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
Prior art keywords
aon
ush2a
mrna
skipping
vector
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JP2019506546A
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English (en)
Japanese (ja)
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JP7043082B2 (ja
JP2019515688A (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 (enExample) 2020-06-11
JP7043082B2 JP7043082B2 (ja) 2022-03-29

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ID=58672573

Family Applications (1)

Application Number Title Priority Date Filing Date
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 (enExample)
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)
WO (1) WO2017186739A1 (enExample)
ZA (1) ZA201806628B (enExample)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102368920B1 (ko) 2016-04-25 2022-02-28 프로큐알 테라퓨틱스 Ⅱ 비.브이. 안 질환 치료용 올리고뉴클레오타이드
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
EP3507366B1 (en) 2016-09-01 2020-10-07 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
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)
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
US12398393B2 (en) 2019-01-28 2025-08-26 Proqr Therapeutics Ii B.V. RNA-editing oligonucleotides for the treatment of usher syndrome
JP2022520783A (ja) * 2019-02-12 2022-04-01 ウニヴェルシタ デリ ストゥディ ディ トレント Cas12aガイドRNA分子およびその使用
US20220177894A1 (en) 2019-04-02 2022-06-09 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for immunotherapy
TW202043249A (zh) 2019-04-15 2020-12-01 大陸商博雅輯因(北京)生物科技有限公司 編輯rna的方法和組合物
WO2020212567A1 (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
PH12022550059A1 (en) 2019-07-12 2022-11-21 Univ Beijing Targeted rna editing by leveraging endogenous adar using engineered rnas
WO2021018750A1 (en) 2019-07-26 2021-02-04 Proqr Therapeutics Ii B.V. Ophthalmic compositions comprising viscosifying polymers and nucleic acids
WO2021023863A1 (en) * 2019-08-08 2021-02-11 Stichting Katholieke Universiteit Antisense oligonucleotides rescue aberrant splicing of abca4
EP4081638A1 (en) 2019-12-23 2022-11-02 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
TW202138561A (zh) * 2019-12-30 2021-10-16 大陸商博雅輯因(北京)生物科技有限公司 治療Usher綜合症的方法和其組合物
US20230134677A1 (en) 2020-03-04 2023-05-04 Proqr Therapeutics Ii B.V. 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.
US20250154504A1 (en) 2022-02-14 2025-05-15 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
AU2023357356A1 (en) * 2022-10-06 2025-04-10 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for treatment of usher 2a. exon 68
AU2023357354A1 (en) * 2022-10-06 2025-04-10 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for treatment of usher 2a. exons 30-31
CN120129747A (zh) * 2022-10-11 2025-06-10 广州瑞风生物科技有限公司 snRNA核酸分子及其应用
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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ATE432993T1 (de) 1999-10-04 2009-06-15 Univ New Jersey Med Tar rna bindende peptide
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
SI2425814T1 (sl) 2010-09-03 2013-10-30 Novagali Pharma S.A. Emulzija vrste voda-v-olju za zdravljenje bolezni očesa
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
JP7089763B2 (ja) 2015-10-05 2022-06-23 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 トリヌクレオチドリピート伸長が関与する遺伝的疾患の予防または治療における一本鎖アンチセンスオリゴヌクレオチドの使用
KR102368920B1 (ko) 2016-04-25 2022-02-28 프로큐알 테라퓨틱스 Ⅱ 비.브이. 안 질환 치료용 올리고뉴클레오타이드

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