-02 2022-06-09 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for immunotherapy
CA3136735A1 (en) 2019-04-15 2020-10-22 Edigene Inc. Methods and compositions for editing rnas
US20220213478A1 (en) 2019-04-18 2022-07-07 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
AU2020313143B2 (en) 2019-07-12 2024-04-04 Peking University Targeted RNA editing by leveraging endogenous adar using engineered RNAs
CA3143071A1 (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
US20230039928A1 (en) 2019-12-23 2023-02-09 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for nucleotide deamination in the treatment of Stargardt disease
AU2020418026A1 (en) * 2019-12-30 2022-08-18 Edigene Therapeutics (Beijing) Inc. Method for treating usher syndrome and composition thereof
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
WO2021175904A1 (en) 2020-03-04 2021-09-10 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for use in the treatment of usher syndrome
US20240141340A1 (en) 2021-03-05 2024-05-02 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
EP4599060A1 (en) * 2022-10-06 2025-08-13 Stichting Radboud universitair medisch centrum Antisense oligonucleotides for treatment of usher 2a. exons 30-31
EP4603585A4 (en) * 2022-10-11 2026-06-24 Guangzhou Reforgene Medicine Co Ltd RNASN NUCLEIC ACID MOLECULE AND ITS APPLICATION
AU2023382528A1 (en) * 2022-11-15 2025-05-15 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for treatment of usher 2a. exons 39-40
CN121729244A (zh) * 2023-07-04 2026-03-24 广州瑞风生物科技股份有限公司 靶向USH2A pre-mRNA假外显子PE40的snRNA及其应用

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US6503713B1 (en) 1999-10-04 2003-01-07 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
AU2016335032B2 (en) 2015-10-05 2022-04-14 Proqr Therapeutics Ii B.V. Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion
MX2018013003A (es) 2016-04-25 2019-01-28 Proqr Therapeutics Ii Bv Oligonucleotidos para tratar una enfermedad ocular.

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