JP2018538288A5 - - Google Patents

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Publication number
JP2018538288A5
JP2018538288A5 JP2018529221A JP2018529221A JP2018538288A5 JP 2018538288 A5 JP2018538288 A5 JP 2018538288A5 JP 2018529221 A JP2018529221 A JP 2018529221A JP 2018529221 A JP2018529221 A JP 2018529221A JP 2018538288 A5 JP2018538288 A5 JP 2018538288A5
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JP
Japan
Prior art keywords
mrna
pharmaceutical composition
item
composition according
nucleobases
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.)
Ceased
Application number
JP2018529221A
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English (en)
Japanese (ja)
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JP2018538288A (ja
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Publication date
Application filed filed Critical
Priority claimed from PCT/US2016/066414 external-priority patent/WO2017106210A1/en
Publication of JP2018538288A publication Critical patent/JP2018538288A/ja
Publication of JP2018538288A5 publication Critical patent/JP2018538288A5/ja
Priority to JP2022069164A priority Critical patent/JP2022106803A/ja
Ceased legal-status Critical Current

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JP2018529221A 2015-12-14 2016-12-13 アラジール症候群の処置のためのアンチセンスオリゴマー Ceased JP2018538288A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022069164A JP2022106803A (ja) 2015-12-14 2022-04-20 アラジール症候群の処置のためのアンチセンスオリゴマー

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562267210P 2015-12-14 2015-12-14
US62/267,210 2015-12-14
PCT/US2016/066414 WO2017106210A1 (en) 2015-12-14 2016-12-13 Antisense oligomers for treatment of alagille syndrome

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022069164A Division JP2022106803A (ja) 2015-12-14 2022-04-20 アラジール症候群の処置のためのアンチセンスオリゴマー

Publications (2)

Publication Number Publication Date
JP2018538288A JP2018538288A (ja) 2018-12-27
JP2018538288A5 true JP2018538288A5 (https=) 2020-01-30

Family

ID=59057483

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2018529221A Ceased JP2018538288A (ja) 2015-12-14 2016-12-13 アラジール症候群の処置のためのアンチセンスオリゴマー
JP2022069164A Pending JP2022106803A (ja) 2015-12-14 2022-04-20 アラジール症候群の処置のためのアンチセンスオリゴマー

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2022069164A Pending JP2022106803A (ja) 2015-12-14 2022-04-20 アラジール症候群の処置のためのアンチセンスオリゴマー

Country Status (4)

Country Link
EP (1) EP3389671A4 (https=)
JP (2) JP2018538288A (https=)
CA (1) CA3005245A1 (https=)
WO (1) WO2017106210A1 (https=)

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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
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
EP3933041B1 (en) 2015-12-14 2024-01-31 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant retardation
IL314915B1 (en) 2017-01-23 2026-02-01 Regeneron Pharma Variants of 17-beta-hydroxysteroid dehydrogenase and their uses
US11963974B2 (en) 2017-03-10 2024-04-23 National Center For Child Health And Development Antisense oligonucleotide and composition for prevention or treatment of glycogen storage disease type Ia
KR20190139869A (ko) 2017-04-11 2019-12-18 리제너론 파마슈티칼스 인코포레이티드 하이드록시스테로이드 (17-베타) 탈수소효소(hsd17b) 패밀리의 구성원의 조절인자의 활성도를 스크리닝하기 위한 검정
US11197884B2 (en) 2017-08-18 2021-12-14 Ionis Pharmaceuticals, Inc. Modulation of the notch signaling pathway for treatment of respiratory disorders
SG11202001590RA (en) 2017-08-25 2020-03-30 Stoke Therapeutics Inc Antisense oligomers for treatment of conditions and diseases
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
CN111511915A (zh) * 2018-03-09 2020-08-07 第一三共株式会社 糖原病Ia型治疗药
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
US20210238257A1 (en) * 2018-04-27 2021-08-05 The Regents Of The University Of California De Novo Formation of the Biliary System by Hepatocyte Transdifferentiation
US12060558B2 (en) 2018-05-04 2024-08-13 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
AU2019336793B2 (en) * 2018-09-06 2024-11-21 The Regents Of The University Of California RNA and DNA base editing via engineered ADAR recruitment
KR20210134003A (ko) 2019-02-27 2021-11-08 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 안티센스 올리고머
BR112022022889A2 (pt) 2020-05-11 2023-04-04 Stoke Therapeutics Inc Oligômeros antissentido de opa1 para tratamento de condições e doenças

Family Cites Families (16)

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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
US20040102401A1 (en) * 2002-11-22 2004-05-27 Isis Pharmaceuticals Inc. Modulation of jagged 1 expression
GB0326578D0 (en) * 2003-11-14 2003-12-17 Univ Belfast Cancer diagnosis and therapy
WO2007047913A2 (en) 2005-10-20 2007-04-26 Isis Pharmaceuticals, Inc Compositions and methods for modulation of lmna expression
WO2007048629A2 (en) * 2005-10-28 2007-05-03 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Modulation of rna silencing efficiency by argonaute proteins
US20080280848A1 (en) * 2005-10-28 2008-11-13 Volker Patzel Structures of Active Guide Rna Molecules and Method of Selection
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
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

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