JP2019515688A5 - - Google Patents

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Publication number
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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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
Application granted granted Critical
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
GB201607141 2016-04-25
GB1607141.7 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 (OSRAM) 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 (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
US10617707B2 (en) 2016-04-25 2020-04-14 Proqr Therapeutics Ii B.V. Oligonucleotides to treat eye disease
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
ES2837076T3 (es) 2016-09-01 2021-06-29 Proqr Therapeutics Ii Bv Oligonucleótidos para la edición de ARN de cadena sencilla modificados químicamente
GB201706009D0 (en) 2017-04-13 2017-05-31 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of stargardt disease
EP3728595A1 (en) 2017-12-21 2020-10-28 CRISPR Therapeutics AG Materials and methods for treatment of usher syndrome type 2a and/or non-syndromic autosomal recessive retinitis pigmentosa (arrp)
JP7402163B2 (ja) 2017-12-21 2023-12-20 クリスパー セラピューティクス アーゲー 2a型アッシャー症候群の処置のための材料および方法
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
CA3127527A1 (en) * 2019-02-12 2020-08-20 Universita Degli Studi Di Trento Cas12a guide rna molecules and uses thereof
WO2020201144A1 (en) 2019-04-02 2020-10-08 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)
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
PH12022550059A1 (en) 2019-07-12 2022-11-21 Univ Beijing Targeted rna editing by leveraging endogenous adar using engineered rnas
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
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
CN114829598A (zh) * 2019-12-30 2022-07-29 北京辑因医疗科技有限公司 一种治疗Usher综合征的方法和其组合物
CA3166720A1 (en) 2020-03-04 2021-09-10 Jim SWILDENS 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.
WO2023152371A1 (en) 2022-02-14 2023-08-17 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
EP4599061A1 (en) * 2022-10-06 2025-08-13 Stichting Radboud universitair medisch centrum Antisense oligonucleotides for treatment of usher 2a. exon 68
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

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU7856600A (en) 1999-10-04 2001-05-10 University Of Medicine And Dentistry Of New Jersey Novel carbamates and ureas
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
PL2425814T3 (pl) 2010-09-03 2013-11-29 Santen Sas Emulsja typu woda-w-oleju do leczenia choroby oka
CA2952289C (en) * 2014-07-10 2024-07-02 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for the treatment of type 2 Usher syndrome
US9927034B2 (en) * 2015-08-25 2018-03-27 Mueller International, Llc Valve seat stiffener
EP3359667A1 (en) 2015-10-05 2018-08-15 ProQR Therapeutics II B.V. Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion
US10617707B2 (en) 2016-04-25 2020-04-14 Proqr Therapeutics Ii B.V. Oligonucleotides to treat eye disease

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