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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- JP
- Japan
- Prior art keywords
- seq
- mrna
- protein
- pharmaceutical composition
- composition according
- Prior art date
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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 (29)
| 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 |
| AU2016334804B2 (en) | 2015-10-09 | 2022-03-31 | University Of Southampton | Modulation of gene expression and screening for deregulated protein expression |
| ES2882500T3 (es) | 2015-12-14 | 2021-12-02 | Cold Spring Harbor Laboratory | Oligómeros antisentido para el tratamiento del síndrome de Dravet |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| SG11201906735RA (en) | 2017-01-23 | 2019-08-27 | Regeneron Pharma | Hydroxysteroid 17-beta dehydrogenase 13 (hsd17b13) variants and uses thereof |
| JP2020516283A (ja) | 2017-04-11 | 2020-06-11 | リジェネロン・ファーマシューティカルズ・インコーポレイテッドRegeneron Pharmaceuticals, Inc. | ヒドロキシステロイド(17−ベータ)デヒドロゲナーゼ(hsd17b)ファミリーのメンバーのモジュレーターの活性をスクリーニングするためのアッセイ |
| SI3673080T1 (sl) | 2017-08-25 | 2024-03-29 | Stoke Therapeutics, Inc. | Protismiselni oligomeri za zdravljenje bolezenskih stanj in bolezni |
| EP3461837A1 (en) * | 2017-09-28 | 2019-04-03 | Secarna Pharmaceuticals GmbH & Co. KG | Inhibitor inhibiting the expression of pprx1 |
| KR20240125690A (ko) | 2017-10-11 | 2024-08-19 | 리제너론 파마슈티칼스 인코포레이티드 | Pnpla3 i148m 변이를 발현하는 환자의 간 질환의 치료에서의 hsd17b13의 저해 |
| EP3700570B1 (en) * | 2017-10-23 | 2025-01-08 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases |
| BR112020016524A2 (pt) | 2018-02-14 | 2021-09-28 | Deep Genomics Incorporated | Terapia com oligonucleotídeos para a doença de wilson |
| CA3091789A1 (en) * | 2018-02-21 | 2019-08-29 | Bristol-Myers Squibb Company | Camk2d antisense oligonucleotides and uses thereof |
| CN112020556A (zh) | 2018-03-21 | 2020-12-01 | 瑞泽恩制药公司 | 第13型17β羟基类固醇脱氢酶(HSD17B13)iRNA组成物及其使用方法 |
| 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 |
| KR20210134003A (ko) | 2019-02-27 | 2021-11-08 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 안티센스 올리고머 |
| 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 |
| CA3147970A1 (en) * | 2019-08-19 | 2021-02-25 | Isabel AZNAREZ | Compositions and methods for modulating splicing and protein expression |
| AU2020349991A1 (en) * | 2019-09-20 | 2022-04-07 | Ractigen Therapeutics | Nucleic acid molecule for treating immune thrombocytopenia and application thereof |
| JP7757277B2 (ja) * | 2019-10-14 | 2025-10-21 | アストラゼネカ・アクチエボラーグ | Pnpla3発現のモジュレーター |
| EP4069844A4 (en) * | 2019-12-04 | 2024-03-20 | Pai, Athma A. | ANTI-SLC6A1 OLIGONUCLEOTIDES AND RELATED METHODS |
| TWI817107B (zh) * | 2020-04-15 | 2023-10-01 | 中央研究院 | 寡核苷酸共軛物及其製備與應用 |
| BR112022022889A2 (pt) | 2020-05-11 | 2023-04-04 | Stoke Therapeutics Inc | Oligômeros antissentido de opa1 para tratamento de condições e doenças |
| WO2021231698A1 (en) * | 2020-05-15 | 2021-11-18 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl) |
| WO2022018187A1 (en) * | 2020-07-23 | 2022-01-27 | F. Hoffmann-La Roche Ag | Oligonucleotides targeting rna binding protein sites |
| WO2022109030A1 (en) * | 2020-11-17 | 2022-05-27 | Skyhawk Therapeutics, Inc. | Rna-targeting splicing modifiers for treatment of pnpla3-associated conditions and diseases |
| US20250333742A1 (en) * | 2024-04-25 | 2025-10-30 | Insitro, Inc. | Compositions and methods for treating metabolic dysfunction-associated steatotic liver disease |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| JPH08501686A (ja) | 1992-09-25 | 1996-02-27 | ローン−プーラン・ロレ・ソシエテ・アノニム | 中枢神経系、特に脳における細胞への外来遺伝子の転移のためのアデノウィルスベクター |
| 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 |
| WO2004001010A2 (en) * | 2002-06-21 | 2003-12-31 | Ptc Therapeutics, Inc. | METHODS FOR IDENTIFYING SMALL MOLECULES THAT MODULATE PREMATURE TRANSLATION TERMINATION AND NONSENSE MEDIATED mRNA DECAY |
| US7645584B2 (en) * | 2005-04-01 | 2010-01-12 | University Of Florida Research Foundation, Inc. | Biomarkers of liver injury |
| WO2007047913A2 (en) | 2005-10-20 | 2007-04-26 | Isis Pharmaceuticals, Inc | Compositions and methods for modulation of lmna expression |
| CN102006890A (zh) | 2007-12-04 | 2011-04-06 | 阿尔尼拉姆医药品有限公司 | 靶向脂质 |
| CN115227709A (zh) | 2009-06-17 | 2022-10-25 | 冷泉港实验室 | 用于在对象中调节smn2剪接的组合物和方法 |
| 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 |
| US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| ES2779302T3 (es) | 2013-09-04 | 2020-08-14 | Cold Spring Harbor Laboratory | Reducción de la degradación de ARNm con mediación sin sentido |
-
2016
- 2016-12-14 EP EP16876543.6A patent/EP3389672A4/en not_active Withdrawn
- 2016-12-14 CA CA3005090A patent/CA3005090A1/en active Pending
- 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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