JP2019500348A5 - - Google Patents

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Publication number
JP2019500348A5
JP2019500348A5 JP2018529249A JP2018529249A JP2019500348A5 JP 2019500348 A5 JP2019500348 A5 JP 2019500348A5 JP 2018529249 A JP2018529249 A JP 2018529249A JP 2018529249 A JP2018529249 A JP 2018529249A JP 2019500348 A5 JP2019500348 A5 JP 2019500348A5
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JP
Japan
Prior art keywords
seq
mrna
protein
optn
pharmaceutical composition
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JP2018529249A
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English (en)
Japanese (ja)
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JP7036723B2 (ja
JP2019500348A (ja
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Priority claimed from PCT/US2016/066691 external-priority patent/WO2017106370A1/en
Publication of JP2019500348A publication Critical patent/JP2019500348A/ja
Publication of JP2019500348A5 publication Critical patent/JP2019500348A5/ja
Priority to JP2022000109A priority Critical patent/JP2022046724A/ja
Application granted granted Critical
Publication of JP7036723B2 publication Critical patent/JP7036723B2/ja
Expired - Fee Related legal-status Critical Current
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JP2018529249A 2015-12-14 2016-12-14 眼疾患の処置のための組成物および方法 Expired - Fee Related JP7036723B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022000109A JP2022046724A (ja) 2015-12-14 2022-01-04 眼疾患の処置のための組成物および方法

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562267259P 2015-12-14 2015-12-14
US62/267,259 2015-12-14
US201662318958P 2016-04-06 2016-04-06
US62/318,958 2016-04-06
PCT/US2016/066691 WO2017106370A1 (en) 2015-12-14 2016-12-14 Compositions and methods for treatment of eye diseases

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022000109A Division JP2022046724A (ja) 2015-12-14 2022-01-04 眼疾患の処置のための組成物および方法

Publications (3)

Publication Number Publication Date
JP2019500348A JP2019500348A (ja) 2019-01-10
JP2019500348A5 true JP2019500348A5 (enExample) 2020-01-30
JP7036723B2 JP7036723B2 (ja) 2022-03-15

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ID=59057531

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2018529249A Expired - Fee Related JP7036723B2 (ja) 2015-12-14 2016-12-14 眼疾患の処置のための組成物および方法
JP2022000109A Pending JP2022046724A (ja) 2015-12-14 2022-01-04 眼疾患の処置のための組成物および方法

Family Applications After (1)

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JP2022000109A Pending JP2022046724A (ja) 2015-12-14 2022-01-04 眼疾患の処置のための組成物および方法

Country Status (4)

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EP (1) EP3390634A4 (enExample)
JP (2) JP7036723B2 (enExample)
CA (1) CA3005128A1 (enExample)
WO (1) WO2017106370A1 (enExample)

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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
KR20220105174A (ko) 2015-10-09 2022-07-26 유니버시티 오브 사우스앰톤 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝
WO2017106377A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
WO2018136702A1 (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
GB201706009D0 (en) * 2017-04-13 2017-05-31 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of stargardt disease
KR20240035631A (ko) 2017-08-25 2024-03-15 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
CN111183234A (zh) 2017-10-11 2020-05-19 雷杰纳荣制药公司 在表达pnpla3 i148m变异的患者的肝病治疗中对hsd17b13的抑制
CN120290560A (zh) * 2017-10-23 2025-07-11 斯托克制药公司 用于治疗基于无义介导的rna衰变的病况和疾病的反义寡聚体
RU177889U1 (ru) * 2017-11-30 2018-03-15 Общество с ограниченной ответственностью "Вертикаль-М" (ООО "Вертикаль-М") Дренаж для хирургического лечения глаукомы
KR20250154534A (ko) 2018-03-21 2025-10-28 리제너론 파마슈티칼스 인코포레이티드 17β-하이드록시스테로이드 데하이드로게나제 유형 13 (HSD17B13) iRNA 조성물 및 그것의 사용 방법
WO2019204514A1 (en) * 2018-04-17 2019-10-24 Limelight Bio, Inc. Trans-splicing molecules
WO2019213525A1 (en) 2018-05-04 2019-11-07 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
AU2019287635A1 (en) * 2018-06-14 2020-12-17 Ionis Pharmaceuticals, Inc. Compounds and methods for increasing STMN2 expression
US11279929B2 (en) 2018-07-03 2022-03-22 Hoffmann-La Roche, Inc. Oligonucleotides for modulating Tau expression
JP2022523065A (ja) * 2019-01-25 2022-04-21 ナヤン セラピューティクス， インク． Ｎｒ２ｅ３発現抑制オリゴヌクレオチド、それを含む組成物、及びそれらの使用方法
JP2022543474A (ja) * 2019-08-08 2022-10-12 ユーシーエル ビジネス リミテッド アンチセンスオリゴヌクレオチドは、ａｂｃａ４の異常スプライシングをレスキューする
JP2022544702A (ja) * 2019-08-19 2022-10-20 ストーク セラピューティクス，インク． スプライシングおよびタンパク質発現を調節するための組成物および方法
AU2021265838A1 (en) * 2020-04-29 2022-10-20 Saliogen Therapeutics, Inc. Compositions and methods for treatment of inherited macular degeneration
KR20230022409A (ko) 2020-05-11 2023-02-15 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 opa1 안티센스 올리고머
JP2023553710A (ja) 2020-12-18 2023-12-25 エフ． ホフマン－ラ ロシュ アーゲー プログラニュリンを標的とするためのアンチセンスオリゴヌクレオチド
CN118434858A (zh) * 2021-12-07 2024-08-02 豪夫迈·罗氏有限公司 靶向actl6b的反义寡核苷酸
JP2025506826A (ja) * 2022-02-25 2025-03-13 北京中因科技有限公司 発現カセット組み合わせ及びその使用
CA3252043A1 (en) 2022-05-13 2023-11-16 Ascidian Therapeutics, Inc. ABCA4 TRANS-SPLICING MOLECULES
EP4619047A1 (en) * 2022-11-17 2025-09-24 Skip Therapeutics Ltd. Compositions and methods for treating retinal diseases
WO2024223696A1 (en) 2023-04-24 2024-10-31 Universiteit Gent Compounds to treat inherited retinal disease
WO2025171404A1 (en) * 2024-02-09 2025-08-14 Utr Therapeutics Inc. Destabilized are 3' utrs of tead1 and yap1 as pan cancer therapeutics

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RU2597972C2 (ru) * 2010-10-22 2016-09-20 Курна Инк. Лечение заболеваний, связанных с геном альфа-l-идуронидазы (idua), путем ингибирования природного антисмыслового транскрипта гена idua
WO2014121287A2 (en) * 2013-02-04 2014-08-07 Isis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
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
EP3201339A4 (en) * 2014-10-03 2018-09-19 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output

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