PL2320952T3 - ULEPSZONE CZĄSTECZKI CZĄSTECZKI TRANS-SPLICINGUJĄCEJ PRE-mRNA (RTM) I ICH ZASTOSOWANIE - Google Patents
ULEPSZONE CZĄSTECZKI CZĄSTECZKI TRANS-SPLICINGUJĄCEJ PRE-mRNA (RTM) I ICH ZASTOSOWANIEInfo
- Publication number
- PL2320952T3 PL2320952T3 PL09802455.7T PL09802455T PL2320952T3 PL 2320952 T3 PL2320952 T3 PL 2320952T3 PL 09802455 T PL09802455 T PL 09802455T PL 2320952 T3 PL2320952 T3 PL 2320952T3
- Authority
- PL
- Poland
- Prior art keywords
- rtm
- molecules
- improved pre
- splicing molecule
- mrna trans
- Prior art date
Links
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
- C12N15/63—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
-
- 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
- A61P17/00—Drugs for dermatological disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P17/00—Drugs for dermatological disorders
- A61P17/02—Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P17/00—Drugs for dermatological disorders
- A61P17/06—Antipsoriatics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
- A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/78—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
- C12N2740/13041—Use of virus, viral particle or viral elements as a vector
- C12N2740/13043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
- C12N2840/445—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor for trans-splicing, e.g. polypyrimidine tract, branch point splicing
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Wood Science & Technology (AREA)
- Immunology (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Dermatology (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Toxicology (AREA)
- Transplantation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP20080013671 EP2151248A1 (en) | 2008-07-30 | 2008-07-30 | Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PL2320952T3 true PL2320952T3 (pl) | 2016-11-30 |
Family
ID=39797958
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL09802455.7T PL2320952T3 (pl) | 2008-07-30 | 2009-07-30 | ULEPSZONE CZĄSTECZKI CZĄSTECZKI TRANS-SPLICINGUJĄCEJ PRE-mRNA (RTM) I ICH ZASTOSOWANIE |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US8735366B2 (pl) |
| EP (2) | EP2151248A1 (pl) |
| JP (1) | JP5735912B2 (pl) |
| ES (1) | ES2579477T3 (pl) |
| PL (1) | PL2320952T3 (pl) |
| WO (1) | WO2010012472A1 (pl) |
Families Citing this family (39)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9303078B2 (en) | 2009-10-08 | 2016-04-05 | Association Institut De Myologie | Nucleic acid molecules and methods for exchanging exon(s) by transsplicing |
| KR101828100B1 (ko) * | 2011-05-02 | 2018-02-09 | 포스 애널리터컬 에이/에스 | 분광측정장치 |
| SG2014012207A (en) * | 2011-08-12 | 2014-05-29 | Virxsys Corp | Compositions and methods for inducing apoptosis |
| RS54944B1 (sr) | 2012-07-12 | 2016-11-30 | Proqr Therapeutics Ii Bv | Oligonukleotidi za pravljenje promena u sekvencama ciljanih rnk molekula prisutnih u živoj ćeliji |
| US20150209448A1 (en) * | 2012-07-12 | 2015-07-30 | Proqr Therapeutics N.V. | Exon replacement with stabilized artificial rnas |
| GB201219762D0 (en) | 2012-11-02 | 2012-12-19 | Bauer Johann | A RNA trans-splicing molecule (RTM) for use in the treatment of cancer |
| EP3099801B1 (en) | 2014-01-31 | 2020-03-18 | Factor Bioscience Inc. | Synthetic rna for use in the treatment of dystrophic epidermolysis bullosa |
