AU2020286462A1 - Methods for the treatment of alpha-1 antitrypsin deficiency (AATD) - Google Patents

Methods for the treatment of alpha-1 antitrypsin deficiency (AATD) Download PDF

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Publication number
AU2020286462A1
AU2020286462A1 AU2020286462A AU2020286462A AU2020286462A1 AU 2020286462 A1 AU2020286462 A1 AU 2020286462A1 AU 2020286462 A AU2020286462 A AU 2020286462A AU 2020286462 A AU2020286462 A AU 2020286462A AU 2020286462 A1 AU2020286462 A1 AU 2020286462A1
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AU
Australia
Prior art keywords
aat
drug substance
dose
aat rnai
rnai drug
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Pending
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AU2020286462A
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English (en)
Inventor
Dawn CHRISTIANSON
Bruce GIVEN
James Hamilton
Zhen Li
Tao Pei
Christine Wooddell
Rui ZHU
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Arrowhead Pharmaceuticals Inc
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Arrowhead Pharmaceuticals Inc
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Publication of AU2020286462A1 publication Critical patent/AU2020286462A1/en
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
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    • 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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    • C12N2310/30—Chemical structure
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    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2310/35—Nature of the modification
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen

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  • Inorganic Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2020286462A 2019-06-06 2020-06-05 Methods for the treatment of alpha-1 antitrypsin deficiency (AATD) Pending AU2020286462A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962858059P 2019-06-06 2019-06-06
US62/858,059 2019-06-06
PCT/US2020/036359 WO2020247774A1 (en) 2019-06-06 2020-06-05 Methods for the treatment of alpha-1 antitrypsin deficiency (aatd)

Publications (1)

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AU2020286462A1 true AU2020286462A1 (en) 2022-01-27

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AU2020286462A Pending AU2020286462A1 (en) 2019-06-06 2020-06-05 Methods for the treatment of alpha-1 antitrypsin deficiency (AATD)

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Country Link
US (1) US12582668B2 (https=)
EP (1) EP3980540A4 (https=)
JP (2) JP7697889B2 (https=)
KR (1) KR20220019255A (https=)
CN (1) CN114222820A (https=)
AU (1) AU2020286462A1 (https=)
BR (1) BR112021024534A2 (https=)
CA (1) CA3141762A1 (https=)
CO (1) CO2022000016A2 (https=)
MX (1) MX2021015003A (https=)
TW (1) TW202113081A (https=)
WO (1) WO2020247774A1 (https=)

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JP2022535127A (ja) 2022-08-04
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US12582668B2 (en) 2026-03-24
CA3141762A1 (en) 2020-12-10
US20220305046A1 (en) 2022-09-29
BR112021024534A2 (pt) 2022-02-08
WO2020247774A1 (en) 2020-12-10
JP7697889B2 (ja) 2025-06-24
EP3980540A1 (en) 2022-04-13
JP2025096283A (ja) 2025-06-26
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CN114222820A (zh) 2022-03-22
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