CN116368227A - 用于治疗与scn2a中功能失去突变相关的障碍的组合物和方法 - Google Patents

用于治疗与scn2a中功能失去突变相关的障碍的组合物和方法 Download PDF

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CN116368227A
CN116368227A CN202180064122.5A CN202180064122A CN116368227A CN 116368227 A CN116368227 A CN 116368227A CN 202180064122 A CN202180064122 A CN 202180064122A CN 116368227 A CN116368227 A CN 116368227A
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antisense oligonucleotide
intron
scn2a
sequence
mrna
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CN202180064122.5A
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Chinese (zh)
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S·彼得鲁
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Praxis Precision Pharmaceuticals
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Florey Institute of Neuroscience and Mental Health
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Priority claimed from AU2020902550A external-priority patent/AU2020902550A0/en
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Publication of CN116368227A publication Critical patent/CN116368227A/zh
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
CN202180064122.5A 2020-07-22 2021-07-22 用于治疗与scn2a中功能失去突变相关的障碍的组合物和方法 Pending CN116368227A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
AU2020902550A AU2020902550A0 (en) 2020-07-22 Compositions and methods for treating disorders associated with loss-of-function mutations in SCN2A
AU2020902550 2020-07-22
PCT/AU2021/050788 WO2022016222A1 (en) 2020-07-22 2021-07-22 Compositions and methods for treating disorders associated with loss-of-function mutations in scn2a

Publications (1)

Publication Number Publication Date
CN116368227A true CN116368227A (zh) 2023-06-30

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CN202180064122.5A Pending CN116368227A (zh) 2020-07-22 2021-07-22 用于治疗与scn2a中功能失去突变相关的障碍的组合物和方法

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US (1) US20230272387A1 (enExample)
EP (1) EP4185697A4 (enExample)
JP (1) JP2023534720A (enExample)
KR (1) KR20230095056A (enExample)
CN (1) CN116368227A (enExample)
AU (1) AU2021311137A1 (enExample)
BR (1) BR112023000988A2 (enExample)
CA (1) CA3186629A1 (enExample)
CL (1) CL2023000208A1 (enExample)
CO (1) CO2023001922A2 (enExample)
EC (1) ECSP23012641A (enExample)
IL (1) IL299999A (enExample)
MX (1) MX2023000907A (enExample)
PE (1) PE20230982A1 (enExample)
WO (1) WO2022016222A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4473113A4 (en) * 2022-02-04 2026-03-25 Praxis Prec Medicines Inc TREATMENT METHODS FOR DISORDERS RELATED TO SCN2A
KR20250180156A (ko) 2024-06-21 2025-12-31 중앙대학교 산학협력단 뇌전증의 예방 또는 치료용 조성물, 및 뇌전증 치료제 스크리닝 방법과 이의 조성물

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE1252330T1 (de) * 1999-11-26 2003-11-27 Mcgill University, Montreal Loci der idiopathischen epilepsie, mutationen derselben und verfahren zu deren verwendung zur feststellung, prognose und behandlung von epilepsie
EP1268856A2 (de) * 2000-04-07 2003-01-02 Epigenomics AG Detektion von snp's und cytosin-methylierungen
AU2001276919A1 (en) * 2000-07-13 2002-01-30 Genaissance Pharmaceuticals, Inc. Haplotypes of the mmp13 gene
CA2541836A1 (en) * 2003-10-13 2005-04-21 Bionomics Limited A diagnostic method for neonatal or infantile epilepsy syndromes
CN101148665A (zh) * 2005-12-05 2008-03-26 广州医学院第二附属医院 钠离子通道scn1a基因突变及基因突变检测方法和用途
WO2010151671A2 (en) * 2009-06-24 2010-12-29 Curna, Inc. Treatment of tumor necrosis factor receptor 2 (tnfr2) related diseases by inhibition of natural antisense transcript to tnfr2
EP2490699A1 (en) * 2009-10-20 2012-08-29 Santaris Pharma A/S Oral delivery of therapeutically effective lna oligonucleotides
US20150141320A1 (en) * 2012-05-16 2015-05-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
CN109843914B (zh) * 2016-07-06 2024-03-15 沃泰克斯药物股份有限公司 用于治疗疼痛相关病症的材料和方法
KR20190030833A (ko) * 2017-09-15 2019-03-25 가톨릭대학교 산학협력단 신장암 환자의 예후 진단 및 치료 전략 결정용 연령 특이적 마커
HUE070436T2 (hu) * 2017-10-23 2025-06-28 Stoke Therapeutics Inc Antiszensz oligomerek nonszensz-mediált RNS-lebomláson alapuló állapotok és betegségek kezelésére
US11713463B2 (en) * 2018-01-17 2023-08-01 The Florey Institute Of Neuroscience And Mental Health Compositions and methods for increasing expression of SCN2A
BR112021003224A2 (pt) * 2018-08-20 2021-07-20 Rogcon, Inc. oligonucleotídeos antissentido direcionados a scn2a para o tratamento de encefalopatias por scn1a
US20220090087A1 (en) * 2019-01-23 2022-03-24 The Florey Institute Of Neuroscience And Mental Health Antisense oligonucleotides targeting scn2a retained introns

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CA3186629A1 (en) 2022-01-27
BR112023000988A2 (pt) 2023-03-28
EP4185697A1 (en) 2023-05-31
JP2023534720A (ja) 2023-08-10
ECSP23012641A (es) 2023-03-31
CO2023001922A2 (es) 2023-06-09
WO2022016222A1 (en) 2022-01-27
EP4185697A4 (en) 2025-01-01
PE20230982A1 (es) 2023-06-21
MX2023000907A (es) 2023-04-27
IL299999A (en) 2023-03-01
KR20230095056A (ko) 2023-06-28
CL2023000208A1 (es) 2023-07-21
US20230272387A1 (en) 2023-08-31
AU2021311137A1 (en) 2023-03-23

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