JP2019500348A5 - - Google Patents
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- JP2019500348A5 JP2019500348A5 JP2018529249A JP2018529249A JP2019500348A5 JP 2019500348 A5 JP2019500348 A5 JP 2019500348A5 JP 2018529249 A JP2018529249 A JP 2018529249A JP 2018529249 A JP2018529249 A JP 2018529249A JP 2019500348 A5 JP2019500348 A5 JP 2019500348A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- mrna
- protein
- optn
- pharmaceutical composition
- 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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Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022000109A JP2022046724A (ja) | 2015-12-14 | 2022-01-04 | 眼疾患の処置のための組成物および方法 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562267259P | 2015-12-14 | 2015-12-14 | |
| US62/267,259 | 2015-12-14 | ||
| US201662318958P | 2016-04-06 | 2016-04-06 | |
| 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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022000109A Division JP2022046724A (ja) | 2015-12-14 | 2022-01-04 | 眼疾患の処置のための組成物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019500348A JP2019500348A (ja) | 2019-01-10 |
| JP2019500348A5 true JP2019500348A5 (https=) | 2020-01-30 |
| JP7036723B2 JP7036723B2 (ja) | 2022-03-15 |
Family
ID=59057531
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018529249A Expired - Fee Related JP7036723B2 (ja) | 2015-12-14 | 2016-12-14 | 眼疾患の処置のための組成物および方法 |
| JP2022000109A Pending JP2022046724A (ja) | 2015-12-14 | 2022-01-04 | 眼疾患の処置のための組成物および方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022000109A Pending JP2022046724A (ja) | 2015-12-14 | 2022-01-04 | 眼疾患の処置のための組成物および方法 |
Country Status (4)
| Country | Link |
|---|---|
| EP (1) | EP3390634A4 (https=) |
| JP (2) | JP7036723B2 (https=) |
| CA (1) | CA3005128A1 (https=) |
| WO (1) | WO2017106370A1 (https=) |
Families Citing this family (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| AU2015327836B2 (en) | 2014-10-03 | 2021-07-01 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| CN108603230A (zh) | 2015-10-09 | 2018-09-28 | 南安普敦大学 | 基因表达的调节与蛋白质表达失调的筛选 |
| 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 |
| RU2019126483A (ru) | 2017-01-23 | 2021-02-24 | Ридженерон Фармасьютикалз, Инк. | Варианты 17-бета-гидроксистероиддегидрогеназы 13 (hsd17b13) и их применение |
| KR20190139869A (ko) | 2017-04-11 | 2019-12-18 | 리제너론 파마슈티칼스 인코포레이티드 | 하이드록시스테로이드 (17-베타) 탈수소효소(hsd17b) 패밀리의 구성원의 조절인자의 활성도를 스크리닝하기 위한 검정 |
| GB201706009D0 (en) * | 2017-04-13 | 2017-05-31 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of stargardt disease |
| KR102318434B1 (ko) | 2017-08-25 | 2021-11-01 | 스톡 테라퓨틱스, 인크. | 병태 및 질환 치료용 안티센스 올리고머 |
| AU2018348195B2 (en) | 2017-10-11 | 2025-05-15 | Regeneron Pharmaceuticals, Inc. | Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation |
| HUE070436T2 (hu) * | 2017-10-23 | 2025-06-28 | Stoke Therapeutics Inc | Antiszensz oligomerek nonszensz-mediált RNS-lebomláson alapuló állapotok és betegségek kezelésére |
| RU177889U1 (ru) * | 2017-11-30 | 2018-03-15 | Общество с ограниченной ответственностью "Вертикаль-М" (ООО "Вертикаль-М") | Дренаж для хирургического лечения глаукомы |
| AU2019239971B2 (en) | 2018-03-21 | 2025-09-11 | Regeneron Pharmaceuticals, Inc. | 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof |
| KR102866133B1 (ko) | 2018-04-17 | 2025-09-30 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 트랜스-스플라이싱 분자 |
| CA3099280A1 (en) | 2018-05-04 | 2019-11-07 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| CA3103429A1 (en) * | 2018-06-14 | 2019-12-19 | Don W. Cleveland | Compounds and methods for increasing stmn2 expression |
| PE20210346A1 (es) * | 2018-07-03 | 2021-02-25 | Hoffmann La Roche | Oligonucleotidos para modular la expresion de tau |
| 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 |
| AU2020227825B2 (en) | 2019-02-27 | 2026-03-26 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of conditions and diseases |
| US20220290154A1 (en) * | 2019-08-08 | 2022-09-15 | Stichting Radboud Universitair Medisch Centrum | Antisense oligonucleotides rescue aberrant splicing of ABCA4 |
| EP4017979A4 (en) * | 2019-08-19 | 2024-03-27 | Stoke Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR MODULATING PROTEIN SPLICING AND EXPRESSION |
| AU2021265838A1 (en) * | 2020-04-29 | 2022-10-20 | Saliogen Therapeutics, Inc. | Compositions and methods for treatment of inherited macular degeneration |
| CN115867657A (zh) | 2020-05-11 | 2023-03-28 | 斯托克制药公司 | 用于治疗疾患和疾病的opa1反义寡聚物 |
| AR124378A1 (es) | 2020-12-18 | 2023-03-22 | Hoffmann La Roche | Oligonucleótidos antisentido dirigida a progranulina |
| WO2023104693A1 (en) * | 2021-12-07 | 2023-06-15 | F. Hoffmann-La Roche Ag | Antisense oligonucleotides targeting actl6b |
| WO2023160454A1 (zh) * | 2022-02-25 | 2023-08-31 | 北京中因科技有限公司 | 表达盒组合及其应用 |
| EP4522758A4 (en) | 2022-05-13 | 2026-05-06 | 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 |
| WO2025238644A1 (en) * | 2024-05-16 | 2025-11-20 | Skip Therapeutics Ltd. | Compositions and methods for treating retinal diseases |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR101865433B1 (ko) * | 2010-10-22 | 2018-07-13 | 큐알엔에이, 인크. | 알파-l 이두로니다아제 (idua)에 대한 자연 안티센스 전사체의 저해에 의한 idua 관련된 질환의 치료 |
| WO2014121287A2 (en) * | 2013-02-04 | 2014-08-07 | Isis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
| EP3041958B1 (en) * | 2013-09-04 | 2019-12-04 | Cold Spring Harbor Laboratory | Reducing nonsense-mediated mrna decay |
| GB201410693D0 (en) * | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| AU2015327836B2 (en) * | 2014-10-03 | 2021-07-01 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
-
2016
- 2016-12-14 JP JP2018529249A patent/JP7036723B2/ja not_active Expired - Fee Related
- 2016-12-14 CA CA3005128A patent/CA3005128A1/en active Pending
- 2016-12-14 WO PCT/US2016/066691 patent/WO2017106370A1/en not_active Ceased
- 2016-12-14 EP EP16876610.3A patent/EP3390634A4/en not_active Withdrawn
-
2022
- 2022-01-04 JP JP2022000109A patent/JP2022046724A/ja active Pending
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