JP2023514204A - Ｃｄｋｌ５欠損障害を処置するための遺伝子治療 - Google Patents

Ｃｄｋｌ５欠損障害を処置するための遺伝子治療 Download PDF

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JP2023514204A
JP2023514204A JP2022548652A JP2022548652A JP2023514204A JP 2023514204 A JP2023514204 A JP 2023514204A JP 2022548652 A JP2022548652 A JP 2022548652A JP 2022548652 A JP2022548652 A JP 2022548652A JP 2023514204 A JP2023514204 A JP 2023514204A
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raav
promoter
sequence
cdkl5
seq
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JP2023514204A5 (https=
JPWO2021163322A5 (https=
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シャリル リン ファイフ－マリチッチ，
マシュー スコット フラー，
マーガレット キャロライン ライト，
ローレライ イオアナ ストイカ，
スチュワート クレイグ，
ショーン クリストファー ドーハティ，
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ウルトラジェニックス ファーマシューティカル インコーポレイテッド
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Publication of JP2023514204A5 publication Critical patent/JP2023514204A5/ja
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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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    • 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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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
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    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1205—Phosphotransferases with an alcohol group as acceptor (2.7.1), e.g. protein kinases
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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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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    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11—Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11022—Cyclin-dependent kinase (2.7.11.22)

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2022548652A 2020-02-14 2021-02-11 Ｃｄｋｌ５欠損障害を処置するための遺伝子治療 Pending JP2023514204A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202062976483P 2020-02-14 2020-02-14
US62/976,483 2020-02-14
US202063090492P 2020-10-12 2020-10-12
US63/090,492 2020-10-12
PCT/US2021/017656 WO2021163322A1 (en) 2020-02-14 2021-02-11 Gene therapy for treating cdkl5 deficiency disorder

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JP2023514204A true JP2023514204A (ja) 2023-04-05
JP2023514204A5 JP2023514204A5 (https=) 2024-02-20
JPWO2021163322A5 JPWO2021163322A5 (https=) 2024-02-20

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US (1) US20230054144A1 (https=)
EP (1) EP4103724A1 (https=)
JP (1) JP2023514204A (https=)
KR (1) KR20220140537A (https=)
CN (1) CN115151648A (https=)
AU (1) AU2021218703A1 (https=)
BR (1) BR112022015921A2 (https=)
CA (1) CA3164714A1 (https=)
IL (1) IL294781A (https=)
MX (1) MX2022009982A (https=)
TW (1) TWI904135B (https=)
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WO2022225978A1 (en) * 2021-04-21 2022-10-27 The Regents Of The University Of California Use of a split dcas fusion protein system for epigenetic editing
EP4387641A4 (en) * 2021-08-18 2025-07-09 Ultragenyx Pharmaceutical Inc RECOMBINANT AAV FORMULATIONS
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CN116350801A (zh) 2022-11-22 2023-06-30 四川至善唯新生物科技有限公司 一种重组腺相关病毒载体的药物组合物及其用途
CN116377050A (zh) * 2022-12-16 2023-07-04 湖南家辉生物技术有限公司 一种发育性癫痫性脑病2型致病基因cdkl5突变位点的应用及其检测试剂和应用
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BR112022015921A2 (pt) 2022-10-04
TWI904135B (zh) 2025-11-11
KR20220140537A (ko) 2022-10-18
AU2021218703A1 (en) 2022-07-28
US20230054144A1 (en) 2023-02-23
MX2022009982A (es) 2022-09-12
TW202144577A (zh) 2021-12-01
WO2021163322A1 (en) 2021-08-19
CA3164714A1 (en) 2021-08-19
CN115151648A (zh) 2022-10-04
EP4103724A1 (en) 2022-12-21
IL294781A (en) 2022-09-01

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