JP2018538287A5 - - Google Patents

Download PDF

Info

Publication number
JP2018538287A5
JP2018538287A5 JP2018529219A JP2018529219A JP2018538287A5 JP 2018538287 A5 JP2018538287 A5 JP 2018538287A5 JP 2018529219 A JP2018529219 A JP 2018529219A JP 2018529219 A JP2018529219 A JP 2018529219A JP 2018538287 A5 JP2018538287 A5 JP 2018538287A5
Authority
JP
Japan
Prior art keywords
mrna
protein
item
pharmaceutical composition
composition according
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.)
Withdrawn
Application number
JP2018529219A
Other languages
English (en)
Japanese (ja)
Other versions
JP2018538287A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2016/066417 external-priority patent/WO2017106211A1/en
Publication of JP2018538287A publication Critical patent/JP2018538287A/ja
Publication of JP2018538287A5 publication Critical patent/JP2018538287A5/ja
Priority to JP2022000102A priority Critical patent/JP2022046723A/ja
Withdrawn legal-status Critical Current

Links

JP2018529219A 2015-12-14 2016-12-13 多発性嚢胞腎の処置のためのアンチセンスオリゴマー Withdrawn JP2018538287A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022000102A JP2022046723A (ja) 2015-12-14 2022-01-04 多発性嚢胞腎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
US201562267252P 2015-12-14 2015-12-14
US62/267,252 2015-12-14
PCT/US2016/066417 WO2017106211A1 (en) 2015-12-14 2016-12-13 Antisense oligomers for treatment of polycystic kidney disease

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022000102A Division JP2022046723A (ja) 2015-12-14 2022-01-04 多発性嚢胞腎の処置のためのアンチセンスオリゴマー

Publications (2)

Publication Number Publication Date
JP2018538287A JP2018538287A (ja) 2018-12-27
JP2018538287A5 true JP2018538287A5 (enExample) 2020-01-30

Family

ID=59057523

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2018529219A Withdrawn JP2018538287A (ja) 2015-12-14 2016-12-13 多発性嚢胞腎の処置のためのアンチセンスオリゴマー
JP2022000102A Pending JP2022046723A (ja) 2015-12-14 2022-01-04 多発性嚢胞腎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
JP2022000102A Pending JP2022046723A (ja) 2015-12-14 2022-01-04 多発性嚢胞腎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) EP3389782A4 (enExample)
JP (2) JP2018538287A (enExample)
CA (1) CA3005247A1 (enExample)
WO (1) WO2017106211A1 (enExample)

Families Citing this family (12)

* 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
EP3673080B1 (en) 2017-08-25 2023-10-18 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
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
BR112023015636A2 (pt) * 2021-02-03 2024-01-02 Stoke Therapeutics Inc Composições para tratamento de condições e doenças associadas à expressão de policistina
AU2024304872A1 (en) * 2023-06-16 2025-12-18 PYC Therapeutics Limited Compositions and methods for treatment of kidney disease

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040102402A1 (en) * 2002-11-22 2004-05-27 Isis Pharmaceuticals Inc. Modulation of tissue factor expression
US8007790B2 (en) * 2006-04-03 2011-08-30 Stowers Institute For Medical Research Methods for treating polycystic kidney disease (PKD) or other cyst forming diseases
US8771946B2 (en) * 2006-07-24 2014-07-08 Athena Diagnostics, Inc. PKD mutations and evaluation of same
US20150141320A1 (en) * 2012-05-16 2015-05-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
DK3041958T3 (da) * 2013-09-04 2020-03-09 Cold Spring Harbor Laboratory Reducering af nonsense-medieret mrna-degradering
AU2015327836B2 (en) * 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output

Similar Documents

Publication Publication Date Title
JP2018538287A5 (enExample)
JP2018538288A5 (enExample)
JP2019501892A5 (enExample)
JP2019500347A5 (enExample)
JP2019500350A5 (enExample)
JP2022062140A5 (enExample)
JP7420679B2 (ja) 状態および疾患の処置のためのアンチセンスオリゴマー
JP2019500346A5 (enExample)
JP2019500349A5 (enExample)
JP2017536338A5 (enExample)
EP2516647B1 (en) Molecule for treating an inflammatory disorder
JP2019500345A5 (enExample)
KR20080031164A (ko) ＳＲ 단백질의 결합의 방해에 의해 그리고 이차 ＲＮＡ구조의 방해에 의한 ｐｒｅ－ｍＲＮＡ에서 엑손 인식의조절
US9353169B2 (en) Means and methods for modulating NOTCH3 protein expression and/or the coding region of NOTCH3; compositions and use thereof in the treatment of CADASIL
CA3140045A1 (en) Splice modulating oligonucleotides targeting receptor for advanced glycation end products and methods of use
JPWO2019181946A1 (ja) 毒性が軽減した核酸
US20200407721A1 (en) Compounds of chemically modified oligonucleotides and methods of use thereof
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
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
JPWO2019213525A5 (enExample)
EP3746555B1 (en) Inhibition of a lncrna for treatment of neuroblastoma
KR20250122542A (ko) 넌센스 매개 rna 분해 기반 병태 및 질환의 치료를 위한 안티센스 올리고머
JP2025533501A (ja) ポンペ病マウスモデルの生成、特徴決定および使用方法
JPWO2022169947A5 (enExample)
CN118853661A (zh) 编辑rna的方法、组合物及其应用