JP6985283B2 - Ｓｅｒｐｉｎｃ１関連障害を処置するための方法および組成物 - Google Patents

Ｓｅｒｐｉｎｃ１関連障害を処置するための方法および組成物 Download PDF

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JP6985283B2
JP6985283B2 JP2018549141A JP2018549141A JP6985283B2 JP 6985283 B2 JP6985283 B2 JP 6985283B2 JP 2018549141 A JP2018549141 A JP 2018549141A JP 2018549141 A JP2018549141 A JP 2018549141A JP 6985283 B2 JP6985283 B2 JP 6985283B2
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pharmaceutical composition
hemophilia
nucleotides
sense strand
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JP2019504112A5 (OSRAM
JP2019504112A (ja
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アキン・アキンク
ベニー・ソレンセン
プシュカル・ガーグ
ガブリエル・ロビー
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Genzyme Corp
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Genzyme Corp
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
    • A—HUMAN NECESSITIES
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
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    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
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    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
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    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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    • 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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/00—Structure or type of the nucleic acid
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JP2018549141A 2015-12-07 2016-12-07 Ｓｅｒｐｉｎｃ１関連障害を処置するための方法および組成物 Active JP6985283B2 (ja)

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JP2021189823A JP7422120B2 (ja) 2015-12-07 2021-11-24 Ｓｅｒｐｉｎｃ１関連障害を処置するための方法および組成物
JP2024002987A JP2024046760A (ja) 2015-12-07 2024-01-12 Ｓｅｒｐｉｎｃ１関連障害を処置するための方法および組成物

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201562264013P 2015-12-07 2015-12-07
US62/264,013 2015-12-07
US201662315228P 2016-03-30 2016-03-30
US62/315,228 2016-03-30
US201662366304P 2016-07-25 2016-07-25
US62/366,304 2016-07-25
US201662429241P 2016-12-02 2016-12-02
US62/429,241 2016-12-02
PCT/US2016/065245 WO2017100236A1 (en) 2015-12-07 2016-12-07 Methods and compositions for treating a serpinc1-associated disorder

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JP2019504112A5 JP2019504112A5 (OSRAM) 2020-11-26
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JP2021189823A Active JP7422120B2 (ja) 2015-12-07 2021-11-24 Ｓｅｒｐｉｎｃ１関連障害を処置するための方法および組成物
JP2024002987A Pending JP2024046760A (ja) 2015-12-07 2024-01-12 Ｓｅｒｐｉｎｃ１関連障害を処置するための方法および組成物

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JP2024002987A Pending JP2024046760A (ja) 2015-12-07 2024-01-12 Ｓｅｒｐｉｎｃ１関連障害を処置するための方法および組成物

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US (2) US11091759B2 (OSRAM)
EP (2) EP4424828A1 (OSRAM)
JP (3) JP6985283B2 (OSRAM)
KR (3) KR20210157474A (OSRAM)
CN (2) CN120284999A (OSRAM)
AU (3) AU2016368289C1 (OSRAM)
BR (1) BR112018011450A2 (OSRAM)
CA (1) CA3007014A1 (OSRAM)
CO (1) CO2018006590A2 (OSRAM)
DK (1) DK3386518T3 (OSRAM)
ES (1) ES3041380T3 (OSRAM)
FI (1) FI3386518T3 (OSRAM)
HR (1) HRP20251125T1 (OSRAM)
IL (2) IL259795B2 (OSRAM)
LT (1) LT3386518T (OSRAM)
MX (2) MX2018006989A (OSRAM)
PL (1) PL3386518T3 (OSRAM)
PT (1) PT3386518T (OSRAM)
RS (1) RS67250B1 (OSRAM)
RU (2) RU2754188C2 (OSRAM)
SG (2) SG11201804729RA (OSRAM)
SI (1) SI3386518T1 (OSRAM)
TW (3) TW202423457A (OSRAM)
WO (1) WO2017100236A1 (OSRAM)

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