AU2020407119A1 - Methods for treating Huntington's disease - Google Patents

Methods for treating Huntington's disease Download PDF

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Publication number
AU2020407119A1
AU2020407119A1 AU2020407119A AU2020407119A AU2020407119A1 AU 2020407119 A1 AU2020407119 A1 AU 2020407119A1 AU 2020407119 A AU2020407119 A AU 2020407119A AU 2020407119 A AU2020407119 A AU 2020407119A AU 2020407119 A1 AU2020407119 A1 AU 2020407119A1
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AU
Australia
Prior art keywords
nucleic acid
seq
sequence
isolated nucleic
mirna
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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.)
Pending
Application number
AU2020407119A
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English (en)
Inventor
SiewHui LOW
Philippe Moullier
Bin Xiao
Xiao Xiao
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.)
University of North Carolina at Chapel Hill
AskBio Inc
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University of North Carolina at Chapel Hill
AskBio 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 University of North Carolina at Chapel Hill, AskBio Inc filed Critical University of North Carolina at Chapel Hill
Publication of AU2020407119A1 publication Critical patent/AU2020407119A1/en
Assigned to THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL, AskBio Inc. reassignment THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL Amend patent request/document other than specification (104) Assignors: ASKLEPIOS BIOPHARMACEUTICAL, INC., THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL
Pending legal-status Critical Current

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • C—CHEMISTRY; METALLURGY
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    • 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
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    • 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
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • C12N9/0077—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14) with a reduced iron-sulfur protein as one donor (1.14.15)
    • C12N9/0081—Cholesterol monooxygenase (cytochrome P 450scc)(1.14.15.6)
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
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    • C12N2320/31—Combination therapy
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    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
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    • 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
    • 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
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • C—CHEMISTRY; METALLURGY
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    • 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
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/156—Polymorphic or mutational markers
    • 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
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/158—Expression markers

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Neurology (AREA)
  • Plant Pathology (AREA)
  • Neurosurgery (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
AU2020407119A 2019-12-20 2020-12-18 Methods for treating Huntington's disease Pending AU2020407119A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962951582P 2019-12-20 2019-12-20
US62/951,582 2019-12-20
PCT/US2020/066039 WO2021127455A1 (en) 2019-12-20 2020-12-18 Methods for treating huntington's disease

Publications (1)

Publication Number Publication Date
AU2020407119A1 true AU2020407119A1 (en) 2022-07-14

Family

ID=76478540

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2020407119A Pending AU2020407119A1 (en) 2019-12-20 2020-12-18 Methods for treating Huntington's disease

Country Status (7)

Country Link
US (1) US20230340470A1 (enExample)
EP (1) EP4076656A4 (enExample)
JP (2) JP7730327B2 (enExample)
CN (1) CN115443339B (enExample)
AU (1) AU2020407119A1 (enExample)
CA (1) CA3163034A1 (enExample)
WO (1) WO2021127455A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2023211606A1 (en) * 2022-01-27 2024-07-11 AskBio Inc. Compositions for treating neurological disease

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003084977A1 (en) * 2002-04-04 2003-10-16 Xiao Xiao Gene expression control system and its use in recombinant virus packaging cell lines
US8795987B2 (en) * 2005-02-11 2014-08-05 International Business Machines Corporation Ribonucleic acid interference molecules of Oryza sativa
EP2325315B1 (en) * 2005-10-28 2014-05-07 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of huntingtin gene
EP2422819B1 (en) 2006-01-26 2017-03-01 Ionis Pharmaceuticals, Inc. Compositions and their uses directed to Huntingtin
US9920317B2 (en) * 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
MX391904B (es) 2013-11-11 2025-03-12 Sangamo Biosciences Inc Un represor genetico para usarse en el tratamiento de la enfermedad de huntington.
BR112016025849A2 (pt) * 2014-05-08 2017-10-17 Chdi Foundation Inc métodos e composições para o tratamento da doença de huntington
AU2015274457A1 (en) * 2014-06-11 2017-01-05 Circuit Therapeutics, Inc. Optogenetic therapies for movement disorders
US10457940B2 (en) * 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
WO2019217483A1 (en) * 2018-05-07 2019-11-14 Spark Therapeutics, Inc. Plasmid free aav vector producing cell lines
EP4022057A1 (en) 2019-08-27 2022-07-06 Vertex Pharmaceuticals Incorporated Compositions and methods for treatment of disorders associated with repetitive dna

Also Published As

Publication number Publication date
US20230340470A1 (en) 2023-10-26
CN115443339A (zh) 2022-12-06
CN115443339B (zh) 2026-06-02
EP4076656A1 (en) 2022-10-26
CA3163034A1 (en) 2021-06-24
EP4076656A4 (en) 2024-07-03
JP7730327B2 (ja) 2025-08-27
JP2023507741A (ja) 2023-02-27
JP2025166191A (ja) 2025-11-05
WO2021127455A1 (en) 2021-06-24

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DA3 Amendments made section 104

Free format text: THE NATURE OF THE AMENDMENT IS: TO ADD THE NAMES OF CO-INVENTORS XIAO, BIN AND MOULLIER, PHILIPPE

HB Alteration of name in register

Owner name: THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL

Free format text: FORMER NAME(S): ASKLEPIOS BIOPHARMACEUTICAL, INC. ; THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL

Owner name: ASKBIO INC.

Free format text: FORMER NAME(S): ASKLEPIOS BIOPHARMACEUTICAL, INC. ; THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL