JP2019500345A5 - - Google Patents
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- JP2019500345A5 JP2019500345A5 JP2018529229A JP2018529229A JP2019500345A5 JP 2019500345 A5 JP2019500345 A5 JP 2019500345A5 JP 2018529229 A JP2018529229 A JP 2018529229A JP 2018529229 A JP2018529229 A JP 2018529229A JP 2019500345 A5 JP2019500345 A5 JP 2019500345A5
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Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562267238P | 2015-12-14 | 2015-12-14 | |
| US62/267,238 | 2015-12-14 | ||
| US201662319015P | 2016-04-06 | 2016-04-06 | |
| US62/319,015 | 2016-04-06 | ||
| PCT/US2016/066564 WO2017106283A1 (en) | 2015-12-14 | 2016-12-14 | Compositions and methods for treatment of liver diseases |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019500345A JP2019500345A (ja) | 2019-01-10 |
| JP2019500345A5 true JP2019500345A5 (cg-RX-API-DMAC7.html) | 2020-01-30 |
Family
ID=59057460
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018529229A Pending JP2019500345A (ja) | 2015-12-14 | 2016-12-14 | 肝臓病の処置のための組成物および方法 |
Country Status (4)
| Country | Link |
|---|---|
| EP (1) | EP3389672A4 (cg-RX-API-DMAC7.html) |
| JP (1) | JP2019500345A (cg-RX-API-DMAC7.html) |
| CA (1) | CA3005090A1 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2017106283A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| CN107109411B (zh) | 2014-10-03 | 2022-07-01 | 冷泉港实验室 | 核基因输出的定向增加 |
| KR102422625B1 (ko) | 2015-10-09 | 2022-07-20 | 유니버시티 오브 사우스앰톤 | 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝 |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| KR102604132B1 (ko) | 2015-12-14 | 2023-11-17 | 콜드스프링하버러보러토리 | 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머 |
| CN118325899A (zh) | 2017-01-23 | 2024-07-12 | 瑞泽恩制药公司 | Hsd17b13变体及其应用 |
| RU2019135845A (ru) | 2017-04-11 | 2021-05-11 | Ридженерон Фармасьютикалз, Инк. | Анализы для скрининга активности модуляторов членов семейства (17-бета)гидроксистероиддегидрогеназ (hsd17b) |
| CN111278991B (zh) | 2017-08-25 | 2022-04-01 | 斯托克制药公司 | 用于治疗病况和疾病的反义寡聚体 |
| EP3461837A1 (en) * | 2017-09-28 | 2019-04-03 | Secarna Pharmaceuticals GmbH & Co. KG | Inhibitor inhibiting the expression of pprx1 |
| EP4234719B1 (en) | 2017-10-11 | 2025-11-26 | Regeneron Pharmaceuticals, Inc. | Inhibition of hsd17b13 in the treatment of liver disease in patients expressing the pnpla3 i148m variation |
| RS66633B1 (sr) | 2017-10-23 | 2025-08-29 | Stoke Therapeutics Inc | Antisens oligomeri za lečenje bolesti i stanja na bazi besmisleno posredovanog raspadanja rnk |
| WO2019161105A1 (en) * | 2018-02-14 | 2019-08-22 | Deep Genomics Incorporated | Oligonucleotide therapy for wilson disease |
| CN112020559A (zh) * | 2018-02-21 | 2020-12-01 | 百时美施贵宝公司 | Camk2d反义寡核苷酸及其用途 |
| BR112020018758A2 (pt) | 2018-03-21 | 2021-01-26 | Regeneron Pharmaceuticals, Inc. | agente de ácido ribonucleico de fita dupla, célula, vetor, composição farmacêutica, e, métodos para inibição da expressão de 17¿-hidroxiesteroide desidrogenases tipo 13, para tratamento de um indivíduo, para prevenção de um sintoma em um indivíduo, para redução do risco de desenvolver doença hepática crônica, para inibição da progressão de esteatose, para inibição do acúmulo de gotículas de lipídios |
| US12060558B2 (en) | 2018-05-04 | 2024-08-13 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| TWI869213B (zh) * | 2018-09-19 | 2025-01-01 | 美商Ionis製藥公司 | Pnpla3表現之調節劑 |
| US10913951B2 (en) | 2018-10-31 | 2021-02-09 | University of Pittsburgh—of the Commonwealth System of Higher Education | Silencing of HNF4A-P2 isoforms with siRNA to improve hepatocyte function in liver failure |
