PL2320952T3 - ULEPSZONE CZĄSTECZKI CZĄSTECZKI TRANS-SPLICINGUJĄCEJ PRE-mRNA (RTM) I ICH ZASTOSOWANIE - Google Patents

ULEPSZONE CZĄSTECZKI CZĄSTECZKI TRANS-SPLICINGUJĄCEJ PRE-mRNA (RTM) I ICH ZASTOSOWANIE

Info

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
Application number
PL09802455.7T
Other languages
English (en)
Inventor
Lloyd G. Mitchell
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed filed Critical
Publication of PL2320952T3 publication Critical patent/PL2320952T3/pl

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    • 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)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Zoology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Dermatology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Transplantation (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)
PL09802455.7T 2008-07-30 2009-07-30 ULEPSZONE CZĄSTECZKI CZĄSTECZKI TRANS-SPLICINGUJĄCEJ PRE-mRNA (RTM) I ICH ZASTOSOWANIE PL2320952T3 (pl)

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 (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011042556A1 (en) * 2009-10-08 2011-04-14 Association Institut De Myologie Nucleic acid molecules and methods for exchanging exon(s) by transsplicing
CN102906535B (zh) * 2011-05-02 2016-01-20 福斯分析股份公司 光谱仪
CA2844930A1 (en) * 2011-08-12 2013-02-21 Virxsys Corporation Compositions and methods for inducing apoptosis
NZ703824A (en) 2012-07-12 2018-06-29 Proqr Therapeutics Ii Bv Oligonucleotides for making a change in the sequence of a target rna molecule present in a living cell
WO2014011050A1 (en) * 2012-07-12 2014-01-16 Proqr Therapeutics B.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
DK3690056T5 (da) 2014-01-31 2024-08-26 Factor Bioscience Inc Fremgangsmåder og produkter til fremstilling og indgivelse af nukleinsyre
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
CN107109411B (zh) 2014-10-03 2022-07-01 冷泉港实验室 核基因输出的定向增加
AU2016218977C1 (en) 2015-02-13 2023-03-23 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
IL308174A (en) 2015-10-09 2024-01-01 Univ Southampton Gene expression modulation and dysregulated protein expression scanning
AU2016355343B2 (en) * 2015-11-19 2023-12-14 Lloyd G. Mitchell Compositions and methods for correction of heritable ocular disease
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
EP3390636B1 (en) 2015-12-14 2021-05-19 Cold Spring Harbor Laboratory Antisense oligomers for treatment of dravet syndrome
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 DIAGNOSIS OF DISEASES ASSOCIATED WITH THE COL6 GENE AND RELATED METHODS OF TREATMENT
WO2018035377A1 (en) 2016-08-17 2018-02-22 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
GB2599884B (en) 2017-08-25 2022-08-31 Stoke Therapeutics Inc Antisense oligomers for treatment of conditions and diseases
IT201700104587A1 (it) * 2017-09-19 2019-03-19 Holostem Terapie Avanzate S R L Usi terapeutici di lembi di cellule geneticamente modificate
MX2020010959A (es) 2018-04-17 2021-01-15 Univ Pennsylvania Moleculas de empalme trans.
JP2021523227A (ja) 2018-05-04 2021-09-02 ストーク セラピューティクス，インク． コレステリルエステル蓄積症の処置のための方法及び組成物
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
KR20230022409A (ko) 2020-05-11 2023-02-15 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 opa1 안티센스 올리고머
WO2024068898A1 (en) 2022-09-30 2024-04-04 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
WO2024118946A1 (en) * 2022-11-30 2024-06-06 Amber Bio Inc. Methods and compositions for targeted trans-splicing

Family Cites Families (16)

* Cited by examiner, † Cited by third party
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
DK0725788T3 (da) 1993-10-27 1999-08-23 Ribozyme Pharm Inc 2'-amido- og 2'-peptidomodificerede oligonukleotider
US6083702A (en) 1995-12-15 2000-07-04 Intronn Holdings Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
US20030077754A1 (en) 1995-12-15 2003-04-24 Mitchell Lloyd G. Methods and compositions for use in spliceosome mediated RNA trans-splicing
JP4321877B2 (ja) * 1995-12-15 2009-08-26 バークシス コーポレイション トランス―スプライスにより生成される治療用分子
US20030027250A1 (en) 1995-12-15 2003-02-06 Mitchell Lloyd G. Methods and compositions for use in spliceosome mediated RNA trans-splicing
US6280978B1 (en) * 1995-12-15 2001-08-28 Intronn Holdings, Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
US20060088938A1 (en) 1995-12-15 2006-04-27 Mitchell Lloyd G Methods and compositions for use in spliceosome mediated RNA trans-splicing in plants
US20020115207A1 (en) 1995-12-15 2002-08-22 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
WO1998037340A1 (en) 1997-02-20 1998-08-27 Sram Corporation Discontinuous mechanical advantage front shifting for bicycles
WO2003069311A2 (en) 2002-02-12 2003-08-21 Intronn, Inc. 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
WO2003104416A2 (en) * 2002-06-05 2003-12-18 Intronn, Inc. 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

Also Published As

Publication number Publication date
ES2579477T3 (es) 2016-08-11
EP2320952A1 (en) 2011-05-18
JP2011529333A (ja) 2011-12-08
WO2010012472A1 (en) 2010-02-04
US8735366B2 (en) 2014-05-27
EP2151248A1 (en) 2010-02-10
US20130059901A1 (en) 2013-03-07
JP5735912B2 (ja) 2015-06-17
EP2320952B1 (en) 2016-05-04

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