AU2019326551A1 - Recombinant virus products and methods for inhibiting expression of dystrophia myotonica protein kinase and/or interfering with a trinucleotide repeat expansion in the 3' untranslated region of the DMPK gene - Google Patents

Recombinant virus products and methods for inhibiting expression of dystrophia myotonica protein kinase and/or interfering with a trinucleotide repeat expansion in the 3' untranslated region of the DMPK gene Download PDF

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Publication number
AU2019326551A1
AU2019326551A1 AU2019326551A AU2019326551A AU2019326551A1 AU 2019326551 A1 AU2019326551 A1 AU 2019326551A1 AU 2019326551 A AU2019326551 A AU 2019326551A AU 2019326551 A AU2019326551 A AU 2019326551A AU 2019326551 A1 AU2019326551 A1 AU 2019326551A1
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AU
Australia
Prior art keywords
aav
dmpk
seq
viral vector
gene
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Abandoned
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AU2019326551A
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English (en)
Inventor
Kelly Reed Clark
Scott Quenton HARPER
Nicolas Sebastien Wein
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Nationwide Childrens Hospital Inc
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Nationwide Childrens Hospital Inc
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Publication date
Application filed by Nationwide Childrens Hospital Inc filed Critical Nationwide Childrens Hospital Inc
Publication of AU2019326551A1 publication Critical patent/AU2019326551A1/en
Priority to AU2025283617A priority Critical patent/AU2025283617A1/en
Abandoned legal-status Critical Current

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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
    • C12N15/11—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
    • C12N15/1137—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 enzymes
    • 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
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2019326551A 2018-08-22 2019-08-22 Recombinant virus products and methods for inhibiting expression of dystrophia myotonica protein kinase and/or interfering with a trinucleotide repeat expansion in the 3' untranslated region of the DMPK gene Abandoned AU2019326551A1 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2025283617A AU2025283617A1 (en) 2018-08-22 2025-12-19 Recombinant Virus Products And Methods For Inhibiting Expression Of Dystrophia Myotonica Protein Kinase And/Or Interfering With A Trinucleotide Repeat Expansion In The 3' Untranslated Region Of The Dmpk Gene

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862721105P 2018-08-22 2018-08-22
US62/721,105 2018-08-22
PCT/US2019/047779 WO2020041634A1 (en) 2018-08-22 2019-08-22 Recombinant virus products and methods for inhibiting expression of dystrophia myotonica protein kinase and/or interfering with a trinucleotide repeat expansion in the 3' untranslated region of the dmpk gene

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2025283617A Division AU2025283617A1 (en) 2018-08-22 2025-12-19 Recombinant Virus Products And Methods For Inhibiting Expression Of Dystrophia Myotonica Protein Kinase And/Or Interfering With A Trinucleotide Repeat Expansion In The 3' Untranslated Region Of The Dmpk Gene

Publications (1)

Publication Number Publication Date
AU2019326551A1 true AU2019326551A1 (en) 2021-03-18

Family

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Family Applications (2)

Application Number Title Priority Date Filing Date
AU2019326551A Abandoned AU2019326551A1 (en) 2018-08-22 2019-08-22 Recombinant virus products and methods for inhibiting expression of dystrophia myotonica protein kinase and/or interfering with a trinucleotide repeat expansion in the 3' untranslated region of the DMPK gene
AU2025283617A Pending AU2025283617A1 (en) 2018-08-22 2025-12-19 Recombinant Virus Products And Methods For Inhibiting Expression Of Dystrophia Myotonica Protein Kinase And/Or Interfering With A Trinucleotide Repeat Expansion In The 3' Untranslated Region Of The Dmpk Gene

Family Applications After (1)

Application Number Title Priority Date Filing Date
AU2025283617A Pending AU2025283617A1 (en) 2018-08-22 2025-12-19 Recombinant Virus Products And Methods For Inhibiting Expression Of Dystrophia Myotonica Protein Kinase And/Or Interfering With A Trinucleotide Repeat Expansion In The 3' Untranslated Region Of The Dmpk Gene

Country Status (6)

Country Link
US (2) US12492402B2 (https=)
EP (1) EP3841209A4 (https=)
JP (3) JP2021534755A (https=)
AU (2) AU2019326551A1 (https=)
CA (1) CA3110466A1 (https=)
WO (1) WO2020041634A1 (https=)

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AU2023228712A1 (en) 2022-03-04 2024-10-17 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising engineered small nuclear rna (snrna)
WO2023196862A1 (en) * 2022-04-06 2023-10-12 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
TW202409289A (zh) * 2022-06-27 2024-03-01 美商安斯泰來基因治療股份有限公司 用於治療肌強直性營養不良之組合物及方法
WO2024086633A1 (en) * 2022-10-19 2024-04-25 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of dm1 protein kinase (dmpk) compositions thereof, and methods of use
AR131170A1 (es) * 2022-12-02 2025-02-19 Humble Bee Ltd Polipéptido recombinante
US20240384271A1 (en) * 2023-04-05 2024-11-21 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
WO2025137219A1 (en) * 2023-12-21 2025-06-26 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
EP4692348A1 (en) * 2024-08-07 2026-02-11 Nocturna Therapeutics SL Artificial rnas for treating repeat expansion disorders

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WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
CA2207927A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
WO1997008298A1 (en) 1995-08-30 1997-03-06 Genzyme Corporation Chromatographic purification of adenovirus and aav
US6632670B1 (en) 1995-09-08 2003-10-14 Genzyme Corporation AAV vectors for gene therapy
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
JP2001500497A (ja) 1996-09-06 2001-01-16 トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組換えアデノ随伴ウイルスに指図される遺伝子治療の方法
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
DK1009808T3 (da) 1997-09-05 2013-01-21 Genzyme Corp Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
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
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Also Published As

Publication number Publication date
US20260049320A1 (en) 2026-02-19
EP3841209A1 (en) 2021-06-30
CA3110466A1 (en) 2020-02-27
JP2025166094A (ja) 2025-11-05
JP2024105681A (ja) 2024-08-06
AU2025283617A1 (en) 2026-02-19
JP2021534755A (ja) 2021-12-16
WO2020041634A1 (en) 2020-02-27
US12492402B2 (en) 2025-12-09
EP3841209A4 (en) 2023-01-04
US20220033823A1 (en) 2022-02-03

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