AU2018352236B2 - Treatment of amyotrophic lateral sclerosis (ALS) - Google Patents

Treatment of amyotrophic lateral sclerosis (ALS) Download PDF

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Publication number
AU2018352236B2
AU2018352236B2 AU2018352236A AU2018352236A AU2018352236B2 AU 2018352236 B2 AU2018352236 B2 AU 2018352236B2 AU 2018352236 A AU2018352236 A AU 2018352236A AU 2018352236 A AU2018352236 A AU 2018352236A AU 2018352236 B2 AU2018352236 B2 AU 2018352236B2
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Australia
Prior art keywords
aav
sequence
viral genome
limiting example
promoter
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Expired - Fee Related
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AU2018352236A
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AU2018352236A1 (en
Inventor
Qingmin Chen
Joan R. Coates
Jinzhao Hou
Mathieu E. NONNENMACHER
Holger Patzke
Dinah Wen-Yee Sah
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Voyager Therapeutics Inc
University of Missouri St Louis
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Voyager Therapeutics Inc
University of Missouri Columbia
University of Missouri St Louis
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    • 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
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    • C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
    • C12Y115/01001—Superoxide dismutase (1.15.1.1)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2267/03—Animal model, e.g. for test or diseases
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    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
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    • 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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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
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  • Chemical Kinetics & Catalysis (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (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)
AU2018352236A 2017-10-16 2018-10-16 Treatment of amyotrophic lateral sclerosis (ALS) Expired - Fee Related AU2018352236B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762572702P 2017-10-16 2017-10-16
US62/572,702 2017-10-16
PCT/US2018/055999 WO2019079240A1 (en) 2017-10-16 2018-10-16 TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)

Publications (2)

Publication Number Publication Date
AU2018352236A1 AU2018352236A1 (en) 2020-04-23
AU2018352236B2 true AU2018352236B2 (en) 2025-04-10

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AU2018352236A Expired - Fee Related AU2018352236B2 (en) 2017-10-16 2018-10-16 Treatment of amyotrophic lateral sclerosis (ALS)

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US (3) US11434502B2 (https=)
EP (2) EP3697905A1 (https=)
JP (2) JP7502991B2 (https=)
CN (2) CN119242711A (https=)
AU (1) AU2018352236B2 (https=)
CA (1) CA3077426A1 (https=)
TW (2) TW202413649A (https=)
WO (1) WO2019079240A1 (https=)

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CA3193811A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
CN119876138A (zh) 2014-11-14 2025-04-25 沃雅戈治疗公司 调节性多核苷酸
CA3024448C (en) 2016-05-18 2025-09-09 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
CA3024449A1 (en) 2016-05-18 2017-11-23 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
JP2020518259A (ja) * 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ハンチントン病治療組成物および方法
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
SG11202006677XA (en) * 2018-01-25 2020-08-28 Biogen Ma Inc Methods of treating spinal muscular atrophy
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
EP3861010A1 (en) 2018-10-02 2021-08-11 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
MA54383A (fr) 2018-12-06 2021-10-13 Biogen Ma Inc Protéine neurofilamenteuse pour guider une intervention thérapeutique dans la sclérose laterale amyotrophique
WO2020223296A1 (en) * 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
CN110452910B (zh) * 2019-08-27 2023-05-30 深圳市宝安区妇幼保健院 一种fus突变基因、检测引物和试剂盒
CN114901822A (zh) * 2019-12-23 2022-08-12 维萨梅布有限公司 用于同时调节基因表达的组合物和方法
CN116096734A (zh) 2020-05-13 2023-05-09 沃雅戈治疗公司 Aav衣壳的向性的重定向
MX2024004936A (es) 2021-11-02 2024-05-08 Voyager Therapeutics Inc Variantes de capsides de aav y sus usos.
WO2023092002A2 (en) * 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Compositions and methods for treating amyotrophic lateral sclerosis and disorders associatedwith the spinal cord
WO2024226761A2 (en) 2023-04-26 2024-10-31 Voyager Therapeutics, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
WO2025045130A1 (zh) * 2023-08-29 2025-03-06 石药集团中奇制药技术（石家庄）有限公司 一种抑制sod1基因表达的dsrna分子及其应用

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