JP2019500346A5 - - Google Patents

Download PDF

Info

Publication number
JP2019500346A5
JP2019500346A5 JP2018529237A JP2018529237A JP2019500346A5 JP 2019500346 A5 JP2019500346 A5 JP 2019500346A5 JP 2018529237 A JP2018529237 A JP 2018529237A JP 2018529237 A JP2018529237 A JP 2018529237A JP 2019500346 A5 JP2019500346 A5 JP 2019500346A5
Authority
JP
Japan
Prior art keywords
mrna
peroxisome proliferator
activated receptor
seq
pharmaceutical composition
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
JP2018529237A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019500346A (ja
JP7049247B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2016/066576 external-priority patent/WO2017106292A1/en
Publication of JP2019500346A publication Critical patent/JP2019500346A/ja
Publication of JP2019500346A5 publication Critical patent/JP2019500346A5/ja
Priority to JP2022013876A priority Critical patent/JP2022062143A/ja
Application granted granted Critical
Publication of JP7049247B2 publication Critical patent/JP7049247B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2018529237A 2015-12-14 2016-12-14 腎臓病の処置のための組成物と方法 Expired - Fee Related JP7049247B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022013876A JP2022062143A (ja) 2015-12-14 2022-02-01 腎臓病の処置のための組成物と方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562267242P 2015-12-14 2015-12-14
US62/267,242 2015-12-14
PCT/US2016/066576 WO2017106292A1 (en) 2015-12-14 2016-12-14 Compositions and methods for treatment of kidney diseases

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022013876A Division JP2022062143A (ja) 2015-12-14 2022-02-01 腎臓病の処置のための組成物と方法

Publications (3)

Publication Number Publication Date
JP2019500346A JP2019500346A (ja) 2019-01-10
JP2019500346A5 true JP2019500346A5 (enExample) 2020-01-30
JP7049247B2 JP7049247B2 (ja) 2022-04-06

Family

ID=59057520

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2018529237A Expired - Fee Related JP7049247B2 (ja) 2015-12-14 2016-12-14 腎臓病の処置のための組成物と方法
JP2022013876A Pending JP2022062143A (ja) 2015-12-14 2022-02-01 腎臓病の処置のための組成物と方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2022013876A Pending JP2022062143A (ja) 2015-12-14 2022-02-01 腎臓病の処置のための組成物と方法

Country Status (4)

Country Link
EP (1) EP3390666A4 (enExample)
JP (2) JP7049247B2 (enExample)
CA (1) CA3005249A1 (enExample)
WO (1) WO2017106292A1 (enExample)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015187825A2 (en) 2014-06-03 2015-12-10 University Of Massachusetts Compositions and methods for modulating dysferlin expression
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
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
EP3390636B1 (en) 2015-12-14 2021-05-19 Cold Spring Harbor Laboratory Antisense oligomers for treatment of dravet syndrome
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
US12163129B2 (en) 2018-06-08 2024-12-10 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
CN112805299B (zh) 2018-10-05 2025-09-16 西雅图儿童医院以西雅图儿童研究机构名义经营 针对原发性免疫缺陷、胱氨酸病和威尔逊病的新生儿筛查
WO2020154462A1 (en) * 2019-01-23 2020-07-30 Rogcon U.R., Inc. Antisense oligonucleotides targeting scn2a retained introns
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
US11940448B2 (en) 2020-03-31 2024-03-26 Seattle Children's Hospital Proteomic screening for lysosomal storage diseases
US12523662B2 (en) 2020-04-02 2026-01-13 Seattle Children's Hospital Antibodies that specifically bind peptides associated with the primary immunodeficiencies: Wiskott-Aldrich syndrome and x-linked agammaglobulinemia
AU2021270720A1 (en) 2020-05-11 2022-12-08 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030224514A1 (en) * 2002-05-31 2003-12-04 Isis Pharmaceuticals Inc. Antisense modulation of PPAR-delta expression
WO2006055786A2 (en) * 2004-11-19 2006-05-26 Acadia Pharmaceuticals Inc. Methods to identify ligands of hormone nuclear receptors
US20110269735A1 (en) * 2010-04-19 2011-11-03 Celera Corporation Genetic polymorphisms associated with statin response and cardiovascular diseases, methods of detection and uses thereof
WO2012138487A2 (en) * 2011-04-07 2012-10-11 The Board Of Regents Of The University Of Texas System Oligonucleotide modulation of splicing
US9909128B2 (en) * 2012-11-15 2018-03-06 The Regents Of The University Of California Splice modulating oligonucleotides that inhibit cancer
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
JP2019500346A5 (enExample)
JP2019501892A5 (enExample)
JP2019500347A5 (enExample)
JP2019500349A5 (enExample)
JP2018538288A5 (enExample)
JP2018538287A5 (enExample)
JP2024056778A5 (enExample)
JP2019500350A5 (enExample)
US20230203483A1 (en) Compositions and Methods Using SNRNA Components
JP2017536338A5 (enExample)
JP2019500345A5 (enExample)
EP2027267B1 (en) Means and method for inducing exon-skipping
AU2017379073B2 (en) Compositions and methods for treating alpha-1 antitrypsin deficiency
CN107406852B (zh) 用于莱伯氏先天性黑蒙的寡核苷酸疗法
AU2010335039B2 (en) Molecule for treating an inflammatory disorder
KR20080031164A (ko) ＳＲ 단백질의 결합의 방해에 의해 그리고 이차 ＲＮＡ구조의 방해에 의한 ｐｒｅ－ｍＲＮＡ에서 엑손 인식의조절
CN113195721A (zh) 治疗α-1抗胰蛋白酶缺乏症的组合物和方法
EP3353301A1 (en) Antisense oligonucleotides and uses thereof
US20200407721A1 (en) Compounds of chemically modified oligonucleotides and methods of use thereof
JP7760130B2 (ja) Ａｐｏｃ３を標的としたアンチセンス核酸
US20250327093A1 (en) Viral vector and cancer cell proliferation inhibitor comprising the same
JPWO2019213525A5 (enExample)
EP3690046A2 (en) Composition for treatment of hemophilia, comprising crispr/cas system having coagulation factor viii gene inversion correction potential
CA2999192C (en) Antisense oligonucleotides hybridizing with a key element of the polyadenylation region of a dux4 pre-mrna and uses thereof
JPWO2022169947A5 (enExample)