JP2019500345A5 - - Google Patents

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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
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JP
Japan
Prior art keywords
seq
mrna
protein
pharmaceutical composition
composition according
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Pending
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JP2018529229A
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English (en)
Japanese (ja)
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JP2019500345A (ja
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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

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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

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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)

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EP (1) EP3389672A4 (enExample)
JP (1) JP2019500345A (enExample)
CA (1) CA3005090A1 (enExample)
WO (1) WO2017106283A1 (enExample)

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CN116059236A (zh) 2015-12-14 2023-05-05 冷泉港实验室 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体
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EP3461837A1 (en) * 2017-09-28 2019-04-03 Secarna Pharmaceuticals GmbH & Co. KG Inhibitor inhibiting the expression of pprx1
AU2018348195B2 (en) 2017-10-11 2025-05-15 Regeneron Pharmaceuticals, Inc. Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation
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KR20220104677A (ko) * 2019-08-19 2022-07-26 스톡 테라퓨틱스, 인크. 스플라이싱 및 단백질 발현을 조절하기 위한 조성물 및 방법
CA3148158A1 (en) * 2019-09-20 2021-03-25 Longcheng Li Nucleic acid molecule for treating thrombocytopenia and use thereof
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
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
US12338437B2 (en) 2020-05-11 2025-06-24 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases
WO2021231698A1 (en) * 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
EP4185696A1 (en) 2020-07-23 2023-05-31 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
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