CA3005128A1 - Compositions and methods for treatment of eye diseases - Google Patents
Compositions and methods for treatment of eye diseases Download PDFInfo
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- CA3005128A1 CA3005128A1 CA3005128A CA3005128A CA3005128A1 CA 3005128 A1 CA3005128 A1 CA 3005128A1 CA 3005128 A CA3005128 A CA 3005128A CA 3005128 A CA3005128 A CA 3005128A CA 3005128 A1 CA3005128 A1 CA 3005128A1
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- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
- C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
- C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
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| US62/318,958 | 2016-04-06 | ||
| PCT/US2016/066691 WO2017106370A1 (en) | 2015-12-14 | 2016-12-14 | Compositions and methods for treatment of eye diseases |
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| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| EP3201339A4 (en) | 2014-10-03 | 2018-09-19 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| KR20220105174A (ko) | 2015-10-09 | 2022-07-26 | 유니버시티 오브 사우스앰톤 | 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝 |
| WO2017106377A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| WO2018136702A1 (en) | 2017-01-23 | 2018-07-26 | Regeneron Pharmaceuticals, Inc. | Hydroxysteroid 17-beta dehydrogenase 13 (hsd17b13) variants and uses thereof |
| CA3059348A1 (en) | 2017-04-11 | 2018-10-18 | Regeneron Pharmaceuticals, Inc. | Assays for screening activity of modulators of members of the hydroxysteroid (17-beta) dehydrogenase (hsd17b) family |
| GB201706009D0 (en) * | 2017-04-13 | 2017-05-31 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of stargardt disease |
| KR20240035631A (ko) | 2017-08-25 | 2024-03-15 | 스톡 테라퓨틱스, 인크. | 병태 및 질환 치료용 안티센스 올리고머 |
| CN111183234A (zh) | 2017-10-11 | 2020-05-19 | 雷杰纳荣制药公司 | 在表达pnpla3 i148m变异的患者的肝病治疗中对hsd17b13的抑制 |
| CN120290560A (zh) * | 2017-10-23 | 2025-07-11 | 斯托克制药公司 | 用于治疗基于无义介导的rna衰变的病况和疾病的反义寡聚体 |
| RU177889U1 (ru) * | 2017-11-30 | 2018-03-15 | Общество с ограниченной ответственностью "Вертикаль-М" (ООО "Вертикаль-М") | Дренаж для хирургического лечения глаукомы |
| KR20250154534A (ko) | 2018-03-21 | 2025-10-28 | 리제너론 파마슈티칼스 인코포레이티드 | 17β-하이드록시스테로이드 데하이드로게나제 유형 13 (HSD17B13) iRNA 조성물 및 그것의 사용 방법 |
| WO2019204514A1 (en) * | 2018-04-17 | 2019-10-24 | Limelight Bio, Inc. | Trans-splicing molecules |
| WO2019213525A1 (en) | 2018-05-04 | 2019-11-07 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| AU2019287635A1 (en) * | 2018-06-14 | 2020-12-17 | Ionis Pharmaceuticals, Inc. | Compounds and methods for increasing STMN2 expression |
| US11279929B2 (en) | 2018-07-03 | 2022-03-22 | Hoffmann-La Roche, Inc. | Oligonucleotides for modulating Tau expression |
| JP2022523065A (ja) * | 2019-01-25 | 2022-04-21 | ナヤン セラピューティクス， インク． | Ｎｒ２ｅ３発現抑制オリゴヌクレオチド、それを含む組成物、及びそれらの使用方法 |
| 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 |
| JP2022544702A (ja) * | 2019-08-19 | 2022-10-20 | ストーク セラピューティクス，インク． | スプライシングおよびタンパク質発現を調節するための組成物および方法 |
| AU2021265838A1 (en) * | 2020-04-29 | 2022-10-20 | Saliogen Therapeutics, Inc. | Compositions and methods for treatment of inherited macular degeneration |
| KR20230022409A (ko) | 2020-05-11 | 2023-02-15 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 opa1 안티센스 올리고머 |
| JP2023553710A (ja) | 2020-12-18 | 2023-12-25 | エフ． ホフマン－ラ ロシュ アーゲー | プログラニュリンを標的とするためのアンチセンスオリゴヌクレオチド |
| CN118434858A (zh) * | 2021-12-07 | 2024-08-02 | 豪夫迈·罗氏有限公司 | 靶向actl6b的反义寡核苷酸 |
| JP2025506826A (ja) * | 2022-02-25 | 2025-03-13 | 北京中因科技有限公司 | 発現カセット組み合わせ及びその使用 |
| CA3252043A1 (en) | 2022-05-13 | 2023-11-16 | Ascidian Therapeutics, Inc. | ABCA4 TRANS-SPLICING MOLECULES |
| EP4619047A1 (en) * | 2022-11-17 | 2025-09-24 | Skip Therapeutics Ltd. | Compositions and methods for treating retinal diseases |
| WO2024223696A1 (en) | 2023-04-24 | 2024-10-31 | Universiteit Gent | Compounds to treat inherited retinal disease |
| WO2025171404A1 (en) * | 2024-02-09 | 2025-08-14 | Utr Therapeutics Inc. | Destabilized are 3' utrs of tead1 and yap1 as pan cancer therapeutics |
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| RU2597972C2 (ru) * | 2010-10-22 | 2016-09-20 | Курна Инк. | Лечение заболеваний, связанных с геном альфа-l-идуронидазы (idua), путем ингибирования природного антисмыслового транскрипта гена idua |
| WO2014121287A2 (en) * | 2013-02-04 | 2014-08-07 | Isis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
| WO2015035091A1 (en) * | 2013-09-04 | 2015-03-12 | Cold Spring Harbor Laboratory | Reducing nonsense-mediated mrna decay |
| GB201410693D0 (en) * | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| EP3201339A4 (en) * | 2014-10-03 | 2018-09-19 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
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| JP2022046724A (ja) | 2022-03-23 |
| EP3390634A4 (en) | 2019-08-14 |
| JP7036723B2 (ja) | 2022-03-15 |
| JP2019500348A (ja) | 2019-01-10 |
| WO2017106370A1 (en) | 2017-06-22 |
| EP3390634A1 (en) | 2018-10-24 |
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