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
Anticipated expiration legal-status Critical

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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 (https=) 2020-01-30
JP7036723B2 JP7036723B2 (ja) 2022-03-15

Family

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)

Application Number Title Priority Date Filing Date
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)

Country Link
EP (1) EP3390634A4 (https=)
JP (2) JP7036723B2 (https=)
CA (1) CA3005128A1 (https=)
WO (1) WO2017106370A1 (https=)

Families Citing this family (31)

* 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
EP3359685B1 (en) 2015-10-09 2026-01-28 University Of Southampton Modulation of gene expression for deregulated protein expression
EP3933041B1 (en) 2015-12-14 2024-01-31 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant retardation
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
IL314915B1 (en) 2017-01-23 2026-02-01 Regeneron Pharma Variants of 17-beta-hydroxysteroid dehydrogenase and their uses
KR20190139869A (ko) 2017-04-11 2019-12-18 리제너론 파마슈티칼스 인코포레이티드 하이드록시스테로이드 (17-베타) 탈수소효소(hsd17b) 패밀리의 구성원의 조절인자의 활성도를 스크리닝하기 위한 검정
GB201706009D0 (en) * 2017-04-13 2017-05-31 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of stargardt disease
SG11202001590RA (en) 2017-08-25 2020-03-30 Stoke Therapeutics Inc Antisense oligomers for treatment of conditions and diseases
US10961583B2 (en) 2017-10-11 2021-03-30 Regeneron Phramaceuticals, Inc. Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation
GB2610100B (en) * 2017-10-23 2023-08-16 Stoke Therapeutics Inc Antisense oligomers for treatment of non-sense mediated RNA decay based conditions and diseases
RU177889U1 (ru) * 2017-11-30 2018-03-15 Общество с ограниченной ответственностью "Вертикаль-М" (ООО "Вертикаль-М") Дренаж для хирургического лечения глаукомы
JP7507093B2 (ja) 2018-03-21 2024-06-27 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 １７β－水酸化ステロイド脱水素酵素１３型（ＨＳＤ１７Ｂ１３）ｉＲＮＡ組成物およびその使用方法
CA3097004A1 (en) * 2018-04-17 2019-10-24 The Trustees Of The University Of Pennsylvania Trans-splicing molecules
US12060558B2 (en) 2018-05-04 2024-08-13 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
CA3103429A1 (en) * 2018-06-14 2019-12-19 Don W. Cleveland Compounds and methods for increasing stmn2 expression
SG11202012759XA (en) 2018-07-03 2021-01-28 Hoffmann La Roche Oligonucleotides for modulating tau expression
WO2020154686A1 (en) * 2019-01-25 2020-07-30 Nayan Therapeutics, Inc. Nr2e3 expression reducing oligonucleotides, compositions containing the same, and methods of their use
KR20210134003A (ko) 2019-02-27 2021-11-08 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 안티센스 올리고머
WO2021023863A1 (en) * 2019-08-08 2021-02-11 Stichting Katholieke Universiteit Antisense oligonucleotides rescue aberrant splicing of abca4
EP4017979A4 (en) * 2019-08-19 2024-03-27 Stoke Therapeutics, Inc. COMPOSITIONS AND METHODS FOR MODULATING PROTEIN SPLICING AND EXPRESSION
WO2021222654A1 (en) * 2020-04-29 2021-11-04 Saliogen Therapeutics, Inc. Compositions and methods for treatment of inherited macular degeneration
BR112022022889A2 (pt) 2020-05-11 2023-04-04 Stoke Therapeutics Inc Oligômeros antissentido de opa1 para tratamento de condições e doenças
TW202229553A (zh) 2020-12-18 2022-08-01 瑞士商赫孚孟拉羅股份公司 靶向顆粒蛋白前體之反義寡核苷酸
EP4444882A1 (en) * 2021-12-07 2024-10-16 F. Hoffmann-La Roche AG Antisense oligonucleotides targeting actl6b
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
AU2023269865A1 (en) 2022-05-13 2024-11-21 Ascidian Therapeutics, Inc. Abca4 trans-splicing molecules
CN120225227A (zh) * 2022-11-17 2025-06-27 斯基普治疗有限公司 用于治疗视网膜疾病的组合物和方法
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
WO2025238644A1 (en) * 2024-05-16 2025-11-20 Skip Therapeutics Ltd. Compositions and methods for treating retinal diseases

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9222088B2 (en) * 2010-10-22 2015-12-29 Curna, Inc. Treatment of alpha-L-iduronidase (IDUA) related diseases by inhibition of natural antisense transcript to IDUA
ES2817050T3 (es) * 2013-02-04 2021-04-06 Ionis Pharmaceuticals Inc Compuestos antisentido selectivos y usos de los mismos
PL3041958T4 (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

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