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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JP7051683B2 (ja
JP2019500350A (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 (cg-RX-API-DMAC7.html) 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 (cg-RX-API-DMAC7.html)
JP (2) JP7051683B2 (cg-RX-API-DMAC7.html)
CA (1) CA3005131A1 (cg-RX-API-DMAC7.html)
WO (1) WO2017106375A1 (cg-RX-API-DMAC7.html)

Families Citing this family (15)

* 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
CN107109411B (zh) 2014-10-03 2022-07-01 冷泉港实验室 核基因输出的定向增加
KR102422625B1 (ko) 2015-10-09 2022-07-20 유니버시티 오브 사우스앰톤 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝
KR102604132B1 (ko) 2015-12-14 2023-11-17 콜드스프링하버러보러토리 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
CN118325899A (zh) 2017-01-23 2024-07-12 瑞泽恩制药公司 Hsd17b13变体及其应用
RU2019135845A (ru) 2017-04-11 2021-05-11 Ридженерон Фармасьютикалз, Инк. Анализы для скрининга активности модуляторов членов семейства (17-бета)гидроксистероиддегидрогеназ (hsd17b)
CN111278991B (zh) 2017-08-25 2022-04-01 斯托克制药公司 用于治疗病况和疾病的反义寡聚体
EP4234719B1 (en) 2017-10-11 2025-11-26 Regeneron Pharmaceuticals, Inc. Inhibition of hsd17b13 in the treatment of liver disease in patients expressing the pnpla3 i148m variation
BR112020018758A2 (pt) 2018-03-21 2021-01-26 Regeneron Pharmaceuticals, Inc. agente de ácido ribonucleico de fita dupla, célula, vetor, composição farmacêutica, e, métodos para inibição da expressão de 17¿-hidroxiesteroide desidrogenases tipo 13, para tratamento de um indivíduo, para prevenção de um sintoma em um indivíduo, para redução do risco de desenvolver doença hepática crônica, para inibição da progressão de esteatose, para inibição do acúmulo de gotículas de lipídios
US12060558B2 (en) 2018-05-04 2024-08-13 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
WO2021034985A1 (en) * 2019-08-19 2021-02-25 Stoke Therapeutics, Inc. Compositions and methods for modulating splicing and protein expression
CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物
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
SG166778A1 (en) * 2006-10-11 2010-12-29 Max Planck Gesellschaft Influenza targets
US20120252877A1 (en) * 2009-08-14 2012-10-04 Theramind Research, Llc Methods and compositions for treatment of tuberous sclerosis complex
JP2013503646A (ja) * 2009-09-08 2013-02-04 ラボラトリー コーポレイション オブ アメリカ ホールディングス 自閉症スペクトラム障害を診断するための組成物および方法
PL3041958T3 (pl) * 2013-09-04 2020-11-02 Cold Spring Harbor Laboratory Redukcja rozpadu mRNA mediowanego sekwencjami nonsensownymi
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
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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