JPWO2021034985A5 - - Google Patents

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Publication number
JPWO2021034985A5
JPWO2021034985A5 JP2022511203A JP2022511203A JPWO2021034985A5 JP WO2021034985 A5 JPWO2021034985 A5 JP WO2021034985A5 JP 2022511203 A JP2022511203 A JP 2022511203A JP 2022511203 A JP2022511203 A JP 2022511203A JP WO2021034985 A5 JPWO2021034985 A5 JP WO2021034985A5
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JP
Japan
Prior art keywords
fold
premrna
composition
target protein
exon
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.)
Pending
Application number
JP2022511203A
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English (en)
Japanese (ja)
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JP2022544702A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/047081 external-priority patent/WO2021034985A1/en
Publication of JP2022544702A publication Critical patent/JP2022544702A/ja
Publication of JPWO2021034985A5 publication Critical patent/JPWO2021034985A5/ja
Pending legal-status Critical Current

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JP2022511203A 2019-08-19 2020-08-19 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 Pending JP2022544702A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962888887P 2019-08-19 2019-08-19
US62/888,887 2019-08-19
US202063049262P 2020-07-08 2020-07-08
US63/049,262 2020-07-08
PCT/US2020/047081 WO2021034985A1 (en) 2019-08-19 2020-08-19 Compositions and methods for modulating splicing and protein expression

Publications (2)

Publication Number Publication Date
JP2022544702A JP2022544702A (ja) 2022-10-20
JPWO2021034985A5 true JPWO2021034985A5 (enrdf_load_stackoverflow) 2023-08-28

Family

ID=74659533

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022511203A Pending JP2022544702A (ja) 2019-08-19 2020-08-19 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

Country Status (11)

Country Link
US (1) US20220290142A1 (enrdf_load_stackoverflow)
EP (1) EP4017979A4 (enrdf_load_stackoverflow)
JP (1) JP2022544702A (enrdf_load_stackoverflow)
KR (1) KR20220104677A (enrdf_load_stackoverflow)
CN (1) CN114746550A (enrdf_load_stackoverflow)
AU (1) AU2020334067A1 (enrdf_load_stackoverflow)
BR (1) BR112022002905A2 (enrdf_load_stackoverflow)
CA (1) CA3147970A1 (enrdf_load_stackoverflow)
IL (1) IL290595A (enrdf_load_stackoverflow)
MX (1) MX2022002198A (enrdf_load_stackoverflow)
WO (1) WO2021034985A1 (enrdf_load_stackoverflow)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7652713B2 (ja) * 2019-05-27 2025-03-27 ビジョン ファーマ ピーティーワイ リミテッド 新規の網膜色素変性処置
CA3153071A1 (en) * 2019-09-03 2021-03-11 The Regents Of The University Of Colorado A Body Corporate Systems, methods, and compositions for the rapid early-detection of host rna biomarkers of infection and early identification of covid-19 coronavirus infection in humans
BR112022022893A2 (pt) * 2020-05-11 2023-03-14 The Florey Inst Of Neuroscience And Mental Health Composições e métodos para tratamento de distúrbios associados a mutações de perda de função em syngap1
EP4359538A2 (en) * 2021-06-21 2024-05-01 Stoke Therapeutics, Inc. Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases
EP4373845A4 (en) * 2021-07-23 2025-06-25 The Children's Medical Center Corporation Zinc finger ccch-type containing 14 (zc3h14) mutants and methods of use
US20230265436A1 (en) * 2022-02-24 2023-08-24 Q-State Biosciences, Inc. Therapeutics for syngap haploinsufficiency
WO2023196841A2 (en) * 2022-04-05 2023-10-12 The Johns Hopkins University Methods and materials for treating syngap1-associated neurodevelopmental disorders
JP2025514346A (ja) * 2022-04-28 2025-05-02 アキュラステム インコーポレイテッド Syf2アンチセンスオリゴヌクレオチド
WO2023220727A1 (en) * 2022-05-13 2023-11-16 The Trustees Of The University Of Pennsylvania Compositions for treating syngap-1 related neurodevelopmental disorders
JP2025522625A (ja) * 2022-06-28 2025-07-15 エージェンシー フォー サイエンス,テクノロジー アンド リサーチ オリゴヌクレオチド
WO2025090628A1 (en) * 2023-10-23 2025-05-01 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising small nuclear rna (snrna) for treating genetic epilepsies
GB202401412D0 (en) * 2024-02-02 2024-03-20 Harness Therapeutics Ltd Functional nucleic acid

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8637478B2 (en) * 2007-11-13 2014-01-28 Isis Pharmaceuticals, Inc. Compounds and methods for modulating protein expression
WO2010051632A1 (en) * 2008-11-07 2010-05-14 Centre Hospitalier Universitaire Sainte-Justine Syngap1 dysfunctions and uses thereof in diagnostic and therapeutic applications for mental retardation
AU2015327836B2 (en) * 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
AU2016334804B2 (en) * 2015-10-09 2022-03-31 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
EP3390666A4 (en) * 2015-12-14 2019-08-07 Cold Spring Harbor Laboratory COMPOSITIONS AND METHODS OF TREATING NURSE DISEASES
EP3933041B1 (en) * 2015-12-14 2024-01-31 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant retardation
EP3390634A4 (en) * 2015-12-14 2019-08-14 Cold Spring Harbor Laboratory COMPOSITIONS AND METHODS FOR THE TREATMENT OF EYE DISEASES
CA3005090A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of liver diseases
JP2018538287A (ja) * 2015-12-14 2018-12-27 コールド スプリング ハーバー ラボラトリー 多発性嚢胞腎の処置のためのアンチセンスオリゴマー
EP3390635A4 (en) * 2015-12-14 2019-05-01 Cold Spring Harbor Laboratory Antisense oligomers for the treatment of a tuberous sclerosis complex
EA201991309A1 (ru) * 2016-11-28 2019-11-29 Способы модуляции сплайсинга рнк
CA3073515A1 (en) * 2017-08-25 2019-02-28 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
SMT202500127T1 (it) * 2017-10-23 2025-05-12 Stoke Therapeutics Inc Oligomeri antisenso per il trattamento di malattie e condizioni basate sul decadimento dell’rna mediato da un nonsenso

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