ES2809199T3 - Modulación de la expresión de UBE3A-ATS - Google Patents
Modulación de la expresión de UBE3A-ATS Download PDFInfo
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- ES2809199T3 ES2809199T3 ES18188705T ES18188705T ES2809199T3 ES 2809199 T3 ES2809199 T3 ES 2809199T3 ES 18188705 T ES18188705 T ES 18188705T ES 18188705 T ES18188705 T ES 18188705T ES 2809199 T3 ES2809199 T3 ES 2809199T3
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- ES
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- ube3a
- modified oligonucleotide
- modified
- antisense
- ats
- Prior art date
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Classifications
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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| US201361755617P | 2013-01-23 | 2013-01-23 | |
| US201361772925P | 2013-03-05 | 2013-03-05 |
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| US10400243B2 (en) | 2014-11-25 | 2019-09-03 | Ionis Pharmaceuticals, Inc. | Modulation of UBE3A-ATS expression |
| EP4154914B1 (en) | 2015-05-07 | 2024-08-28 | University of South Florida | Modified ube3a gene for a gene therapy approach for angelman syndrome |
| UA125963C2 (uk) * | 2015-11-12 | 2022-07-20 | Ф. Хоффманн-Ля Рош Аг | Олігонуклеотид для індукції батьківської експресії ube3a |
| AU2017368050A1 (en) | 2016-11-29 | 2019-06-20 | Puretech Lyt, Inc. | Exosomes for delivery of therapeutic agents |
| WO2019109001A1 (en) | 2017-12-01 | 2019-06-06 | The Texas A&M University System | Angelman syndrome antisense treatment |
| US20210054370A1 (en) * | 2018-02-27 | 2021-02-25 | The University Of North Carolina At Chapel Hill | Methods and compositions for treating angelman syndrome |
| WO2019241624A1 (en) | 2018-06-14 | 2019-12-19 | Ovid Therapeutics Inc. | Use of mir-92a or mir-145 in the treatment of angelman syndrome |
| EP3833398A4 (en) * | 2018-09-21 | 2021-12-22 | University of Connecticut | COMPOSITIONS AND METHODS OF RESTORING PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN'S SYNDROME IN HUMANS |
| US12360123B2 (en) | 2019-01-17 | 2025-07-15 | Hoffmann-La Roche Inc. | E3 ubiquitin ligase (UBE3A) protein targets |
| CA3135180A1 (en) | 2019-03-29 | 2020-10-08 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating ube3a-ats |
| EA202193214A1 (ru) * | 2019-05-22 | 2022-03-14 | Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл | Гены ube3a и кассеты экспрессии и их использование |
| WO2021011802A1 (en) * | 2019-07-16 | 2021-01-21 | The University Of North Carolina At Chapel Hill | Methods and compositions for unsilencing imprinted genes |
| GB201912103D0 (en) * | 2019-08-22 | 2019-10-09 | Univ Oxford Innovation Ltd | Method of haplotyping |
| EP4076471A4 (en) | 2019-12-18 | 2023-12-27 | National University of Singapore | Method for treating angelman syndrome and related disorders |
| US20230167438A1 (en) * | 2020-04-28 | 2023-06-01 | The Trustees Of The University Of Pennsylvania | Compositions and uses thereof for treatment of angelman syndrome |
| US20240018522A1 (en) * | 2020-10-26 | 2024-01-18 | Remix Therapeutics Inc. | Oligonucleotides useful for modulation of splicing |
| EP4288538A2 (en) * | 2021-02-03 | 2023-12-13 | The Board Of Regents Of The University Of Texas System | Gene therapy for angelman syndrome |
| US20240240179A1 (en) * | 2021-05-05 | 2024-07-18 | The General Hospital Corporation | Therapy for treatment of prader-willi syndrome |
| WO2023168000A1 (en) * | 2022-03-03 | 2023-09-07 | The Regents Of The University Of California | Compositions and methods for treatment of angelman syndrome |
| IL316964A (en) | 2022-05-27 | 2025-01-01 | Zero Carbon Systems Inc | High-output moving plate direct air capture system |
| TW202536178A (zh) * | 2023-11-09 | 2025-09-16 | 美商Ionis製藥公司 | 用於調節ｕｂｅ３ａ－ａｔｓ之方法 |
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| WO2025217466A1 (en) * | 2024-04-11 | 2025-10-16 | Ultragenyx Pharmaceutical Inc. | Dosing regimens for antisense treatment of angelman syndrome |
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2013
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- 2013-06-25 PT PT181887050T patent/PT3461895T/pt unknown
- 2013-06-25 EP EP24188665.4A patent/EP4458361A3/en active Pending
- 2013-06-25 AU AU2013280474A patent/AU2013280474B2/en active Active
- 2013-06-25 EP EP18188705.0A patent/EP3461895B1/en not_active Revoked
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- 2013-06-25 US US14/409,348 patent/US9617539B2/en active Active
- 2013-06-25 CA CA2877905A patent/CA2877905A1/en active Pending
- 2013-06-25 DK DK18188705.0T patent/DK3461895T3/da active
- 2013-06-25 DK DK13809694.6T patent/DK2864479T3/en active
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- 2013-06-25 SI SI201331770T patent/SI3461895T1/sl unknown
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Also Published As
| Publication number | Publication date |
|---|---|
| PL3461895T3 (pl) | 2021-01-11 |
| SI3461895T1 (sl) | 2020-10-30 |
| EP3770258B1 (en) | 2024-07-17 |
| EP4458361A2 (en) | 2024-11-06 |
| HUE051698T2 (hu) | 2021-03-29 |
| EP3461895B1 (en) | 2020-07-01 |
| US20150191723A1 (en) | 2015-07-09 |
| AU2013280474B2 (en) | 2019-01-03 |
| WO2014004572A3 (en) | 2014-05-01 |
| EP3770258A1 (en) | 2021-01-27 |
| LT3461895T (lt) | 2020-09-10 |
| AU2013280474A1 (en) | 2015-01-22 |
| DK3461895T3 (da) | 2020-07-20 |
| EP3461895A1 (en) | 2019-04-03 |
| JP6294876B2 (ja) | 2018-03-14 |
| US9617539B2 (en) | 2017-04-11 |
| DK2864479T3 (en) | 2018-10-22 |
| EP2864479A4 (en) | 2016-06-08 |
| JP2015529635A (ja) | 2015-10-08 |
| ES2987225T3 (es) | 2024-11-14 |
| EP4458361A3 (en) | 2025-01-22 |
| EP2864479A2 (en) | 2015-04-29 |
| PT3461895T (pt) | 2020-09-08 |
| ES2688831T3 (es) | 2018-11-07 |
| CA2877905A1 (en) | 2014-01-03 |
| EP2864479B1 (en) | 2018-08-15 |
| WO2014004572A2 (en) | 2014-01-03 |
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