AU2017330411B2 - AAV treatment of huntington's disease - Google Patents
AAV treatment of huntington's disease Download PDFInfo
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- AU2017330411B2 AU2017330411B2 AU2017330411A AU2017330411A AU2017330411B2 AU 2017330411 B2 AU2017330411 B2 AU 2017330411B2 AU 2017330411 A AU2017330411 A AU 2017330411A AU 2017330411 A AU2017330411 A AU 2017330411A AU 2017330411 B2 AU2017330411 B2 AU 2017330411B2
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- 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
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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- 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
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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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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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
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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
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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
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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
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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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- C12N2320/30—Special therapeutic applications
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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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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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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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
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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
- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
- C12N2750/14171—Demonstrated in vivo effect
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2800/00—Nucleic acids vectors
- C12N2800/10—Plasmid DNA
- C12N2800/106—Plasmid DNA for vertebrates
- C12N2800/107—Plasmid DNA for vertebrates for mammalian
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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)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Epidemiology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Psychology (AREA)
- Immunology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| AU2025200036A AU2025200036A1 (en) | 2016-09-22 | 2025-01-03 | Aav treatment of huntington’s disease |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662398487P | 2016-09-22 | 2016-09-22 | |
| US62/398,487 | 2016-09-22 | ||
| PCT/US2017/052902 WO2018057855A1 (en) | 2016-09-22 | 2017-09-22 | Aav treatment of huntington's disease |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2025200036A Division AU2025200036A1 (en) | 2016-09-22 | 2025-01-03 | Aav treatment of huntington’s disease |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| AU2017330411A1 AU2017330411A1 (en) | 2019-04-11 |
| AU2017330411B2 true AU2017330411B2 (en) | 2024-10-10 |
Family
ID=61691085
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2017330411A Active AU2017330411B2 (en) | 2016-09-22 | 2017-09-22 | AAV treatment of huntington's disease |
| AU2025200036A Pending AU2025200036A1 (en) | 2016-09-22 | 2025-01-03 | Aav treatment of huntington’s disease |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2025200036A Pending AU2025200036A1 (en) | 2016-09-22 | 2025-01-03 | Aav treatment of huntington’s disease |
Country Status (19)
| Country | Link |
|---|---|
| US (4) | US10457940B2 (enExample) |
| EP (1) | EP3515505A4 (enExample) |
| JP (4) | JP6884430B2 (enExample) |
| KR (1) | KR102271675B1 (enExample) |
| CN (2) | CN110035776B (enExample) |
| AU (2) | AU2017330411B2 (enExample) |
| BR (2) | BR122020019033B1 (enExample) |
| CA (1) | CA3037929A1 (enExample) |
| CL (1) | CL2019000732A1 (enExample) |
| CO (1) | CO2019003846A2 (enExample) |
| IL (3) | IL265528B2 (enExample) |
| MX (2) | MX2019003376A (enExample) |
| MY (1) | MY195386A (enExample) |
| PE (2) | PE20191351A1 (enExample) |
| PH (1) | PH12019500626A1 (enExample) |
| RU (1) | RU2749971C2 (enExample) |
| SA (1) | SA519401379B1 (enExample) |
| WO (1) | WO2018057855A1 (enExample) |
| ZA (1) | ZA201902226B (enExample) |
Families Citing this family (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10975391B2 (en) | 2014-04-25 | 2021-04-13 | University Of Massachusetts | Recombinant AAV vectors useful for reducing immunity against transgene products |
| AU2017268382B2 (en) | 2016-05-18 | 2023-09-28 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's disease |
| US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| WO2018204803A1 (en) * | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| EP3754019B1 (en) * | 2018-02-13 | 2024-01-31 | Toray Industries, Inc. | Use of a kit or device and method for detecting dementia |
| JP7607872B2 (ja) * | 2018-03-30 | 2025-01-06 | ユニバーシティ オブ ジュネーブ | マイクロrna発現構築物及びその使用 |
| EP3788141A4 (en) | 2018-04-30 | 2023-03-08 | The Brigham and Women's Hospital, Inc. | COMPOSITIONS AND THERAPEUTIC METHODS OF MICRORNA GENE DELIVERY |
| EP3794125A4 (en) * | 2018-05-15 | 2022-07-13 | University of Massachusetts | MODIFIED AAV CONSTRUCTS AND USES THEREOF |
| US12391947B2 (en) | 2018-11-29 | 2025-08-19 | University Of Massachusetts | Modulation of SPTLC1 via recombinant adeno-associated vectors |
| US20220098592A1 (en) * | 2019-01-09 | 2022-03-31 | Universidade De Coimbra | Double stranded rna and uses thereof |
| WO2021087036A1 (en) * | 2019-11-01 | 2021-05-06 | Alnylam Pharmaceuticals, Inc. | HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF |
| CA3163034A1 (en) * | 2019-12-20 | 2021-06-24 | SiewHui LOW | Methods for treating huntington's disease |
| IL301525A (en) * | 2020-09-21 | 2023-05-01 | Asklepios Biopharmaceutical Inc | Methods for treating neurological disease |
| EP4277915A4 (en) * | 2021-01-13 | 2025-03-05 | Dignity Health | MODULATION OF CHITINASE PROTEIN EXPRESSION |
| CN115141848A (zh) * | 2021-03-30 | 2022-10-04 | 南京大学 | 一种用于治疗亨廷顿病的rna递送系统 |
| CN115487315B (zh) * | 2022-04-20 | 2023-08-04 | 暨南大学 | 治疗亨廷顿病的药物 |
| US20240360443A1 (en) | 2023-04-09 | 2024-10-31 | Dystrogen Gene Therapies Inc. | Nucleic acid molecule and use of the nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide cag repeats |
| WO2025027116A1 (en) | 2023-08-01 | 2025-02-06 | Institut Curie | Nanoparticles comprising nucleic acid sequences encoding cyclic gmp-amp synthase |
| WO2025078598A1 (en) * | 2023-10-11 | 2025-04-17 | Uniqure Biopharma B.V. | Novel nucleic acid regulation of huntingtin gene |
| WO2025160434A1 (en) * | 2024-01-26 | 2025-07-31 | Genzyme Corporation | Artificial micrornas targeting huntington's disease |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20110212520A1 (en) * | 2002-08-05 | 2011-09-01 | University Of Iowa Research Foundation | Rna interference suppression of neurodegenerative diseases and methods of use thereof |
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