KR102719222B1 - 신경 세로이드 지질갈색소증에 대한 유전자 요법 - Google Patents

신경 세로이드 지질갈색소증에 대한 유전자 요법 Download PDF

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KR102719222B1
KR102719222B1 KR1020197036041A KR20197036041A KR102719222B1 KR 102719222 B1 KR102719222 B1 KR 102719222B1 KR 1020197036041 A KR1020197036041 A KR 1020197036041A KR 20197036041 A KR20197036041 A KR 20197036041A KR 102719222 B1 KR102719222 B1 KR 102719222B1
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aav
tpp1
raav
mice
vector
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KR20200023280A (ko
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제임스 엠. 윌슨
네이선 카츠
크리스티안 힌더러
줄리엣 호르도
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더 트러스티스 오브 더 유니버시티 오브 펜실베니아
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    • C—CHEMISTRY; METALLURGY
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    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
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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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
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    • C12N15/86—Viral vectors
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/485—Exopeptidases (3.4.11-3.4.19)
    • 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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    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16111—Cytomegalovirus, e.g. human herpesvirus 5
    • C12N2710/16141—Use of virus, viral particle or viral elements as a vector
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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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
    • C12Y304/14009—Tripeptidyl-peptidase I (3.4.14.9)

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Plant Pathology (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
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KR1020197036041A 2017-05-11 2018-05-11 신경 세로이드 지질갈색소증에 대한 유전자 요법 Active KR102719222B1 (ko)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762504817P 2017-05-11 2017-05-11
US62/504,817 2017-05-11
US201762599816P 2017-12-18 2017-12-18
US62/599,816 2017-12-18
US201862652006P 2018-04-03 2018-04-03
US62/652,006 2018-04-03
PCT/US2018/032278 WO2018209205A1 (en) 2017-05-11 2018-05-11 Gene therapy for neuronal ceroid lipofuscinoses

Publications (2)

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KR20200023280A KR20200023280A (ko) 2020-03-04
KR102719222B1 true KR102719222B1 (ko) 2024-10-21

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US (3) US11591614B2 (https=)
EP (1) EP3621612A4 (https=)
JP (2) JP7273730B2 (https=)
KR (1) KR102719222B1 (https=)
AU (1) AU2018265531B2 (https=)
BR (1) BR112019023303A2 (https=)
CA (1) CA3061655A1 (https=)
IL (2) IL316926A (https=)
RU (1) RU2019139555A (https=)
SG (2) SG11201910015SA (https=)
WO (1) WO2018209205A1 (https=)

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CA3041548A1 (en) * 2016-11-04 2018-05-11 The Children's Hospital Of Philadelphia Gene transfer compositions, methods and uses for treating neurodegenerative diseases
KR102883778B1 (ko) 2017-09-22 2025-11-12 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Ii형 점액다당류증의 치료를 위한 유전자 요법
BR112021009370A2 (pt) * 2018-11-14 2021-08-17 Regenxbio Inc. método de tratamento da doença de batten cln2, composição farmacêutica e kit
JP7616995B2 (ja) * 2018-11-28 2025-01-17 プリベイル セラピューティクス，インコーポレーテッド 神経変性疾患のための遺伝子治療
WO2020206098A1 (en) * 2019-04-03 2020-10-08 Regenxbio Inc. Gene therapy for eye pathologies
JP2022527116A (ja) * 2019-04-08 2022-05-30 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア Ｔｐｐ１を発現するａａｖの投与による眼のリソソーム蓄積症の治療
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AU2021325891A1 (en) * 2020-08-10 2023-04-06 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disorders
AU2021358781A1 (en) * 2020-10-07 2023-05-18 Tern Therapeutics, Llc Gene therapy for ocular manifestations of cln2 disease
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
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IL316926A (en) 2025-01-01
KR20200023280A (ko) 2020-03-04
SG11201910015SA (en) 2019-11-28
US20260078406A1 (en) 2026-03-19
US20200239908A1 (en) 2020-07-30
JP7273730B2 (ja) 2023-05-15
EP3621612A4 (en) 2021-06-16
WO2018209205A1 (en) 2018-11-15
CA3061655A1 (en) 2018-11-15
SG10201912401QA (en) 2020-02-27
US12428653B2 (en) 2025-09-30
US11591614B2 (en) 2023-02-28
RU2019139555A (ru) 2021-06-11
AU2018265531A1 (en) 2019-11-14
WO2018209205A9 (en) 2019-11-21
RU2019139555A3 (https=) 2021-10-12
EP3621612A1 (en) 2020-03-18
BR112019023303A2 (pt) 2020-06-16
IL270422A (en) 2020-01-30
US20230383312A1 (en) 2023-11-30
IL270422B1 (en) 2024-12-01
IL270422B2 (en) 2025-04-01
JP2023099113A (ja) 2023-07-11
JP2020519292A (ja) 2020-07-02
AU2018265531B2 (en) 2024-07-11

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