IL278019A - trans splicing molecules - Google Patents

trans splicing molecules

Info

Publication number
IL278019A
IL278019A IL278019A IL27801920A IL278019A IL 278019 A IL278019 A IL 278019A IL 278019 A IL278019 A IL 278019A IL 27801920 A IL27801920 A IL 27801920A IL 278019 A IL278019 A IL 278019A
Authority
IL
Israel
Prior art keywords
trans
splicing molecules
splicing
molecules
Prior art date
Application number
IL278019A
Other languages
English (en)
Hebrew (he)
Original Assignee
Univ Pennsylvania
Ascidian Therapeutics Inc
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 by Univ Pennsylvania, Ascidian Therapeutics Inc filed Critical Univ Pennsylvania
Publication of IL278019A publication Critical patent/IL278019A/en

Links

Classifications

    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/71—Fusion polypeptide containing domain for protein-protein interaction containing domain for transcriptional activaation, e.g. VP16
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/85—Fusion polypeptide containing an RNA binding domain
    • 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
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
IL278019A 2018-04-17 2020-10-13 trans splicing molecules IL278019A (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862658658P 2018-04-17 2018-04-17
US201862658667P 2018-04-17 2018-04-17
PCT/US2019/027981 WO2019204514A1 (en) 2018-04-17 2019-04-17 Trans-splicing molecules

Publications (1)

Publication Number Publication Date
IL278019A true IL278019A (en) 2020-11-30

Family

ID=68239871

Family Applications (1)

Application Number Title Priority Date Filing Date
IL278019A IL278019A (en) 2018-04-17 2020-10-13 trans splicing molecules

Country Status (11)

Country Link
US (2) US11993776B2 (enExample)
EP (1) EP3781213A4 (enExample)
JP (2) JP7561624B2 (enExample)
KR (2) KR20250150157A (enExample)
CN (1) CN112449605A (enExample)
AU (1) AU2019255708A1 (enExample)
BR (1) BR112020021228A2 (enExample)
CA (1) CA3097004A1 (enExample)
IL (1) IL278019A (enExample)
MX (2) MX2020010959A (enExample)
WO (1) WO2019204514A1 (enExample)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3097004A1 (en) 2018-04-17 2019-10-24 The Trustees Of The University Of Pennsylvania Trans-splicing molecules
US20220204989A1 (en) * 2019-04-17 2022-06-30 The Trustees Of The University Of Pennsylvania Triple helix terminator for efficient rna trans-splicing
AU2022214429A1 (en) 2021-02-01 2023-09-14 Tern Therapeutics, Llc Gene therapy for neuronal ceroid lipofuscinoses
AU2022337146A1 (en) 2021-09-03 2024-03-14 Tacit Therapeutics, Inc. Rna editing via recruitment of spliceosome components
GB2634434A (en) * 2022-05-13 2025-04-09 Ascidian Therapeutics Inc ABCA4 trans-splicing molecules
EP4551703A2 (en) * 2022-07-07 2025-05-14 Aldevron, LLC Ocular vectors and uses thereof
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
KR20250157548A (ko) * 2023-02-15 2025-11-04 아시디엔 테라퓨틱스, 인크. Htt 트랜스 스플라이싱 분자
WO2025166360A1 (en) * 2024-02-03 2025-08-07 Duke University Compositions for and methods of engineering the transcriptome

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US6280978B1 (en) 1995-12-15 2001-08-28 Intronn Holdings, Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
CA2267795C (en) 1996-10-04 2011-04-05 Intronn Llc Sample collection devices and methods using markers and the use of such markers as controls in sample validation, laboratory evaluation and/or accreditation
US6929907B2 (en) 1999-12-31 2005-08-16 North Carolina State University Methods and compositions for determining the purity of chemically synthesized nucleic acids
WO2003003014A1 (en) 2001-06-29 2003-01-09 Veri-Q, Inc. Methods and compositions for determining the purity of and purifying chemically synthesized nucleic acids
CA2475802A1 (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
CA2477295A1 (en) 2002-02-25 2003-09-04 Intronn, Inc. Trans-splicing mediated imaging of gene expression
JP2005528912A (ja) 2002-06-05 2005-09-29 イントロン，インコーポレーテッド 幹細胞におけるスプライセオソーム介在ｒｎａトランススプライシング
AU2003237449A1 (en) 2002-06-05 2003-12-22 Virxsys Corporation Spliceosome mediated RNA trans-splicing and correction of factor VIII genetic defects
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CA3097004A1 (en) 2018-04-17 2019-10-24 The Trustees Of The University Of Pennsylvania Trans-splicing molecules
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US20220204989A1 (en) 2019-04-17 2022-06-30 The Trustees Of The University Of Pennsylvania Triple helix terminator for efficient rna trans-splicing
CN113795155A (zh) 2019-05-01 2021-12-14 宝洁公司 产生短链脂肪酸的益生菌菌株和包含该益生菌菌株的组合物
GB2634434A (en) 2022-05-13 2025-04-09 Ascidian Therapeutics Inc ABCA4 trans-splicing molecules

Also Published As

Publication number Publication date
BR112020021228A2 (pt) 2021-03-02
EP3781213A1 (en) 2021-02-24
US20240318186A1 (en) 2024-09-26
JP7561624B2 (ja) 2024-10-04
US20210155938A1 (en) 2021-05-27
AU2019255708A1 (en) 2020-11-26
CN112449605A (zh) 2021-03-05
KR102866133B1 (ko) 2025-09-30
EP3781213A4 (en) 2022-10-19
MX2025007209A (es) 2025-07-01
US12442003B2 (en) 2025-10-14
CA3097004A1 (en) 2019-10-24
JP2021520837A (ja) 2021-08-26
MX2020010959A (es) 2021-01-15
US11993776B2 (en) 2024-05-28
WO2019204514A1 (en) 2019-10-24
KR20210018222A (ko) 2021-02-17
JP2024099770A (ja) 2024-07-25
KR20250150157A (ko) 2025-10-17

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