AU2017222653B2 - Recombinant virus products and methods for inducing DUX4 exon skipping - Google Patents

Recombinant virus products and methods for inducing DUX4 exon skipping Download PDF

Info

Publication number
AU2017222653B2
AU2017222653B2 AU2017222653A AU2017222653A AU2017222653B2 AU 2017222653 B2 AU2017222653 B2 AU 2017222653B2 AU 2017222653 A AU2017222653 A AU 2017222653A AU 2017222653 A AU2017222653 A AU 2017222653A AU 2017222653 B2 AU2017222653 B2 AU 2017222653B2
Authority
AU
Australia
Prior art keywords
dux4
seq
aav
set out
nucleotide sequence
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
AU2017222653A
Other languages
English (en)
Other versions
AU2017222653A1 (en
Inventor
Scott Quenton HARPER
Nicolas Sebastien Wein
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Nationwide Childrens Hospital Inc
Original Assignee
Nationwide Childrens Hospital 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 Nationwide Childrens Hospital Inc filed Critical Nationwide Childrens Hospital Inc
Publication of AU2017222653A1 publication Critical patent/AU2017222653A1/en
Application granted granted Critical
Publication of AU2017222653B2 publication Critical patent/AU2017222653B2/en
Priority to AU2023216733A priority Critical patent/AU2023216733B2/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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
    • C12N15/11—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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Veterinary Medicine (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Analytical Chemistry (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2017222653A 2016-02-26 2017-02-24 Recombinant virus products and methods for inducing DUX4 exon skipping Active AU2017222653B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2023216733A AU2023216733B2 (en) 2016-02-26 2023-08-14 Recombinant Virus Products And Methods For Inducing DUX4 Exon Skipping

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662300500P 2016-02-26 2016-02-26
US62/300,500 2016-02-26
PCT/US2017/019422 WO2017147467A1 (en) 2016-02-26 2017-02-24 Recombinant virus products and methods for inducing dux4 exon skipping

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2023216733A Division AU2023216733B2 (en) 2016-02-26 2023-08-14 Recombinant Virus Products And Methods For Inducing DUX4 Exon Skipping

Publications (2)

Publication Number Publication Date
AU2017222653A1 AU2017222653A1 (en) 2018-09-13
AU2017222653B2 true AU2017222653B2 (en) 2023-06-01

Family

ID=59685610

Family Applications (2)

Application Number Title Priority Date Filing Date
AU2017222653A Active AU2017222653B2 (en) 2016-02-26 2017-02-24 Recombinant virus products and methods for inducing DUX4 exon skipping
AU2023216733A Active AU2023216733B2 (en) 2016-02-26 2023-08-14 Recombinant Virus Products And Methods For Inducing DUX4 Exon Skipping

Family Applications After (1)

Application Number Title Priority Date Filing Date
AU2023216733A Active AU2023216733B2 (en) 2016-02-26 2023-08-14 Recombinant Virus Products And Methods For Inducing DUX4 Exon Skipping

Country Status (5)

Country Link
US (2) US11180755B2 (https=)
EP (1) EP3420109A4 (https=)
JP (1) JP6966463B2 (https=)
AU (2) AU2017222653B2 (https=)
WO (1) WO2017147467A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019051290A1 (en) * 2017-09-07 2019-03-14 The Children's Medical Center Corporation COMPOSITIONS AND METHODS FOR THE TREATMENT OF FACIO-SCAPULO-HUMERAL DYSTROPHY
EP3906308A1 (en) * 2018-12-31 2021-11-10 Research Institute at Nationwide Children's Hospital Dux4 rna silencing using rna targeting crispr-cas13b
US20220364086A1 (en) * 2019-07-12 2022-11-17 Daiichi Sankyo Company, Limited ANTISENSE OLIGONUCLEOTIDE CAPABLE OF ALTERING SPLICING OF DUX4 pre-mRNA
IL303230A (en) * 2020-11-30 2023-07-01 Res Inst Nationwide Childrens Hospital Compositions and methods for treating facioscapulohumeral muscular dystrophy (fshd)
WO2022240760A2 (en) 2021-05-10 2022-11-17 Entrada Therapeutics, Inc. COMPOSITIONS AND METHODS FOR MODULATING mRNA SPLICING
AU2023228712A1 (en) 2022-03-04 2024-10-17 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising engineered small nuclear rna (snrna)
WO2026011009A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Compositions and methods for muscle disorders
WO2026030242A1 (en) 2024-07-29 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of facioscapulohumeral muscular dystrophy

