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 (enExample) | 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 (enExample) |
| JP (1) | JP2019500345A (enExample) |
| CA (1) | CA3005090A1 (enExample) |
| WO (1) | WO2017106283A1 (enExample) |
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 |
| 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 |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| 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 |
| 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 |
| KR20240035631A (ko) | 2017-08-25 | 2024-03-15 | 스톡 테라퓨틱스, 인크. | 병태 및 질환 치료용 안티센스 올리고머 |
| EP3461837A1 (en) * | 2017-09-28 | 2019-04-03 | Secarna Pharmaceuticals GmbH & Co. KG | Inhibitor inhibiting the expression of pprx1 |
| 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 |
| WO2019161105A1 (en) | 2018-02-14 | 2019-08-22 | Deep Genomics Incorporated | Oligonucleotide therapy for wilson disease |
| JP7500426B2 (ja) * | 2018-02-21 | 2024-06-17 | ブリストル－マイヤーズ スクイブ カンパニー | Ｃａｍｋ２ｄアンチセンスオリゴヌクレオチドおよびその使用 |
| KR20250154534A (ko) | 2018-03-21 | 2025-10-28 | 리제너론 파마슈티칼스 인코포레이티드 | 17β-하이드록시스테로이드 데하이드로게나제 유형 13 (HSD17B13) iRNA 조성물 및 그것의 사용 방법 |
| WO2019213525A1 (en) | 2018-05-04 | 2019-11-07 | 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 |
| 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 |
| PE20220604A1 (es) * | 2019-09-20 | 2022-04-25 | Ractigen Therapeutics | Molecula de acido nucleico para el tratamiento de la trombocitopenia y uso de la misma |
| EP4045062A1 (en) * | 2019-10-14 | 2022-08-24 | Astrazeneca AB | Modulators of pnpla3 expression |
| EP4069844A4 (en) * | 2019-12-04 | 2024-03-20 | Pai, Athma A. | ANTI-SLC6A1 OLIGONUCLEOTIDES AND RELATED METHODS |
| KR20230022409A (ko) | 2020-05-11 | 2023-02-15 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 opa1 안티센스 올리고머 |
| WO2021231698A1 (en) * | 2020-05-15 | 2021-11-18 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl) |
| TW202219273A (zh) * | 2020-07-23 | 2022-05-16 | 瑞士商赫孚孟拉羅股份公司 | 靶向ｒｎａ結合蛋白位點之寡核苷酸 |
| 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 |
| ATE404683T1 (de) | 1992-09-25 | 2008-08-15 | Aventis Pharma Sa | Adenovirus vektoren für die übertragung fremder gene in zellen des zentralen nervensystems, insbesondere im gehirn |
| 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 |
| ES2732071T3 (es) * | 2005-04-01 | 2019-11-20 | Univ Florida | Biomarcadores de lesiones hepáticas |
| US8258109B2 (en) | 2005-10-20 | 2012-09-04 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of LMNA expression |
| US20090247608A1 (en) | 2007-12-04 | 2009-10-01 | Alnylam Pharmaceuticals, Inc. | Targeting Lipids |
| LT3305302T (lt) | 2009-06-17 | 2018-12-10 | Biogen Ma Inc. | Junginiai ir būdai smn2 splaisingo moduliavimui subjekte |
| US8853377B2 (en) * | 2010-11-30 | 2014-10-07 | 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 |
| EP2734208B1 (en) | 2011-07-19 | 2017-03-01 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| WO2015035091A1 (en) | 2013-09-04 | 2015-03-12 | Cold Spring Harbor Laboratory | Reducing nonsense-mediated mrna decay |
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- 2016-12-14 EP EP16876543.6A patent/EP3389672A4/en not_active Withdrawn
- 2016-12-14 WO PCT/US2016/066564 patent/WO2017106283A1/en not_active Ceased
- 2016-12-14 JP JP2018529229A patent/JP2019500345A/ja active Pending
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