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
- Authority
- JP
- Japan
- Prior art keywords
- aon
- ush2a
- mrna
- skipping
- vector
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Granted
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- 108091034117 Oligonucleotide Proteins 0.000 claims description 77
- 239000000074 antisense oligonucleotide Substances 0.000 claims description 72
- 238000012230 antisense oligonucleotides Methods 0.000 claims description 72
- 101000805941 Homo sapiens Usherin Proteins 0.000 claims description 48
- 108020004999 messenger RNA Proteins 0.000 claims description 24
- 238000000034 method Methods 0.000 claims description 24
- 239000000203 mixture Substances 0.000 claims description 23
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 17
- 201000010099 disease Diseases 0.000 claims description 16
- 239000013598 vector Substances 0.000 claims description 13
- 239000013603 viral vector Substances 0.000 claims description 13
- 239000003814 drug Substances 0.000 claims description 11
- 238000000338 in vitro Methods 0.000 claims description 11
- 102000051625 human USH2A Human genes 0.000 claims description 10
- 239000008194 pharmaceutical composition Substances 0.000 claims description 9
- 208000014769 Usher Syndromes Diseases 0.000 claims description 8
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- 101100247004 Rattus norvegicus Qsox1 gene Proteins 0.000 description 1
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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 (enExample) | 2020-06-11 |
| JP7043082B2 JP7043082B2 (ja) | 2022-03-29 |
Family
ID=58672573
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019506546A Active 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)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX2018013003A (es) | 2016-04-25 | 2019-01-28 | Proqr Therapeutics Ii Bv | Oligonucleotidos para tratar una enfermedad ocular. |
| WO2017220751A1 (en) | 2016-06-22 | 2017-12-28 | Proqr Therapeutics Ii B.V. | Single-stranded rna-editing oligonucleotides |
| 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 |
| 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) |
| CA3084632A1 (en) * | 2017-12-21 | 2019-06-27 | Crispr Therapeutics Ag | 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 |
| WO2020157008A1 (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 |
| CA3132178A1 (en) | 2019-04-02 | 2020-10-08 | Aliye Seda Yilmaz-Elis | Antisense oligonucleotides for immunotherapy |
| WO2020211780A1 (en) | 2019-04-15 | 2020-10-22 | Edigene 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 |
| US20220265695A1 (en) | 2019-07-26 | 2022-08-25 | Proqr Therapeutics Ii B.V. | Opthalmic compositions comprising viscosifying polymers and nucleic acids |
| JP2022543474A (ja) * | 2019-08-08 | 2022-10-12 | ユーシーエル ビジネス リミテッド | アンチセンスオリゴヌクレオチドは、ａｂｃａ４の異常スプライシングをレスキューする |
| EP4081638A1 (en) | 2019-12-23 | 2022-11-02 | ProQR Therapeutics II B.V. | Antisense oligonucleotides for nucleotide deamination in the treatment of stargardt disease |
| PE20250386A1 (es) * | 2019-12-30 | 2025-02-11 | Edigene Therapeutics Beijing Inc | Metodo para el tratamiento del sindrome de usher y composicion del mismo |
| 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 |
| US20230134677A1 (en) | 2020-03-04 | 2023-05-04 | 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. |
| US20250154504A1 (en) | 2022-02-14 | 2025-05-15 | Proqr Therapeutics Ii B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
| WO2024074670A1 (en) * | 2022-10-06 | 2024-04-11 | 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 |
| 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 |
| WO2024105063A1 (en) * | 2022-11-15 | 2024-05-23 | 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)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1218544B1 (en) | 1999-10-04 | 2009-06-03 | The University of Medicine and Dentistry of New Jersey | TAR RNA binding peptides |
| 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 |
| PT2425814E (pt) | 2010-09-03 | 2013-09-02 | Novagali Pharma Sa | Emulsão de tipo água em óleo para tratamento de uma doença ocular |
| CA2952289C (en) * | 2014-07-10 | 2024-07-02 | Stichting Katholieke Univ | Antisense oligonucleotides for the treatment of type 2 Usher syndrome |
| US9927034B2 (en) | 2015-08-25 | 2018-03-27 | Mueller International, Llc | Valve seat stiffener |
| KR20180053753A (ko) | 2015-10-05 | 2018-05-23 | 프로큐알 테라퓨틱스 Ⅱ 비.브이. | 삼핵산 반복 확장과 관련된 유전질환의 예방 또는 치료용 단일 가닥 안티센스 올리고뉴클레오타이드의 용도 |
| MX2018013003A (es) | 2016-04-25 | 2019-01-28 | Proqr Therapeutics Ii Bv | Oligonucleotidos para tratar una enfermedad ocular. |
-
2017
- 2017-04-25 MX MX2018013003A patent/MX2018013003A/es unknown
- 2017-04-25 EA EA201892431A patent/EA201892431A1/ru unknown
- 2017-04-25 JP JP2019506546A patent/JP7043082B2/ja active Active
- 2017-04-25 AU AU2017257292A patent/AU2017257292A1/en not_active Abandoned
- 2017-04-25 CN CN201780025611.3A patent/CN109072239A/zh active Pending
- 2017-04-25 US US16/096,038 patent/US10617707B2/en active Active
- 2017-04-25 KR KR1020187031243A patent/KR102368920B1/ko not_active Expired - Fee Related
- 2017-04-25 DK DK17721984.7T patent/DK3448999T3/da active
- 2017-04-25 EP EP17721984.7A patent/EP3448999B1/en active Active
- 2017-04-25 ES ES17721984T patent/ES2801823T3/es active Active
- 2017-04-25 WO PCT/EP2017/059830 patent/WO2017186739A1/en not_active Ceased
- 2017-04-25 CA CA3021899A patent/CA3021899A1/en active Pending
- 2017-04-25 PL PL17721984T patent/PL3448999T3/pl unknown
-
2018
- 2018-10-05 ZA ZA2018/06628A patent/ZA201806628B/en unknown
- 2018-10-08 IL IL262199A patent/IL262199B/en unknown
-
2020
- 2020-03-06 US US16/811,848 patent/US11123360B2/en not_active Expired - Fee Related
-
2021
- 2021-09-15 US US17/475,795 patent/US20210401871A1/en not_active Abandoned
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