JP2019500350A5 - - Google Patents

Download PDF

Info

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
Authority
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
Other languages
English (en)
Japanese (ja)
Other versions
JP7051683B2 (ja
JP2019500350A (ja
Filing date
Publication date
Application filed filed Critical
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

Links

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
AU2015327836B2 (en) 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
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
EP3390636B1 (en) 2015-12-14 2021-05-19 Cold Spring Harbor Laboratory Antisense oligomers for treatment of dravet syndrome
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
WO2018136758A1 (en) 2017-01-23 2018-07-26 Regeneron Pharmaceuticals, Inc. Hsd17b13 variants and uses thereof
MX2019012169A (es) 2017-04-11 2019-12-11 Regeneron Pharma Ensayos para evaluar la actividad de moduladores de miembros de la familia de hidroxiesteroide (17-beta) deshidrogenasa (hsd17b).
EP3673080B1 (en) 2017-08-25 2023-10-18 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
JP7434151B2 (ja) 2017-10-11 2024-02-20 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 Ｐｎｐｌａ３ ｉ１４８ｍの変異を発現している患者の肝疾患の治療におけるｈｓｄ１７ｂ１３の阻害
AU2019239971B2 (en) 2018-03-21 2025-09-11 Regeneron Pharmaceuticals, Inc. 17beta-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
CN113748209A (zh) 2019-02-27 2021-12-03 斯托克制药公司 用于治疗病况和疾病的反义寡聚体
KR20220104677A (ko) * 2019-08-19 2022-07-26 스톡 테라퓨틱스, 인크. 스플라이싱 및 단백질 발현을 조절하기 위한 조성물 및 방법
AU2021270720A1 (en) 2020-05-11 2022-12-08 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
US20120252877A1 (en) * 2009-08-14 2012-10-04 Theramind Research, Llc Methods and compositions for treatment of tuberous sclerosis complex
WO2011031786A2 (en) * 2009-09-08 2011-03-17 Laboratory Corporation Of America Holdings Compositions and methods for diagnosing autism spectrum disorders
DK3041958T3 (da) * 2013-09-04 2020-03-09 Cold Spring Harbor Laboratory Reducering af nonsense-medieret mrna-degradering
GB201410693D0 (en) * 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
AU2015327836B2 (en) * 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
CA3005246A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of central nervous system diseases

Similar Documents

Publication Publication Date Title
JP2019500350A5 (enExample)
JP2019501892A5 (enExample)
JP2018538288A5 (enExample)
JP2018538287A5 (enExample)
JP2019500349A5 (enExample)
JP2019500347A5 (enExample)
JP2019500346A5 (enExample)
US6653467B1 (en) Medicament for treatment of Duchenne muscular dystrophy
EP1191098B1 (en) Pharmaceutical composition comprising dystrophin exon 45 for treatment of duchenne muscular dystrophy
US20200155606A1 (en) Crispr/rna-guided nuclease systems and methods
JP2017536338A5 (enExample)
JP2017519766A5 (enExample)
JP2019500345A5 (enExample)
WO2022147573A1 (en) Programmable rna editing in vivo via recruitment of endogenous adars
WO2021113264A1 (en) Engineering circular guide rnas
US20200407721A1 (en) Compounds of chemically modified oligonucleotides and methods of use thereof
JP2002325582A (ja) デュシェンヌ型筋ジストロフィー治療剤
JP2024079818A (ja) 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
EP3594346A1 (en) Antisense oligonucleotide and composition for prevention or treatment of glycogen storage disease type ia
WO2013134403A1 (en) Method of treating neurodegenerative diseases with microrna regulators
JP2024524974A (ja) ナンセンス変異依存ｒｎａ分解機構に基づく病態及び疾患の処置のためのアンチセンスオリゴマー
JPWO2021158858A5 (enExample)
TW202242113A (zh) 用於治療與多囊蛋白表現相關之病況及疾病之組合物
US20220213484A1 (en) Angptl2 antisense oligonucleotides and uses thereof
EP3690046A2 (en) Composition for treatment of hemophilia, comprising crispr/cas system having coagulation factor viii gene inversion correction potential