Family Cites Families (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus 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
WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
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
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
US9080170B2 (en) 2010-03-17 2015-07-14 Association Institut De Myologie Modified U7 snRNAs for treatment of neuromuscular diseases
ES2596977T3 (es) 2010-09-02 2017-01-13 Université de Mons Agentes útiles en el tratamiento de la distrofia muscular facioescapulohumeral
EP2500434A1 (en) 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
EP2736539B1 (en) 2011-07-25 2017-08-23 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
WO2013120038A2 (en) 2012-02-10 2013-08-15 Regents Of The University Of Minnesota Morpholino targeting dux4 for treating fshd
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
WO2015141521A1 (ja) 2014-03-21 2015-09-24 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及び記録媒体
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
LINDSAY M WALLACE, JIAN LIU, JACQUELINE S DOMIRE, SARA E GARWICK-COPPENS, SUSAN M GUCKES, JERRY R MENDELL, KEVIN M FLANIGAN, SCOTT: "RNA Interference Inhibits DUX4-induced Muscle Toxicity In Vivo: Implications for a Targeted FSHD Therapy", MOLECULAR THERAPY, NO LONGER PUBLISHED BY ELSEVIER, vol. 20, no. 7, 1 July 2012 (2012-07-01), pages 1417 - 1423, XP055169548, ISSN: 15250016, DOI: 10.1038/mt.2012.68 *

Also Published As

Publication number Publication date
JP2019511916A (ja) 2019-05-09
US12084658B2 (en) 2024-09-10
WO2017147467A1 (en) 2017-08-31
EP3420109A1 (en) 2019-01-02
CA3015633A1 (en) 2017-08-31
AU2023216733B2 (en) 2025-12-04
US20190024083A1 (en) 2019-01-24
US20220267768A1 (en) 2022-08-25
AU2023216733A1 (en) 2023-09-28
AU2017222653A1 (en) 2018-09-13
JP6966463B2 (ja) 2021-11-17
EP3420109A4 (en) 2019-11-06
US11180755B2 (en) 2021-11-23

Similar Documents

Publication Publication Date Title
US20240318201A1 (en) Recombinant virus products and methods for inhibition of expression of dux4
AU2023216733B2 (en) Recombinant Virus Products And Methods For Inducing DUX4 Exon Skipping
US11939579B2 (en) Modified U6 promoter system for tissue specific expression
WO2019070741A1 (en) MIARN DECIBLATION SYSTEM FOR SPECIFIC INTERFERENCE OF A FABRIC
CA3015633C (en) Recombinant virus products and methods for inducing dux4 exon skipping
HK40108731A (en) Modified u6 promoter system for tissue specific expression
HK40119328A (en) Recombinant virus products and methods for inhibition of expresssion of dux4
HK40001945A (en) Modified u6 promoter system for tissue specific expression
HK40001945B (en) Modified u6 promoter system for tissue specific expression

Legal Events

Date Code Title Description
DA3 Amendments made section 104

Free format text: THE NATURE OF THE AMENDMENT IS: AMEND THE NAME OF THE INVENTOR TO READ HARPER, SCOTT QUENTON AND WEIN, NICOLAS SEBASTIEN

FGA Letters patent sealed or granted (standard patent)