| CN111849968A (zh) * | 2019-04-30 | 2020-10-30 | 中美瑞康核酸技术(南通)研究院有限公司 | 寡核苷酸分子及其在急性间歇性卟啉症治疗中的应用 |
| WO2021034985A1 (en) * | 2019-08-19 | 2021-02-25 | Stoke Therapeutics, Inc. | Compositions and methods for modulating splicing and protein expression |
| WO2021052470A1 (zh) * | 2019-09-20 | 2021-03-25 | 中美瑞康核酸技术(南通)研究院有限公司 | 用于治疗血小板减少症的核酸分子及其应用 |
| CN114728017B (zh) * | 2019-10-14 | 2025-10-28 | 阿斯利康(瑞典)有限公司 | Pnpla3表达的调节剂 |
| WO2021113755A2 (en) * | 2019-12-04 | 2021-06-10 | Pai Athma A | Anti-slc6a1 oligonucleotides and related methods |
| 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 |
| WO2021231698A1 (en) * | 2020-05-15 | 2021-11-18 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl) |
| CN116209761A (zh) * | 2020-07-23 | 2023-06-02 | 豪夫迈·罗氏有限公司 | 靶向rna结合蛋白位点的寡核苷酸 |
| WO2022109030A1 (en) * | 2020-11-17 | 2022-05-27 | Skyhawk Therapeutics, Inc. | Rna-targeting splicing modifiers for treatment of pnpla3-associated conditions and diseases |
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| US4866042A (en) | 1987-11-18 | 1989-09-12 | Neuwelt Edward A | Method for the delivery of genetic material across the blood brain barrier |
| US6294520B1 (en) | 1989-03-27 | 2001-09-25 | Albert T. Naito | Material for passage through the blood-brain barrier |
| US5151510A (en) | 1990-04-20 | 1992-09-29 | Applied Biosystems, Inc. | Method of synethesizing sulfurized oligonucleotide analogs |
| NZ256018A (en) | 1992-09-25 | 1997-07-27 | Rhone Poulenc Rorer Sa | Recombinant adenoviral vectors and their use in directing expression and transcription of selected nucleotide sequences in cells of the central nervous system |
| US5656612A (en) | 1994-05-31 | 1997-08-12 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide modulation of raf gene expression |
| FR2727867B1 (fr) | 1994-12-13 | 1997-01-31 | Rhone Poulenc Rorer Sa | Transfert de genes dans les motoneurones medullaires au moyen de vecteurs adenoviraux |
| US6936589B2 (en) | 2001-09-28 | 2005-08-30 | Albert T. Naito | Parenteral delivery systems |
| AU2003247610A1 (en) * | 2002-06-21 | 2004-01-06 | Ptc Therapeutics, Inc. | METHODS FOR IDENTIFYING SMALL MOLECULES THAT MODULATE PREMATURE TRANSLATION TERMINATION AND NONSENSE MEDIATED mRNA DECAY |
| AU2006232370B2 (en) * | 2005-04-01 | 2011-10-06 | Banyan Biomarkers | Biomakers of liver injury |
| US8258109B2 (en) | 2005-10-20 | 2012-09-04 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of LMNA expression |
| WO2009073809A2 (en) | 2007-12-04 | 2009-06-11 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugates as delivery agents for oligonucleotides |
| JP5707396B2 (ja) | 2009-06-17 | 2015-04-30 | アイシス ファーマシューティカルズ， インコーポレーテッド | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| WO2012075040A2 (en) * | 2010-11-30 | 2012-06-07 | Shire Human Genetic Therapies, Inc. | mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES |
| US20140128449A1 (en) * | 2011-04-07 | 2014-05-08 | The Board Of Regents Of The University Of Texas System | Oligonucleotide modulation of splicing |
| ES2626488T3 (es) | 2011-07-19 | 2017-07-25 | Wave Life Sciences Pte. Ltd. | Procedimientos para la síntesis de ácidos nucleicos funcionalizados |
| PL3041958T3 (pl) | 2013-09-04 | 2020-11-02 | Cold Spring Harbor Laboratory | Redukcja rozpadu mRNA mediowanego sekwencjami nonsensownymi |
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- 2016-12-14 CA CA3005090A patent/CA3005090A1/en active Pending
- 2016-12-14 JP JP2018529229A patent/JP2019500345A/ja active Pending
- 2016-12-14 WO PCT/US2016/066564 patent/WO2017106283A1/en not_active Ceased
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