IL315378A - Preparations and methods involving small nuclear RNA (snRNA) - Google Patents
Preparations and methods involving small nuclear RNA (snRNA)Info
- Publication number
- IL315378A IL315378A IL315378A IL31537824A IL315378A IL 315378 A IL315378 A IL 315378A IL 315378 A IL315378 A IL 315378A IL 31537824 A IL31537824 A IL 31537824A IL 315378 A IL315378 A IL 315378A
- Authority
- IL
- Israel
- Prior art keywords
- snrna
- compositions
- methods
- small nuclear
- nuclear rna
- Prior art date
Links
- 108091029842 small nuclear ribonucleic acid Proteins 0.000 title 2
- 102000039471 Small Nuclear RNA Human genes 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Neurology (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurosurgery (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Virology (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Epidemiology (AREA)
- Psychology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202263316659P | 2022-03-04 | 2022-03-04 | |
| US202263379983P | 2022-10-18 | 2022-10-18 | |
| PCT/US2023/063798 WO2023168458A1 (en) | 2022-03-04 | 2023-03-06 | Compositions and methods comprising engineered small nuclear rna (snrna) |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL315378A true IL315378A (en) | 2024-11-01 |
Family
ID=85792399
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL315378A IL315378A (en) | 2022-03-04 | 2023-03-06 | Preparations and methods involving small nuclear RNA (snRNA) |
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)
| 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)
| 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) |
-
2023
- 2023-03-06 AU AU2023228712A patent/AU2023228712A1/en active Pending
- 2023-03-06 KR KR1020247032973A patent/KR20240164824A/ko active Pending
- 2023-03-06 CA CA3245423A patent/CA3245423A1/en active Pending
- 2023-03-06 IL IL315378A patent/IL315378A/en unknown
- 2023-03-06 WO PCT/US2023/063798 patent/WO2023168458A1/en not_active Ceased
- 2023-03-06 EP EP23714451.4A patent/EP4486888A1/en active Pending
- 2023-03-06 CN CN202380037529.8A patent/CN119137273A/zh active Pending
- 2023-03-06 JP JP2024552401A patent/JP2025507958A/ja active Pending
- 2023-03-06 US US18/179,110 patent/US20230279397A1/en active Pending
- 2023-08-10 US US18/447,651 patent/US12037588B2/en active Active
-
2024
- 2024-06-04 US US18/733,477 patent/US20250136981A1/en active Pending
- 2024-09-04 MX MX2024010833A patent/MX2024010833A/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EP4486888A1 (en) | 2025-01-08 |
| US12037588B2 (en) | 2024-07-16 |
| US20230279397A1 (en) | 2023-09-07 |
| US20250136981A1 (en) | 2025-05-01 |
| WO2023168458A1 (en) | 2023-09-07 |
| JP2025507958A (ja) | 2025-03-21 |
| US20240018521A1 (en) | 2024-01-18 |
| KR20240164824A (ko) | 2024-11-20 |
| CN119137273A (zh) | 2024-12-13 |
| CA3245423A1 (en) | 2023-09-07 |
| AU2023228712A1 (en) | 2024-10-17 |
| MX2024010833A (es) | 2024-12-06 |
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