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 (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
CN107109411B (zh) 2014-10-03 2022-07-01 冷泉港实验室 核基因输出的定向增加
CN108603230A (zh) 2015-10-09 2018-09-28 南安普敦大学 基因表达的调节与蛋白质表达失调的筛选
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
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
CA3049980A1 (en) 2017-01-23 2018-07-26 Regeneron Pharmaceuticals, Inc. Hydroxysteroid 17-beta dehydrogenase 13 (hsd17b13) variants and uses thereof
CA3059348A1 (en) 2017-04-11 2018-10-18 Regeneron Pharmaceuticals, Inc. Assays for screening activity of modulators of members of the hydroxysteroid (17-beta) dehydrogenase (hsd17b) family
KR20260009385A (ko) 2017-08-25 2026-01-19 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
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
MY207352A (en) 2018-03-21 2025-02-21 Regeneron Pharma 17-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof
JP2021523227A (ja) 2018-05-04 2021-09-02 ストーク セラピューティクス，インク． コレステリルエステル蓄積症の処置のための方法及び組成物
KR20210134003A (ko) 2019-02-27 2021-11-08 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 안티센스 올리고머
KR20220104677A (ko) * 2019-08-19 2022-07-26 스톡 테라퓨틱스, 인크. 스플라이싱 및 단백질 발현을 조절하기 위한 조성물 및 방법
US12338437B2 (en) 2020-05-11 2025-06-24 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases
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
US20110150897A1 (en) * 2006-10-11 2011-06-23 Meyer Thomas F Influenza targets
WO2011020118A1 (en) * 2009-08-14 2011-02-17 Theramind Research, Llc Methods and compositions for treatment of tuberous sclerosis complex
EP3135772B1 (en) * 2009-09-08 2019-10-23 Laboratory Corporation of America Holdings Method for diagnosing autism spectrum disorders
WO2015035091A1 (en) * 2013-09-04 2015-03-12 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay
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
WO2017106382A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of central nervous system diseases

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