| 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 |
| CA2976376A1 (en) | 2015-02-13 | 2016-08-18 | Factor Bioscience Inc. | Nucleic acid products and methods of administration thereof |
| GB201504124D0 (en) | 2015-03-11 | 2015-04-22 | Proqr Therapeutics B V | Oligonucleotides |
| GB201513151D0 (en) * | 2015-07-27 | 2015-09-09 | Univ Singapore | Functional gene replacement therapy |
| 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 |
| CA3005474A1 (en) | 2015-11-19 | 2017-05-26 | The Trustees Of The University Of Pennsylvania | Compositions and methods for correction of heritable ocular disease |
| WO2017106377A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| KR101926331B1 (ko) * | 2016-04-12 | 2018-12-07 | (주)안트로젠 | 수포성 표피박리증 완화 또는 개선용 중간엽줄기세포-하이드로겔-생분해성 또는 중간엽줄기세포-하이드로겔-비분해성 지지체 조성물 |
| EP3481432A4 (en) | 2016-07-05 | 2020-05-06 | The U.S.A. as represented by the Secretary, Department of Health and Human Services | Diagnosing col6-related disorders and methods for treating same |
| IL308824A (en) | 2016-08-17 | 2024-01-01 | Factor Bioscience Inc | Nucleic acid products and methods of administration thereof |
| IT201700094210A1 (it) * | 2017-08-17 | 2019-02-17 | Holostem Terapie Avanzate S R L | Metodo per produrre lembi cellulari |
| KR102318434B1 (ko) | 2017-08-25 | 2021-11-01 | 스톡 테라퓨틱스, 인크. | 병태 및 질환 치료용 안티센스 올리고머 |
| IT201700104587A1 (it) * | 2017-09-19 | 2019-03-19 | Holostem Terapie Avanzate S R L | Usi terapeutici di lembi di cellule geneticamente modificate |
| KR102866133B1 (ko) | 2018-04-17 | 2025-09-30 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 트랜스-스플라이싱 분자 |
| CA3099280A1 (en) | 2018-05-04 | 2019-11-07 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| AU2020227825B2 (en) | 2019-02-27 | 2026-03-26 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of conditions and diseases |
| BR112021020539A2 (pt) * | 2019-04-17 | 2022-01-04 | Univ Pennsylvania | Terminador de hélice tripla para trans-splicing de rna eficiente |
| CA3145425A1 (en) | 2019-07-03 | 2021-01-07 | Factor Bioscience Inc. | Cationic lipids and uses thereof |
| US10501404B1 (en) | 2019-07-30 | 2019-12-10 | Factor Bioscience Inc. | Cationic lipids and transfection methods |
| CN115023499A (zh) | 2019-08-16 | 2022-09-06 | 麻省理工学院 | 使用CRISPR/Cas13的靶向反式剪接 |
| CN115867657A (zh) | 2020-05-11 | 2023-03-28 | 斯托克制药公司 | 用于治疗疾患和疾病的opa1反义寡聚物 |
| WO2022220968A1 (en) * | 2021-04-15 | 2022-10-20 | U1 Bio, Inc. | High efficiency trans-splicing for replacement of targeted rna sequences in human cells |
| AU2022337146A1 (en) | 2021-09-03 | 2024-03-14 | Tacit Therapeutics, Inc. | Rna editing via recruitment of spliceosome components |
| EP4522758A4 (en) | 2022-05-13 | 2026-05-06 | Ascidian Therapeutics Inc | Abca4 trans-splicing molecules |
| EP4594505A1 (en) | 2022-09-30 | 2025-08-06 | Centre National de la Recherche Scientifique | Therapy by trans-splicing of opa1 pre-messenger rnas for the treatment of diseases associated with opa1 gene mutations |
| WO2024112957A1 (en) | 2022-11-23 | 2024-05-30 | Amber Bio Inc. | Gene-modifying endonucleases |
| CN120569476A (zh) * | 2022-11-30 | 2025-08-29 | 安铂生物公司 | 用于靶向反式剪接的方法和组合物 |
| EP4689118A1 (en) * | 2023-03-31 | 2026-02-11 | Duke University | Compositions for and methods of engineering the transcriptome |
