JP2019500350A5 - - Google Patents

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Publication number
JP2019500350A5
JP2019500350A5 JP2018529258A JP2018529258A JP2019500350A5 JP 2019500350 A5 JP2019500350 A5 JP 2019500350A5 JP 2018529258 A JP2018529258 A JP 2018529258A JP 2018529258 A JP2018529258 A JP 2018529258A JP 2019500350 A5 JP2019500350 A5 JP 2019500350A5
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JP
Japan
Prior art keywords
mrna
pharmaceutical composition
composition according
item
tuberin
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.)
Granted
Application number
JP2018529258A
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English (en)
Japanese (ja)
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JP2019500350A (ja
JP7051683B2 (ja
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Publication date
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Priority claimed from PCT/US2016/066705 external-priority patent/WO2017106375A1/en
Publication of JP2019500350A publication Critical patent/JP2019500350A/ja
Publication of JP2019500350A5 publication Critical patent/JP2019500350A5/ja
Priority to JP2022053948A priority Critical patent/JP2022088533A/ja
Application granted granted Critical
Publication of JP7051683B2 publication Critical patent/JP7051683B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2018529258A 2015-12-14 2016-12-14 結節性硬化症の処置のためのアンチセンスオリゴマー Expired - Fee Related JP7051683B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022053948A JP2022088533A (ja) 2015-12-14 2022-03-29 結節性硬化症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
US201562267212P 2015-12-14 2015-12-14
US62/267,212 2015-12-14
PCT/US2016/066705 WO2017106375A1 (en) 2015-12-14 2016-12-14 Antisense oligomers for treatment of tuberous sclerosis complex

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022053948A Division JP2022088533A (ja) 2015-12-14 2022-03-29 結節性硬化症の処置のためのアンチセンスオリゴマー

Publications (3)

Publication Number Publication Date
JP2019500350A JP2019500350A (ja) 2019-01-10
JP2019500350A5 true JP2019500350A5 (enExample) 2020-01-30
JP7051683B2 JP7051683B2 (ja) 2022-04-11

Family

ID=59057534

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2018529258A Expired - Fee Related JP7051683B2 (ja) 2015-12-14 2016-12-14 結節性硬化症の処置のためのアンチセンスオリゴマー
JP2022053948A Pending JP2022088533A (ja) 2015-12-14 2022-03-29 結節性硬化症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
JP2022053948A Pending JP2022088533A (ja) 2015-12-14 2022-03-29 結節性硬化症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) EP3390635A4 (enExample)
JP (2) JP7051683B2 (enExample)
CA (1) CA3005131A1 (enExample)
WO (1) WO2017106375A1 (enExample)

Families Citing this family (16)

* 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
KR20240125690A (ko) 2017-10-11 2024-08-19 리제너론 파마슈티칼스 인코포레이티드 Pnpla3 i148m 변이를 발현하는 환자의 간 질환의 치료에서의 hsd17b13의 저해
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
KR20210134003A (ko) 2019-02-27 2021-11-08 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 안티센스 올리고머
CA3147970A1 (en) * 2019-08-19 2021-02-25 Isabel AZNAREZ Compositions and methods for modulating splicing and protein expression
BR112022022889A2 (pt) 2020-05-11 2023-04-04 Stoke Therapeutics Inc Oligômeros antissentido de opa1 para tratamento de condições e doenças
EP4560019A1 (en) * 2023-11-22 2025-05-28 Sylentis S.A.U. Sirna and compositions for prophylactic and therapeutic treatment of ocular retinal conditions
EP4560020A1 (en) * 2023-11-22 2025-05-28 Sylentis S.A.U. Sirna and compositions for prophylactic and therapeutic treatment of ocular retinal conditions

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2087110A2 (en) * 2006-10-11 2009-08-12 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Influenza targets
WO2011020118A1 (en) 2009-08-14 2011-02-17 Theramind Research, Llc Methods and compositions for treatment of tuberous sclerosis complex
CA3047466A1 (en) * 2009-09-08 2011-03-17 Laboratory Corporation Of America Holdings Compositions and methods for diagnosing autism spectrum disorders
ES2779302T3 (es) * 2013-09-04 2020-08-14 Cold Spring Harbor Laboratory Reducción de la degradación de ARNm con mediación sin sentido
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
EP4104867A3 (en) * 2015-12-14 2023-03-01 Cold Spring Harbor Laboratory Compositions and methods for treatment of central nervous system diseases

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