JP7036723B2 - 眼疾患の処置のための組成物および方法 - Google Patents

眼疾患の処置のための組成物および方法 Download PDF

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JP7036723B2
JP7036723B2 JP2018529249A JP2018529249A JP7036723B2 JP 7036723 B2 JP7036723 B2 JP 7036723B2 JP 2018529249 A JP2018529249 A JP 2018529249A JP 2018529249 A JP2018529249 A JP 2018529249A JP 7036723 B2 JP7036723 B2 JP 7036723B2
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JP2019500348A (ja
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アズナレズ,イザベル
エム. ナッシュ,ヒュー
アゼアリアン,サッサン
ダブリュ. ホール,サミュエル
クライナー,エイドリアン
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コールド スプリング ハーバー ラボラトリー
ストーク セラピューティクス,インク.
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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)

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US201562267259P 2015-12-14 2015-12-14
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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

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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
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
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
RU2019126483A (ru) 2017-01-23 2021-02-24 Ридженерон Фармасьютикалз, Инк. Варианты 17-бета-гидроксистероиддегидрогеназы 13 (hsd17b13) и их применение
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
KR102318434B1 (ko) 2017-08-25 2021-11-01 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
HUE070436T2 (hu) * 2017-10-23 2025-06-28 Stoke Therapeutics Inc Antiszensz oligomerek nonszensz-mediált RNS-lebomláson alapuló állapotok és betegségek kezelésére
RU177889U1 (ru) * 2017-11-30 2018-03-15 Общество с ограниченной ответственностью "Вертикаль-М" (ООО "Вертикаль-М") Дренаж для хирургического лечения глаукомы
AU2019239971B2 (en) 2018-03-21 2025-09-11 Regeneron Pharmaceuticals, Inc. 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof
KR102866133B1 (ko) 2018-04-17 2025-09-30 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 트랜스-스플라이싱 분자
CA3099280A1 (en) 2018-05-04 2019-11-07 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
PE20210346A1 (es) * 2018-07-03 2021-02-25 Hoffmann La Roche Oligonucleotidos para modular la expresion de tau
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
AU2020227825B2 (en) 2019-02-27 2026-03-26 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
US20220290154A1 (en) * 2019-08-08 2022-09-15 Stichting Radboud Universitair Medisch Centrum 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
AU2021265838A1 (en) * 2020-04-29 2022-10-20 Saliogen Therapeutics, Inc. Compositions and methods for treatment of inherited macular degeneration
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
AR124378A1 (es) 2020-12-18 2023-03-22 Hoffmann La Roche Oligonucleótidos antisentido dirigida a progranulina
WO2023104693A1 (en) * 2021-12-07 2023-06-15 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting actl6b
WO2023160454A1 (zh) * 2022-02-25 2023-08-31 北京中因科技有限公司 表达盒组合及其应用
EP4522758A4 (en) 2022-05-13 2026-05-06 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
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
KR101865433B1 (ko) * 2010-10-22 2018-07-13 큐알엔에이, 인크. 알파-l 이두로니다아제 (idua)에 대한 자연 안티센스 전사체의 저해에 의한 idua 관련된 질환의 치료
WO2014121287A2 (en) * 2013-02-04 2014-08-07 Isis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
EP3041958B1 (en) * 2013-09-04 2019-12-04 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay
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

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Journal of the Neurological Sciences,Vol.309,2011年,p.16-17
NATURE,2010年,Vol 465,p.223-227

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WO2017106370A1 (en) 2017-06-22
EP3390634A1 (en) 2018-10-24
CA3005128A1 (en) 2017-06-22
JP2019500348A (ja) 2019-01-10
EP3390634A4 (en) 2019-08-14

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