JP2019500345A5 - - Google Patents
Download PDFInfo
- Publication number
- 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
- Authority
- JP
- Japan
- Prior art keywords
- seq
- mrna
- protein
- pharmaceutical composition
- composition according
- 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.)
- Pending
Links
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 (https=) | 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 (https=) |
| JP (1) | JP2019500345A (https=) |
| CA (1) | CA3005090A1 (https=) |
| WO (1) | WO2017106283A1 (https=) |
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 |
| CN107109411B (zh) | 2014-10-03 | 2022-07-01 | 冷泉港实验室 | 核基因输出的定向增加 |
| EP3359685B1 (en) | 2015-10-09 | 2026-01-28 | University Of Southampton | Modulation of gene expression for deregulated protein expression |
| EP3933041B1 (en) | 2015-12-14 | 2024-01-31 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant retardation |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| IL314915B1 (en) | 2017-01-23 | 2026-02-01 | Regeneron Pharma | Variants of 17-beta-hydroxysteroid dehydrogenase and their uses |
| KR20190139869A (ko) | 2017-04-11 | 2019-12-18 | 리제너론 파마슈티칼스 인코포레이티드 | 하이드록시스테로이드 (17-베타) 탈수소효소(hsd17b) 패밀리의 구성원의 조절인자의 활성도를 스크리닝하기 위한 검정 |
| SG11202001590RA (en) | 2017-08-25 | 2020-03-30 | Stoke Therapeutics Inc | Antisense oligomers for treatment of conditions and diseases |
| EP3461837A1 (en) * | 2017-09-28 | 2019-04-03 | Secarna Pharmaceuticals GmbH & Co. KG | Inhibitor inhibiting the expression of pprx1 |
| US10961583B2 (en) | 2017-10-11 | 2021-03-30 | Regeneron Phramaceuticals, Inc. | Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation |
| GB2610100B (en) | 2017-10-23 | 2023-08-16 | Stoke Therapeutics Inc | Antisense oligomers for treatment of non-sense mediated RNA decay based conditions and diseases |
| CN112041439A (zh) * | 2018-02-14 | 2020-12-04 | 深度基因组学公司 | 用于威尔森病的寡核苷酸疗法 |
| KR20200140805A (ko) * | 2018-02-21 | 2020-12-16 | 브리스톨-마이어스 스큅 컴퍼니 | Camk2d 안티센스 올리고뉴클레오티드 및 그의 용도 |
| JP7507093B2 (ja) | 2018-03-21 | 2024-06-27 | 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 | 17β-水酸化ステロイド脱水素酵素13型(HSD17B13)iRNA組成物およびその使用方法 |
| US12060558B2 (en) | 2018-05-04 | 2024-08-13 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| TWI856973B (zh) * | 2018-09-19 | 2024-10-01 | 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 |
| EP4017979A4 (en) * | 2019-08-19 | 2024-03-27 | Stoke Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR MODULATING PROTEIN SPLICING AND EXPRESSION |
| US20230212565A1 (en) * | 2019-09-20 | 2023-07-06 | Ractigen Therapeutics | Nucleic acid molecule for treating thrombocytopenia and application thereof |
| EP4045062A1 (en) * | 2019-10-14 | 2022-08-24 | Astrazeneca AB | Modulators of pnpla3 expression |
| WO2021113773A2 (en) | 2019-12-04 | 2021-06-10 | Pai Athma A | Identifying non-productive splice sites |
| 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 |
| EP4150078A1 (en) * | 2020-05-15 | 2023-03-22 | 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 |
| WO2025226980A2 (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 |
| EP0669987B1 (en) | 1992-09-25 | 2008-08-13 | Aventis Pharma S.A. | Adenovirus vectors for the transfer of foreign genes into cells of the central nervous system, particularly in brain |
| 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 |
| JP5202296B2 (ja) * | 2005-04-01 | 2013-06-05 | ユニバーシティ オブ フロリダ リサーチ ファウンデーション,インコーポレイティド | 肝臓傷害のバイオマーカー |
| WO2007047913A2 (en) | 2005-10-20 | 2007-04-26 | Isis Pharmaceuticals, Inc | Compositions and methods for modulation of lmna expression |
| WO2009082607A2 (en) | 2007-12-04 | 2009-07-02 | Alnylam Pharmaceuticals, Inc. | Targeting lipids |
| PT3305302T (pt) | 2009-06-17 | 2018-12-14 | Biogen Ma Inc | Composições e métodos de modulação de excisões de smn2 em um sujeito |
| 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 |
| AU2012284265B2 (en) | 2011-07-19 | 2017-08-17 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| PL3041958T4 (pl) | 2013-09-04 | 2020-11-02 | Cold Spring Harbor Laboratory | Redukcja rozpadu mRNA mediowanego sekwencjami nonsensownymi |
-
2016
- 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 EP EP16876543.6A patent/EP3389672A4/en not_active Withdrawn
- 2016-12-14 JP JP2018529229A patent/JP2019500345A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2019500345A5 (https=) | ||
| AU2016200344B2 (en) | Compositions and methods for modulation of smn2 splicing in a subject | |
| JP7049247B2 (ja) | 腎臓病の処置のための組成物と方法 | |
| JP6006120B2 (ja) | 対立遺伝子多様体の選択的低減 | |
| JP6018506B2 (ja) | 対立遺伝子多様体の選択的低減 | |
| CN103038349B (zh) | 调节通过miR-378实现的代谢 | |
| JP2019501892A5 (https=) | ||
| JP7054675B2 (ja) | 網膜色素変性症18と網膜色素変性症13の処置のための組成物と方法 | |
| US20120149757A1 (en) | Compositions and methods for modulation of smn2 splicing | |
| KR20080031164A (ko) | SR 단백질의 결합의 방해에 의해 그리고 이차 RNA구조의 방해에 의한 pre-mRNA에서 엑손 인식의조절 | |
| US20200181613A1 (en) | Compositions and methods for modulation of smn2 splicing | |
| JP2019500346A5 (https=) | ||
| JP2019500345A (ja) | 肝臓病の処置のための組成物および方法 | |
| JP2018538288A5 (https=) | ||
| JP2011513238A (ja) | 平滑筋の増殖および分化を調節するマイクロrnaならびにこれらの使用 | |
| CN116209761A (zh) | 靶向rna结合蛋白位点的寡核苷酸 | |
| JP2018538287A5 (https=) | ||
| KR20230047453A (ko) | 대사 증후군 치료를 위한 조성물 및 방법 | |
| JP2018538287A (ja) | 多発性嚢胞腎の処置のためのアンチセンスオリゴマー | |
| Gibson | Engineered microRNA therapeutics | |
| Lim et al. | Genetic approaches for the treatment of facioscapulohumeral muscular dystrophy | |
| Ho et al. | Modification of phosphorothioate oligonucleotides yields potent analogs with minimal toxicity for antisense experiments in the CNS | |
| Latronico et al. | Therapeutic applications of noncoding RNAs | |
| WO2025103391A1 (zh) | 用于抑制lpa的基因表达的sirna,含其的组合物及其用途 | |
| Bischof et al. | Exploiting the hypoxia sensitive non-coding genome for organ-specific physiologic reprogramming |