JP2025507958A5 - - Google Patents

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Publication number
JP2025507958A5
JP2025507958A5 JP2024552401A JP2024552401A JP2025507958A5 JP 2025507958 A5 JP2025507958 A5 JP 2025507958A5 JP 2024552401 A JP2024552401 A JP 2024552401A JP 2024552401 A JP2024552401 A JP 2024552401A JP 2025507958 A5 JP2025507958 A5 JP 2025507958A5
Authority
JP
Japan
Prior art keywords
rna
nucleic acid
sequence
targeting
acid molecule
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
JP2024552401A
Other languages
English (en)
Japanese (ja)
Other versions
JP2025507958A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2023/063798 external-priority patent/WO2023168458A1/en
Publication of JP2025507958A publication Critical patent/JP2025507958A/ja
Publication of JP2025507958A5 publication Critical patent/JP2025507958A5/ja
Pending legal-status Critical Current

Links

JP2024552401A 2022-03-04 2023-03-06 操作された核内低分子RNA(snRNA)を含む組成物および方法 Pending JP2025507958A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202263316659P 2022-03-04 2022-03-04
US63/316,659 2022-03-04
US202263379983P 2022-10-18 2022-10-18
US63/379,983 2022-10-18
PCT/US2023/063798 WO2023168458A1 (en) 2022-03-04 2023-03-06 Compositions and methods comprising engineered small nuclear rna (snrna)

Publications (2)

Publication Number Publication Date
JP2025507958A JP2025507958A (ja) 2025-03-21
JP2025507958A5 true JP2025507958A5 (enExample) 2026-03-09

Family

ID=85792399

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024552401A Pending JP2025507958A (ja) 2022-03-04 2023-03-06 操作された核内低分子RNA(snRNA)を含む組成物および方法

Country Status (10)

Country Link
US (3) US20230279397A1 (enExample)
EP (1) EP4486888A1 (enExample)
JP (1) JP2025507958A (enExample)
KR (1) KR20240164824A (enExample)
CN (1) CN119137273A (enExample)
AU (1) AU2023228712A1 (enExample)
CA (1) CA3245423A1 (enExample)
IL (1) IL315378A (enExample)
MX (1) MX2024010833A (enExample)
WO (1) WO2023168458A1 (enExample)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2023228712A1 (en) 2022-03-04 2024-10-17 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising engineered small nuclear rna (snrna)
AU2023364059A1 (en) 2022-10-18 2025-05-08 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising programmable snrnas for rna editing
KR20250115422A (ko) 2022-12-01 2025-07-30 리제너론 파마슈티칼스 인코포레이티드 반복적 요소의 적절한 패키징을 위한 아데노 연관 바이러스 벡터
WO2025072530A2 (en) 2023-09-26 2025-04-03 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising small nuclear rna (snrna) for treating dmd
WO2025090633A1 (en) 2023-10-23 2025-05-01 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising small nuclear rna (snrna) for treating genetic epilepsies
WO2025126158A2 (en) 2023-12-15 2025-06-19 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising small nuclear rna (snrna) targeting sod1
WO2025160364A2 (en) 2024-01-26 2025-07-31 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising small nuclear rna (snrna) for the treatment of pompe disease