| WO2025128343A1 (en) | 2023-12-11 | 2025-06-19 | Just-Evotec Biologics, Inc. | Protein expression using trans-splicing and split selectable markers |
| WO2026012439A1 (en) * | 2024-07-11 | 2026-01-15 | Sichuan Real & Best Biotech Co., Ltd. | Rna trans-splicing molecules and systems |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4902505A (en) | 1986-07-30 | 1990-02-20 | Alkermes | Chimeric peptides for neuropeptide delivery through the blood-brain barrier |
| US4904582A (en) | 1987-06-11 | 1990-02-27 | Synthetic Genetics | Novel amphiphilic nucleic acid conjugates |
| ATE174600T1 (de) | 1993-10-27 | 1999-01-15 | Ribozyme Pharm Inc | 2'-amido-und 2'-peptido-modifizierte oligonukleotide |
| US20060088938A1 (en) | 1995-12-15 | 2006-04-27 | Mitchell Lloyd G | Methods and compositions for use in spliceosome mediated RNA trans-splicing in plants |
| US6280978B1 (en) * | 1995-12-15 | 2001-08-28 | Intronn Holdings, Llc | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
| US20020115207A1 (en) | 1995-12-15 | 2002-08-22 | Mitchell Lloyd G. | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
| US6083702A (en) | 1995-12-15 | 2000-07-04 | Intronn Holdings Llc | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
| US20030027250A1 (en) * | 1995-12-15 | 2003-02-06 | Mitchell Lloyd G. | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
| ATE354957T1 (de) | 1995-12-15 | 2006-03-15 | Intronn Inc | Durch trans-spaltung erhaltene therapeutische molekule |
| US20030077754A1 (en) | 1995-12-15 | 2003-04-24 | Mitchell Lloyd G. | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
| GB9602326D0 (en) | 1996-02-06 | 1996-04-03 | Cruachem Ltd | Compounds |
| US6282976B1 (en) | 1997-02-20 | 2001-09-04 | Sram Corporation | Discontinuous mechanical advantage front shifting for bicycles |
| AU2003215249B2 (en) | 2002-02-12 | 2008-07-03 | Virxsys Corporation | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
| US7399753B2 (en) | 2002-02-25 | 2008-07-15 | Virxsys Corporation | Trans-splicing mediated photodynamic therapy |
| AU2003247505A1 (en) * | 2002-06-05 | 2003-12-22 | University Of Iowa Research Foundation | Spliceosome mediated rna trans-splicing in stem cells |
| US20040018622A1 (en) | 2002-07-17 | 2004-01-29 | Mitchell Lloyd G. | Spliceosome mediated RNA trans-splicing for correction of skin disorders |
-
2008
- 2008-07-30 EP EP20080013671 patent/EP2151248A1/en not_active Withdrawn
-
2009
- 2009-07-30 WO PCT/EP2009/005538 patent/WO2010012472A1/en not_active Ceased
- 2009-07-30 US US13/056,449 patent/US8735366B2/en active Active
- 2009-07-30 ES ES09802455.7T patent/ES2579477T3/es active Active
- 2009-07-30 EP EP09802455.7A patent/EP2320952B1/en active Active
- 2009-07-30 PL PL09802455.7T patent/PL2320952T3/pl unknown
- 2009-07-30 JP JP2011520382A patent/JP5735912B2/ja not_active Expired - Fee Related
Also Published As
| Publication number | Publication date |
|---|---|
| ES2579477T3 (es) | 2016-08-11 |
| US20130059901A1 (en) | 2013-03-07 |
| WO2010012472A1 (en) | 2010-02-04 |
| JP5735912B2 (ja) | 2015-06-17 |
| US8735366B2 (en) | 2014-05-27 |
| EP2320952A1 (en) | 2011-05-18 |
| EP2151248A1 (en) | 2010-02-10 |
| JP2011529333A (ja) | 2011-12-08 |
| EP2320952B1 (en) | 2016-05-04 |
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