JP2019500345A5 - - Google Patents

Download PDF

Info

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
Application number
JP2018529229A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019500345A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2016/066564 external-priority patent/WO2017106283A1/en
Publication of JP2019500345A publication Critical patent/JP2019500345A/ja
Publication of JP2019500345A5 publication Critical patent/JP2019500345A5/ja
Pending legal-status Critical Current

Links

JP2018529229A 2015-12-14 2016-12-14 肝臓病の処置のための組成物および方法 Pending JP2019500345A (ja)

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)

* Cited by examiner, † Cited by third party
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)

* Cited by examiner, † Cited by third party
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

Similar Documents

Publication Publication Date Title
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
CN103038349B (zh) 调节通过miR-378实现的代谢
JP2019501892A5 (enExample)
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
JP2019500347A5 (enExample)
JP2019500346A5 (enExample)
JP2019500345A (ja) 肝臓病の処置のための組成物および方法
JP2018538288A5 (enExample)
US20170088835A1 (en) Compositions and methods for modulation of smn2 splicing
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
TW202221120A (zh) 用於治療代謝症候群之組成物及方法
CN116209761A (zh) 靶向rna结合蛋白位点的寡核苷酸
JP2018538287A5 (enExample)
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
WO2026067818A1 (zh) 一种用于调控SCN1A基因mRNA和蛋白表达的方法和化合物