Family Cites Families (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IT1283876B1 (it) 1996-01-12 1998-05-07 Univ Roma Molecole chimeriche ribozima-snrna ad attivita' catalitica per rna a localizzazione nucleare
US5814500A (en) 1996-10-31 1998-09-29 The Johns Hopkins University School Of Medicine Delivery construct for antisense nucleic acids and methods of use
US6692910B2 (en) 1997-07-28 2004-02-17 The University Of Connecticut Inhibition of a target messenger RNA with a modified U1 small nuclear RNA
US7056502B2 (en) 2000-04-28 2006-06-06 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids
WO2002083908A1 (es) 2001-03-30 2002-10-24 Fundacion Para La Investigacion Medica Aplicada Procedimiento de inhibicion reversible de la expresion genica mediante ribonucleoproteinas modificadas
ITRM20020253A1 (it) 2002-05-08 2003-11-10 Univ Roma Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche.
FR2874384B1 (fr) 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
US8822168B2 (en) 2005-03-11 2014-09-02 The Trustees Of The University Of Pennsylvania Assays for detecting small nuclear ribonucleoprotein particle assembly and survival of motor neurons activity
US9040491B2 (en) 2006-08-30 2015-05-26 The Trustees Of The University Of Pennsylvania Compositions, methods and kits based on small nuclear RNAs
US8343941B2 (en) 2007-03-30 2013-01-01 Rutgers, The State University Of New Jersey Compositions and methods for gene silencing
US9441221B2 (en) 2007-03-30 2016-09-13 Rutgers, The State University Of New Jersey Compositions and methods for gene silencing
EP2142672B1 (en) 2007-03-30 2012-09-05 Rutgers, The State University of New Jersey Compositions and methods for gene silencing
US8907075B2 (en) 2007-03-30 2014-12-09 Samuel Ian Gunderson Compositions and methods for gene silencing
ES2714007T3 (es) 2007-04-09 2019-05-24 Univ Florida Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso
US9080170B2 (en) 2010-03-17 2015-07-14 Association Institut De Myologie Modified U7 snRNAs for treatment of neuromuscular diseases
IT1400425B1 (it) 2010-06-08 2013-05-31 Amsterdam Molecular Therapeutics Bv Modified snrnas for use in therapy.
IT1405793B1 (it) 2010-10-15 2014-01-24 Univ Ferrara Molecola di u1snrna umano modificato, gene codificante per la molecola di u1snrna umano modificato, vettore di espressione includente il gene, e loro uso in terapia genica
US9303258B2 (en) 2011-04-05 2016-04-05 The Regents Of The University Of California Method and compositions comprising small RNA agonist and antagonists to modulate inflammation
MX388221B (es) 2013-04-20 2025-03-19 Res Institute At Nationwide Children´S Hospital ADMINISTRACIÓN DE VIRUS ADENO-ASOCIADO RECOMBINANTE DE CONSTRUCCIONES DE POLINUCLÉOTIDOS U7snRNA DIRIGIDA AL EXÓN 2.
CA2957661A1 (en) 2014-08-09 2016-02-18 Kevin FLANIGAN Methods and materials for activating an internal ribosome entry site in exon 5 of the dmd gene
US9669109B1 (en) 2015-11-24 2017-06-06 Universita Di Ferrara Modified human U1snRNA molecule, a gene encoding for the modified human U1snRNA molecule, an expression vector including the gene, and the use thereof in gene therapy of familial dysautonomia and spinal muscular atrophy
FR3044926B1 (fr) 2015-12-09 2020-01-31 Genethon Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine
JP6966463B2 (ja) 2016-02-26 2021-11-17 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 組換えウイルス産物及びdux4エクソンスキッピングを誘導するための方法
JP2021534755A (ja) 2018-08-22 2021-12-16 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 筋強直性ジストロフィープロテインキナーゼの発現を抑制および/またはdmpk遺伝子の3’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法
CN113924364A (zh) 2019-03-08 2022-01-11 罗格斯新泽西州立大学 治疗亨廷顿病的组合物和方法
CA3134683A1 (en) 2019-03-26 2020-10-01 Rutgers, The State University Of New Jersey Compositions and methods for treating neurodegenerative disorders
WO2020214763A1 (en) * 2019-04-18 2020-10-22 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
US20220348958A1 (en) 2019-07-23 2022-11-03 Oxford Biomedica (Uk) Limited Enhancing Production of Lentiviral Vectors
WO2021026075A1 (en) 2019-08-02 2021-02-11 Research Institute At Nationwide Children's Hospital Exon 44-targeted nucleic acids and recombinant adeno-associated virus comprising said nucleic acids for treatment of dystrophin-based myopathies
US20230059753A1 (en) 2019-12-09 2023-02-23 Astellas Pharma Inc. Antisense guide rna with added functional region for editing target rna
CN115777020A (zh) * 2020-04-22 2023-03-10 塑造治疗公司 使用snrna组分的组合物和方法
AU2021349277A1 (en) 2020-09-28 2023-05-11 Research Institute At Nationwide Children's Hospital Products and methods for treating muscular dystrophy
IL303230A (en) 2020-11-30 2023-07-01 Res Inst Nationwide Childrens Hospital Compositions and methods for treating facioscapulohumeral muscular dystrophy (fshd)
AU2022262420A1 (en) 2021-04-23 2023-11-16 Research Institute At Nationwide Children's Hospital Products and methods for treating muscular dystrophy
AU2023228712A1 (en) 2022-03-04 2024-10-17 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising engineered small nuclear rna (snrna)

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